4372 results
Citius Oncology Announces Preliminary Topline Phase 1 Data from Study of LYMPHIR™ (E7777) Dosing Prior to Commercial CAR‑T Therapy in High‑Risk Diffuse Large B‑Cell Lymphoma
PR Newswire
2026-03-04
car
Topline data of investigator‑initiated study at the University of Minnesota and City of Hope demonstrates 86% overall response rate (OR), including 57% complete response (CR) and 29% partial response (PR) LYMPHIR was well-tolerated with no dose-limiting toxicities observed CRANFORD, N.J.,...
Prime to test FDA flexibility with 2-patient gene editing submission
Biopharma Dive
2026-03-04
gene editing
The planned filing comes amid heightened scrutiny of the FDA’s rare disease stance and a year after Prime deprioritized the program for economic reasons.
8-K - Ocugen, Inc. (0001372299) (Filer)
SEC Filing (8-K)
2026-03-04
ocugen
Filed: 2026-03-04 AccNo: 0001104659-26-023288 Size: 14 MB Item 2.02: Results of Operations and Financial Condition Item 9.01: Financial Statements and Exhibits
uniQure: Worst Case Scenario Becomes Reality
uniQure
2026-03-04
amt-130
uniQure’s AMT-130 faces FDA efficacy doubts after 12-month data. QURE's upside now hinges on political pressure. Read here for more analysis.
Affinia Therapeutics Granted FDA Fast Track Designation for AFTX-201 as a Treatment for People Living with BAG3-Associated Dilated Cardiomyopathy (DCM)
Affinia Therapeutics
2026-03-04
aftx-201
gene therapies
An investigational medicine that addresses the genetic root cause of BAG3 DCM, AFTX-201 is being evaluated in the UPBEAT© clinical trial AFTX-201 is designed using Affinia’s proprietary capsid engineered for efficient cardiac transduction at doses that are 5-10-fold lower than doses of gene therapies using conventional capsids WALTHAM, Mass. – March 4, 2026 – Affinia […] The post Affinia Therapeutics Granted FDA Fast Track Designation for AFTX-201 as a Treatment for People Living with BAG3-Associated Dilated Cardiomyopathy (DCM) appeared first on AffiniaTX .
Multiple Myeloma Market 2026: Gilead Sciences Acquires Arcellx for $7.8B to Control CAR-T Therapy
Gilead Sciences
2026-03-04
car
multiple myeloma
Market Size and Growth Multiple Myeloma Market reached US 22 3 billion in 2024 and is expected to reach US 34 58 billion by 2032 growing at a CAGR of 5 6 during the forecast period 2025 2032 Request Executive ...
REGENXBIO Announces Presentations at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
REGENXBIO
2026-03-04
rgx-202
gene therapy
duchenne muscular dystrophy
ROCKVILLE, Md., March 4, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations on its RGX-202 investigational gene therapy for Duchenne muscular dystrophy at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Orlando, FL
UniQure’s Delay, REGENXBIO’s Rejection Explained, Sarepta’s Ingram Steps Down, More
uniQure
2026-03-04
uniqure
regenxbio
gene therapies
UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question the FDA’s decision-making processes; CBER director Vinay Prasad is under probe for allegedly fostering a toxic workplace; Sarepta CEO Doug Ingram is stepping down after several years of tumult at the top of the muscular dystrophy–focused company; and Eli Lilly again tops Novo Nordisk in a weight loss trial.
Cryoport Reports Fourth Quarter and Full-Year 2025 Financial Results
Cryoport Inc
2026-03-03
financial results
Fate Therapeutics to Participate in Leerink Partners 2026 Global Healthcare Conference
Fate Therapeutics
2026-03-03
ipsc
SAN DIEGO, March 03, 2026 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases,
Etranacogene Dezaparvovec May Yield Long-Term Factor IX Expression Improvement in Hemophilia B
Hematology Advisor News
2026-03-03
hemophilia b
Researchers determined 1 infusion of etranacogene dezaparvovec may lead to long-term sustained endogenous factor IX expression and low ABRs in hemophilia B.
Kyverna Therapeutics to Participate in Upcoming March Investor Conferences
Kyverna Therapeutics
2026-03-03
cell therapies
EMERYVILLE, Calif., March 03, 2026 (GLOBE NEWSWIRE) -- Kyverna Therapeutics, Inc. (Nasdaq: KYTX), a clinical-stage biopharmaceutical company developing cell therapies for patients with autoimmune diseases, today announced that company management will participate in the following upcoming
Hematologic Malignancies Market to Reach USD 128.48 Billion by 2032, Driven by Immunotherapy, CAR-T, and Precision Oncology - Credence Research Inc.
PR Newswire
2026-03-03
car
The new report "Hematologic Malignancies Market – Growth, Share, Opportunities, Competitive Analysis, and Forecast 2024–2032" has been added to the library of Credence Research Inc. LONDON, March 3, 2026 /PRNewswire/ -- The Hematologic Malignancies Market is projected to grow from USD...
Passage Bio Reports Fourth Quarter and Full Year 2025 Financial Results and Provides Recent Business Highlights
passage bio
2026-03-03
financial results
Abeona Therapeutics® Announces Date of Fourth Quarter / Full Year 2025 Financial Results and Participation in Upcoming 2026 Leerink Global Healthcare Conference
Abeona Therapeutics
2026-03-03
financial results
8-K - Atara Biotherapeutics, Inc. (0001604464) (Filer)
SEC Filing (8-K)
2026-03-03
atara biotherapeutics
Filed: 2026-03-03 AccNo: 0001193125-26-086793 Size: 144 KB Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
Intellia Therapeutics Presents Longer-Term Clinical Data for Lonvoguran Ziclumeran (lonvo-z); Hereditary Angioedema (HAE) Patient-Focused Research at AAAAI 2026
Intellia Therapeutics Inc.
2026-03-03
hereditary angioedema
Presentations include three-year follow-up data from patients receiving a one-time 50 milligram (mg) dose of lonvo-z and survey findings highlighting patients’ continued treatment burden and unmet needs CAMBRIDGE, Mass., March 03, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc.
Gilead Sciences Arcellx Deal Deepens Oncology Bet And Tests HIV Reliance
Gilead Sciences
2026-03-03
cell therapy
Gilead Sciences has entered a definitive agreement to acquire Arcellx, Inc. in a cash deal valued at $7.8b. The transaction expands Gilead's presence in oncology and cell therapy, areas that differ from its core HIV franchise. For investors watching NasdaqGS:GILD, this move comes as the shares trade around $150.03, with a 1 year return of 33.0% and a 3 year return of 105.6%. Over 5 years, the stock shows a very large gain of 180.7%, and the company currently carries a value score of 4,...
ImmunityBio to Provide a Business Update and Review Financial Results for FY 2025
Immunity Bio
2026-03-03
financial results
The post ImmunityBio to Provide a Business Update and Review Financial Results for FY 2025 appeared first on ImmunityBio .
Quell takes new Treg into clinic after transplant study halt
PharmaPhorum
2026-03-03
car
Quell Therapeutics changes tack, starting trials of a CAR-Treg in rheumatoid arthritis and systemic sclerosis and parking its transplant therapy.
CD70-Directed HIT Cell Therapy Could Boost Immunotherapy for Solid Tumors
Inside Precision Medicine
2026-03-03
cell therapy
HLA-independent T cell (HIT) receptors that can sense a very small number of the target molecule CD70 enabled the killing of heterogeneous kidney, pancreatic, and ovarian tumors in mouse models. The post CD70-Directed HIT Cell Therapy Could Boost Immunotherapy for Solid Tumors appeared first on Inside Precision Medicine .
Rigel Provides Fourth Quarter and Full Year 2025 Financial Results and Provides Business Update
PR Newswire
2026-03-03
financial results
Fourth quarter 2025 total revenues of approximately $69.8 million, including record net product sales of $65.4 million and contract revenues of $4.4 million 2025 total revenues of approximately $294.3 million, including net product sales of $232.0 million and contract revenues of $62.3...
Pierre Fabre seeks to revive US approval chances for spurned cell therapy
Biopharma Dive
2026-03-03
ebvallo
cell therapy
pierre fabre
Ebvallo is one of a series of rare disease treatments recently turned back by the FDA amid shifting guidance that has surprised some companies and frustrated investors.
The Progeria Research Foundation and Forge Biologics Announce Manufacturing Partnership to Advance Gene Therapy for Children with Progeria
Forge Biologics
2026-03-03
gene therapy
“The era of Progeria gene therapy has arrived. Our hope is that SamPro-2 will give children and young adults with Progeria the longer, healthier lives they deserve,” said Leslie Gordon, M.D., Ph.D., co-founder and medical director of PRF and the mother of Sam Berns who had Progeria. The post The Progeria Research Foundation and Forge Biologics Announce Manufacturing Partnership to Advance Gene Therapy for Children with Progeria appeared first on Forge Biologics .
Janssen Pharmaceutica describes new IL-17A/ IL-17RA interaction inhibitors
Janssen Global Services
2026-03-03
lupus
multiple sclerosis
Janssen Pharmaceutica NV has identified interleukin-17A (IL-17A)/IL-17 receptor A (IL-17RA) interaction inhibitors reported to be useful for the treatment of psoriasis, rheumatoid arthritis, radiographic axial spondyloarthritis (ankylosing spondylitis), multiple sclerosis, asthma, chronic obstructive pulmonary disease, atopic dermatitis and systemic lupus erythematosus, among others.
REGENXBIO’s Hunter Rejection Hinged on Inadequate Controls, Surrogate Endpoint
BioSpace
2026-03-03
gene therapy
In a complete response letter published by the FDA on Monday, the agency said a resubmission for REGENXBIO’s Hunter syndrome gene therapy should provide evidence of normalized or improved biomarker levels or neurodevelopmental outcomes.
Prime Medicine Reports Full Year 2025 Financial Results and Provides Business Updates
prime medicine
2026-03-03
bla
wilson disease
financial results
On track to file IND and/or CTA for Wilson Disease and AATD programs in 1H 2026 and mid-2026, respectively; initial clinical data for both expected in 2027 -- -- Ongoing engagement with FDA for PM359 in CGD; plan to submit BLA following final alignment -- -- Prime Medicine reported cash, cash
CG Oncology, Inc. (CGON) Stock Analysis: A 39% Potential Upside In The Battle Against Bladder Cancer
CG Oncology
2026-03-03
bladder cancer
CG Oncology, Inc. (CGON) Stock Analysis: A 39% Potential Upside in the Battle Against Bladder Cancer
Intellia’s CRISPR Gene Therapy Free To Move Forward After FDA Fully Lifts Hold
BioSpace
2026-03-03
crispr
gene therapy
The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme elevations in one patient, who died a few days later.
Rocket Pharmaceuticals to Participate in Upcoming Investor Conferences
Rocket Pharmaceuticals
2026-03-03
pipeline
CRANBURY, N.J. --(BUSINESS WIRE)--Aug. 28, 2025-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the Company will participate in the
Atara Biotherapeutics Provides Regulatory Update on Tabelecleucel
Atara Biotherapeutics
2026-03-03
tabelecleucel
The road ahead for uniQure: FDA says more data needed for AMT-130 gene therapy
uniQure
2026-03-02
amt-130
gene therapy
The FDA wants more data before approving AMT-130 for Huntington’s disease in the U.S. On March 2026, uniQure shared in an update that current Phase 1/2 data weren’t enough for the agency. A new randomized, sham-controlled trial may be required.
FDA calls for fresh study of uniQure’s Huntington’s gene therapy, shares slide
uniQure
2026-03-02
gene therapy
By Puyaan Singh and Christy Santhosh March 2 (Reuters) - UniQure said the U.S. drug regulator called for a new study to support the approval of its gene therapy for a brain disorder as the data from a previous trial was insufficient, sending the Dutc...
Allogene Therapeutics to Report Fourth Quarter and Full Year 2025 Financial Results and Provide Business Update
Allogene
2026-03-02
car
allogeneic
financial results
Conference Call and Webcast Scheduled for March 12, 2026 at 2:00 p.m. PT / 5:00 p.m. ET SOUTH SAN FRANCISCO, Calif., March 02, 2026 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T)
Uniqure Crashes On Another Twist In Its Gene Therapy Saga
uniQure
2026-03-02
gene therapy
Uniqure stock collapsed Monday after the FDA said the company will need to run a final-phase study of its gene therapy.
8-K - Intellia Therapeutics, Inc. (0001652130) (Filer)
SEC Filing (8-K)
2026-03-02
intellia therapeutics
Filed: 2026-03-02 AccNo: 0001193125-26-084309 Size: 234 KB Item 1.01: Entry into a Material Definitive Agreement Item 7.01: Regulation FD Disclosure Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
Precision BioSciences Receives $7.5M in Proceeds from TG Therapeutics for Azer-cel Clinical Milestone in Multiple Sclerosis
precision biosciences
2026-03-02
gene editing
multiple sclerosis
– Milestone payments include $5.25 million cash and $2.25 million purchase of Precision stock – – Cash received supports Precision’s expected cash runway through 2028 and strategic focus on in vivo gene editing pipeline – DURHAM, N.C. --(BUSINESS WIRE)--Mar. 2, 2026-- Precision BioSciences, Inc.
UniQure says FDA wants another study of Huntington’s gene therapy
uniQure
2026-03-02
gene therapy
Agency staff "strongly recommended" a sham surgery-controlled trial be conducted before an approval filing, a stance one analyst called a "worst case scenario" for UniQure.
Anixa Biosciences to Host 2026 Annual Meeting of Stockholders and Provide Corporate Update Highlighting Recent Clinical and Regulatory Progress
anixa biosciences
2026-03-02
car
cell therapies
Presentation to Review Ovarian Cancer CAR-T Survival Observations and Dose Escalation, as well as Final Phase 1 Breast Cancer Vaccine Data SAN JOSE, Calif. , March 2, 2026 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that it will host its 2026 Annual Meeting of Stockholders (the "Meeting") on Tuesday, March 10, 2026, at 10:00 a.m. Pacific Time in a virtual format. The Meeting will be open to all interested parties, including non-stockholders. Following the formal business and voting portion of the Meeting, Anixa Chairman and CEO Dr. Amit Kumar will deliver an investor presentation and corporate update. The presentation will include a review of recent clinical milestones across the Company's oncology pipeline, including: Encouraging survival observations and regulatory approval for substantial dose escalation in the Company's ongoing Phase 1 ovarian cancer CAR-T trial; and Final Phase 1 data from the breast cancer vaccine program, which met primary endpoints and demonstrated favorable tolerability and protocol-defined immune responses in the majority of…
UniQure says FDA wants another study of Huntington’s gene therapy
Biopharma Dive
2026-03-02
gene therapy
Agency staff "strongly recommended" a sham surgery-controlled trial be conducted before an approval filing, a stance one analyst called a "worst case scenario" for UniQure.
CG Oncology (CGON) Valuation Check As Bladder Cancer Trial Timelines Accelerate And Cash Runway Extends To 2029
CG Oncology
2026-03-02
bladder cancer
CG Oncology (CGON) has drawn fresh attention after updating timelines for topline data from key Phase 2 and Phase 3 bladder cancer trials, while also highlighting a cash runway that management expects will extend into 2029. See our latest analysis for CG Oncology. The recent update on expedited trial timelines and a cash runway into 2029 comes after a strong run in the shares, with the latest close at $58.8, a 90 day share price return of 42.13% and a 1 year total shareholder return of...
UniQure Stock Drops 36% After Earnings. Bad News From the FDA Gets Even Worse.
uniQure
2026-03-02
amt-130
gene therapy
The U.S. Food and Drug Administration rejects the biotech company’s AMT-130 gene therapy.
UniQure shares crash 40% as FDA rejects early approval path for Huntington's gene therapy
uniQure
2026-03-02
gene therapy
Shares of uniQure plummeted more than 40% in premarket trading Monday after the company confirmed the FDA has
XellSmart Receives Fourth Consecutive US FDA & China NMPA IND Clearance for MSA-P iPSC-derived Cell Therapy
PR Newswire
2026-03-02
ipsc
cell therapy
SUZHOU, China, March 2, 2026 /PRNewswire/ -- In February 2026, XellSmart Pharmaceutical Co., Ltd. (XellSmart) announced that it had secured its fourth US FDA and China NMPA clearance for Phase I/II registrational clinical trial entry with its independently developed, off-the-shelf,...
UniQure Eyes Phase 3 for Huntington’s Gene Therapy Despite Makary’s Apparent Criticism
BioSpace
2026-03-02
amt-130
gene therapy
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed to the biotech that a sham surgery–controlled study is needed before submitting the gene therapy for approval.
Anixa Biosciences to Host 2026 Annual Meeting of Stockholders and Provide Corporate Update Highlighting Recent Clinical and Regulatory Progress
PR Newswire
2026-03-02
car
Presentation to Review Ovarian Cancer CAR-T Survival Observations and Dose Escalation, as well as Final Phase 1 Breast Cancer Vaccine Data SAN JOSE, Calif., March 2, 2026 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on...
uniQure NV (QURE) Q4 2025 Earnings Call Highlights: Breakthroughs and Challenges in Gene Therapy
uniQure
2026-03-02
gene therapy
uniQure NV (QURE) reports promising data on Huntington's disease treatment while navigating regulatory hurdles and financial setbacks.
FDA urges new trial for experimental Huntington's gene therapy AMT-130
uniQure
2026-03-02
amt-130
gene therapy
FDA minutes say AMT-130 Phase I/II data are not enough to support an application, and uniQure plans a new sham-controlled trial and Q2 Type B meeting.
Candid Therapeutics Strikes Up Merger With Rallybio to Advance Its Autoimmune Drug Pipeline
MedCity News
2026-03-02
myasthenia gravis
Along with the reverse merger, Candid Therapeutics announced $505 million in private financing to support its pipeline of T cell engagers for autoimmune disease. Lead program cizutamig is on track to begin global Phase 2 testing in myasthenia gravis and interstitial lung disease. The post Candid Therapeutics Strikes Up Merger With Rallybio to Advance Its Autoimmune Drug Pipeline appeared first on MedCity News .
CAR Macrophages Show Preclinical Efficacy in Lung Cancer Brain Metastases
Inside Precision Medicine
2026-03-02
car
Researchers engineered CAR macrophages capable of crossing the blood–brain barrier and attacking lung cancer brain metastases. The preclinical results suggest a safer, brain-penetrant alternative to CAR T therapy for CNS disease. The post CAR Macrophages Show Preclinical Efficacy in Lung Cancer Brain Metastases appeared first on Inside Precision Medicine .
News
Genetix Biotherapeutics
2026-03-02
bluebird bio
genetix biotherapeutics
bluebird bio Rebrands to Genetix Biotherapeutics, Returning to Its Foundational Roots
UniQure stock plunges after FDA rejects gene therapy data, Sell-side Comments
uniQure
2026-03-02
gene therapy
UniQure stock plunges after FDA rejects gene therapy data, Sell-side Comments
uniQure Announces 2025 Financial Results and Provides Recent Company Updates
uniQure
2026-03-02
amt-130
financial results
Held Type A meeting with FDA to discuss AMT-130 for Huntington’s disease; Company evaluating Phase III development considerations and plans to request...
New FDA knockback hits uniQure and Huntington patients hard
PharmaPhorum
2026-03-02
amt-130
gene therapy
uniQure looks like it may have to run another trial of its Huntington's disease gene therapy AMT-130, delaying the highly anticipated programme.
UniQure slides after FDA says trial data insufficient for brain disorder therapy filing
uniQure
2026-03-02
gene therapy
March 2 (Reuters) - UniQure said on Monday the U.S. drug regulator had informed that data from an early- to mid-stage trial of its brain disorder gene therapy would not support its approval application, sending its shares down 40% in premarket tradin...
CGON: Credo shows high efficacy and durability, with regulatory and commercial plans advancing
CG Oncology
2026-03-02
bladder cancer
Credo demonstrates strong efficacy and durability in multiple bladder cancer settings, with a 75.5% CR rate in BOND-003 and promising adjuvant and combination data. Regulatory filings are underway, with commercial launch preparations targeting a concentrated market and leveraging a robust team.Base…
FDA tells uniQure early trial data insufficient for brain disorder therapy filing
uniQure
2026-03-02
gene therapy
UniQure NASDAQ:QURE said on Monday the U.S. health regulator has informed the company that data from an early- to mid-stage trial of its gene therapy for a brain disorder would not support a marketing application for the treatment.The company's shares slumped nearly 44% in premarket trading.UniQure…
CG Oncology (CGON) Valuation Check As Bladder Cancer Trial Timelines Accelerate And Cash Runway Extends To 2029
CG Oncology
2026-03-02
bladder cancer
U.S. stocks, Saudi stocks, stock trading and investment platforms
The Multiple Sclerosis Association of America (MSAA) Launches Campaign Focused on Self-Advocacy Strategies in Recognition of MS Awareness Month
PR Newswire
2026-03-02
multiple sclerosis
CHERRY HILL, N.J., March 2, 2026 /PRNewswire/ -- The Multiple Sclerosis Association of America (MSAA) proudly recognizes March as MS Awareness Month and is pleased to highlight several programs to support the MS community. This month's awareness campaign, "Your MS, Your Voice: The Power...
Intellia Therapeutics Announces FDA Lift of Clinical Hold on MAGNITUDE Phase 3 Clinical Trial in ATTR-CM
Intellia Therapeutics Inc.
2026-03-02
crispr
gene editing
clinical hold
CAMBRIDGE, Mass., March 02, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, today announced that the U.S.
FDA calls for fresh study of uniQure's Huntington's gene therapy, shares slide
uniQure
2026-03-02
gene therapy
By Puyaan Singh and Christy Santhosh UniQure NASDAQ:QURE said the U.S. drug regulator called for a new study to support the approval of its gene therapy for a brain disorder as the data from a previous trial was insufficient, sending the Dutch drugmaker's U.S.-listed shares more than 36% lower o…
UniQure says FDA prefers randomized trial for Huntington's treatment
Endpoints News
2026-03-02
amt-130
UniQure's stock dropped again Monday morning after it received negative feedback from the FDA on its Huntington’s disease program. Regulators do not believe that the application for the program, known as AMT-130, is “sufficient” to ...
FDA lifts hold on second Intellia Phase 3 gene editing trial
Endpoints News
2026-03-02
gene editing
The FDA has allowed Intellia to restart a Phase 3 study of a gene editing therapy for a heart disease caused by misfolded proteins, lifting a hold that was ordered after a patient in the ...
enGene Maps BLA by End-2026, Targets 2027 Launch for Lead NMIBC Therapy at Oppenheimer Conference
enGene Google
2026-02-28
bla
detalimogene voraplasmid
enGene (NASDAQ:ENGN) Chief Executive Officer Ron Cooper outlined the company’s near-term regulatory and clinical plans for its lead program, detalimogene voraplasmid, during a presentation at Oppenheimer’s 36th Annual Healthcare Life Sciences Conference. Cooper said the company expects to provide an
Does The Share Price Of uniQure (QURE) Reflect Its Gene Therapy Prospects?
uniQure
2026-02-28
gene therapy
If you are wondering whether uniQure’s current share price reflects its true worth, this breakdown is designed to help you weigh what you are paying against what you are getting. The stock closed at US$15.63, with returns of a 33.3% decline over 7 days, a 32.1% decline over 30 days, a 33.2% decline year to date, but an 18.9% gain over the past year, which may catch the eye of investors thinking about both risk and recovery potential. Recent news around uniQure has focused on its position in...
Ultra-Sensitive CAR T Cells May Unlock Potential Therapeutic Strategy for Solid Tumors
GenEng News
2026-02-27
car
Researchers have developed a new form of highly sensitive CAR T cells, known as HIT T cells, that aims to overcome one of the biggest barriers in solid tumor immunotherapies, which is the way that solid tumors lack a single, widely shared surface target. The post Ultra-Sensitive CAR T Cells May Unlock Potential Therapeutic Strategy for Solid Tumors appeared first on GEN - Genetic Engineering and Biotechnology News .
ImmunityBio to Provide a Business Update and Review Financial Results for FY 2025 on Tuesday, March 3, 2026
Immunity Bio
2026-02-27
financial results
The post ImmunityBio to Provide a Business Update and Review Financial Results for FY 2025 on Tuesday, March 3, 2026 appeared first on ImmunityBio .
8-K - Inmune Bio, Inc. (0001711754) (Filer)
SEC Filing (8-K)
2026-02-27
inmune bio
Filed: 2026-02-27 AccNo: 0001213900-26-021120 Size: 18 MB Item 7.01: Regulation FD Disclosure Item 9.01: Financial Statements and Exhibits
8-K - CG Oncology, Inc. (0001991792) (Filer)
SEC Filing (8-K)
2026-02-27
cg oncology
Filed: 2026-02-27 AccNo: 0001193125-26-079452 Size: 348 KB Item 2.02: Results of Operations and Financial Condition Item 9.01: Financial Statements and Exhibits
UniQure falls further on Makary comments
Biopharma Dive
2026-02-27
gene therapy
Remarks the FDA commissioner made during a CNBC appearance seemed to stoke investor fears that UniQure's gene therapy for Huntington's won't get approved.
CG Oncology Reports 2025 Year End Financial Results and Provides Business Updates
CG Oncology
2026-02-27
financial results
PIVOT-006 Phase 3 topline data evaluating cretostimogene monotherapy as an adjuvant therapy in intermediate-risk NMIBC expected first half 2026CORE-008 Cohort CX Phase 2 first results of combination cretostimogene with gemcitabine in high-risk (HR) NMIBC expected first half 2026Well-positioned to deliver on key milestones with approximately $903.0 million cash, cash equivalents and marketable securities sufficient to fund operations into the first half of 2029 IRVINE, Calif., Feb. 27, 2026 (GLOB
CG Oncology Reports Strong Cash Position and Upcoming Milestones for Cretostimogene Development in NMIBC Trials
CG Oncology
2026-02-27
bladder cancer
financial results
CG Oncology reported financial results and upcoming data releases for cretostimogene, targeting NMIBC treatments with significant cash reserves.Quiver AI SummaryCG Oncology, Inc. announced its financial results for the fourth quarter and year ended December 31, 2025, highlighting key advancements in their clinical trials for cretostimogene, an investigational immunotherapy for non-muscle invasive bladder cancer (NMIBC). Topline data from the PIVO
CG Oncology details cretostimogene bladder program
CG Oncology
2026-02-27
bla
bladder cancer
CG Oncology outlines late-stage trials and BLA plans for cretostimogene, an intravesical oncolytic immunotherapy targeting high- and intermediate-risk non-muscle invasive bladder cancer.
CG Oncology lines up 2026 NMIBC trial readouts with $903M cash
CG Oncology
2026-02-27
bla
Net loss hit $161.0M in 2025 as CG Oncology lifted cash to $903.0M, funding operations into 1H 2029 and outlining key NMIBC trial and BLA milestones in 2026.
Azitra, Inc. Announces Full Year 2025 Results and Provides Business Updates
PR Newswire
2026-02-27
financial results
BRANFORD, Conn., Feb. 27, 2026 /PRNewswire/ -- Azitra, Inc. ("Azitra") (NYSE American: AZTR), a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today reported financial results for the full year ended December 31, 2025, and...
uniQure N.V. (QURE) Investors: April 13, 2026, Deadline in Securities Fraud Class Action Lawsuit Filed by Kessler Topaz Meltzer & Check, LLP
uniQure
2026-02-27
bla
amt-130
gene therapy
accelerated approval
Did you buy QURE ordinary shares between September 24, 2025, and October 31, 2025? Affected uniQure N.V. Investor Summary Who: uniQure N.V. (NASDAQ: QURE) What: Securities fraud class action lawsuit filed Class Period: September 24, 2025, and October 31, 2025 Deadline to Seek Lead Plaintiff Status: April 13, 2026 Key Lawsuit Allegations: Material misstatements and/or omissions concerning the company’s Huntington’s disease gene therapy drug. Investor Action: Contact Kessler Topaz Meltzer (2) Defendants downplayed the likelihood that, despite purportedly highly successful results from the Pivotal Study, uniQure would have to delay its BLA timeline to perform additional studies to supplement its BLA submission; and (3) as a result, Defendants’ statements about uniQure’s business, operations, and prospects lacked a reasonable basis. Why did uniQure’s Share Price Drop? Investors learned the truth about the company’s prospects and the BLA timeline for AMT-130 on November 3, 2025, when uniQure revealed that “the FDA currently no longer agrees that the data from the Phase I/II studies of AMT-130 in comparison to an external control, as per the prespecified protocols and statistical…
Opus Genetics Announces Initial Clinical Data from Phase 1/2 OPGx-BEST1 Gene Therapy Study at the Macula Society Annual Meeting
opus genetics
2026-02-27
gene therapy
First-in-Human Success for Prenatal Stem Cell Therapy in Spina Bifida
Inside Precision Medicine
2026-02-27
cell therapy
For the first time ever, six children with spina bifida have been treated with a stem cell therapy before birth. The post First-in-Human Success for Prenatal Stem Cell Therapy in Spina Bifida appeared first on Inside Precision Medicine .
After Sarepta’s Annus Horribilis, Elevidys Sales Expected To Continue Downward Spiral
BioSpace
2026-02-26
elevidys
gene therapy
duchenne muscular dystrophy
After a rocky 2025, Sarepta Therapeutics’ executives admit they have work to do to bring patients back into the fold as sales of Duchenne muscular dystrophy gene therapy Elevidys continue to decline.
Neurogene Announces FDA Breakthrough Therapy Designation for NGN-401 Gene Therapy for Rett Syndrome
neurogene
2026-02-26
gene therapy
rett syndrome
Breakthrough Therapy designation granted based on interim NGN-401 Phase 1/2 data demonstrating clinically meaningful, durable and multidomain functional improvements On track to complete dosing in Embolden™ registrational trial of NGN-401 in second quarter of 2026 Plans to present additional
Atara Climbs Amid Report of FDA Inconsistency Leading to Cell Therapy’s Rejection
BioSpace
2026-02-26
ebvallo
cell therapy
The rejection of Atara Biotherapeutics’ Ebvallo in January was a “complete reversal” of the conesensus FDA reviewers had come to, according to a former agency employee, who said manufacturing problems were the only approvability barrier for the drug.
A2 Biotherapeutics Doses First Patient in EVEREST-2 Study with A2B543, a Logic-Gated CAR T Cell Therapy Enhanced with a Membrane-Tethered IL-12 Booster
A2 Bio
2026-02-26
car
cell therapy
The post A2 Biotherapeutics Doses First Patient in EVEREST-2 Study with A2B543, a Logic-Gated CAR T Cell Therapy Enhanced with a Membrane-Tethered IL-12 Booster appeared first on A2 Biotherapeutics .
Ryoncil Profits Underpinning Substantial Growth Pipeline
Mesoblast
2026-02-26
ryoncil
Corporate Presentation and Half Year Financial Results
Mesoblast
2026-02-26
financial results
Pfizer Advances Beam’s Gene Editor After Pulling Hemophilia Gene Therapy
BioSpace
2026-02-26
gene therapy
Pfizer will be responsible for all global clinical and regulatory activities for Beam Therapeutics’ liver-directed gene editor.
Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2025 Financial Results and Highlights Recent Progress
Rocket Pharmaceuticals
2026-02-26
pdufa
kresladi
financial results
Pivotal Phase 2 trial of RP-A501 for Danon disease to resume in 1H 2026 KRESLADI ™ for severe LAD-I on track for March 28, 2026 PDUFA date Dosing of first patient in Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy anticipated in mid-2026 Cash, cash equivalents and investments of
ENGN: Detalimogene shows 62% six-month CR rate in NMIBC, with launch targeted for 2027
enGene Google
2026-02-26
bla
gene therapy
detalimogene voraplasmid
Detalimogene voraplasmid is advancing as a non-viral gene therapy for NMIBC, showing strong efficacy and tolerability in pivotal trials, with a six-month CR rate of 62%. The product offers significant advantages in ease of use, manufacturing, and cost, with a BLA filing planned for late 2024 and po…
Boehringer drug approved in 44 days; BioMarin shelves Roctavian
BioMarin Pharmaceutical
2026-02-26
roctavian
gene therapy
Boehringer’s lung cancer medicine Hernexeos is the second “national priority” voucher winner to gain a speedy approval. Elsewhere, a landmark gene therapy was withdrawn and a Pfizer deal paid dividends.
Lineage Cell Therapeutics to Report Fourth Quarter and Full Year 2025 Financial Results and Provide Business Update on March 5, 2026
lineage cell therapeutics
2026-02-26
allogeneic
cell therapies
financial results
CARLSBAD, Calif. --(BUSINESS WIRE)--Feb. 26, 2026-- Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic, or “off the shelf”, cell therapies for serious medical conditions, today announced that it will report its fourth quarter
Fate Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results and Business Updates
Fate Therapeutics
2026-02-26
car
cell therapy
financial results
Outpatient treatment enabled under FT819-102 autoimmune basket protocol with patients now treated with FT819 off-the-shelf CAR T-cell therapy as same-day hospital discharge Four systemic sclerosis patients and the first idiopathic inflammatory myopathy patient treated with FT819; First systemic
GEMMABio Announces First Patient Dosed in CHARISMA Trial of GB221, Investigational Next‑Generation Gene Therapy for Spinal Muscular Atrophy Type 1 (SMA1)
PR Newswire
2026-02-26
gene therapy
spinal muscular atrophy
U.S. FDA grants GB221 rare pediatric disease designation PHILADELPHIA, Feb. 26, 2026 /PRNewswire/ -- Gemma Biotherapeutics ("GEMMABio"), a clinical‑stage global genetic medicines company, announced today that the first patient has been dosed in the Phase 1/2 CHARISMA clinical trial of...
UniQure falls after FDA commissioner’s comments on rare disease therapy
uniQure
2026-02-26
amt-130
gene therapy
accelerated approval
uniQure (QURE) shares fell ~28% on Thursday after FDA Commissioner Marty Makary, in a potential reference to Danish biotech’s gene therapy AMT-130, suggested his agency will not approve drugs that are associated with patient morbidity. His comments come at a time when uniQure (QURE) is seeking FDA accelerated approval for AMT-130, which is designed...
Intellia Therapeutics Announces Fourth Quarter and Full-Year 2025 Financial Results and Business Updates
Intellia Therapeutics Inc.
2026-02-26
bla
financial results
HAELO Phase 3 clinical data for lonvo-z in HAE expected by mid-2026; BLA submission in second half of 2026; anticipated U.S. launch in first half of 2027 Process underway to reactivate global sites for MAGNITUDE-2 Phase 3 clinical trial of nex-z in ATTRv-PN; enrollment completion expected in second
Boehringer drug approved in 44 days; BioMarin shelves Roctavian
Biopharma Dive
2026-02-26
roctavian
gene therapy
Boehringer’s lung cancer medicine Hernexeos is the second “national priority” voucher winner to gain a speedy approval. Elsewhere, a landmark gene therapy was withdrawn and a Pfizer deal paid dividends.
Sarepta Therapeutics to Present New Long-Term and Safety Data Across Gene Therapy and Exon-Skipping Programs at 2026 Muscular Dystrophy Association Clinical & Scientific Congress
Sarepta Therapeutics
2026-02-26
gene therapy
Several abstracts, including a late-breaking podium presentation and posters, bring forward accumulating long-term efficacy, safety and caregiver-reported insights that deepen understanding of dystrophin restoration and its impact in Duchenne CAMBRIDGE, Mass. --(BUSINESS WIRE)--Feb.
UniQure stock tumbles on FDA comments about gene therapy trial
uniQure
2026-02-26
gene therapy
UniQure stock tumbles on FDA comments about gene therapy trial
uniQure's AMT-130: Assessing the FDA Hurdle and What's Priced In
uniQure
2026-02-26
amt-130
The market's reaction to recent FDA comments has been severe and swift. UniQure's stock fell ~28% on Thursday after FDA Commissioner Marty Makary appeared t | Bitget crypto news!
CG Oncology, Inc. unveils five cretostimogene research posters at ASCO GU in San Francisco
CG Oncology
2026-02-26
bladder cancer
CG Oncology, Inc. reveals five cretostimogene posters at ASCO GU in San Francisco, highlighting advances in bladder cancer research.
Opinion: Balancing Safety Access in Rare Disease—Lessons From Sarepta
BioSpace
2026-02-25
elevidys
Regulators overseeing rare disease treatments need better tools to weigh competing risks in real time. Sarepta Therapeutics’ Elevidys is a prime example of why.
Celldex Completes Enrollment in Global Phase 3 Studies (EMBARQ-CSU1 and EMBARQ-CSU2) of Barzolvolimab in Chronic Spontaneous UrticariaDownload PDF
Celldex Therapeutics
2026-02-25
bla
Enrollment completed six months ahead of guidance, driven by significant unmet need for better treatments in CSU Topline data expected Q4 2026 BLA submission planned for 2027 HAMPTON, N.J., Feb. 25, 2026 (GLOBE NEWSWIRE) -- Celldex (NASDAQ:CLDX) announced today the completion of enrollment in the Download PDF
Gilead’s Arcellx Deal And HIV Data Reframe Growth And Valuation Story
Gilead Sciences
2026-02-25
car
cell therapy
cell therapies
Gilead Sciences (NasdaqGS:GILD) has agreed to acquire Arcellx in a $7.8b deal, expanding its presence in cell therapies for blood cancers. The acquisition adds a near-approval CAR T cell therapy candidate to Gilead’s oncology portfolio. Separately, Gilead reported new Phase 3 data for an investigational once daily, single tablet HIV regimen, showing noninferior efficacy and high tolerability versus existing treatments. For investors watching Gilead Sciences at a share price of $147.12,...
Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2025 Financial Results and Recent Corporate Developments
Sarepta Therapeutics
2026-02-25
elevidys
financial results
Net product revenues for the full year 2025 totaled $1,864.3 million, consisting of $965.6 million of PMO net product revenue and $898.7 million of ELEVIDYS net product revenue Net product revenues for the fourth quarter 2025 totaled $369.6 million, consisting of $259.2 million of PMO net product
Sarepta CEO Doug Ingram to step down
Endpoints News
2026-02-25
duchenne muscular dystrophy
Sarepta CEO Doug Ingram is stepping down from his role by the end of the year, ending a brash tenure in which he brought several Duchenne muscular dystrophy products to market and later presided over ...
Immuno-gene therapy for brain cancer tumors now on FDA fast track
Rare Cancer News
2026-02-25
gene therapy
An “immuno-gene” therapy that turns a virus into a cancer-fighting agent has received a fast-track designation from the U.S. Food and Drug Administration (FDA). The experimental treatment, known as SRN-101, is being developed for recurrent high-grade glioma, an aggressive form of brain cancer that has returned after initial treatment and currently has few effective options. […] The post Immuno-gene therapy for brain cancer tumors now on FDA fast track appeared first on Rare Cancer News .
MIMEDX Announces Record Fourth Quarter and Full Year 2025 Operating and Financial Results
mimedx
2026-02-25
financial results
Net Sales Grew 27% Year-Over-Year for the Fourth Quarter and 20% for the Full Year Fourth Quarter GAAP Net Income and Earnings Per Share were $15 million and $0.10, Respectively Fourth Quarter Adjusted EBITDA 1 was $29 Million, or 25% of Net Sales Company Estimates 2026 Net Sales in a Range of
Immuno-Oncology Investments Accelerate as Pipeline Catalysts Multiply in 2026
PR Newswire
2026-02-25
car
cell therapy
Issued on behalf of Oncolytics Biotech Inc. VANCOUVER, BC, Feb. 25, 2026 /PRNewswire/ -- Equity Insider News Commentary, The immuno-oncology market is forecast to grow from $65.22 billion in 2025 to $170.19 billion by 2032, fueled by checkpoint inhibitors, CAR-T cell therapy, and cancer...
Celldex Reports Fourth Quarter and Year End 2025 Financial Results and Provides Corporate UpdateDownload PDF
Celldex Therapeutics
2026-02-25
bla
financial results
Enrollment completed in both Phase 3 chronic spontaneous urticaria global registration studies (EMBARQ-CSU 1 and 2) six months ahead of guidance; Topline data expected in Q4 26; BLA submission planned for 2027 Phase 3 cold urticaria and symptomatic dermographism study (EMBARQ-ColdU and -SD) Download PDF
REGENXBIO to Host Conference Call on March 5 to Discuss Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights
REGENXBIO
2026-02-25
financial results
ROCKVILLE, Md., Feb. 25, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Thursday, March 5, at 8:00 a.m. ET to discuss its financial results for the fourth quarter and full year ended December 31, 2025, and operational highlights.
Penumbra, Inc. Reports Fourth Quarter and Full Year 2025 Financial Results
PR Newswire
2026-02-25
financial results
ALAMEDA, Calif., Feb. 25, 2026 /PRNewswire/ -- Penumbra, Inc. (NYSE: PEN), the world's leading thrombectomy company, today reported financial results for the fourth quarter and full year ended December 31, 2025. Financial Highlights: Revenue of $385.4 million for the fourth quarter of...
Harbour Cofounds Cancer Startup, Hands Off Clinical-Stage Antibody
BioSpace
2026-02-25
melanoma
Solstice Oncology will gain an exclusive worldwide license to Harbour BioMed’s porustobrt, an anti-CTLA-4 antibody currently being studied for melanoma, colorectal cancer and other malignancies in China.
Mahzi Therapeutics Announces First Patient Dosed in Phase 1/2 UNITE Study of MZ-1866 for Pitt Hopkins Syndrome
PR Newswire
2026-02-25
gene therapy
First Investigational Gene Therapy for Pitt Hopkins Syndrome Intended to Address Underlying Disease Biology SOUTH SAN FRANCISCO, Calif., Feb. 25, 2026 /PRNewswire/ -- Mahzi Therapeutics Inc., a clinical-stage biotechnology company developing precision therapies for neurogenetic disorders,...
Lilly Bests Novo Again, Rare Disease Week Goes Regulatory, More CDC Leadership Upheaval
BioSpace
2026-02-25
car
Eli Lilly notches another win over Novo Nordisk, as Zepbound bests CagriSema in a head-to-head trial sponsored by Novo; The FDA kicked off Rare Disease Week, providing draft guidance on its new plausible mechanism pathway, while a bipartisan senate hearing on Thursday will focus on the authorization process for rare conditions; Another leadership change shakes up CDC; and Gilead acquires CAR T partner Arcellx for nearly $8 billion.
Patient dies in MacroGenics' cancer study, FDA puts trial on hold
Endpoints News
2026-02-24
clinical hold
The FDA has placed a partial clinical hold on MacroGenics’ Phase 2 study in gynecologic cancers after one patient died and three others experienced life-threatening side effects, the company disclosed Tuesday. The patient who died ...
IN8bio to Present at TD Cowen 46th Annual Health Care Conference
In8Bio
2026-02-24
cell therapies
NEW YORK, Feb. 24, 2026 (GLOBE NEWSWIRE) -- IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta (γδ) T cell therapies for cancer and autoimmune diseases, today announced that William Ho, CEO and co-founder, will be presenting at the TD Cowen 46
Intellia Therapeutics to Participate in Upcoming Investor Conferences
Intellia Therapeutics Inc.
2026-02-24
crispr
gene editing
CAMBRIDGE, Mass., Feb. 24, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that management will be participating in the upcoming investor conferences:
Gilead adds to cell therapy portfolio with $8bn acquisition of Arcellx
Gilead Sciences
2026-02-24
car
cell therapy
anitocabtagene autoleucel
Deal builds on the companies’ 2022 collaboration over CAR T-cell therapy anitocabtagene autoleucel (anito-cel).
Beam Designs New Approach for Direct Correction of Mutations Causing PKU
GenEng News
2026-02-24
base editing
lipid nanoparticle
Beam’s liver-targeted genetic disease program, BEAM-304, leverages Beam’s proprietary and clinically validated base editing technology and lipid nanoparticle delivery capabilities to correct mutations in the phenylalanine hydroxylase gene that cause PKU. The post Beam Designs New Approach for Direct Correction of Mutations Causing PKU appeared first on GEN - Genetic Engineering and Biotechnology News .
Iovance Announces Positive Results from the First Clinical Trial for TIL Cell Therapy in Soft Tissue Sarcomas
Iovance Biotherapeutics
2026-02-24
cell therapy
50% Objective Response Rate (ORR) in Advanced Sarcomas Significant Market Opportunity with More than 8,000 Patients Diagnosed Annually in the U.S. and Europe SAN CARLOS, Calif., Feb. 24, 2026 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company
BMRN Beats on Q4 Earnings, to Withdraw Roctavian From Market
BioMarin Pharmaceutical
2026-02-24
roctavian
gene therapy
BioMarin beats Q4 earnings estimates as Voxzogo drives year-over-year revenue growth. The company decides to voluntarily withdraw its hemophilia gene therapy.
Axogen, Inc. Reports Fourth Quarter and Full-Year 2025 Financial Results
axogen
2026-02-24
financial results
Gilead's $7.8 Billion Bet on Arcellx: A New Era for Multiple Myeloma Therapy Begins
Gilead Sciences
2026-02-24
multiple myeloma
Gilead Sciences is set to acquire Arcellx for up to $7.8B, gaining full control of their promising multiple myeloma therapy, anito-cel.With FDA review underway, Gilead aims to expand its oncology pipeline. #CancerResearch
BioMarin fails to find buyer, pulls Roctavian gene therapy off market
Endpoints News
2026-02-24
roctavian
gene therapy
BioMarin's previous CEO wanted to offer money-back guarantees to insurers for covering its hemophilia gene therapy Roctavian. Investors in the company may wish that such a promise on performance could have ...
FDA reviewing new iberdomide combo for hard-to-treat multiple myeloma
Rare Cancer News
2026-02-24
multiple myeloma
The U.S. Food and Drug Administration (FDA) is reviewing an application from Bristol Myers Squibb (BMS) seeking approval of its investigational oral therapy iberdomide for the treatment of people with hard-to-manage multiple myeloma. The application seeks approval of iberdomide in combination with two standard myeloma therapies, Darzalex (daratumumab) and dexamethasone, for people with relapsed or […] The post FDA reviewing new iberdomide combo for hard-to-treat multiple myeloma appeared first on Rare Cancer News .
Gilead Sciences Acquiring Arcellx to Consolidate Anito-Cel Ownership, UBS Securities Says
Gilead Sciences
2026-02-24
anitocabtagene autoleucel
Gilead Sciences' planned acquisition of Arcellx is its way to consolidate ownership of anitocabtagene autoleucel, or anito-cel, UBS Securities said in a Monday note. Gilead said Monday that it...
BioMarin pulls hemophilia gene therapy Roctavian, taking $240M hit after divestiture efforts flounder
BioMarin Pharmaceutical
2026-02-24
roctavian
gene therapy
hemophilia a
Just three months after further scaling back its support for the struggling hemophilia A gene therapy Roctavian, the company is walking away altogether by pulling the treatment from the market.&nbs | Just three months after further scaling back its support for the struggling hemophilia A gene therapy Roctavian, the company is walking away altogether by pulling the treatment from the market.
FDA Grants Accelerated Approval to Loargys for Arginase 1 Deficiency
Hematology Advisor News
2026-02-24
accelerated approval
Pegzilarginase is an enzyme replacement therapy that provides an exogenous source of the deficient human arginase 1 enzyme to reduce blood arginine levels.
Immedica wins accelerated approval for once-rejected rare disease drug
Endpoints News
2026-02-24
accelerated approval
Swedish biotech Immedica won an accelerated approval from the FDA, resurrecting the first treatment to address persistently elevated levels of plasma arginine, which is the primary driver of the rare disease ARG1-D. Monday's approval of ...
8-K - Kyverna Therapeutics, Inc. (0001994702) (Filer)
SEC Filing (8-K)
2026-02-24
kyverna therapeutics
Filed: 2026-02-24 AccNo: 0001193125-26-065320 Size: 196 KB Item 5.02: Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers: Compensatory Arrangements of Certain Officers Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
Beam Therapeutics' new PKU program; Protara's bladder cancer data
Endpoints News
2026-02-24
gene editing
bladder cancer
Plus, news about Bristol Myers Squibb, Catalent, Angelini Pharma, Quiver Bioscience and Brainomix: 🥼 Beam Therapeutics' gene editing plans: The company said it will develop multiple gene editing therapies tailored to ...
Real-World Austrian Data Show Limits of Chemotherapy in High-Risk DLBCL
Hematology Advisor News
2026-02-24
car
cell therapy
Patients meeting eligibility criteria for CAR T-cell therapy experienced extremely poor survival with historical standard-of-care regimens.
BMRN Beats on Q4 Earnings, to Withdraw Roctavian From Market
BioMarin Pharmaceutical
2026-02-24
roctavian
BioMarin Pharmaceutical BMRN reported fourth-quarter 2025 adjusted earnings per share of 46 cents, beating the Zacks Consensus Estimate of 25 cents. However, earnings declined 50% year over year, largely due to a $119.2 million inventory write-off charge tied to the company’s decision to voluntaril…
Drug-Controlled CAR T Cells May Enable Safer Immunotherapy
GenEng News
2026-02-24
car
Researchers developed a CAR T technology, called DROP-CAR, that incorporates human protein components, and uses a clinically approved, non-immunosuppressive drug to remotely and reversibly switch off the CAR T cells by disrupting tumor cell binding. The post Drug-Controlled CAR T Cells May Enable Safer Immunotherapy appeared first on GEN - Genetic Engineering and Biotechnology News .
Beam Therapeutics Announces $500 Million Strategic Financing Facility with Sixth Street
beam therapeutics
2026-02-24
sickle cell disease
$100 Million Funded at Close with up to an Additional $400 Million Available Under Facility with Seven-Year Term Financing Bolsters Balance Sheet with Long-term, Non-dilutive Capital to Support Anticipated Launch of Risto-cel in Sickle Cell Disease (SCD) CAMBRIGE, Mass., Feb.
Gilead’s Arcellx Deal Deepens Oncology Focus And Raises Valuation Questions
Gilead Sciences
2026-02-24
car
cell therapy
multiple myeloma
Gilead Sciences (NasdaqGS:GILD) has agreed to acquire cancer therapy developer Arcellx in a deal valued at US$7.8b. The transaction will give Gilead full control of anito cel, a late stage CAR T cell therapy for multiple myeloma. An FDA approval decision for anito cel is expected later this year. This is Gilead's largest acquisition since 2020 and expands its presence in oncology and cell therapy. Gilead Sciences is best known for its antiviral and HIV treatments. In recent years it has...
Nanoscope Therapeutics Post-Hoc Clinical Analyses to be Presented at 2026 Macula Society Annual Meeting
Nanoscope Therapeutics
2026-02-24
mco-010
gene therapy
retinitis pigmentosa
Dr. Christine N. Kay to Present Multivariant Analysis of 3-Year RESTORE and REMAIN Data, Identifying Patient and Disease Characteristics Associated with Best Outcomes in Retinitis Pigmentosa Patients Following MCO-010 Optogenetic Gene Therapy DALLAS, Feb. 24, 2026 — Nanoscope Therapeutics, Inc., a biotechnology company committed to developing and commercializing novel, disease-agnostic therapies for patients with photoreceptor loss and […] The post Nanoscope Therapeutics Post-Hoc Clinical Analyses to be Presented at 2026 Macula Society Annual Meeting appeared first on Nanoscope Therapeutics .
Beam Therapeutics Reports Fourth Quarter and Year-End 2025 Financial Results and Announces New Liver-Targeted Genetic Disease Program in Phenylketonuria (PKU)
beam therapeutics
2026-02-24
phenylketonuria
financial results
alpha-1 antitrypsin deficiency
New Program Designed as Platform-based Approach for Direct Correction of Mutations Causing PKU; Investigational New Drug (IND) Filing for BEAM‑304 Anticipated in 2026 Updated Phase 1/2 Data and Next Steps for Pivotal Development for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) on Track for Q1
Sarepta Therapeutics Announces Commercial Launch of ELEVIDYS in Japan
Sarepta Therapeutics
2026-02-24
elevidys
gene therapy
duchenne muscular dystrophy
Japan now offers ELEVIDYS gene therapy to children with Duchenne muscular dystrophy aged 3 years to less than 8 years - Company is eligible to receive a $40 million milestone payment upon first commercial sale in Japan CAMBRIDGE, Mass. --(BUSINESS WIRE)--Feb. 24, 2026-- Sarepta Therapeutics, Inc.
Krystal Biotech RMAT Win Shifts Focus To Broader Gene Therapy Pipeline
Krystal Biotech
2026-02-24
gene therapy
Krystal Biotech received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for KB707 in advanced non small cell lung cancer. The company reported progress across its gene therapy pipeline, including pivotal ophthalmology trials with data expected next year. These updates extend Krystal Biotech’s focus beyond rare skin diseases into larger indications such as lung cancer. For investors following Krystal Biotech, ticker NasdaqGS:KRYS, the RMAT news comes with the shares at $274.59...
Iovance Biotherapeutics Highlights Strong Fourth Quarter and Full Year 2025 Results, Business Achievements and Corporate Updates
Iovance Biotherapeutics
2026-02-24
amtagvi
lifileucel
30% Quarterly Revenue Growth Driven by Amtagvi Demand Gross Margin Increased to 50% FY25 Revenue of $264M Achieved Annual Guidance U.S. FDA Fast Track Designation Granted for Lifileucel in Second-Line Advanced Non-Small Cell Lung Cancer SAN CARLOS, Calif., Feb.
8-K - IOVANCE BIOTHERAPEUTICS, INC. (0001425205) (Filer)
SEC Filing (8-K)
2026-02-24
iovance biotherapeutics
Filed: 2026-02-24 AccNo: 0001104659-26-018837 Size: 9 MB Item 2.02: Results of Operations and Financial Condition Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
Gilead to acquire cell therapy partner Arcellx for $7.8B
Endpoints News
2026-02-23
car
cell therapy
multiple myeloma
Gilead Sciences is buying its CAR-T collaborator Arcellx as its closely-watched multiple myeloma cell therapy nears potential approval and launch. The deal is worth $7.8 billion, when calculated from $115 per ...
Gilead Doubles Down on CAR T With $7.8B Arcellx Acquisition
Gilead Sciences
2026-02-23
car
multiple myeloma
The centerpiece of the takeover is anito-cel, a CAR T therapy under development for relapsed or refractory multiple myeloma. An FDA decision on the therapy is expected by December 2026.
Gilead to buy Arcellx in $7.8B wager on multiple myeloma cell therapy
Gilead Sciences
2026-02-23
cell therapy
multiple myeloma
The acquisition bolsters Gilead’s sputtering cell therapy business with a medication the company expects to become a “foundational treatment” for the blood cancer, its CEO said.
US Gilead Sciences acquires cancer drug developer Arcellx for $7.8B
Gilead Sciences
2026-02-23
cell therapy
Data shows biopharmaceutical giant aims to maximize the long-term potential of cell therapy 'anito-cel' - Anadolu Ajansı
Gilead to Acquire Arcellx for $7.8B, Adding Anito-Cel to Cancer Pipeline
GenEng News
2026-02-23
car
bcma
cell therapy
multiple myeloma
chimeric antigen receptor
Gilead Sciences has agreed to acquire Arcellx for $7.8 billion, the companies said today, in a deal intended to give the buyer full control of the buyout target’s lead candidate, a BCMA-directed chimeric antigen receptor (CAR) T-cell therapy for multiple myeloma that Arcellx and Kite, a Gilead Company, have spent the past 3+ years partnering to co-develop and co-commercialize. The post Gilead to Acquire Arcellx for $7.8B, Adding Anito-Cel to Cancer Pipeline appeared first on GEN - Genetic Engineering and Biotechnology News .
U.S. FDA has granted accelerated approval of Loargys® (pegzilarginase-nbln) for the treatment of hyperargininemia in patients 2 years and older with Arginase 1 Deficiency (ARG1-D)
PR Newswire
2026-02-23
accelerated approval
LOARGYS is the first and only therapy proven to lower arginine in patients 2 years of age and older living with ARG1-D ARG1-D is an ultra-rare, debilitating, and progressive metabolic disease resulting in persistent elevation of plasma arginine, also known as hyperargininemia FDA...
BREAKING: Gilead Sciences to Acquire Arcellx a Staggering $7.8 Billion CAR-T Cell Therapy Deal
Gilead Sciences
2026-02-23
car
bcma
cell therapy
multiple myeloma
anitocabtagene autoleucel
Today, February 23rd, 2026, Gilead Sciences announced a definitive agreement to acquire Arcellx in a transaction valued at approximately $7.8 billion, providing Gilead with full ownership of anitocabtagene autoleucel (anito-cel), an investigational BCMA-directed CAR-T therapy for patients with relapsed or refractory multiple myeloma. Arcellx is a clinical-stage biotech company headquartered in Rockville, Maryland, founded in […]
Gilead to buy Arcellx in nearly $8B deal
Gilead Sciences
2026-02-23
car
multiple myeloma
Gilead Sciences is buying Arcellx in a deal worth $7.8 billion, picking up its partner on a CAR-T therapy for multiple myeloma.
BioMarin Q4 2025 slides: 17% revenue growth amid ROCTAVIAN charges
BioMarin Pharmaceutical
2026-02-23
roctavian
BioMarin Q4 2025 slides: 17% revenue growth amid ROCTAVIAN charges
8-K - Atara Biotherapeutics, Inc. (0001604464) (Filer)
SEC Filing (8-K)
2026-02-23
atara biotherapeutics
Filed: 2026-02-23 AccNo: 0001193125-26-062534 Size: 283 KB Item 1.01: Entry into a Material Definitive Agreement Item 3.02: Unregistered Sales of Equity Securities Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
SEC Filing (8-K)
2026-02-23
ultragenyx
Filed: 2026-02-23 AccNo: 0001193125-26-062568 Size: 153 KB Item 8.01: Other Events
I have multiple myeloma, but I don’t have an expiration date
Rare Cancer News
2026-02-23
multiple myeloma
The entrance to my local supermarket becomes a danger zone at this time of year. The danger is deceptive: girls dressed in brown and green, selling seriously addictive products. Yes, it’s Girl Scout Cookies season in my part of the U.S. If you don’t think Girl Scout Cookies are addictive, then you’ve never had a […] The post I have multiple myeloma, but I don’t have an expiration date appeared first on Rare Cancer News .
First test of gene therapy for rare form of autism is underway
Endpoints News
2026-02-23
gene therapy
Two years ago, a small biotech startup got permission to test a gene therapy for a rare and disabling form of autism. The plan: Deliver a gene that’s vital for building and maintaining connections between ...
Neurogene Announces First Participant Dosed in Embolden™ Registrational Trial of NGN-401 Gene Therapy for Rett Syndrome
neurogene
2026-02-23
bla
phase 1
phase 1/2
gene therapy
rett syndrome
Single trial to support future BLA submission for NGN-401 in patients ages ≥ 3 years Rapid trial execution underway with 12 of 13 clinical sites initiated Expect to complete trial enrollment in three to six months Neurogene management to present Phase 1/2 interim data update at Stifel Healthcare
Gilead Doubles Down on CAR T With $7.8B Arcellx Acquisition
BioSpace
2026-02-23
car
multiple myeloma
The centerpiece of the takeover is anito-cel, a CAR T therapy under development for relapsed or refractory multiple myeloma. An FDA decision on the therapy is expected by December 2026.
Sickle Cell Gene Therapies Casgevy and Lyfgenia Still Lacking Traction 2 Years In
BioSpace
2026-02-23
casgevy
lyfgenia
gene therapies
sickle cell disease
The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
Gilead Sciences Is Acquiring Arcellx and Partnered Multiple Myeloma Drug in $7.8B Deal
Gilead Sciences
2026-02-23
arcellx
carvykti
cell therapy
legend biotech
multiple myeloma
Gilead Sciences and Arcellx have been collaborating on the multiple myeloma cell therapy anito-cel since 2022. This therapy was designed to provide better safety, which would be an advantage over Carvykti, the blockbuster multiple myeloma product marketed by Johnson & Johnson and Legend Biotech.
Anixa Biosciences Announces Dr. Jose Conejo-Garcia will Keynote the South Carolina Clinical & Translational Research Institute 2026 Retreat
anixa biosciences
2026-02-23
car
cell therapy
cell therapies
Dr. Conejo-Garcia is the co-inventor of Anixa's FSHR-mediated CAR-T technology SAN JOSE, Calif. , Feb. 23, 2026 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that Jose Conejo-Garcia, M.D., Ph.D., Professor of Immunology in the Department of Integrative Immunobiology at the Duke University School of Medicine and the co-inventor of Anixa's CAR-T technology, will be a keynote speaker at the South Carolina Clinical & Translational Research (SCTR) Institute 2026 Retreat: Cell-Based Therapies from Discovery to Translation, on Friday, March 6, 2026. Dr. Conejo-Garcia's keynote presentation will include a discussion of Anixa's ongoing Phase 1 clinical trial of liraltagene autoleucel, or lira-cel, which utilizes the FSHR-mediated CAR-T therapy first discovered by Dr. Conejo-Garcia and his team at The Wistar Institute. The SCTR 2026 Retreat will bring together pioneers in research, clinical care, and industry to showcase breakthrough innovations in advanced cell therapy, immune therapy, oncology, and cellular product manufacturing, and engage in empowering…
Roche to stop work on bone health treatment for Duchenne
Endpoints News
2026-02-23
duchenne muscular dystrophy
Roche is no longer recruiting patients for a Phase 2 trial studying its immune disease drug Enspryng in Duchenne muscular dystrophy, and will end work developing the treatment for bone health in those patients. ...
Beam Therapeutics to Host Investor Webcast for Fourth Quarter and Year-End 2025 Financial Results and New Liver-targeted Genetic Disease Program
beam therapeutics
2026-02-23
base editing
financial results
CAMBRIDGE, Mass., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will announce its fourth quarter and full year 2025 financial results and a new
uniQure N.V. to Report Q4 and Full Year 2025 Financial Results on March 2, 2026
uniQure
2026-02-23
gene therapy
financial results
uniQure will announce its 2025 financial results on March 2, 2026, followed by a conference call.Quiver AI SummaryuniQure N.V., a gene therapy company, will release its financial results for the fourth quarter and full year of 2025 on March 2, 2026, prior to market opening. Following the release, management will hold a conference call at 8:00 a.m. ET, which will be available via webcast on the company's website. Analysts can participate in the Q&
Ultragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2026-02-23
aav
bla
dtx401
gene therapy
glycogen storage disease
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Gilead to acquire Arcellx in deal worth $7.8bn
PMLive Healthcare
2026-02-23
multiple myeloma
The US FDA has accepted a licence application for drug for relapsed/refractory multiple myeloma
Gilead inks $7.8B Arcellx buyout to steer CAR-T into J&J showdown
Gilead Sciences
2026-02-23
car
cell therapy
Gilead Sciences has struck a de | Gilead has struck a deal to buy Arcellx for $7.8 billion to take full control of a CAR-T cell therapy that is on the cusp of approval.
Evaluating uniQure (QURE) After New Class Action Allegations Over AMT-130 FDA Disclosures
uniQure
2026-02-23
amt-130
gene therapy
Why the new class action matters for uniQure investors Kessler Topaz Meltzer & Check has filed a securities fraud class action against uniQure (NasdaqGS:QURE), alleging misleading statements about FDA alignment and timelines for AMT-130, its Huntington’s disease gene therapy program. See our latest analysis for uniQure. uniQure’s latest share price of US$23.43 comes after a 7 day share price return of 14.29%. However, the 90 day share price return of a 15.66% decline and 5 year total...
uniQure to Announce 2025 Financial Results
uniQure
2026-02-23
gene therapy
financial results
uniQure to host earnings call on Monday, March 2, 2026 at 8:00 a.m. ET ~LEXINGTON, Mass. and AMSTERDAM, Feb. 23, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report fourth quarter and full year of 2025 financial results before market open on Monday, March 2, 2026. Management will then host a conference call at 8:00 a.m. ET. The event will be webcast under the Events & Presenta
Gilead to acquire cancer therapy developer Arcellx for up to $7.8 billion
Gilead Sciences
2026-02-23
cell therapy
Gilead Sciences said on Monday it will buy cancer therapy partner Arcellx for an implied equity value of $7.8 billion, expanding their cell therapy development collaboration that started in 2022.
Gilead Sciences to Acquire Arcellx to Maximize Long-Term Potential of Anito-cel (1)
Gilead Sciences
2026-02-23
bla
multiple myeloma
– Builds on Successful 2022 Collaboration on Anito-cel, a Potentially Transformative Treatment for Patients with Multiple Myeloma –– FDA Accepted Anito-cel BLA for the Treatment of Adult Patients with Relapsed/Refractory Multiple Myeloma –– Provides Gilead with Full Control of Anito-cel, Accelera...
Intellia Therapeutics to Hold Conference Call to Discuss Second Quarter 2025 Earnings and Company Updates
Intellia Therapeutics Inc.
2026-02-23
crispr
gene editing
CAMBRIDGE, Mass. , July 31, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, will present its second quarter 2025 financial results and operational highlights in a
REGENXBIO Secures Legal Win in Gene Therapy Patent Battle vs. Sarepta
BioSpace
2026-02-23
aav
gene therapy
A lower court had previously ruled for Sarepta in the companies’ long-running dispute, finding that REGENXBIO’s AAV patent was invalid because its elements were naturally occurring. The appeals court on Friday said that this original decision adopts a “narrow” view of the invention.
Assessing CG Oncology (CGON) Valuation After Strong Recent Share Price Momentum
CG Oncology
2026-02-23
bladder cancer
CG Oncology stock: what recent performance means for investors CG Oncology (CGON) has drawn fresh attention after a strong past 3 months, with the share price up about 32%, adding to solid gains over the past year. For investors, that move puts a spotlight on how the company’s late stage bladder cancer pipeline, current revenue base, and ongoing losses fit together at the current share price. See our latest analysis for CG Oncology. The recent 32.3% 3 month share price return, alongside a...
FDA unveils long-awaited guidance on new pathway for individualized therapies
Endpoints News
2026-02-23
gene therapies
The FDA is spelling out the details of a new pathway to help speed personalized cell and gene therapies to market for rare diseases. Monday's long-awaited draft guidance outlines the agency’s “plausible mechanism” framework, a ...
Gilead buys cell therapy partner Arcellx for up to $7.8bn
PharmaPhorum
2026-02-23
car
cell therapy
cell therapies
multiple myeloma
Gilead has shored up its position in cell therapies with a deal to acquire Arcellx, taking full control of their partnered multiple myeloma CAR-T.
FDA’s Bespoke Pathway To Focus on Gene Editing and RNA-Based Treatments for Rare Diseases
BioSpace
2026-02-23
gene editing
The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay Prasad in November, was criticized for lacking detailed guidance. Agency leaders elucidated on the pathway for personalized medicines on Monday.
Genprex Strengthens Intellectual Property Portfolio with Japanese and EU Patents for Reqorsa® Gene Therapy in Combination with Immunotherapies to Treat Cancer
Genprex
2026-02-23
gene therapy
Japanese Patent Covers Acclaim-3 Clinical Trial Combining REQORSA Gene Therapy with Genentech, Inc.’s Tecentriq® AUSTIN, Texas — (Feb. 23, 2026) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that the Japanese Patent Office has issued a favorable Appeal Decision […]
StockWatch: FDA Reversal Boosts Moderna, But Not Other Vaccine Companies
GenEng News
2026-02-22
accelerated approval
Moderna proposed a revised regulatory pathway based on age, agreeing to seek full approval for adults ages 50–64 and accelerated approval for adults ages 65 and older, with the accelerated approval subject to a post-marketing requirement that Moderna conduct an additional study in older adults. The post StockWatch: FDA Reversal Boosts Moderna, But Not Other Vaccine Companies appeared first on GEN - Genetic Engineering and Biotechnology News .
Biotech Investor Adds $3 Million to Krystal Biotech as Revenue Hits $389 Million in 2025
Krystal Biotech
2026-02-22
gene therapies
Krystal Biotech develops redosable gene therapies targeting rare skin diseases, with a late-stage clinical pipeline and expanding portfolio.
Avoro Capital Advisors LLC Increases Stake in uniQure NV
uniQure
2026-02-21
gene therapy
On December 31, 2025, Avoro Capital Advisors LLC (Trades, Portfolio) executed a significant transaction involving uniQure NV, a gene therapy company. The firm a
Jefferies Raises its Price Target on Krystal Biotech, Inc. (KRYS) to $371 and Maintains a Buy Rating
Krystal Biotech
2026-02-21
vyjuvek
Krystal Biotech, Inc. (NASDAQ:KRYS) is among the 11 Best All-Time High Stocks to Buy According to Wall Street. On February 17, 2026, Jefferies analyst Roger Song raised the price target on Krystal Biotech, Inc. (NASDAQ:KRYS) to $371 from $310 and maintained a Buy rating. Roger Song noted another strong quarter and full year, with Vyjuvek […]
Amicus Therapeutics Announces Full-Year 2025 Financial Results and Corporate Updates
amicus therapeutics
2026-02-20
financial results
Riding the Gene Therapy Rollercoaster Into 2026
Inside Precision Medicine
2026-02-20
aav
gene editing
gene therapy
gene therapies
It has been a tough, if interesting, year for the gene therapy space, with many ups and downs culminating in some optimism and increased funding and investment as 2025 drew to a close. The sector is expanding beyond traditional AAV gene therapies, with new vectors being trialed and the gene editing space expanding, with new and innovative companies. The post Riding the Gene Therapy Rollercoaster Into 2026 appeared first on Inside Precision Medicine .
InventHelp Inventor Develops New Seasoning to Enhance Flavor & Relieve Pain (SGM-586)
PR Newswire
2026-02-20
ind
PITTSBURGH, Feb. 20, 2026 /PRNewswire/ -- "I wanted to create a healthier way to enjoy a delicious blend of flavors and spices when cooking," said an inventor, from Indianapolis¸ Ind., "so I invented the TRIO ALTERNATIVE. My formula would also provide therapeutic benefits to help relieve...
Amicus Therapeutics Announces Full-Year 2025 Financial Results and Corporate Updates
BioMarin Pharmaceutical
2026-02-20
financial results
Amicus Therapeutics, a global biotechnology company focused on developing transformative medicines for rare diseases, has released its financial results for the full year ending December 31, 2025. The company reported significant growth in revenue and a strong cash position, alongside notable corpo…
Mesoblast 2026 Half Year Financial Results Webcast
Mesoblast
2026-02-20
financial results
When Antibodies Become the Drug Factory
Inside Precision Medicine
2026-02-20
gene therapy
Gene therapy is transforming biologic medicines from repeated injections into living, self-sustaining treatments. The post When Antibodies Become the Drug Factory appeared first on Inside Precision Medicine .
Fierce Pharma Asia—Gilead's synthetic lethality deal; A Tokyo biotech IPO; Novo’s Ozempic China sales dip
Gilead Sciences
2026-02-20
cell therapy
Gilead has penned a potential $1.5 billion synthetic lethality deal with a Chinese biotech. A cell therapy company could end a one-year-plus biotech IPO drought in Tokyo. | Gilead penned a potential $1.5 billion synthetic lethality deal with a Chinese biotech. A cell therapy company could end a one-year-plus biotech IPO drought in Tokyo. Novo Nordisk faces increased GLP-1 competition in China. And more.
Mayo Clinic, NIH Chronicity Indices Comparably Predict Kidney Failure Risk in Lupus Nephritis
Hematology Advisor News
2026-02-20
lupus
The NIH-CI and MCCS are independent predictors of renal outcomes in lupus nephritis that provide additional prognostic value than routine clinical data.
PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2025 Financial Results
PR Newswire
2026-02-19
financial results
– Full-year 2025 product and royalty revenue of $831M, exceeding guidance – – Strong Sephience™ (sepiapterin) uptake since 2H 2025 launch with fourth quarter and 2025 revenue of $92M and $111M, respectively – – Cash of $1.95B as of December 31, 2025 – WARREN, N.J., Feb. 19, 2026...
Intellia Therapeutics to Hold Conference Call to Discuss Fourth Quarter and Full-Year 2025 Financial Results and Business Updates
Intellia Therapeutics Inc.
2026-02-19
crispr
gene editing
financial results
CAMBRIDGE, Mass., Feb. 19, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that the company will host a conference call on February 26, 2026, at 8 a.m.
Muscle Invasive Bladder Cancer Market: Pharma Pipeline Fuels Rapid Expansion by 2034 – DelveInsight | CG Oncology, Pfizer, UroGen Pharma, Janssen R&D, Protara Therapeutics, Binhui Biopharmaceutical
CG Oncology
2026-02-19
bladder cancer
The Key Muscle Invasive Bladder Cancer Companies in the market include - CG Oncology, Pfizer, UroGen Pharma, Janssen Research & Development, LLC, Protara Therapeutics, Binhui Biopharmaceutical, SURGE Therapeutics, Pfizer, AstraZeneca, ImmunityBio, In...
Why Analysts See Krystal Biotech (KRYS) Story Shifting After New Data And Target Resets
Krystal Biotech
2026-02-19
vyjuvek
Krystal Biotech’s modeled fair value has shifted from US$273.60 to US$291.22, with the discount rate moving from 7.06% to 7.02% and revenue growth assumptions holding essentially flat at 33.39% versus 33.38%. This reset aligns with recent Street commentary that links a more constructive stance to Vyjuvek traction, progress in the ophthalmology pipeline, and early cystic fibrosis data, while still recognizing that much depends on future clinical and commercial execution. Continue reading to...
Is Krystal Biotech (KRYS) Priced Right After A 61% One-Year Share Gain?
Krystal Biotech
2026-02-19
gene therapy
If you are wondering whether Krystal Biotech's share price reflects its true worth, this article will walk through how the current market price compares with a range of valuation checks. The stock last closed at US$283.29, with returns of 3.9% over 7 days, 14.7% year to date, and 60.8% over the past year. This performance has put it firmly on many investors' radar. Recent interest in Krystal Biotech has been shaped by ongoing attention to its gene therapy pipeline and regulatory milestones...
PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2025 Financial Results
ptc biotherapeutics
2026-02-19
financial results
– Full-year 2025 product and royalty revenue of $831M, exceeding guidance – – Strong Sephience™ (sepiapterin) uptake since 2H 2025 launch with fourth quarter and 2025 revenue of $92M and $111M, respectively – – Cash of $1.95B as of December 31, 2025 – WARREN, N.J., Feb.
J&J Plans $1B Investment in PA Cell Therapy Manufacturing Facility
BioSpace
2026-02-19
cell therapy
cell therapies
Once fully operational, the Pennsylvania site will employ more than 500 people and make cell therapies for thousands of patients a year.
Kyowa Kirin : Transcript of Results Briefing Fiscal 2025
Kyowa Kirin
2026-02-19
financial results
Kyowa Kirin Co., Ltd. Financial Results Briefing for the Fiscal Year 2025 February 10, 2026 Event Summary [Event Name] Financial Results Briefing...
NeuExcell Therapeutics' brain cancer therapy tests debated field
Endpoints News
2026-02-18
gene therapy
A Chinese company says it slowed an aggressive form of cancer in the first human test of a gene therapy technique that aims to coax the brain into healing itself. Shanghai-based NeuExcell Therapeutics reported preliminary ...
ImmunityBio Receives Authorization from the European Commission for ANKTIVA® with BCG for Non-Muscle Invasive Bladder Cancer Carcinoma in Situ, Expanding Global Access to 33 Countries
Immunity Bio
2026-02-18
bladder cancer
The post ImmunityBio Receives Authorization from the European Commission for ANKTIVA® with BCG for Non-Muscle Invasive Bladder Cancer Carcinoma in Situ, Expanding Global Access to 33 Countries appeared first on ImmunityBio .
Updated: Novartis signs macrocyclic deal with Unnatural Products
Endpoints News
2026-02-18
ind
Novartis will pay $100 million in upfront and pre-IND milestone payments to tap into a macrocyclic peptide platform from Unnatural Products, a deal that could lead to new cardiovascular disease medicines. The move allows the ...
Precision BioSciences Announces Presentation at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference
precision biosciences
2026-02-18
gene editing
DURHAM, N.C. --(BUSINESS WIRE)--Feb. 18, 2026-- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that new preclinical study data
EyePoint scopes out new commercial chief to prepare for Duravyu launch
Genentech
2026-02-18
wet age-related macular degeneration
As it looks ahead to the potential launch of its sustained-delivery treatment Duravyu for wet age-related macular degeneration (AMD) and diabetic macular edema (DME), EyePoint Pharmaceuticals has t | As it looks ahead to the potential launch of its sustained-delivery treatment Duravyu for wet age-related macular degeneration and diabetic macular edema, EyePoint Pharmaceuticals has tapped a new commercial leader with experience heading up ophthalmology franchises at Genentech, Novartis and other drugmakers.
CIMG Inc. Reports Fiscal Year 2025 Financial Results
PR Newswire
2026-02-18
financial results
HONG KONG, Feb. 18, 2026 /PRNewswire/ -- CIMG Inc. ("CIMG" or the "Company") (Nasdaq: IMG), a business group specializing in digital health and sales development, which utilizes technologies and marketing networks to enhance its business partners' sales growth and commercial value, today...
Buy Rating on Krystal Biotech Driven by Vyjuvek’s Global Momentum and Upside from Enhanced NK and Corneal Abrasion Pipeline
Krystal Biotech
2026-02-18
vyjuvek
Analyst Yigal Nochomovitz of Citi reiterated a Buy rating on Krystal Biotech, boosting the price target to $371.00. Yigal Nochomovitz has given his Buy rating due t...
J&J pours $1B into new cell therapy site in Pennsylvania
Endpoints News
2026-02-18
cell therapy
Johnson & Johnson said it will spend more than $1 billion to build a cell therapy manufacturing plant in Pennsylvania as part of its broader commitment unveiled last year to invest more ...
Stem Cell Therapy Market to Reach USD 45.2B by 2036 (8.4% CAGR) | U.S., Germany & Japan Lead Growth | Mesenchymal Stem Cells Hold 45% Share -- Future Market Insights
PR Newswire
2026-02-18
mesenchymal
cell therapy
NEWARK, Del., Feb. 18, 2026 /PRNewswire/ -- Future Market Insights (FMI) reports that the global stem cell therapy market was valued at USD 18.7 billion in 2025 and is projected to expand to USD 20.1 billion in 2026 and USD 45.2 billion by 2036, advancing at a CAGR of 8.4% over the...
Affinia Therapeutics Receives European Medicines Agency Orphan Drug Designation for AFTX-201 in BAG3-Associated Dilated Cardiomyopathy (DCM)
Affinia Therapeutics
2026-02-18
aftx-201
BAG3 DCM is a devastating genetic heart disease caused by a mutation in the BAG3 gene and resulting in loss of heart function at a young age; despite current standard of care, almost 25% of patients require a heart transplant AFTX-201 addresses the genetic root cause of BAG3 DCM and is designed using Affinia’s proprietary […] The post Affinia Therapeutics Receives European Medicines Agency Orphan Drug Designation for AFTX-201 in BAG3-Associated Dilated Cardiomyopathy (DCM) appeared first on AffiniaTX .
Hansa Biopharma's Biologics License Application (BLA) for imlifidase accepted by the FDA
PR Newswire
2026-02-18
bla
LUND, Sweden, Feb. 18, 2026 /PRNewswire/ -- Hansa Biopharma AB, ("Hansa" or "the Company"), (Nasdaq Stockholm: HNSA), today announced that its Biologics License Application (BLA) for imlifidase has been accepted by the U.S. Food and Drug Administration (FDA). FDA's filing review was...
Reassessing Krystal Biotech (KRYS) After Strong 1-Year Rally And Vyjuvek Pipeline Focus
Krystal Biotech
2026-02-18
vyjuvek
If you are wondering whether Krystal Biotech's share price still lines up with its underlying value, you are not alone. A closer look at its valuation can help frame that question more clearly. The stock recently closed at US$287.84, with returns of 6.3% over 7 days, 1.0% over 30 days, 16.6% year to date, 83.7% over 1 year and 276.0% over 5 years. These figures may change how some investors view its potential and risk. Recent news coverage around Krystal Biotech has focused on its position...
Reinventing Gene Editing for Safer, More Precise Therapies
Inside Precision Medicine
2026-02-18
gene editing
Asked and Answered—Damian Doherty Chats with Amanda Smith, PhD The post Reinventing Gene Editing for Safer, More Precise Therapies appeared first on Inside Precision Medicine .
Why uniQure (QURE) Is Down 12.0% After AMT-130 Lawsuits Challenge Its FDA Milestone Story
uniQure
2026-02-18
amt-130
gene therapy
fabry disease
accelerated approval
In early February 2026, multiple law firms filed securities fraud class action lawsuits against uniQure N.V., alleging that the company misled investors about U.S. FDA alignment, pivotal study design, and the likelihood of accelerated approval for its Huntington’s disease gene therapy candidate, AMT-130, during late 2025. At the same time, uniQure reported new Phase I/IIa data on its Fabry disease program AMT-191, underscoring a broader pipeline but also highlighting clinical and safety...
Emicizumab Prophylaxis May Be Safe, Effective in Infants With Hemophilia A
Hematology Advisor News
2026-02-18
hemophilia a
Emicizumab prophylaxis was considered potentially safe and effective in a real-world, international study of infants with hemophilia A.
Court Rejects Two Moderna Defenses in mRNA Vaccine Patent Row With Arbutus
BioSpace
2026-02-18
lipid nanoparticle
Arbutus alleges that Moderna’s COVID-19 vaccine infringes on patents protecting its lipid nanoparticle delivery technology.
Ocular’s Hydrogel Beats Regeneron’s Eylea in Wet AMD Study, Plots Path to FDA
BioSpace
2026-02-17
wet age-related macular degeneration
Following the successful late-stage study in wet age-related macular degeneration, Ocular plans to meet with the FDA to determine a regulatory path for Axpaxli.
Krystal Biotech Reports $107.1 Million in 4Q Revenue and FDA Designations for Innovative Gene Therapies
Krystal Biotech
2026-02-17
vyjuvek
gene therapies
financial results
Krystal Biotech reported $107.1 million in Q4 VYJUVEK revenue, with significant progress in multiple gene therapies and strong financials.Quiver AI SummaryKrystal Biotech, Inc. announced its fourth quarter and full year 2025 financial results, reporting $107.1 million in VYJUVEK revenue for the quarter and a total of $730.3 million since its U.S. launch. The company received an RMAT designation from the FDA for KB707 to treat advanced non-small c
Ocular Therapeutix’s Axpaxli could become first choice in wet AMD after showing superiority over Eylea
Endpoints News
2026-02-17
wet age-related macular degeneration
Ocular Therapeutix’ investigational drug could become the preferred therapy for wet age-related macular degeneration after data released Tuesday showed statistical superiority to Regeneron and Bayer’s current bestseller Eylea. However, the data from the trial appear ...
Cryoport to Report Fourth Quarter and Full Year 2025 Financial Results on March 3, 2026
Cryoport Inc
2026-02-17
financial results
Ray Therapeutics Appoints Michael Murtagh as Chief Regulatory Officer
ray therapeutics
2026-02-17
gene therapies
Industry veteran brings global regulatory expertise to support development of innovative optogenetic gene therapies
Blood test helps predict myeloma relapse risk without biopsy
Rare Cancer News
2026-02-17
multiple myeloma
A new, ultrasensitive blood test checks for cancer cells remaining in the body after multiple myeloma treatment using blood samples instead of more invasive bone marrow biopsies, helping doctors assess relapse risk and monitor treatment response. Quest Diagnostics said its Flow Cytometry MRD for Myeloma test detects minimal residual disease (MRD), the small number of cancer […] The post Blood test helps predict myeloma relapse risk without biopsy appeared first on Rare Cancer News .
KRYS Stock Rallies On Upbeat Earnings: Jefferies Ups Price Target On Sustained Vyjuvek Optimism
Krystal Biotech
2026-02-17
vyjuvek
Jefferies’ new price target represents a potential upside of about 34% from the stock’s closing price on Friday.
Boehringer Ingelheim stops work on inhaled cystic fibrosis gene therapy
Endpoints News
2026-02-17
gene therapy
Boehringer Ingelheim stopped development of an inhaled cystic fibrosis gene therapy that was in early-stage clinical trials. The German pharma company told Endpoints News that it decided to terminate a study of the gene therapy ...
Selecting the optimal cell therapy manufacturing platform
Biopharma Dive
2026-02-17
cell therapy
Navigating modular vs. integrated platforms—find the best path for your cell therapy program.
MIMEDX to Host Fourth Quarter and Full Year 2025 Operating and Financial Results Conference Call on February 25
mimedx
2026-02-17
financial results
MARIETTA, Ga., Feb. 17, 2026 (GLOBE NEWSWIRE) -- MiMedx Group, Inc. (Nasdaq: MDXG) (“MIMEDX” or the “Company”) today announced that it will report its operating and financial results for the fourth quarter and full year ended December 31, 2025 after the market close on Wednesday, February 25, 2026.
Gilead’s HIV Advances And Yescarta Label Shift Refocus Investment Thesis
Gilead Sciences
2026-02-17
car
yescarta
cell therapy
Gilead Sciences (NasdaqGS:GILD) plans to present late breaking data at CROI 2026 on a new investigational single tablet HIV regimen. The company will also share the first HIV cure clinical program data from South Africa at the same conference. Kite, Gilead's cell therapy unit, supported the largest metabolomic study in CAR T cell therapy to date. The FDA updated the label for Yescarta, removing major use limitations for the CAR T cell therapy. For investors tracking Gilead, these updates...
Shaping The Future of Obesity Care A Discussion with Annegret Van der Aa
Inside Precision Medicine
2026-02-17
crispr
CRISPR-based tools are changing the face of rapid and point-of-care diagnostics. These five players are driving the field with more flexible and decentralized tests.. The post Shaping The Future of Obesity Care A Discussion with Annegret Van der Aa appeared first on Inside Precision Medicine .
Krystal Biotech Q4 Earnings Call Highlights
Krystal Biotech
2026-02-17
vyjuvek
Krystal Biotech (NASDAQ:KRYS) reported fourth-quarter 2025 net revenue from VYJUVEK of $107.1 million, extending what management described as continued launch momentum in the U.S. and early traction in Europe and Japan. Total net VYJUVEK revenue since launch has now surpassed $730 million, the compa
BioMarin to Host Fourth Quarter and Full-Year 2025 Financial Results Conference Call and Webcast on Monday, February 23, 2026, at 4: 30pm ET
BioMarin Pharmaceutical
2026-02-17
financial results
SAN RAFAEL, Calif., Feb. 17, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. today announced that Alexander Hardy, President and Chief Executive Officer of BioMarin, will host a conference call and...
Promising Biotech Stocks Worth Watching
Krystal Biotech
2026-02-17
krystal biotech
vertex pharmaceuticals
Danaher, Vertex Pharmaceuticals, Moderna, argenex, and Krystal Biotech are the five Biotech stocks to watch today, according to MarketBeat's stock screener tool. Biotech stocks are shares of publicly traded companies whose core business is applying biological science and technology to develop drugs
Gilead Sciences Data And Label Updates Test HIV And CAR T Valuation
Gilead Sciences
2026-02-17
car
yescarta
cell therapy
central nervous system lymphoma
Gilead Sciences and Kite are highlighting new late stage HIV and CAR T cell therapy data, along with regulatory updates, at upcoming medical meetings in 2026. Gilead Sciences plans to present Phase 3 results for a new single tablet HIV regimen and updates on HIV cure research at CROI 2026. Kite received an FDA label update for Yescarta that removes a key limitation of use in relapsed or refractory primary central nervous system lymphoma, expanding eligible patients for this CAR T cell...
Krystal Biotech Q4 2025 slides: VYJUVEK drives revenue growth, pipeline advances By Investing.com
Krystal Biotech
2026-02-17
vyjuvek
Krystal Biotech Q4 2025 slides: VYJUVEK drives revenue growth, pipeline advances
Zemcelpro® (dorocubicel) receives NUB Status 1 in Germany
Excellthera
2026-02-17
cell therapy
The NUB designation for Zemcelpro® (dorocubicel) enables hospitals to immediately apply for temporary, supplementary reimbursement for its use. MONTREAL, February 16, 2026 – ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and metabolic fitness, and its wholly owned subsidiary Cordex Biologics (Cordex), announced that Zemcelpro® (dorocubicel), also known as UM171 Cell Therapy, has […]
BioMarin to Host Fourth Quarter and Full-Year 2025 Financial Results Conference Call and Webcast on Monday, February 23, 2026, at 4:30pm ET
BioMarin Pharmaceutical
2026-02-17
financial results
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Alexander Hardy, President and Chief Executive Officer of BioMarin, will host a conference call and webcast on Monday, February 23, 2026, at 4:30 p.m. ET to discuss fourth quarter and full-year 2025 financial results and provide a general business update.
Alzheimer’s Risk Increased by Exposure to Particulate Air Pollution
Inside Precision Medicine
2026-02-17
alzheimer's disease
Air pollution exposure significantly but modestly increases a person's risk for developing Alzheimer's disease in a way that is mostly independent of other risk factors. The post Alzheimer’s Risk Increased by Exposure to Particulate Air Pollution appeared first on Inside Precision Medicine .
Jefferies sets a $28 PT for enGene Holdings Inc. (ENGN)
enGene Google
2026-02-16
bladder cancer
We recently published an article titled 10 Best Low Volatility Canadian Stocks to Buy. On January 30, Jefferies initiated coverage of enGene Holdings Inc. (NASDAQ:ENGN) with a Buy rating and a $28 price target, highlighting the company’s ongoing pivotal study in high-risk non-muscle invasive bladder cancer (NMIBC), with a key data update expected in the […]
Jefferies Sets a $28 PT for enGene Holdings Inc. (ENGN)
enGene Google
2026-02-16
bladder cancer
We recently published an article titled 10 Best Low Volatility Canadian Stocks to Buy. On January 30, Jefferies initiated coverage of enGene Holdings Inc. (NASDAQ:ENGN) with a Buy rating and a $28 price target, highlighting the company’s ongoing pivotal study in high-risk non-muscle invasive bladder cancer (NMIBC), with a key data update expected in the […]
Trial testing cell therapy for hard-to-treat AML now full in both countries
Rare Cancer News
2026-02-16
cell therapy
acute myeloid leukemia
Enrollment is now complete for an early clinical study assessing the safety and tolerability of SENTI-202, Senti Biosciences’ cell therapy candidate for adults with hard-to-treat acute myeloid leukemia (AML), a type of blood cancer. The Phase 1 trial (NCT06325748) enrolled an estimated 21 people, ages 18 to 74, at sites in the U.S. and Australia. […] The post Trial testing cell therapy for hard-to-treat AML now full in both countries appeared first on Rare Cancer News .
CAR T-Cell Therapy Market worth $13.78 billion by 2031 | MarketsandMarkets™
PR Newswire
2026-02-16
car
cell therapy
DELRAY BEACH, Fla., Feb. 16, 2026 /PRNewswire/ -- According to MarketsandMarkets™, the CAR T-Cell Therapy Market is estimated to reach USD 13.78 billion by 2031 from USD 7.24 billion in 2026, at a CAGR of 13.7%. Browse 300 market data Tables and 30 Figures spread through 280 Pages and...
Roche announces positive phase III results for Gazyva/Gazyvaro in primary membranous nephropathy, marking a significant milestone in this autoimmune disease
Roche Group
2026-02-16
lupus
MAJESTY, the first global phase III study in primary membranous nephropathy, met its primary endpoint of complete remission at two years Up to 30% of people with membranous nephropathy progress to kidney failure over 10 years despite current treatment approaches; achieving complete remission can help delay or prevent this1,2Gazyva/Gazyvaro could become the first approved treatment for primary membranous nephropathy, having already achieved positive results in lupus nephritis, systemic lupus eryt
Atamyo Therapeutics Announces the Dosing of a Fourth Patient with ATA-200 in an Ongoing LGMD-R5 Clinical Trial, Supported by CureSCG
atamyo therapeutics
2026-02-16
gene therapy
Atamyo Therapeutics announces continued progress in its ongoing clinical trial of ATA-200, with the recent dosing of a fourth patient, marking an important milestone in the development of its gene therapy for γ-sarcoglycan related limb […]
GRAIL to Announce Fourth Quarter and Full Year 2025 Financial Results
PR Newswire
2026-02-16
financial results
MENLO PARK, Calif., Feb. 16, 2026 /PRNewswire/ -- GRAIL, Inc. (Nasdaq: GRAL), a healthcare company whose mission is to detect cancer early when it can be cured, announced today that it will issue financial results for the fourth quarter and full year 2025 following the close of market on...
Did Gilead’s (GILD) Strong Quarter, Bigger Dividend and Yescarta Win Just Redefine Its Story?
Gilead Sciences
2026-02-15
car
yescarta
cell therapy
In February 2026, Gilead Sciences reported higher fourth-quarter and full-year 2025 revenue and earnings, raised its quarterly dividend to US$0.82 per share, completed a multi-year US$4.15 billion buyback program, and received FDA approval to remove key prescribing limitations for its Yescarta CAR T-cell therapy in certain lymphoma patients. Together, these moves highlight Gilead’s emphasis on returning cash to shareholders while trying to widen its oncology footprint alongside its...
UniQure Balances Fabry Trial Progress With Safety Review And Legal Risks
uniQure
2026-02-15
gene therapy
fabry disease
gene therapies
uniQure (NasdaqGS:QURE) released updated clinical data for its gene therapy candidate AMT-191 in Fabry disease and paused new dosing to review safety findings. The company is also facing a newly filed securities fraud class action lawsuit related to past disclosures about its Huntington's disease program. uniQure focuses on gene therapies for serious genetic diseases, so any clinical update or legal development can draw close attention from investors. The fresh AMT-191 data and dosing pause...
Gilead Sciences (GILD) Valuation Check As Strong Results Yeztugo Uptake And Yescarta Expansion Draw Attention
Gilead Sciences
2026-02-14
yescarta
cell therapy
Gilead Sciences (GILD) is back in focus after a busy stretch, including strong Q4 and 2025 results, rapid take up of HIV prevention drug Yeztugo, and an FDA label expansion for cell therapy Yescarta. See our latest analysis for Gilead Sciences. The recent run of earnings, guidance, dividend increase, and the Yescarta label update has coincided with strong momentum, with a 30 day share price return of 24.91% and a 1 year total shareholder return of 53.07% building on a 5 year total shareholder...
UniQure (QURE) Reports Positive Phase I/IIa AMT-191 Data for Fabry Disease, Shows Sustained Enzyme Activity
uniQure
2026-02-14
gene therapy
fabry disease
UniQure (NASDAQ:QURE) is one of the most promising low-cost stocks to buy now. On February 6, UniQure reported positive updated data from its Phase I/IIa trial of AMT-191, which is a gene therapy for Fabry disease. All 11 patients across three different dose levels showed increased enzyme activity, with the longest-treated patient maintaining these high […]
UniQure (QURE) Reports Positive Phase I/IIa AMT-191 Data for Fabry Disease, Shows Sustained Enzyme Activity
uniQure
2026-02-14
fabry disease
Stock screener for investors and traders, financial visualizations.
Vertex, CRISPR Set Lofty Goal for Casgevy Gene Therapy as Patient Starts Ramp
BioSpace
2026-02-13
crispr
casgevy
gene therapy
sickle cell disease
Following over a year of slow uptake, Vertex Pharmaceuticals and CRISPR Therapeutics expect Casgevy revenues to nearly triple in 2026, as patient access to the sickle cell disease and beta thalassemia gene therapy grows.
Gilead Broadens Cancer Reach As HIV Prevention Momentum Builds For 2026
Gilead Sciences
2026-02-13
car
yescarta
cell therapy
central nervous system lymphoma
FDA expands the label for Gilead’s Yescarta CAR T-cell therapy to include relapsed or refractory primary central nervous system lymphoma, the first therapy of its kind for this rare indication. Gilead reports major milestones in HIV prevention, with YES2GO gaining rapid market traction. The company highlights multiple expected product launches across HIV, liver disease, and oncology in 2026. Gilead Sciences, listed as NasdaqGS:GILD, is best known for its antiviral therapies in HIV and liver...
Epigenetic editing startup Moonwalk shifts focus to siRNA for obesity
Endpoints News
2026-02-13
crispr
gene editing
Two years ago, CRISPR gene editing pioneer Feng Zhang and former Illumina chief technology officer Alex Aravanis launched a startup to develop medicines that would change how genes are turned on and ...
Citius Oncology, Inc. Announces First Reported Revenue Following Successful Launch of LYMPHIR™
PR Newswire
2026-02-13
financial results
Company reports $3.9 million in revenue generated from initial sales in December 2025 and provides first fiscal quarter 2026 financial results CRANFORD, N.J., Feb. 13, 2026 /PRNewswire/ -- Citius Oncology, Inc. ("Citius Oncology") (Nasdaq: CTOR), the oncology-focused subsidiary of Citius...
Inmune Bio plans to seek approval of RDEB therapy in UK, US, EU
Epidermolysis Bullosa News
2026-02-13
cordstrom
Inmune Bio is planning to submit applications this year seeking regulatory approval of CORDStrom for RDEB in the U.K., the U.S., and EU.
Ultragenyx cuts jobs as it seeks path to profit in 2027
Biopharma Dive
2026-02-13
gene therapy
Another FDA knockback for a gene therapy has investors and analysts redrawing expectations for the rare disease drug developer.
ONA Summit 2026: Advanced Immunotherapies and the Expanding Role of Oncology Nursing
Hematology Advisor News
2026-02-13
car
cell therapy
The Oncology Nurse Advisor Summit spotlights CAR T-cell therapy, bispecific antibodies, and immunotherapy nursing best practices.
8-K - Longeveron Inc. (0001721484) (Filer)
SEC Filing (8-K)
2026-02-13
longeveron
Filed: 2026-02-13 AccNo: 0001213900-26-016519 Size: 319 KB Item 5.02: Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers: Compensatory Arrangements of Certain Officers Item 9.01: Financial Statements and Exhibits
Perrigo to Release Fourth Quarter and Fiscal Year 2025 Financial Results on February 26, 2026
PR Newswire
2026-02-13
financial results
DUBLIN, Feb. 13, 2026 /PRNewswire/ -- Perrigo Company plc (NYSE: PRGO), a leading provider of consumer health products, today announced that it plans to issue its fourth quarter and fiscal year 2025 financial results on Thursday, February 26th, 2026, and host a conference call beginning...
Does FDA RMAT Status For KB707 In Lung Cancer Change The Bull Case For Krystal Biotech (KRYS)?
Krystal Biotech
2026-02-13
gene therapy
In February 2026, Krystal Biotech announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to KB707, its inhaled, redosable immunotherapy for advanced or metastatic non-small cell lung cancer, based on early KYANITE-1 data showing durable responses and clinically meaningful tumor reductions. This RMAT status gives Krystal Biotech enhanced access to FDA guidance and potential accelerated pathways, highlighting how its gene therapy platform is extending beyond...
Vertex’s CRISPR therapy rebounds in latest earnings
Biopharma Dive
2026-02-13
crispr
casgevy
Sales of Casgevy, a gene-edited treatment developed with CRISPR Therapeutics, more than tripled compared to the third quarter, a performance analysts saw as a bright spot in Vertex's report.
Lyell starts first-of-its-kind CAR-T trial; New coalition aims to boost European biotechs
Biopharma Dive
2026-02-13
car
cell therapy
Lyell’s trial will test its experimental cancer cell therapy directly against marketed counterparts. Elsewhere, Biogen swapped board chairs and venture firms are joining up at a “pivotal time” for Europe.
Updated: Ultragenyx to lay off about 130 employees, resubmit rare disease application again
Endpoints News
2026-02-13
gene therapy
Ultragenyx announced Thursday afternoon that the FDA would not take up its recent gene therapy application. Additionally, it will lay off about 10% of its employees as part of a restructuring, following a bumpy 2025 ...
Krystal Biotech: Flawless Vyjuvek Launch
Krystal Biotech
2026-02-12
vyjuvek
Executive summary Krystal Biotech has successfully transitioned into a commercial-stage biotechnology powerhouse following the landmark regulatory approval and rapid market penetration of its flagship product,...
Lyell starts first head-to-head CAR-T therapy trial
Endpoints News
2026-02-12
car
cell therapy
Some cell therapy makers are employing an unprecedented but welcome strategy to forge the next wave of CAR-T treatments: making them beat the existing ones. Physicians have long preferred studies that pit one treatment directly ...
Yescarta Limitations of Use Removed for Primary CNS Lymphoma
Hematology Advisor News
2026-02-12
yescarta
Based on phase 1 safety data, the FDA has removed the Limitations of Use for Yescarta in patients with relapsed or refractory primary CNS lymphoma.
Vertex Reports Fourth Quarter and Full Year 2025 Financial Results
Vertex
2026-02-12
financial results
— Full year total revenue of $12.0 billion, a 9% increase compared to full year 2024; fourth quarter total revenue of $3.19 billion, a 10% increase compared to fourth quarter 2024 — — Company provides full year 2026 total revenue guidance of $12.95 billion to $13.1 billion, with non-CF products
CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2025 Financial Results
CRISPR
2026-02-12
crispr
financial results
ZUG, Switzerland and BOSTON, Feb. 12, 2026 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) today reported financial results for the fourth quarter and full year ended December 31, 2025. “As we close out the fourth quarter, CRISPR Therapeutics continues to make steady progress across a broad
enGene CEO Talks Detalimogene for Community Urology as LEGEND Study Hits Full Enrollment
enGene Google
2026-02-12
bladder cancer
enGene (NASDAQ:ENGN) CEO Ron Cooper said the company’s lead product candidate, detalimogene, is being developed with an eye toward the practical needs of urologists treating high-risk non-muscle invasive bladder cancer (HR-NMIBC), particularly in the community setting where the majority of patients
CG Oncology's Creto Launch Could See Headwind From Reimbursement Incentives, RBC Says
CG Oncology
2026-02-12
bladder cancer
CG Oncology's future launch of Cretostimogene, or Creto, as a therapy for bladder cancer could face a headwind from reimbursement dynamics, RBC Capital Markets said in a Thursday note. The...
Ultragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2026-02-12
financial results
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Benitec Biopharma Releases Second Quarter 2026 Financial Results and Provides Operational Update
benitec biopharma
2026-02-12
financial results
Lyell Immunopharma Announces Initiation of Patient Dosing in First-of-Its-Kind Phase 3 Head-To-Head CAR T-Cell Clinical Trial in Aggressive Large B-Cell Lymphoma
lyell immunopharma
2026-02-12
car
cd19
cell therapies
b-cell lymphoma
large b-cell lymphoma
PiNACLE – H2H will evaluate the efficacy and safety of rondecabtagene autoleucel (ronde-cel) versus Investigator’s choice of approved CD19 CAR T-cell therapies in patients with aggressive large B-cell lymphoma receiving treatment in the second-line setting The pivotal single-arm trial, PiNACLE,
Caribou Biosciences to Participate in Upcoming Investor Conferences
caribou biosciences
2026-02-12
crispr
BERKELEY, Calif., Feb. 12, 2026 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today announced management will participate in the following investor conferences: Citi 2026 Virtual Oncology Leadership Summit,
Ovarian cancer drug lira-cel extends survival ‘far longer’ than expected
Rare Cancer News
2026-02-11
cell therapy
In an early clinical trial in the U.S., most women with hard-to-treat ovarian cancer given a low dose of cell therapy candidate liraltagene autoleucel, known for short as lira-cel, lived longer than they were expected to, according to new data announced by developer Anixa Biosciences. In fact, one woman’s survival extended more than two years […] The post Ovarian cancer drug lira-cel extends survival ‘far longer’ than expected appeared first on Rare Cancer News .
Real-Word Population Further Validates CAR-HEMATOTOX for CAR-T Therapy
Hematology Advisor News
2026-02-11
car
Utilizing the CAR-HEMATOTOX score among patients with R/R LBCL was reaffirmed in a larger, real-world cohort with respect to neutropenia.
Precision BioSciences Receives U.S. FDA Clearance of Investigational New Drug Application for First-in-Class PBGENE-DMD for Treatment of Duchenne Muscular Dystrophy
precision biosciences
2026-02-11
duchenne muscular dystrophy
– U.S. FDA Study May Proceed notification enables initiation of clinical trial site activation for the FUNCTION-DMD Phase 1/2 clinical study in patients with Duchenne muscular dystrophy (DMD) – – Institutional Review Board (IRB) process is underway at multiple world-class DMD clinical trial sites –
Weight Loss Drama Compounds on Heels of Novo, Lilly Earnings, Plus Gene Therapy News
BioSpace
2026-02-11
gene therapy
A lawsuit and FDA warning ensued after Hims & Hers launched a compounded version of Novo Nordisk’s new obesity pill, more Big Pharma report earnings—including from weight loss rivals Novo and Eli Lilly—and the gene therapy space sees another rejection.
Brad Ringeisen: CRISPR for Disease Elimination and Humanitarian Solutions
Inside Precision Medicine
2026-02-11
crispr
The Innovative Genomics Institute (IGI) executive director and Nobel laureate Jennifer Doudna are turning CRISPR's early promise into durable and accessible treatments for rare, complex, and underserved diseases. The post Brad Ringeisen: CRISPR for Disease Elimination and Humanitarian Solutions appeared first on Inside Precision Medicine .
Roche sees positive results for new multiple sclerosis drug in late-stage trials
Roche Group
2026-02-11
multiple sclerosis
About 1 million people are living with multiple sclerosis in the U.S., and about 10% of those have PPMS, making it the least common form of the disease.
Iovance Biotherapeutics to Report Fourth Quarter and Full Year 2025 Financial Results and Corporate Updates on Tuesday, February 24, 2026
Iovance Biotherapeutics
2026-02-11
financial results
SAN CARLOS, Calif., Feb. 11, 2026 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, will host a conference call and live
Alaunos Therapeutics to Participate in Upcoming Investor Conferences in April
Alaunos
2026-02-11
cell therapy
HOUSTON, March 29, 2023 (GLOBE NEWSWIRE) -- Alaunos Therapeutics, Inc. (“Alaunos” or the “Company”) (Nasdaq: TCRT), a leading T-cell receptor (TCR) cell therapy company advancing a clinical-stage pipeline of therapeutics for solid tumors, today announced that company management will be
Medtronic to announce financial results for its third quarter of fiscal year 2026
PR Newswire
2026-02-11
financial results
GALWAY, Ireland, Feb. 11, 2026 /PRNewswire/ -- Medtronic plc (NYSE: MDT), a global leader in healthcare technology, today announced that it will report financial results on Tuesday, February 17, 2026, for its third quarter of fiscal year 2026, which ended on Friday, January 23, 2026. A...
Sarepta Therapeutics to Announce Fourth Quarter and Full-Year 2025 Financial Results
Sarepta Therapeutics
2026-02-11
financial results
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Feb. 11, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report fourth quarter and full-year 2025 financial results after the Nasdaq Global Market closes on Wednesday, Feb. 25, 2026.
Bag of Tr1Xs: Off-the-Shelf Type 1 Treg Shows Phase I Promise for GvHD
Inside Precision Medicine
2026-02-11
allogeneic
cell therapy
Tr1X Bio demonstrated the safety and effectiveness of allogeneic Tr1 cell therapy through immune reconstitution and engraftment in a first-of-its-kind clinical trial. The post Bag of Tr1Xs: Off-the-Shelf Type 1 Treg Shows Phase I Promise for GvHD appeared first on Inside Precision Medicine .
Yescarta Label Expansion And Dividend Growth Shape Gilead Investment Case
Gilead Sciences
2026-02-11
car
yescarta
cell therapy
central nervous system lymphoma
FDA expands the label for Gilead Sciences' CAR T-cell therapy Yescarta to include relapsed or refractory primary central nervous system lymphoma (PCNSL). The decision removes prior limitations on approval and allows Yescarta to be marketed for this rare, hard to treat cancer. The update marks the first time a CAR T-cell therapy is cleared for use in PCNSL, widening potential access for eligible patients. For investors watching NasdaqGS:GILD, this label expansion aligns with Gilead’s...
In Vivo Is Having a Moment as Cell and Gene Therapy Sector Gathers in San Diego
BioSpace
2026-02-11
gene therapy
A recurring theme Tuesday morning at Phacilitate’s Advanced Therapies Week was the quickly emerging potential of in vivo approaches to cell and gene therapy—a trend also reflected in recent investments by Eli Lilly and Regeneron.
Gilead Sciences 4th-qtr beats expectations but guidance disappoints
Gilead Sciences
2026-02-11
financial results
US antivirals giant Gilead Sciences posted fourth quarter and full year 2025 financial results yesterday that received a mixed market reaction.
California’s CIRM aims to speed therapies’ move from lab to clinic
AADC News/BioNews
2026-02-11
gene therapies
The California Institute for Regenerative Medicine (CIRM) approved a $100 million funding program designed to speed the development of gene therapies for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency. The Rare Disease Acceleration Platform and Innovation and Delivery (RAPID) program will invest the funds over two years to create a scalable, platform-based model […] The post California’s CIRM aims to speed therapies’ move from lab to clinic appeared first on AADC News .
Sanofi invests in China startup GluBio in developing molecular glue for sickle cell
Endpoints News
2026-02-11
gene editing
A small startup based in San Diego and Shanghai believes it has figured out how to boost hemoglobin levels with a pill, potentially offering a cheaper and simpler alternative to expensive and onerous gene editing ...
Gilead Sciences Announces Fourth Quarter and Full Year 2025 Financial Results
Gilead Sciences
2026-02-10
financial results
Gilead Sciences, Inc. (Nasdaq: GILD) announced today its results of operations for the fourth quarter and full year 2025. “Our fourth quarter and full-year r...
American Journal of Preventive Cardiology Publishes Landmark Study on Sex Differences in Homozygous Familial Hypercholesterolemia in First Issue Under New Editor-in-Chief
PR Newswire
2026-02-10
familial hypercholesterolemia
BOULDER, Colo., Feb. 10, 2026 /PRNewswire/ -- The American Journal of Preventive Cardiology (AJPC) is proud to announce the publication of a landmark multinational study examining sex-based differences in homozygous familial hypercholesterolemia (HoFH) in its first issue under the...
Bladder Cancer Market: Rapid Increment Driven by Innovation by 2034 – DelveInsight | Bristol-Myers Squibb Company, GlaxoSmithKline PLC., Johnson & Johnson, Janssen Pharma, F. Hoffmann-La Roche., Merck
Janssen Global Services
2026-02-10
bladder cancer
The Key Bladder Cancer Companies in the market include - Bristol-Myers Squibb Company, GlaxoSmithKline PLC., Johnson & Johnson (Janssen Pharmaceutical), F. Hoffmann-La Roche., Merck & Co. Inc., and others. DelveInsight’s “Bladder Cancer Market Insig...
Myeloma treatment anito-cel shows potential for fewer side effects
Rare Cancer News
2026-02-10
arcellx
kite pharma
cell therapy
cell therapies
anitocabtagene autoleucel
Anito-cel (anitocabtagene autoleucel), a cell therapy in clinical development as a potential treatment for myeloma, may be less likely to cause side effects than similar, approved myeloma cell therapies, according to a preclinical study. Arcellx, which is developing anito-cel alongside Kite Pharma, said in a press release that it shared the findings at the 2026 […] The post Myeloma treatment anito-cel shows potential for fewer side effects appeared first on Rare Cancer News .
PADCEV™ + Keytruda ® Cuts Risk of Recurrence or Death by Nearly 50% in Cisplatin-Eligible Muscle-Invasive Bladder Cancer
astellas
2026-02-10
bladder cancer
In the Phase 3 EV-304 trial, statistically significant improvements in overall survival and pathological complete response were also observed, with over half of patients having no detectable...
Dismay as FDA blocks Regenxbio's Hunter syndrome therapy
PharmaPhorum
2026-02-10
gene therapy
The FDA said it needs more information before it can approve Regenxbio's gene therapy for Hunter syndrome, in a devastating decision for patients.
REGENXBIO Announces Regulatory Update on RGX-121 BLA for MPS II
REGENXBIO
2026-02-10
bla
rgx-121
mucopolysaccharidosis
clemidsogene lanparvovec
FDA issues Complete Response Letter for RGX-121 (clemidsogene lanparvovec) for treatment of Mucopolysaccharidosis II (MPS II) REGENXBIO plans to work with FDA on a path forward with the goal of resubmitting the BLA ROCKVILLE, Md., Feb. 9, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today
Gilead Sciences Announces Fourth Quarter and Full Year 2025 Financial Results
Gilead Sciences
2026-02-10
financial results
Gilead Sciences, Inc., a biopharmaceutical company known for its innovative treatments for life-threatening diseases, has released its financial results for the fourth quarter and full year 2025. The company reported strong performance across several key metrics, reflecting its robust market positi…
Genprex Announces IP Australia’s Intent to Grant Patent for Reqorsa® Gene Therapy in Combination with PD-L1 Antibodies to Treat Cancers
Genprex
2026-02-10
gene therapy
Patent Covers Acclaim-3 Clinical Trial Combining REQORSA Gene Therapy with Genentech, Inc.’s Tecentriq® Company Adds Additional Acclaim-3 Clinical Trial Site AUSTIN, Texas — (Feb. 10, 2026) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that IP Australia, the Australian […]
REGENXBIO’s Hunter Syndrome Gene Therapy Fails to Win FDA Nod
BioSpace
2026-02-10
rgx-121
gene therapy
The FDA recommended that REGENXBIO run a new study, treat more patients and include a placebo arm to support a resubmission for the gene therapy RGX-121.
Made Scientific and Streamline Bio Launch Exclusive Early Adopter Program to Advance AI-Driven Robotic Automation for Cell Therapy Manufacturing
PR Newswire
2026-02-10
cell therapy
Partnership designates Made Scientific as exclusive cell therapy CDMO partner for Streamline Bio's platform, accelerating development of autonomous manufacturing workflows in GMP cleanroom environments PRINCETON, N.J. and CAMBRIDGE, Mass., Feb. 10, 2026 /PRNewswire/ -- Made Scientific, a...
Axogen, Inc. to Report Fourth Quarter and Full Year 2025 Financial Results on February 24, 2026
axogen
2026-02-10
financial results
Genentech plans NDA for multiple sclerosis pill with study win
Genentech
2026-02-10
multiple sclerosis
Genentech Inc. is covering more bases in multiple sclerosis, with its latest swing on fenebrutinib hitting a rare phase III noninferiority win against Ocrevus (ocrelizumab) in primary progressive multiple sclerosis.
uniQure: Strengthening Gene Therapy Platform and Fabry Data Undervalued, Supporting Buy Rating and $70 Target
uniQure
2026-02-10
gene therapy
uniQure, the Healthcare sector company, was revisited by a Wall Street analyst today. Analyst Patrick Trucchio from H.C. Wainwright reiterated a Buy rating on the s...
The 15-20 National Hospital and GenSight Biologics announce the treatment of the first patient in the GS010/LUMEVOQ® REVISE Study
GenSight Biologics
2026-02-10
lumevoq
AstraZeneca raises guidance on stellar cancer drugs growth
PharmaPhorum
2026-02-10
financial results
AZ reports strong financial results for 2025, and provides a first peek at the data for its oral GLP-1 contender elecoglipron.
Following Sarepta Woes, Others Look To Unlock the Next Chapter in DMD Treatment
BioSpace
2026-02-09
elevidys
regenxbio
capricor therapeutics
duchenne muscular dystrophy
With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne muscular dystrophy pipeline, as Sarepta’s Elevidys leaves the door open.
Lilly to acquire Orna Therapeutics to advance cell therapies
orna therapeutics
2026-02-09
car
cell therapies
Orna’s in vivo CAR-T pipeline includes potential best-in-class programs to reset the immune system and address B cell-driven autoimmune diseases Acquisition provides a broad platform for long-term innovation in genetic medicine and in vivo cell engineering INDIANAPOLIS and WATERTOWN, Mass., Feb. 9, 2026 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) and Orna Therapeutics, Inc., a biotechnology company dedicated to engineering immune cells in… The post Lilly to acquire Orna Therapeutics to advance cell therapies appeared first on Orna Therapeutics .
Announcement of Consolidated Financial Results Fiscal 2025
Kyowa Kirin Co.
2026-02-09
financial results
Eli Lilly Expands Its In Vivo Ambitions, Acquiring Cell Therapy Startup Orna for up to $2.4B
MedCity News
2026-02-09
cell therapy
Orna Therapeutics brings Eli Lilly an in vivo cell therapy ready for Phase 1 testing as a potential treatment for autoimmune diseases. Lilly is now the latest pharma company to use M&A to enter this growing field, following deals made in the past year by AstraZeneca, AbbVie, Gilead Sciences, and Bristol Myers Squibb. The post Eli Lilly Expands Its In Vivo Ambitions, Acquiring Cell Therapy Startup Orna for up to $2.4B appeared first on MedCity News .
Lilly joins in vivo CAR-T sprint with $2.4B Orna acquisition
Endpoints News
2026-02-09
car
cell therapy
Eli Lilly will buy Orna Therapeutics to get a hold of its immune system-resetting pipeline and enter the in vivo CAR-T cell therapy race. The Monday morning acquisition amounts to as ...
Eli Lilly Enters CAR T Realm With $2.4B Orna Buy, Starting With Autoimmune
BioSpace
2026-02-09
car
The deal gets Lilly access to Orna’s in vivo CAR T technology. The biotech’s lead asset, which has yet to start clinical testing, is focused on B cell–driven autoimmune diseases.
Ayrmid Appoints ACA Pharma as Exclusive Distributor for OMISIRGE ® across Greater China and Southeast Asia
Gamida Cell
2026-02-09
omisirge
ACA Pharma granted exclusive rights to register and distribute OMISIRGE® across 15 markets, covering both FDA-approved OMISIRGE® indications LONDON – February 9, 2026 – Ayrmid, Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc.
Ultragenyx’s Gene Therapy Shows Long-Term Efficacy in Two Sanfilippo Studies
BioSpace
2026-02-09
ux111
gene therapy
The FDA in July last year declined to approve UX111 for Sanfilippo syndrome, a rare neurodegenerative disorder, citing manufacturing issues. Ultragenyx resubmitted its application ten days ago, expecting a six month review time.
8-K - Ocugen, Inc. (0001372299) (Filer)
SEC Filing (8-K)
2026-02-09
ocugen
Filed: 2026-02-09 AccNo: 0001104659-26-011765 Size: 283 KB Item 5.02: Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers: Compensatory Arrangements of Certain Officers
Lilly strikes again, buying CAR-T firm Orna for $2.4bn
PharmaPhorum
2026-02-09
car
In its second major deal in two days, Eli Lilly has reached an agreement to buy in vivo CAR-T developer Orna Therapeutics for up to $2.4bn.
Bioretec Ltd's financial statements bulletin for January-December 2025 will be published on February 13, 2026
PR Newswire
2026-02-09
financial statements
TAMPERE, Finland, Feb. 9, 2026 /PRNewswire/ -- Bioretec Ltd will publish its financial statements bulletin for the financial year 1 January-31 December 2025 on Friday, 13 February 2026 at approximately 9:30 a.m. EET. The company will arrange a webcast for investors and media on the same...
FDA rejects Regenxbio's Hunter syndrome gene therapy
Endpoints News
2026-02-09
gene therapy
The FDA has rejected Regenxbio’s gene therapy for a serious inherited disease called Hunter syndrome in a decision that could reverberate to other rare disease drugmakers as well. The complete response letter comes shortly after ...
Liver Toxicities Force Pause on Two Dose Groups of UniQure’s Mid-Stage Fabry Study
BioSpace
2026-02-09
aav
gene therapy
fabry disease
The gene therapy uses an AAV vector to restore healthy levels of the alpha-galactosidase enzyme, which is rendered dysfunctional in patients with Fabry disease, leading to the toxic build-up of lipids in cells.
FDA Reverses Course on AMT-130, Citing Insufficient External Data for Submission | NeurologyLive - Clinical Neurology News and Neurology Expert Insights
uniQure
2026-02-09
amt-130
accelerated approval
uniQure navigates FDA's unexpected shift on AMT-130, aiming for accelerated approval for Huntington's disease despite recent regulatory challenges.
SparingVision Successfully Completes PRODYGY Trial Patient Dosing with SPVN06, its Novel Neuroprotective Gene Therapy
Sparing Vision
2026-02-09
gene therapy
The post SparingVision Successfully Completes PRODYGY Trial Patient Dosing with SPVN06, its Novel Neuroprotective Gene Therapy appeared first on SparingVision .
Antibiotic Resistant Genetic Elements Inactivated by CRISPR-Based Tool
GenEng News
2026-02-09
crispr
The Pro-Active Genetics (Pro-AG) tool, named pPro-MobV, is a second-generation technology similar to gene drives, which are currently applied in insect populations to prevent the spread of parasites that cause malaria. The post Antibiotic Resistant Genetic Elements Inactivated by CRISPR-Based Tool appeared first on GEN - Genetic Engineering and Biotechnology News .
Krystal Biotech wins FDA RMAT status for lung cancer therapy
Krystal Biotech
2026-02-09
gene therapies
Krystal Biotech (KRYS) announced on Monday that the U.S. Food and Drug Administration awarded the Regenerative Medicine Advanced Therapy designation for KB707, its immunotherapy candidate targeting non-small cell lung cancer. The regulator grants RMAT designation for gene therapies and other regenerative medicines designed to treat serious or life-...
Anixa Biosciences Reports Encouraging Patient Survival Observations in Ovarian Cancer CAR-T Trial; Achieves Regulatory Approval Enabling Major Dose Escalation
anixa biosciences
2026-02-09
car
Multiple patients substantially exceed expected survival at low dose levels; absence of dose- limiting toxicities supports escalation to doses up to 100x higher Trial expansion reflects growing confidence in intra-peritoneal CAR-T delivery and introduces lymphodepletion to potentially enhance efficacy Anixa to participate in Water Tower Research fireside chat at 11:00am ET on February 10, 2026 to discuss trial observations SAN JOSE, Calif. , Feb. 9, 2026 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today provided an update on patient outcomes observed in its ongoing Phase 1 ovarian cancer CAR-T clinical trial, following regulatory approval of a protocol amendment that enables substantial dose escalation. The ongoing Phase 1 trial is enrolling adult women with recurrent ovarian cancer, who have failed standard of care chemotherapy, and progressed after two or more prior therapies. To date, twelve patients have been treated in the trial at four dosage levels. Of these patients, seven have lived beyond their expected median survival of approximately three to four months,…
Gilead Sciences (GILD) Is Up 7.4% After FDA Expands Yescarta Lymphoma Label Restrictions Removed
Gilead Sciences
2026-02-08
car
yescarta
cell therapy
b-cell lymphoma
large b-cell lymphoma
central nervous system lymphoma
In early February 2026, Kite, a Gilead company, reported that the FDA approved an update to Yescarta’s prescribing information, removing prior use limitations in patients with relapsed or refractory primary central nervous system lymphoma based on Phase 1 data from Dana-Farber Cancer Institute. This change makes Yescarta the only CAR T-cell therapy for relapsed or refractory large B-cell lymphoma without such restrictions in PCNSL, addressing a cancer subset with a five-year survival rate of...
enGene Snags FDA RMAT Designation for Bladder Cancer Gene Therapy Detalimogene
enGene Google
2026-02-08
rmat
phase 1/2
gene therapy
bladder cancer
The gene therapy is currently being evaluated in the phase 1/2 LEGEND clinical trial.
uniQure Announces Updated Preliminary AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease
uniQure
2026-02-06
fabry disease
Supraphysiological expression of α-Gal A activity maintained for over a year in longest treated patient as of data cutoff date ~ ~ Stable Lyso-Gb3...
Experimental Fabry gene therapy lets some patients stop enzyme replacement
uniQure
2026-02-06
gene therapy
Supraphysiological α-Gal A gains held over a year in AMT-191 Fabry trial, with 6 of 11 off ERT as dosing in mid/high cohorts pauses for safety review.
uniQure Announces Updated Preliminary AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease
uniQure
2026-02-06
fabry disease
Supraphysiological expression of α-Gal A activity maintained for over a year in longest treated patient as of data cutoff date ~ ~ Stable Lyso-Gb3...
Ayrmid and Ariti Sign Distribution Agreement for Omisirge ® in Greece and Cyprus
Gamida Cell
2026-02-06
omisirge
LONDON – February 5, 2026 – Ayrmid, Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., today announced that it has entered into an exclusive distribution agreement with Ariti S.A.
uniQure reports promising data from Fabry disease gene therapy trial By Investing.com
uniQure
2026-02-06
gene therapy
fabry disease
uniQure reports promising data from Fabry disease gene therapy trial
UniQure pauses Fabry gene therapy dosing in 2 groups after toxicities emerge
uniQure
2026-02-06
aav
gene therapy
fabry disease
Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to treat the rare condition Fabry disease. | Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to treat the rare condition Fabry disease.
FDA Updates Yescarta Label, Boosts Gilead's (GILD) Market Position
Gilead Sciences
2026-02-06
yescarta
Key Takeaways: The FDA has approved a revision to the prescribing guidelines for Yescarta, a product of Kite, a subsidiary of Gilead Sciences (GILD), enhanc
Gilead Sciences (GILD) Valuation Check After Oncology And HIV Therapy Momentum
Gilead Sciences
2026-02-06
yescarta
Gilead Sciences (GILD) has returned to focus after a series of company-specific catalysts, including its oncology expansion through Repare Therapeutics, FDA approval of HIV prevention drug Yeztugo, and encouraging updates for Trodelvy and Yescarta. See our latest analysis for Gilead Sciences. These company specific catalysts have coincided with a sharp shift in market sentiment, with a 30 day share price return of 23.1% and a 1 year total shareholder return of 56.6% suggesting momentum has...
Gilead (GILD) Gains FDA Nod for Expanded Use of Yescarta in Lymphoma Treatment
Gilead Sciences
2026-02-06
car
yescarta
Key Takeaways: Gilead Sciences' Kite division receives FDA approval for a label update on its CAR-T therapy, Yescarta, enhancing its market potential. Gi
Kaiser Permanente and Risant Health report 2025 financial results
PR Newswire
2026-02-06
financial results
The organizations delivered high-quality, affordable, value-based care for members and patients and worked to improve the health of the communities they serve. OAKLAND, Calif., Feb. 6, 2026 /PRNewswire/ -- Kaiser Foundation Health Plan, Inc., Kaiser Foundation Hospitals, Risant Health,...
Uniqure stock rises after AMT-191 gene therapy shows promising Fabry results By Investing.com
uniQure
2026-02-06
gene therapy
Uniqure stock rises after AMT-191 gene therapy shows promising Fabry results
UniQure pauses higher doses in Fabry study; Aro shares Pompe data
uniQure
2026-02-06
gene therapy
UniQure pauses mid/high-dose Fabry gene therapy after liver enzyme issues in 3 patients. Company reports 6 of 11 patients stopped needing enzyme replacement therapy.
Gilead Sciences Says Yescarta Won FDA Label Update, Removing Use Limits for Relapsed CNS Lymphoma Patients
Gilead Sciences
2026-02-06
yescarta
Gilead Sciences' Kite said Friday that the U.S. Food and Drug Administration approved an update to the prescribing label for Yescarta, removing a prior limitation on use in patients with relapsed...
Gilead wins favorable U.S. label update for Yescarta
Gilead Sciences
2026-02-06
car
yescarta
cell therapy
Gilead (GILD) stock is in focus as the FDA approves a favorable label update for its CAR-T cell therapy Yescarta. Read more here.
Sarepta Saga Has 'Gone on Too Long' as Competitors Catch Up
BioSpace
2026-02-05
gene therapies
After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments around the long-time Wall Street darling, and patients may soon begin looking elsewhere.
Additional Positive Results for Omisirge® in Treating Severe Aplastic Anemia (SAA) Presented at TANDEM
Gamida Cell
2026-02-05
omisirge
Omisirge transplantation shows accelerated immune recovery in SAA New data presented at the 2026 TANDEM Meetings demonstrate that patients with SAA who received Omisirge experienced faster and more robust immune recovery compared with patients receiving alternative transplant graft sources Omisirge...
Mass Spectrometry’s Expanding Frontier
GenEng News
2026-02-05
crispr
Mass spectrometry is now experiencing a renaissance. As drug modalities diversify—peptides, siRNAs, mRNA vaccines, CRISPR components, polymer-intensive manufacturing systems, and volatile organic contaminants—the analytical burden has grown dramatically. The post Mass Spectrometry’s Expanding Frontier appeared first on GEN - Genetic Engineering and Biotechnology News .
Agent Orange Exposure Linked to Higher Risk of Rare Melanoma in U.S. Veterans, Study Finds
PR Newswire
2026-02-05
melanoma
Research analyzing Veterans Health Administration data suggests approximately 30% higher odds of acral melanoma among veterans with Agent Orange exposure WASHINGTON, Feb. 5, 2026 /PRNewswire/ -- A new study published in JAMA Dermatology suggests U.S. veterans exposed to Agent Orange may...
Best-in-Class Real-World Data Support Early Amtagvi® Treatment in Advanced Melanoma
Iovance Biotherapeutics
2026-02-05
amtagvi
melanoma
52% Amtagvi Response Rate with Two or Fewer Prior Lines of Therapy 73% Overall Disease Control Rate SAN CARLOS, Calif., Feb. 05, 2026 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel
Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2025 Financial Results and Corporate Update—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2026-02-05
financial results
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Killer T Cells Targeting Epstein-Barr Virus May Contribute to Multiple Sclerosis
GenEng News
2026-02-05
multiple sclerosis
Immune cells that normally target Epstein-Barr virus may be implicated in the immune activity in multiple sclerosis, according to data from a new study that explores the intersection between viral exposure and autoimmune disease. The post Killer T Cells Targeting Epstein-Barr Virus May Contribute to Multiple Sclerosis appeared first on GEN - Genetic Engineering and Biotechnology News .
PTC Therapeutics to Report Fourth Quarter and Full Year 2025 Financial Results on Thursday, Feb. 19, 2026
ptc biotherapeutics
2026-02-05
financial results
WARREN, N.J., Feb. 5, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the company will host a webcast conference call to report its fourth quarter and full year 2025 financial results and provide an update on the company's business and outlook on Thursday, Feb.
CRISPR Roundup
GenEng News
2026-02-05
crispr
A trio of CRISPR pioneers—Nobel laureate Jennifer Doudna, PhD; Matthew Porteus, MD, PhD, and Rodolphe Barrangou, PhD—offer their views on the commercial potential and challenges of genome editing following a rollercoaster 2025. The post CRISPR Roundup appeared first on GEN - Genetic Engineering and Biotechnology News .
Stem Cell Specialist PrimeGen Takes SPAC Track to Nasdaq
BioSpace
2026-02-05
mesenchymal
The SPAC agreement values PrimeGen US at $1.5 billion in equity and gives it capital to advance its pre-clinical triple-activated mesenchymal stem cell pipeline into the clinic.
Allogene Therapeutics Announces Participation in Upcoming Investor Conferences
Allogene
2026-02-05
car
allogeneic
SOUTH SAN FRANCISCO, Calif., Feb. 05, 2026 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer and autoimmune disease, today announced that it will participate in two
Lilly Axes 3 Therapies Amid Pipeline-in-a-Product Push for GLP-1
BioSpace
2026-02-05
gene therapy
One of the assets shelved by Eli Lilly is a gene therapy for dementia, which it obtained in its $1.04-billion acquisition of Prevail Therapeutics in late 2020.
Ayrmid Reports Additional New Real-World Data on Motixafortide for Stem Cell Mobilization in Sickle Cell Disease and Beta-Thalassemia
Gamida Cell
2026-02-04
gene therapy
gene therapies
sickle cell disease
Key Highlights Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease and beta-thalassemia enabling accelerated access to gene therapies 73% (11 of 15) of patients were able to collect sufficient HSC to enable progress to gene therapy manu...
AbbVie Reports Full-Year and Fourth-Quarter 2025 Financial Results
Abbvie Investors
2026-02-04
financial results
Reports Full-Year Diluted EPS of $2.36 on a GAAP Basis, a Decrease of 1.3 Percent; Adjusted Diluted EPS of $10.00, a Decrease of 1.2 Percent; These Results Include an Unfavorable Impact of $2.76 Per Share Related to 2025 Acquired IPR&D and Milestones Expense Delivers Full-Year Net Revenues of
MiNK Therapeutics Presents New Data of allo-iNKT Cell Therapy as a Potential Disease-Modifying Approach in Idiopathic Pulmonary Fibrosis at Keystone Symposia
Mink Therapeutics
2026-02-04
cell therapy
Human lung tissue analyses identify iNKT cell depletion as a mechanistic feature of advanced IPF Findings extend MiNK’s iNKT platform into chronic fibrotic lung disease and support immune restoration strategies in IPF, a large unmet-need market LEXINGTON, Mass., Feb.
Caribou Biosciences Announces Late-Breaking Presentations at the 2026 Tandem Meetings of ASTCT® and CIBMTR®
caribou biosciences
2026-02-04
car
autologous
cell therapy
Clinical data disclosed in November 2025 to be presented at medical meeting with new supportive translational data for both programs Vispa-cel (CB-010) ANTLER phase 1 translational and clinical data demonstrate efficacy and durability that is on par with autologous CAR-T cell therapy in 2L LBCL
Spur Therapeutics Presents New Phase 1/2 Data on Its Gene Therapy Candidate in Gaucher Disease at 22nd Annual WORLDSymposium™
syncona
2026-02-04
gene therapy
gaucher disease
Arcellx Announces Late-Breaking Presentation at TANDEM Demonstrating Unique, High Target-Specificity of anito-cel’s D-Domain Binder
arcellx, inc
2026-02-04
car
bcma
CAR constructs representative of anito-cel (D-Domain), cilta-cel (dual VHH), and ide-cel (scFv) were assessed preclinically for tonic signaling and off-target Claudin binding - - No tonic signaling or off-target activity was observed with the D-Domain, supporting its unique target-specific binding profile and anito-cel’s potentially best-in-class safety profile - - Specific off-target activity against Claudin-9 was observed with the dual VHH binder, consistent with cilta-cel’s publicly available regulatory assessment reports; the D-Domain and scFv binders did not demonstrate off-target activity - - CAR T activation and cytokine release were observed for dual VHH and scFv binders in the absence of BCMA antigen, but not with the D-Domain, supporting lack of tonic signaling for anito-cel - - Data to be presented February 5 during the 2026 Tandem Meetings in Salt Lake City, UT - Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company focused on delivering a new class of innovative
enGene to Spotlight Bladder Cancer Gene Therapy at Guggenheim Biotech Summit 2026
enGene Google
2026-02-04
gene therapy
bladder cancer
The latest update is out from enGene Holdings ( ($ENGN) ). enGene Holdings Inc. will showcase its non-viral genetic medicine platform and lead bladder cancer progra...
Vyjuvek Growth Powers Krystal Biotech’s (KRYS) Huge Upward Analyst Target Price Revision
Krystal Biotech
2026-02-04
vyjuvek
Krystal Biotech, Inc. (NASDAQ:KRYS) is one of the 10 best NASDAQ growth stocks to buy for the next 10 years. Andrea Tan, an analyst at Goldman Sachs, raised the firm’s price target on Krystal Biotech, Inc. (NASDAQ:KRYS) from $206 to $327 on January 30 while keeping a Buy rating. The firm’s revised price target implies […]
Evolent To Release 2025 Fourth Quarter and Year-End Financial Results on Tuesday, February 24, 2026
PR Newswire
2026-02-04
financial results
WASHINGTON, Feb. 4, 2026 /PRNewswire/ -- Evolent Health, Inc. (NYSE: EVH), a company focused on achieving better health outcomes for people with complex conditions, today announced it will release its 2025 fourth quarter and year-end financial results on Tuesday, February 24, 2026, after...
Cumberland Pharmaceuticals Receives FDA Fast Track Designation for its Ifetroban Duchenne Muscular Dystrophy Program
PR Newswire
2026-02-04
duchenne muscular dystrophy
NASHVILLE, Tenn., Feb. 4, 2026 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on developing new products for rare diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for...
Affinia Therapeutics Announces FDA Acceptance of IND Application to Advance AFTX-201 to a Phase 1/2 Trial for the Treatment of BAG3-Associated Dilated Cardiomyopathy (DCM)
Affinia Therapeutics
2026-02-04
ind
gene therapies
AFTX-201 uses Affinia’s proprietary capsid engineered for efficient cardiac transduction at doses that are 5-10-fold lower than doses of gene therapies using conventional capsids Company on track to initiate the UPBEAT© clinical trial to investigate safety, pharmacodynamics, and preliminary efficacy of AFTX-201 in BAG3 DCM in the first half of 2026 WALTHAM, Mass. – February […] The post Affinia Therapeutics Announces FDA Acceptance of IND Application to Advance AFTX-201 to a Phase 1/2 Trial for the Treatment of BAG3-Associated Dilated Cardiomyopathy (DCM) appeared first on AffiniaTX .
Financial Results Library
astellas
2026-02-04
quarterly
Explore financial results, earnings updates, and annual reports from Astellas — organized by fiscal year and updated quarterly.
Digital Tools Benefits Lauded by U.K. Cell and Gene Therapy Accelerator
GenEng News
2026-02-04
gene therapy
cell therapies
A U.K. center of excellence for gene and cell therapies is promoting the benefits of digital tools for paperless batch management, analytics, and process control. The post Digital Tools Benefits Lauded by U.K. Cell and Gene Therapy Accelerator appeared first on GEN - Genetic Engineering and Biotechnology News .
Financial Results of Astellas for Q3 YTD/FY2025
astellas
2026-02-04
financial results
View Financial Results of Astellas for Q3 YTD/FY2025.
Pfizer’s Metsera Data, 2025 Earnings, the FDA and REGENXBIO, Psychedelics and IPOs
BioSpace
2026-02-04
pdufa
Pfizer announces the first data from its Metsera-acquired pipeline just ahead of its earnings call, where analysts pressed execs for more details; Merck and Roche also released Q4 and full year earnings, with Eli Lilly, Novo Nordisk and others reporting Wednesday; REGENXBIO hits a regulatory snag ahead of its upcoming PDUFA; more.
GSK delivers in new CEO Miels' first financial update
PharmaPhorum
2026-02-04
financial results
Luke Miels' first financial results since taking over as chief executive of GSK revealed a jump in sales and profits.
Autolus Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Autolus Therapeutics
2026-02-04
cell therapies
LONDON and GAITHERSBURG, Md., Feb. 04, 2026 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), a commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies and candidates, today announced that the Compensation Committee of the Company's Board of Directors granted stock option awards to purchase an aggregate of 113,650 American Depositary Shares, representing an equal number of ordinary shares, to 22 employees pursuant to the Company’s 2025 Inducement Plan. The stock options were granted as an inducement material to the individual becoming an employee of Autolus in accordance with Nasdaq Listing Rule 5635(c)(4).
IN8bio to Present at Upcoming Investor and Scientific Conferences in February
In8Bio
2026-02-03
cell therapies
NEW YORK, Feb. 03, 2026 (GLOBE NEWSWIRE) -- IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta (γδ) T cell therapies for cancer and autoimmune diseases, today announced that William Ho, CEO and co-founder, will be presenting the following
A Look At uniQure (QURE) Valuation After FDA Setback And Legal Investigations
uniQure
2026-02-03
amt-130
gene therapy
uniQure (NasdaqGS:QURE) is back in focus after disclosing that the FDA views current AMT-130 gene therapy data as insufficient for a Biologics License Application, clouding timelines and drawing legal and analyst scrutiny. See our latest analysis for uniQure. The FDA setback has come after a volatile period, with a 56.83% 1 year total shareholder return contrasting with an 18.86% 3 month share price decline and a smaller 5.60% year to date share price gain. This suggests that momentum has...
Assessing Krystal Biotech (KRYS) Valuation After Analyst Optimism On Vyjuvek Expansion And Ophthalmic Pipeline
Krystal Biotech
2026-02-03
kb803
vyjuvek
Analyst optimism and product pipeline in focus Krystal Biotech (KRYS) is back in the spotlight after several major research houses highlighted its potential to evolve into a multi product company, pointing to ophthalmic candidates KB803 and KB801 and the planned geographic expansion of Vyjuvek. See our latest analysis for Krystal Biotech. Those upbeat analyst views have landed alongside strong recent momentum, with Krystal Biotech’s share price at $282.27 and a 30 day share price return of...
8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
SEC Filing (8-K)
2026-02-03
ultragenyx
Filed: 2026-02-03 AccNo: 0001193125-26-034474 Size: 174 KB Item 8.01: Other Events
Assessing Krystal Biotech (KRYS) Valuation After Analyst Optimism On Vyjuvek Expansion And Ophthalmic Pipeline
Krystal Biotech
2026-02-03
vyjuvek
U.S. stocks, Saudi stocks, stock trading and investment platforms
8-K - Kyverna Therapeutics, Inc. (0001994702) (Filer)
SEC Filing (8-K)
2026-02-03
kyverna therapeutics
Filed: 2026-02-03 AccNo: 0001193125-26-034464 Size: 422 KB Item 5.02: Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers: Compensatory Arrangements of Certain Officers Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
Vyjuvek Growth Powers Krystal Biotech's (KRYS) Huge Upward Analyst Target Price Revision
Krystal Biotech
2026-02-03
vyjuvek
Stock screener for investors and traders, financial visualizations.
Single Fecal Microbiota Transplant May Boost Response to ICI in NSCLC and Melanoma
Hematology Advisor News
2026-02-03
melanoma
In this phase 2 study, researchers explored whether fecal microbiota transplant could overcome resistance to immune checkpoint inhibition.
FDA OKs multiple myeloma combo for all newly diagnosed patients
Rare Cancer News
2026-02-03
multiple myeloma
A treatment regimen combining Darzalex Faspro (daratumumab and hyaluronidase-fihj) with three other medications is now approved in the U.S. for all people newly diagnosed with multiple myeloma. The combination treatment, known as D-VRd, uses Darzalex Faspro alongside Velcade (bortezomib), Revlimid (lenalidomide), and dexamethasone. It was previously authorized only for newly diagnosed myeloma patients eligible for […] The post FDA OKs multiple myeloma combo for all newly diagnosed patients appeared first on Rare Cancer News .
Atamyo Therapeutics Announces First Three Patients Dosed with ATA-200 Gene Therapy in on-going LGMD-R5 Clinical Trial, and Extension of Partnership with the Dion Foundation
atamyo therapeutics
2026-02-03
gene therapy
Evry, France and Boston, MA (February 3, 2026) – Atamyo Therapeutics and The Dion Foundation for Children with Rare Diseases, today announced the expansion of their partnership to support financing of the first-in-human clinical trial […]
Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA)—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2026-02-03
ux111
gene therapy
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Fate Therapeutics Reports New Employee Inducement Awards Under Nasdaq Listing Rule 5635(c)(4)
Fate Therapeutics
2026-02-03
ipsc
SAN DIEGO, Feb. 03, 2026 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases,
China startup AccurEdit raises $75M for gene editing therapies
Endpoints News
2026-02-03
crispr
gene editing
AccurEdit Therapeutics, a startup based in Suzhou, China, has raised $75 million in Series A funding to advance its growing pipeline of CRISPR-based medicines — among the largest rounds on record for a Chinese gene editing ...
Ultragenyx unwraps data that may be central to its gene therapy resubmission
Endpoints News
2026-02-03
gene therapy
Ultragenyx outlined new data on Tuesday that it hopes will convince the FDA to approve its recently resubmitted rare disease gene therapy. Among 17 patients with Sanfilippo syndrome type A who received the therapy, cognition ...
uniQure (QURE) Navigates Gene Therapy Development as Investors Weigh Long-Term Potential
uniQure
2026-02-02
gene therapy
Apis Capital Advisors’ Apis Flagship Fund released its Q4 2025 investor letter. A copy of the letter can be downloaded here. Apis Flagship Fund delivered strong performance in the fourth quarter of 2025, with the Fund up 10.0% net for the quarter and gaining 55.1% for the full year, significantly outperforming the MSCI ACWI global […]
Chronic HBV Coinfection Linked to Worse Survival Outcomes in Pediatric T-ALL
Hematology Advisor News
2026-02-02
acute lymphoblastic leukemia
Researchers examined whether chronic hepatitis B with acute exacerbation affected prognosis in pediatric patients with T-cell acute lymphoblastic leukemia.
Venglustat Improves Neurological Symptoms in Type 3 Gaucher Disease
Hematology Advisor News
2026-02-02
gaucher disease
Findings from the LEAP2MONO study support the regulatory submission of venglustat for treatment of type 3 Gaucher disease.
CREATE Medicines Demonstrates Complete B Cell Depletion and Repeatable Dosing in Non-human Primates with In Vivo CAR-T Platform
PR Newswire
2026-02-02
car
Industry-leading non-human primate (NHP) data validates CREATE's differentiated receptor design, targeted RNA-LNP platform, and ability to repeat dose Clinical entry planned H2 2026 CAMBRIDGE, Mass., Feb. 2, 2026 /PRNewswire/ -- CREATE Medicines, Inc. ("CREATE"), a clinical-stage...
Adjuvant Systemic Therapy in Resected Mucosal Melanoma Improves Survival
Hematology Advisor News
2026-02-02
melanoma
Adjuvant systemic therapy was associated with improved rates of overall survival among patients who underwent surgical resection for MM.
Anixa Biosciences Secures United States Adopted Names Council Approval of Non-Proprietary Name for its CAR-T Therapy, a Key Step Toward Future Commercialization
PR Newswire
2026-02-02
car
SAN JOSE, Calif., Feb. 2, 2026 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the United States Adopted Names (USAN) Council approved "liraltagene...
Alfa-PROTECT Study Shows Low Rates of AESI Among Children With Hemophilia A
Hematology Advisor News
2026-02-02
hemophilia a
Researchers determined the incidence of adverse events of special interest was low among pediatric patients aged 7 years to less than 12 years treated with damoctocog alfa pegol.
Anixa Biosciences Secures United States Adopted Names Council Approval of Non-Proprietary Name for its CAR-T Therapy, a Key Step Toward Future Commercialization
anixa biosciences
2026-02-02
car
cell therapies
chimeric antigen receptor
SAN JOSE, Calif. , Feb. 2, 2026 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the United States Adopted Names (USAN) Council approved "liraltagene autoleucel" for the non-proprietary name of the Company's novel FSHR-targeted CAR-T therapy for recurrent ovarian cancer. This U.S. approval follows the earlier approval for international use of the name by the International Nonproprietary Names (INN) Expert Committee of the World Health Organization (WHO). The USAN and INN nomenclature schemes for CAR-T cell therapies follow a two-word structure describing the gene and cell component. "The assignment of the non-proprietary name in the U.S. represents an important step in the development and potential future commercialization of our CAR-T therapy. With this approval, and the prior approval by the INN Expert Committee of the WHO, we have the ability to establish a universally recognized and conflict-free non-proprietary drug name for our CAR-T therapy on a world-wide basis," said Dr. Amit Kumar, Chairman and CEO of Anixa. "We remain focused on the…
ImmunityBio Launches Phase 2 Chemotherapy-Free CAR-NK Cell Therapy Trial with ANKTIVA® (ResQ215B) in Indolent Lymphomas
Immunity Bio
2026-02-02
car
car-nk
cell therapy
nk cell therapy
The post ImmunityBio Launches Phase 2 Chemotherapy-Free CAR-NK Cell Therapy Trial with ANKTIVA® (ResQ215B) in Indolent Lymphomas appeared first on ImmunityBio .
uniQure (QURE) Is Down 9.4% After FDA Deems AMT-130 Data Insufficient for BLA Filing
uniQure
2026-02-01
bla
amt-130
gene therapy
In November 2025, uniQure reported that the FDA had informed the company that data from its Phase I/II AMT-130 gene therapy trial were not sufficient to support a Biologics License Application, leaving the timing of any filing uncertain. This FDA feedback places greater emphasis on the robustness of uniQure’s clinical data packages and could reshape perceptions of its gene therapy pipeline. We’ll now examine how the FDA’s stance on AMT-130’s Phase I/II data influences uniQure’s investment...
Clinical Trial Holds Cast Doubt on Approval Chances of Regenxbio Gene Therapy for Rare Disease
MedCity News
2026-02-01
gene therapy
clinical hold
gene therapies
Tests of two Regenxbio gene therapies have been placed under an FDA clinical hold after a patient in one of the studies developed cancer. Our recap of recent regulatory news also includes one clinical hold removed, several complete response letters, and drug approvals in the U.S. and Europe. The post Clinical Trial Holds Cast Doubt on Approval Chances of Regenxbio Gene Therapy for Rare Disease appeared first on MedCity News .
FDA’s Multiple Myeloma Guidance Highlights Decade of Success
BioSpace
2026-01-30
multiple myeloma
New draft guidance from the FDA on multiple myeloma endpoints reflects the new technology available to assess disease and how patient journeys have changed with better treatments.
The week in pharma: action, reaction and insight – week to January 30, 2026
Kyowa Kirin
2026-01-30
ind
clinical hold
The US Food and Drug Administration (FDA) last week lifted the clinical hold on Intellia Therapeutics’ investigational new drug (IND) for its nexiguran ziclumeran (nex-z) for amyloidosis. The US Centers for Medicare and Medicaid Services (CMS) has released the names of 15 more drugs for a third cycle of Medicare price negotiations. On the deal-making front, Moderna signed a commercialization collaboration with Italy’s Recordati on mRNA-3927, and Japan’s Kyowa Kirin revealed that its partner Amgen has terminated the collaboration on dermatology candidate rocatinlimab.
Is BOND-003’s Phase 3 Progress Reshaping The Investment Case For CG Oncology (CGON)?
CG Oncology
2026-01-30
bladder cancer
CG Oncology has been gaining attention as its flagship BOND-003 therapy progresses through phase 3 trials for high-risk BCG-unresponsive non-muscle invasive bladder cancer, aiming to offer a bladder-sparing treatment option. The company’s focus on addressing this difficult-to-treat patient group highlights its potential role in reshaping how bladder cancer is managed if trial outcomes support its approach. Next, we’ll examine how the advanced phase 3 progress of BOND-003 shapes CG Oncology’s...
Adicet Bio to Present at the Guggenheim Emerging Outlook: Biotech Summit 2026
Adicet Bio
2026-01-30
allogeneic
cell therapies
REDWOOD CITY, Calif. --(BUSINESS WIRE)--Jan. 30, 2026-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced that Chen Schor, President and Chief Executive
Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2026-01-30
aav
ux111
gene therapy
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Base Editing Reverses Most Common Inherited Kidney Disease in Mice
Inside Precision Medicine
2026-01-30
base editing
A base editing therapy for autosomal dominant polycystic kidney disease (ADPKD) sets a new precedent for treating genetic conditions affecting the kidneys, which have historically proven difficult to target with this technology. The post Base Editing Reverses Most Common Inherited Kidney Disease in Mice appeared first on Inside Precision Medicine .
enGene initiated with a Buy at Jefferies
enGene Google
2026-01-30
bladder cancer
Jefferies initiated coverage of enGene (ENGN) with a Buy rating and $28 price target The company is running a pivotal high-risk non-muscle invasive bladder cancer s...
8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
SEC Filing (8-K)
2026-01-30
ultragenyx
Filed: 2026-01-30 AccNo: 0001193125-26-030590 Size: 155 KB Item 8.01: Other Events
Ultragenyx resubmits gene therapy for rare neuro disease to FDA
Endpoints News
2026-01-30
aav
ux111
gene therapy
Ultragenyx has resubmitted its approval request for a rare disease gene therapy after the FDA rejected it last summer. UX111 is an AAV gene therapy designed to treat Sanfilippo syndrome type A. The company is ...
I Peace Announces Successful Generation of Human iPS Cells from NKT Cells and Commences Their Availability
PR Newswire
2026-01-30
ipsc
cell therapy
cell therapies
PALO ALTO, Calif., Jan. 30, 2026 /PRNewswire/ -- Leading GMP cell therapy CDMO I Peace, Inc. (https://ipeace.com/en/), specializing in induced pluripotent stem cells (iPSCs) and iPSC-derived cell therapies, announced that it has successfully generated human induced pluripotent stem (iPS)...
[Ad hoc announcement pursuant to Art. 53 LR] Roche reports strong 2025 results with 7% sales growth
Roche Group
2026-01-29
multiple sclerosis
Group sales grew by 7%1 at constant exchange rates (CER; 2% in CHF), driven by strong demand for medicines and diagnostic solutions.Sales in the fourth quarter increased by 8%, reflecting the positive momentum.Pharmaceuticals Division sales increased by 9% (3% in CHF), with Phesgo (breast cancer), Xolair (food allergies), Ocrevus (multiple sclerosis), Hemlibra (haemophilia A) and Vabysmo (severe eye diseases) being the top growth drivers.Diagnostics Division sales grew 2% (-3% in CHF) as demand
Huntington's Groups Urge FDA to Review UniQure's Gene Therapy
uniQure
2026-01-29
amt-130
gene therapy
accelerated approval
UniQure had planned to seek accelerated approval for AMT-130 early 2026, but then said its plans were stalled after unexpected FDA feedback.
AstraZeneca Pledges $15B More in Chinese Investments for Cell Therapies, Radiopharma
BioSpace
2026-01-29
cell therapy
cell therapies
AstraZeneca has risen as one of pharma’s most prolific investors in China, including a $630 million pledge last week for full rights to AbelZeta’s cell therapy for cancer.
Adaptive Biotechnologies (ADPT) Is Up 5.3% After Surging Revenue And Genentech Tie-Up Spotlighted - Has The Bull Case Changed?
Genentech
2026-01-29
cell therapy
Adaptive Biotechnologies, a commercial-stage biotech focused on the adaptive immune system, recently highlighted very large year-on-year revenue growth alongside upcoming presentations at the BTIG and TD Cowen health care conferences. An important angle for investors is how this rapid revenue expansion, combined with its neoantigen T cell therapy collaboration with Genentech, is shaping perceptions of the company’s commercial and scientific progress. With recent share price moves, we’ll...
Roche earnings edge up 5% as forex effects weigh on drug sales growth
Roche Group
2026-01-29
multiple sclerosis
By Ludwig Burger FRANKFURT, Jan 29 (Reuters) - Roche's adjusted operating income rose by a lower-than-expected 5% in 2025 as gains in drugs such as Ocrevus against multiple sclerosis and Hemlibra against haemophilia were blunted by a weak U.S. dollar ...
Summit gets November PDUFA date for its PD-1xVEGF bispecific
Endpoints News
2026-01-29
pdufa
The leader in the next frontier of immuno-oncology officially has a date with the FDA. Summit Therapeutics announced Thursday morning that US regulators have accepted its submission package for the experimental drug ivonescimab, a bispecific ...
Roche earnings edge up 5% as forex effects weigh on drug sales growth
Roche Group
2026-01-29
multiple sclerosis
Roche's adjusted operating income rose by a lower-than-expected 5% in 2025 as gains in drugs such as Ocrevus against multiple sclerosis and Hemlibra against haemophilia were blunted by a weak U.S. dollar weighing on overseas sales.
LYMA REPORTS STRONG 2025 PERFORMANCE WITH $48M REVENUE AS US EXPANSION DRIVES GROWTH
PR Newswire
2026-01-29
financial results
NEW YORK, Jan. 29, 2026 /PRNewswire/ -- LYMA, the luxury regenerative MedTech company, today revealed its financial results for the year ending 31 December 2025, reporting revenues of $48 million and EBITDA of $7 million, marking a year of significant growth driven by successful expansion...
Otsuka preps for July decision on centanafadine for ADHD
PharmaPhorum
2026-01-29
priority review
The FDA has started a priority review of Otsuka's triple-acting drug for ADHD, setting up a decision on the first-in-class drug in July.
FDA Fast Track Accelerates Gamgertamig for ITP
Hematology Advisor News
2026-01-29
fast track
Ongoing multinational basket trials are enrolling patients with active ITP, relapsed/refractory AIHA, or both.
CRISPR Screen Highlights CHD1, MAP3K7 Loss as Immunotherapy Response Indicators
Inside Precision Medicine
2026-01-28
crispr
CRISPR-based functional genomics analysis revealed that reduced CHD1 and MAP3K7 expression enhances tumor sensitivity to immune checkpoint inhibitors, suggesting a way to better identify patients who may benefit from immunotherapy. The post CRISPR Screen Highlights CHD1 , MAP3K7 Loss as Immunotherapy Response Indicators appeared first on Inside Precision Medicine .
Advanced Therapies Week 2026: ‘Solving for Science’ and Weathering Global Complexity
BioSpace
2026-01-28
gene therapies
Phacilitate’s annual event dawns as cell and gene therapies reach a new tipping point: the science has hit new heights just as regulatory and government policies spark momentum and frustration.
BMS-Partnered Cellares Raises $257M for Cell Therapy Manufacturing Expansion
BioSpace
2026-01-28
cell therapy
Cellares, which last year became the first company to receive the FDA's new advanced manufacturing technology designation, expects to support clinical production this year and offer commercial-scale manufacturing services in 2027.
Zachary Roberts: Building a Viable Off-the-Shelf CAR T
Inside Precision Medicine
2026-01-28
car
cell therapies
Cancer immunotherapy’s rise—from checkpoint inhibitors to CAR T—shaped the Allogene Therapeutics chief medical officer's career, leading him to build off-the-shelf cell therapies to expand access for patients worldwide. The post Zachary Roberts: Building a Viable Off-the-Shelf CAR T appeared first on Inside Precision Medicine .
MRI-guided gene therapy targets serotonin in AADC deficiency
AADC News/BioNews
2026-01-28
kebilidi
gene therapy
Researchers have developed a real-time, MRI-guided method to deliver Kebilidi (eladocagene exuparvovec-tneq), an approved gene therapy for people with AADC deficiency, to multiple brain regions, according to a study in nonhuman primates (NHPs). AADC deficiency is characterized by low levels of dopamine and serotonin, two neurotransmitters. While current delivery of Kebilidi into the putamen or […] The post MRI-guided gene therapy targets serotonin in AADC deficiency appeared first on AADC News .
FDA Grants Fast Track Status to BNT113 for HPV16+ Head and Neck Cancer
Hematology Advisor News
2026-01-28
fast track
The FDA grants Fast Track designation to BNT113 for treating HPV16+ head and neck squamous cell carcinoma expressing PD-L1 in the first-line setting.
Roivant to Report Financial Results for the Fourth Quarter and Fiscal Year Ended March 31, 2025 and Provide Business Update on Thursday, May 29, 2025
roivant
2026-01-28
financial results
BASEL, Switzerland and LONDON and NEW YORK, May 15, 2025 (GLOBE NEWSWIRE) -- Roivant (Nasdaq: ROIV) today announced that it will host a live conference call and webcast at 8:00 a.m. ET on Thursday, May 29, 2025, to report its financial results for the fourth quarter and fiscal year ended March 31,
Cellares raises $257M to fund global expansion as it eyes 2027 IPO
Endpoints News
2026-01-28
cell therapy
Clinical-stage cell therapy manufacturer Cellares has raised $257 million in a Series D round as it gears up commercially and prepares to go public. Cellares aims to launch its commercial cell therapy manufacturing services in ...
Scenthound Reflects on Historic Year of Growth; Sets Sights on Continued Rapid Expansion and Innovation
PR Newswire
2026-01-28
fast track
Category Creator in Dog Wellness Remains on Fast Track for Accelerated Expansion Sustainable Franchise Growth Fuels Double-Digit Openings and 100+ Licenses Sold Technology Investments, Membership Growth and Corporate Team Expansion Take Center Stage in 2026 JUPITER, Fla., Jan. 28, 2026...
Lilly bets on Seamless, delving further into genetic medicine for hearing loss
Biopharma Dive
2026-01-28
gene editing
The potentially $1 billion alliance centered around a flexible gene editing technology adds to a portfolio of cutting-edge treatments the company is developing for hearing disorders.
iXCells and Rosebud Biosciences Partner on Organoid-Based Rare Disease Models
GenEng News
2026-01-28
ipsc
The combined workflow brings together iXCells’ scalable, modular iPSC platform, iPSCore, with Rosebud’s AI-driven organoid platform, designed to generate scalable, reproducible human tissue data for drug discovery and safety assessment. The post iXCells and Rosebud Biosciences Partner on Organoid-Based Rare Disease Models appeared first on GEN - Genetic Engineering and Biotechnology News .
Gilead Sciences to Release Fourth Quarter & Full Year 2025 Financial Results on Tuesday, February 10, 2026
Gilead Sciences
2026-01-28
financial results
Roche Makes Obesity Splash, Moderna Cans Vaccine Trials, Sarepta’s New Data, More
BioSpace
2026-01-28
gene therapy
duchenne muscular dystrophy
Roche’s obesity candidate achieves 22.5% weight loss in Phase II; Moderna pulls the plug on late-stage vaccine trials as Health Secretary Robert F. Kennedy’s anti-vaccine policies and rhetoric continues; and embattled gene therapy maker Sarepta announces new data in Duchenne muscular dystrophy.
8-K - REGENXBIO Inc. (0001590877) (Filer)
SEC Filing (8-K)
2026-01-28
regenxbio
Filed: 2026-01-28 AccNo: 0001193125-26-026139 Size: 205 KB Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
Immix Biopharma Receives U.S. FDA Breakthrough Therapy Designation for NXC-201
Immix Biopharma
2026-01-28
bla
nxc-201
– Breakthrough Therapy Designation granted to NXC-201 based on positive NEXICART-2 Phase 2 interim clinical results, presented at the American Society of Hematology (ASH) 2025 annual meeting – – Final data expected this year followed by planned BLA submission – LOS ANGELES, CA, Jan. 28, 2026 (GLOBE NEWSWIRE) — Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” […] The post Immix Biopharma Receives U.S. FDA Breakthrough Therapy Designation for NXC-201 first appeared on IMMIX BIOPHARMA INC .
FDA suspends two Regenxbio gene therapy trials after patient developed brain tumor
Endpoints News
2026-01-28
gene therapy
gene therapies
The FDA has placed studies of two gene therapies from Regenxbio on hold after a five-year-old patient in one of the trials was found to have a brain tumor. The drug regulator's decision is a ...
REGENXBIO Delay Could Put Denali in Pole Position for Hunter Syndrome Approval
BioSpace
2026-01-28
gene therapy
The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
Lilly partners with recombinase-based gene editing startup Seamless Therapeutics
Endpoints News
2026-01-28
gene editing
Seamless Therapeutics, a startup developing a new form of gene editing based on enzymes called recombinases, has struck a partnership with Eli Lilly that could be worth more than $1.12 billion, the company announced Wednesday ...
Lilly Leans Into Hearing Loss in $1.12B+ Gene Editing Pact With Seamless
BioSpace
2026-01-28
gene editing
gene therapy
The deal will help bolster Eli Lilly's growing hearing loss portfolio, which is anchored by the gene therapy AK-OTOF.
J&J’s Darzalex Faspro Notches Another Multiple Myeloma Indication, Pushing Into Frontline
BioSpace
2026-01-28
multiple myeloma
Darzalex Faspro, in combination with an anti-cancer triplet, is the first anti-CD38-based regimen for newly diagnosed patients with multiple myeloma, regardless of eligibility for stem cell transplantation.
Regenxbio gene therapy trials suspended by FDA over safety worries
Biopharma Dive
2026-01-28
gene therapy
The development of a brain tumor in a study participant led regulators to suspend a Hurler syndrome therapy in early testing and a treatment for Hunter syndrome nearing an FDA decision.
REGENXBIO Announces Regulatory Update on Ultra Rare MPS Programs
REGENXBIO
2026-01-28
gene therapy
clinical hold
ROCKVILLE, Md., Jan. 28, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) placed a clinical hold on its investigational gene therapy, RGX-111, for the treatment of MPS I, also known as Hurler syndrome, following preliminary analysis
Anchor Line Partners Supports Stoke Therapeutics' Growth by Securing a 98,500 Square-Foot Lease in Waltham, MA
PR Newswire
2026-01-27
dravet syndrome
New Headquarters Will Support Company's Work to Deliver zorevunersen, a Potential Disease-Modifying Medicine, to Patients with Dravet Syndrome and Expansion of Research Efforts Lease is One of the Largest in Greater Boston Life Sciences Over the Past Year WALTHAM, Mass., Jan. 27, 2026...
AI‑Designed Anti‑CRISPRs Enable Rapid Cas13 Inhibition
GenEng News
2026-01-27
crispr
AI‑designed anti‑CRISPR inhibitors rapidly block Cas13 activity and may offer a programmable way to regulate CRISPR systems. The approach highlights how AI‑accelerated protein design could expand future tools for safer, more controlled gene‑editing applications. The post AI‑Designed Anti‑CRISPRs Enable Rapid Cas13 Inhibition appeared first on GEN - Genetic Engineering and Biotechnology News .
FDA greenlights Life Biosciences' gene therapy study to rewind the age of cells
Endpoints News
2026-01-27
gene therapy
One of the biggest theories about the cause of aging, and how to slow and potentially even reverse it, is about to be put to the test. Life Biosciences, a biotech startup co-founded by Harvard ...
What’s next for cell and gene therapies
Prime Therapeutics
2026-01-27
gene therapies
In the latest episode of the “Pharmacy Friends” podcast, hear from Prime Therapeutics experts about how the fast-moving pipeline is changing how patients, providers and payers prepare for next-generation therapies.
Caregiving for Frontotemporal Dementia: What Families Need to Know
aviadobio
2026-01-27
frontotemporal dementia
The post Caregiving for Frontotemporal Dementia: What Families Need to Know appeared first on AviadoBio .
FDA Lifts One of Two Clinical Holds on Intellia’s ATTR Gene Editing Program
BioSpace
2026-01-27
crispr
gene editing
After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
Opus Genetics Launches Gene Therapy Clinical Trial for MERTK-related Retinitis Pigmentosa
opus genetics
2026-01-27
gene therapy
retinitis pigmentosa
Intellia Therapeutics Announces FDA Lift of Clinical Hold on MAGNITUDE-2 Phase 3 Clinical Trial in ATTRv-PN
Intellia Therapeutics Inc.
2026-01-27
gene editing
clinical hold
Plan to resume MAGNITUDE-2 patient enrollment and dosing FDA engagement ongoing regarding clinical hold on MAGNITUDE Phase 3 clinical trial in ATTR-CM CAMBRIDGE, Mass., Jan. 27, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused
8-K - Intellia Therapeutics, Inc. (0001652130) (Filer)
SEC Filing (8-K)
2026-01-27
intellia therapeutics
Filed: 2026-01-27 AccNo: 0001193125-26-023387 Size: 189 KB Item 7.01: Regulation FD Disclosure Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
FDA lifts hold on an Intellia CRISPR drug trial
Biopharma Dive
2026-01-27
crispr
Intellia is instituting new safeguards following the death of a study participant that led regulators to pause two trials in people with transthyretin amyloidosis. One of those tests remains suspended.
Gilead Sciences to Release Fourth Quarter & Full Year 2025 Financial Results on Tuesday, February 10, 2026
Gilead Sciences
2026-01-27
financial results
Gilead Sciences, Inc. (Nasdaq: GILD) announced today that its fourth quarter and full year 2025 financial results and guidance will be released on Tuesday, F...
CD20-Targeting Bispecific Antibodies May Be Effective in Patients With DLBCL in Real-World Settings
Hematology Advisor News
2026-01-27
cd20
Researchers determined epcoritamab and glofitamab may be effective in treating patients with relapsed or refractory DLBCL in the real-world setting.
FDA lifts hold on one of two Phase 3 gene editing studies by Intellia
Endpoints News
2026-01-27
gene editing
The FDA has allowed Intellia Therapeutics to resume one of its two pivotal trials of a gene editing therapy for transthyretin amyloidosis, which is a disease caused by misfolded proteins. Intellia
enGene: Two Major Catalysts On Deck For 2nd Half Of 2026
enGene Google
2026-01-27
bla
With $202.3M in cash and an expanded $125M debt facility, ENGN is financially equipped to advance pivotal Cohort 1 and pursue BLA filing. Read more on the stock here.
Anixa Biosciences Receives Notice of Allowance from Mexican Institute of Industrial Property (IMPI) for Patent Covering Breast Cancer Vaccine Technology
anixa biosciences
2026-01-27
car
cell therapies
Notice of Allowance marks first Mexican patent to be issued on Anixa Breast Cancer Vaccine Expands global intellectual property coverage in markets with greater late-stage breast cancer diagnoses and higher triple-negative breast cancer incidence rates SAN JOSE, Calif. , Jan. 27, 2026 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the Mexican Institute of Industrial Property (IMPI) has issued a Notice of Allowance for a new patent related to its breast cancer vaccine technology. This patent, exclusively licensed from Cleveland Clinic, will provide composition of matter protection for the Company's novel approach to breast cancer treatment and prevention in Mexico. With this allowance, Anixa continues to expand the international scope of its intellectual property portfolio, reinforcing its leadership in the field of cancer immunotherapy. The Mexican patent complements patents issued in the United States and other key global jurisdictions, and represents an important step toward future regulatory and commercial efforts outside the U.S. "This newly…
Trial of myeloma therapy KLN-1010 gets FDA green light to expand to US
Rare Cancer News
2026-01-27
multiple myeloma
A clinical trial in Australia that’s testing the genetic therapy KLN-1010 in people with hard-to-treat multiple myeloma has won regulatory approval to add new study sites in the U.S. The Phase 1 study, dubbed inMMyCAR (NCT07075185), will soon start enrolling patients in the U.S. It will be recruiting 40 adults with multiple myeloma that is relapsed […] The post Trial of myeloma therapy KLN-1010 gets FDA green light to expand to US appeared first on Rare Cancer News .
Reporter’s Notebook: Gene Editing’s Long Road from Theory to Therapy
Inside Precision Medicine
2026-01-27
gene editing
In these video interviews, leadership from Mammoth Biosciences, Caribou Biosciences, Stylus Medicines, and RheumaGen revealed the state of gene editing as therapeutics. The post Reporter’s Notebook: Gene Editing’s Long Road from Theory to Therapy appeared first on Inside Precision Medicine .
DARZALEX FASPRO®-based quadruplet regimen approved in the U.S. for newly diagnosed patients with multiple myeloma who are transplant ineligible
PR Newswire
2026-01-27
multiple myeloma
Quadruplet regimen demonstrated significantly deeper and more durable responses, higher MRD negativity and improved progression-free survival versus a standard of care Approval marks the twelfth indication for DARZALEX FASPRO® and fifth in the newly diagnosed setting, underscoring its...
Sarepta touts three-year Duchenne gene therapy data after patient deaths
Endpoints News
2026-01-26
gene therapy
duchenne muscular dystrophy
Sarepta said the effects of its Duchenne muscular dystrophy gene therapy were durable three years after treatment, based on longer-term findings from patients in its key clinical trial. The data come after a tumultuous year ...
Sarepta Announces Positive Topline Three-Year EMBARK Results Showing ELEVIDYS Significantly Slows Disease Progression on Key Functional Measures in Ambulatory Duchenne Patients
Sarepta Therapeutics
2026-01-26
elevidys
gene therapy
A t a mean age of 9 years old, ELEVIDYS-treated patients achieved mean North Star Ambulatory Assessment (NSAA) scores above baseline three years after treatment (n=52) ELEVIDYS gene therapy demonstrated 70% or greater reduction in the rate of decline relative to the propensity-weighted external
Sofi-cel gets FDA breakthrough status for aggressive blood cancers
Rare Cancer News
2026-01-26
cell therapy
acute lymphoblastic leukemia
The U.S. Food and Drug Administration (FDA) has granted breakthrough designation to soficabtagene geleucel (sofi-cel), a cell therapy designed to treat relapsed or refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma (T-ALL/LBL), blood cancers caused by the uncontrolled growth of immune cells called T-cells. The FDA grants breakthrough designation to therapies that have the […] The post Sofi-cel gets FDA breakthrough status for aggressive blood cancers appeared first on Rare Cancer News .
Rare Disease Biotechs Stand To Lose $4B if Priority Voucher Program Not Reinstated: Report
BioSpace
2026-01-26
priority review
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Sarepta’s DMD Gene Therapy Staves Off Disease Three Years After Treatment
BioSpace
2026-01-26
gene therapy
After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
InventHelp Inventor Develops New Type of Exerciser (SGM-608)
PR Newswire
2026-01-26
ind
PITTSBURGH, Jan. 26, 2026 /PRNewswire/ -- "I thought there should be a way to exercise the core and the upper and lower body using one piece of equipment," said an inventor, from Indianapolis, Ind., "so I invented the 2 STEP. My design enables you to engage in a variety of...
The crucial role of raw material selection for success in cell therapy manufacturing
Biopharma Dive
2026-01-26
cell therapy
Why raw materials make or break cell therapy manufacturing success.
InventHelp Inventors Develop Protective Cover for Wounds while Showering (SGM-625)
PR Newswire
2026-01-26
ind
PITTSBURGH, Jan. 26, 2026 /PRNewswire/ -- "We thought there could be a better way to keep a surgical wound dry while showering," said one of two inventors, from Valpo, Ind., "so we invented the AQUA SLEEVE. Our protective design prevents water from coming into contact with the surgical...
FDA-Approved WAS Gene Therapy Hits Market in Nonprofit Model
Fondazione Telethon
2026-01-26
gene therapy
Fondazione and Orphan Therapeutics outline a US access model for Waskyra, highlighting new approaches to manufacturing, distribution, and sustainability.
Early Promise for Lexeo’s Connexin-43 Cardiomyopathy Gene Therapy
Inside Precision Medicine
2026-01-26
aav
gene therapy
arrhythmogenic cardiomyopathy
An adeno-associated-viral (AAV) vector gene therapy that can restore connexin-43 to the heart could be a good treatment for people with arrhythmogenic cardiomyopathy, suggest preclinical results. The post Early Promise for Lexeo’s Connexin-43 Cardiomyopathy Gene Therapy appeared first on Inside Precision Medicine .
Autolus (NASDAQ: AUTL) signs 10-year AGC Biologics lentiviral supply pact
Autolus Therapeutics
2026-01-26
lentiviral
Resale prospectus supplement for 54.6M ADSs adds details on a 10-year AGC Biologics supply deal with minimum EUR 25M lentiviral vector purchases.
8-K - BIOMARIN PHARMACEUTICAL INC (0001048477) (Filer)
SEC Filing (8-K)
2026-01-26
biomarin pharmaceutical
Filed: 2026-01-26 AccNo: 0001193125-26-021718 Size: 573 KB Item 2.02: Results of Operations and Financial Condition Item 7.01: Regulation FD Disclosure Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
Immusoft Receives FDA Rare Pediatric Disease Designation for ISP-002 to treat Mucopolysaccharidosis Type II
Immusoft Corporation
2026-01-26
cell therapies
mucopolysaccharidosis
/PRNewswire/ -- Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, today announced that the U.S. Food and Drug...
Catalent plans to close Belgium cell therapy site
Endpoints News
2026-01-26
cell therapy
Catalent plans to close a cell therapy site in Gosselies, Belgium, due to “changing market dynamics and customer needs,” a company spokesperson confirmed to Endpoints News. The contract developer and manufacturer is considering transitioning ...
Making ends meet with multiple myeloma
Rare Cancer News
2026-01-26
multiple myeloma
Today, Jan. 26, is the first day of tax season here in the U.S. Last week, our accountant emailed her annual reminder to collect the many forms that go with our tax return, along with documentation of expenses. So I have to do something I’ve been putting off: the annual update of the medical expense […] The post Making ends meet with multiple myeloma appeared first on Rare Cancer News .
Autolus Therapeutics Signs Long-Term Lentiviral Supply Agreement
Autolus Therapeutics
2026-01-26
lentiviral
Autolus Therapeutics ( ($AUTL) ) has provided an update. On January 21, 2026, Autolus Limited, a subsidiary of Autolus Therapeutics, entered into a new ten-year Mas...
China’s edge in early-stage drugmaking ‘likely to persist,’ Pitchbook says
Biopharma Dive
2026-01-26
gene therapies
The ongoing surge in licensing deals for early drug prospects should continue this year and involve more cell and gene therapies, which have had trouble securing funding in the U.S., the firm said in a new report.
CNI vs Belimumab for Lupus Nephritis May Carry Higher Cancer Risk
Hematology Advisor News
2026-01-26
lupus
Investigators compared the risk of cancer onset in patients with lupus nephritis treatedwith calcineurin inhibitors vs belimumab.
Real World Experience with Ryoncil Shows 84% Early Survival
Mesoblast
2026-01-26
ryoncil
Sarepta, battling slowing sales, claims Duchenne gene therapy’s impact grows with time
Biopharma Dive
2026-01-26
elevidys
gene therapy
On a Monday conference call, Sarepta CEO Doug Ingram said new long-term data should help “rebalance the discussion” surrounding Elevidys.
Eisai and Biogen’s subcutaneous Leqembi given FDA Priority Review for early Alzheimer’s
PMLive Regulatory
2026-01-26
priority review
Administering the drug subcutaneously rather than intravenously showed similar clinical benefits for patients
FDA Approves Waskyra as First Gene Therapy for Wiskott-Aldrich Syndrome
CGTLive
2026-01-25
gene therapy
wiskott-aldrich
etuvetidigene autotemcel
wiskott-aldrich syndrome
The Fondazione Telethon agent, etuvetidigene autotemcel, significantly reduced severe infections and bleeding events in clinical studies.
QURE Investigation Reminder: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2026-01-24
bla
amt-130
gene therapy
RADNOR, PA - January 24, 2026 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Real-World Use of Efanesoctocog Alfa Shows Benefit in Severe Hemophilia A
Hematology Advisor News
2026-01-23
hemophilia a
Efanesoctocog alfa improves hemostasis, reduces bleeding, and enhances quality of life for patients with severe hemophilia A, including those previously excluded from trials.
Roivant to Report Financial Results for the Third Quarter Ended December 31, 2025, and Provide Business Update on Friday, February 6, 2026
roivant
2026-01-23
financial results
BASEL, Switzerland and LONDON and NEW YORK, Jan. 23, 2026 (GLOBE NEWSWIRE) -- Roivant (Nasdaq: ROIV) today announced that it will host a live conference call and webcast at 8:00 a.m. ET on Friday, February 6, 2026, to report its financial results for the second quarter ended December 31, 2026, and
Sarepta Therapeutics to Report 3-Year Topline Data from EMBARK Study of ELEVIDYS Gene Therapy in Ambulatory Individuals with Duchenne Muscular Dystrophy
Sarepta Therapeutics
2026-01-23
elevidys
gene therapy
duchenne muscular dystrophy
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jan. 23, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Jan. 26, 2026 , at 8:30 am Eastern Time , the Company will host a webcast and conference call to present 3-year
Spending Bill Clears House, Keeping Priority Review Voucher Hopes Alive
BioSpace
2026-01-23
priority review
The $1.2 trillion budget package will now move to the Senate, which is expected to hold a vote next week.
8-K - Ocugen, Inc. (0001372299) (Filer)
SEC Filing (8-K)
2026-01-23
ocugen
Filed: 2026-01-23 AccNo: 0001104659-26-006031 Size: 213 KB Item 7.01: Regulation FD Disclosure Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
ImmunityBio Reports Median Overall Survival Not Yet Reached and Lymphopenia Reversed in Recurrent Glioblastoma Patients Receiving ANKTIVA® Plus CAR-NK, Chemo-Free Therapy
Immunity Bio
2026-01-23
car
car-nk
The post ImmunityBio Reports Median Overall Survival Not Yet Reached and Lymphopenia Reversed in Recurrent Glioblastoma Patients Receiving ANKTIVA® Plus CAR-NK, Chemo-Free Therapy appeared first on ImmunityBio .
Macrophage-Targeting CAR T Cell Therapy Improves Survival in Solid Tumor Mouse Models
GenEng News
2026-01-23
car
cell therapy
Experimental CAR T cell immunotherapy targeting tumor-associated macrophages promoted tumor clearance and increased survival in preclinical models of metastatic ovarian and lung cancer, pointing to a new potential strategy for treating advanced-stage solid tumors. The post Macrophage-Targeting CAR T Cell Therapy Improves Survival in Solid Tumor Mouse Models appeared first on GEN - Genetic Engineering and Biotechnology News .
Milestone Reached in Cell and Gene Therapy Robotic Automation and Manufacturing Collaboration
GenEng News
2026-01-23
gene therapy
gene therapies
It has been widely known that manufacturing costs and variability are major challenges to the widespread adoption of cell and gene therapies. These challenges have limited broader access to advanced therapies. The post Milestone Reached in Cell and Gene Therapy Robotic Automation and Manufacturing Collaboration appeared first on GEN - Genetic Engineering and Biotechnology News .
Bladder Cancer Vaccines Market Poised for Rapid Growth at 21.32% CAGR Amid BCG Innovation & Immunotherapy Brea
CG Oncology
2026-01-23
bladder cancer
The global bladder cancer vaccines market reached US 254 million in 2024 up from US 209 million in 2023 and is projected to reach US 1 446 million by 2033 growing at a robust CAGR of 21 32 from 2025 ...
Rare Disease Vouchers Caught in Political Abyss. It Didn’t Have To Be This Way
BioSpace
2026-01-23
priority review
The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
In HelloNation, Personal Injury Lawyer Celeste & Selina Padilla Share Insights on What to Do After a Car Accident in McAllen
PR Newswire
2026-01-23
car
MCALLEN, Texas, Jan. 23, 2026 /PRNewswire/ -- What should someone do immediately after a car accident in McAllen? Celeste and Selina Padilla of Padilla Law Firm, PLLC address this question in HelloNation, offering a clear sequence of actions designed to protect both personal well-being...
NKGen Biotech Names Leading Dementia Expert Bruce L. Miller, M.D. to Scientific Advisory Board
nkgen biotech
2026-01-22
allogeneic
autologous
Dr. Miller’s appointment further enhances NKGen’s scientific and clinical advisory capabilities as the Company advances its research in Alzheimer’s and other related neurodegenerative diseases. SANTA ANA, Calif., January 22, 2026 – NKGen Biotech, Inc. (OTC: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic […] The post NKGen Biotech Names Leading Dementia Expert Bruce L. Miller, M.D. to Scientific Advisory Board first appeared on NKGen Biotech .
Therapies to Watch in 1H 2026
CGTLive
2026-01-22
gene therapy
We highlighted a few investigational cell and gene therapy candidates to keep an eye on in the first half of the new year.
NICE backs Roche lupus drug that supports kidney function
PharmaPhorum
2026-01-22
lupus
Roche's Gazyvaro, a drug that can restore kidney function in lupus nephritis, has been cleared for use by the NHS in England and Wales.
FDA lays out new path to speed development of multiple myeloma drugs
Biopharma Dive
2026-01-22
cell therapies
multiple myeloma
In certain cases, regulators will now be open to clearing therapies based on their ability to eliminate tumor cells, a change that could accelerate cell therapies and other new medicines.
Reducing Costs of Goods in Cell and Gene Therapy
CGTLive
2026-01-22
gene therapy
Deborah Phippard, PhD, shared her thoughts with Renier Brentjens, MD, PhD, on cost reduction for cell and gene therapy products.
Ultragenyx Finishes Rolling Submission of BLA for Glycogen Storage Disease Gene Therapy DTX401
CGTLive
2026-01-22
bla
dtx401
gene therapy
The BLA is supported by 96-week data from the randomized, placebo-controlled phase 3 GlucoGene clinical trial.
Rutgers and Iovance Assess E7 TCR T-Cell Therapy in Phase 1/2 Trial for HPV-Associated Tumors
CGTLive
2026-01-22
tcr t
phase 1/2
cell therapy
Future Predictions for the Field of Cell and Gene Therapy
CGTLive
2026-01-22
gene therapy
Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, discussed their thoughts on the field's future.
New platform aims for personalized gene editing for rare diseases
AADC News/BioNews
2026-01-21
crispr
gene editing
Aurora Therapeutics has launched a personalized gene-editing platform to develop CRISPR-based therapies — ones that precisely correct disease-causing genetic mutations — for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency. The launch was supported by $16 million in seed funding from Menlo Ventures, a California-based company that works with early-stage founders. The tech company was founded […] The post New platform aims for personalized gene editing for rare diseases appeared first on AADC News .
The Bull Case For uniQure (QURE) Could Change Following FDA Pushback On AMT-130 Evidence Standards
uniQure
2026-01-21
amt-130
gene therapies
accelerated approval
In November 2025, uniQure disclosed that the FDA found data from its Phase I/II AMT-130 Huntington’s disease studies insufficient to support a Biologics License Application, and later feedback questioned the adequacy of its external-control dataset for accelerated approval. This setback highlights how emerging regulatory expectations around evidence standards can materially affect the development path of gene therapies even when early efficacy signals appear encouraging. We’ll now examine...
enGene Expands Hercules Debt Facility To $125 Million
enGene Google
2026-01-21
bladder cancer
detalimogene voraplasmid
enGene Holdings, a clinical-stage developer of non-viral genetic medicines, said it has expanded its debt facility with Hercules Capital to up to $125 million, adding what the company described as non-dilutive financing as it prepares for a planned U.S. regulatory submission for its lead program, detalimogene voraplasmid, in high-risk, BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ.
The Implications of the Final Data From Hemgenix’s HOPE-B Trial
CGTLive
2026-01-21
hemgenix
gene therapy
hemophilia b
Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, also discussed open questions that remain for the future of the hemophilia B gene therapy.
8-K - enGene Holdings Inc. (0001980845) (Filer)
SEC Filing (8-K)
2026-01-21
engene
Filed: 2026-01-21 AccNo: 0001193125-26-017226 Size: 250 KB Item 7.01: Regulation FD Disclosure Item 9.01: Financial Statements and Exhibits
enGene Announces Expanded $125 Million Debt Facility with Hercules Capital, Inc.
enGene Google
2026-01-21
bladder cancer
Up to $100 million in additional loan proceeds provide additional financial flexibility to advance detalimogene development and commercialization for bladder cancer$25 million advanced at close of the amendment to refinance existing debt facilityBOSTON & MONTREAL--(BUSINESS WIRE)--enGene Holdings...
Around the Helix: Cell and Gene Therapy Company Updates – January 7, 2026
CGTLive
2026-01-21
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Sofetabart mipitecan for gynecological cancers earns FDA breakthrough status
Rare Cancer News
2026-01-21
breakthrough therapy
The U.S. Food and Drug Administration (FDA) has granted its breakthrough therapy designation to sofetabart mipitecan as a potential treatment for people with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received prior bevacizumab (sold as Avastin, among others) and Elahere (mirvetuximab soravtansine), if eligible. The FDA gives this designation to experimental […] The post Sofetabart mipitecan for gynecological cancers earns FDA breakthrough status appeared first on Rare Cancer News .
Around the Helix: Cell and Gene Therapy Company Updates – January 21, 2026
CGTLive
2026-01-21
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
ImmunityBio rises as route opens to broader Anktiva label
PharmaPhorum
2026-01-21
bladder cancer
Shares in ImmunityBio shot up after it agreed a path with the FDA that should allow it to refile for wider use of its bladder cancer drug Anktiva.
The Impact of Artificial Intelligence on Cell and Gene Therapy
CGTLive
2026-01-21
gene therapy
Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, spoke about the potential of AI to affect development and use of cell and gene therapy products.
8-K - Ocugen, Inc. (0001372299) (Filer)
SEC Filing (8-K)
2026-01-21
ocugen
Filed: 2026-01-21 AccNo: 0001104659-26-005454 Size: 535 KB Item 1.01: Entry into a Material Definitive Agreement Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
JPM 2026: Aurora Therapeutics’ Edward Kaye on Scalable Personalized Gene Editing
Inside Precision Medicine
2026-01-21
gene editing
The CEO has been tasked with bringing Jennifer Doudna and Fyodor Urnov's scientific mission to fruition in the clinic at the newly launched company. The post JPM 2026: Aurora Therapeutics’ Edward Kaye on Scalable Personalized Gene Editing appeared first on Inside Precision Medicine .
J&J praises new FDA guidance to speed development of multiple myeloma drugs
Endpoints News
2026-01-21
multiple myeloma
Johnson & Johnson, which has several drugs approved for multiple myeloma, praised the FDA's just-released draft guidance on how the agency will allow the use of minimal residual disease as a primary endpoint for accelerated ...
Moderna cancer vaccine holds up; IO Biotech considers a sale
Biopharma Dive
2026-01-21
melanoma
Long-term data suggest Moderna’s personalized melanoma shot provides “durable tumor control,” one analyst said. Elsewhere, Insilico Medicine partnered with a startup on a popular drug target.
U.S. FDA Grants to Wugen’s WU-CART-007 Breakthrough Therapy Designation for Treatment of Relapsed or Refractory T Cell Acute Lymphoblastic Leukemia / T Cell Lymphoblastic Lymphoma
Wugen Inc.
2026-01-21
car
allogeneic
wu-cart-007
breakthrough therapy
acute lymphoblastic leukemia
— Breakthrough Therapy Designation granted to WU-CART-007 (Soficabtagene Geleucel “Sofi-cel”), an allogeneic CAR-T therapy for the treatment of T cell malignancies — Pivotal Study of Sofi-cel for Relapsed or Refractory T-Cell ALL/LBL in Pediatric and Adult Patients is ongoing — Company to Initiate an exploratory cohort of Sofi-cel in patients with Minimal Residual Disease ... The post U.S. FDA Grants to Wugen’s WU-CART-007 Breakthrough Therapy Designation for Treatment of Relapsed or Refractory T Cell Acute Lymphoblastic Leukemia / T Cell Lymphoblastic Lymphoma appeared first on Wugen .
Data builds behind Moderna's skin cancer vaccine
PharmaPhorum
2026-01-21
melanoma
Five-year results with Moderna/MSD's melanoma jab show a 49% reduction in disease progression or death, boding well for phase 3 data later this year.
Moderna and Merck announce data from melanoma combination therapy study
PMLive Research
2026-01-21
melanoma
Skin cancer is one of the most common cancers in the US, with melanoma accounting for the majority of skin cancer deaths
Around the Helix: Cell and Gene Therapy Company Updates – December 17, 2025
CGTLive
2026-01-21
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
After Cell Therapy Retreat, Novo Offloads Technologies in Deepened Aspect Pact
BioSpace
2026-01-21
cell therapy
Novo Nordisk pulled back from cell therapies last October, scrapping development of a type 1 diabetes therapy and laying off most employees working on this modality.
Legend Biotech to Present CARVYKTI® Data at 2026 Tandem Meetings Reinforcing Growing Evidence Supporting Earlier Use
Legend Biotech
2026-01-21
carvykti
Around the Helix: Cell and Gene Therapy Company Updates – January 14, 2026
CGTLive
2026-01-21
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Steven W. Pipe, MD, on Integrating Gene Therapy into Clinical Care for Hemophilia B
CGTLive
2026-01-21
gene therapy
hemophilia b
Medlive Expands Advisory Board with Three Leading Oncology Experts
PR Newswire
2026-01-21
multiple myeloma
Appointments strengthen the company's commitment to evidence-based continuing medical education across hepatobiliary cancers, multiple myeloma, and thoracic oncology BOSTON, Jan. 21, 2026 /PRNewswire/ -- Medlive, a provider of trusted education from leading clinician associations and...
Arcellx to Advance the Conversation on Its Platform and the Importance of Early CAR T Access for Patients with Multiple Myeloma During the 2026 Tandem Meetings
arcellx, inc
2026-01-21
car
cell therapy
multiple myeloma
The D-Domain binder has a fast off-rate and contributes to anito-cel’s differentiated pharmacology profile in multiple myeloma - - A simulation model of CAR T use ahead of bispecific antibodies in 4L+ RRMM is estimated to reduce the risk of progression or death by 64% and death by 48% over five years versus the reverse sequence, supporting CAR T before bispecific antibody as the sequence that delivers superior improved long-term outcomes in 4L+ RRMM - - Geographic inequities persist in CAR T access for MM patients marked by long travel, out-of-state care, and bypassed ATCs, highlighting systemic referral gaps and the need for ATC expansion to improve equitable access - Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, is advancing both its scientific foundation for its D-Domain platform technology and commercial preparedness for anito-cel for the
UK and Japan strengthen ties with gene therapy investment
Kyowa Kirin
2026-01-20
gene therapy
GENE therapy development is set for a funding boost as the UK and Japan expand their trade partnership in the life sciences sector.
Topline results: Antibody therapy lowers inflammatory biomarkers in cystic fibrosis
Healio
2026-01-20
cf
cystic fibrosis
In patients with cystic fibrosis, CMTX-101, an immune-enabling antibody therapy, lowered four inflammatory biomarkers and Pseudomonas aeruginosa burden vs. standard of care, according to phase 1b/2a trial topline results.“CMTX-101 could represent an important new tool for those who treat people with [cystic fibrosis (CF)] and other bronchiectatic lung diseases,” David V. Richards, JD,
Komzifti Gains FDA Approval, Giving Kura Oncology and Kyowa Kirin a Key Strategic Win in NPM1-Mutated AML
Kyowa Kirin
2026-01-20
aml
Approval of Komzifti (ziftomenib) provides a new targeted option for a high-risk patient population and strengthens the Kura Oncology–Kyowa Kirin collaboration, which includes a global development and commercialization strategy.
Astellas Takes Aim at XLMTM Again, Now with MyoAAV Capsid-Based Gene Therapy
GenEng News
2026-01-20
aav
gene therapy
The therapy, which Kate calls KT430, is being developed by Astellas through an exclusive license of undisclosed value under the name ASP2957 (MyoAAV3.8-MHCK7-hMTM1). ASP2957 is an investigational, nonreplicating recombinant adeno-associated virus (AAV) with a Kate-engineered capsid that contains a DNA sequence of the human MTM1 gene under the control of an MHCK7 muscle-specific promoter. The post Astellas Takes Aim at XLMTM Again, Now with MyoAAV Capsid-Based Gene Therapy appeared first on GEN - Genetic Engineering and Biotechnology News .
ImmunityBio Advances Regulatory Discussions with FDA on Potential Resubmission Path for ANKTIVA® in BCG-Unresponsive Papillary Bladder Cancer
Immunity Bio
2026-01-20
bladder cancer
The post ImmunityBio Advances Regulatory Discussions with FDA on Potential Resubmission Path for ANKTIVA® in BCG-Unresponsive Papillary Bladder Cancer appeared first on ImmunityBio .
AGC Biologics powers commercial launch of its life-changing gene therapy for rare childhood immune disorder
Fondazione Telethon
2026-01-20
waskyra
lentiviral
gene therapy
ex vivo gene therapy
Waskyra is an ex vivo gene therapy that uses patients’ own CD34+ hematopoietic stem and progenitor cells, genetically engineered with a lentiviral vector
Waters Corporation Schedules Fourth Quarter 2025 Earnings Conference Call
PR Newswire
2026-01-20
financial results
MILFORD, Mass., Jan. 20, 2026 /PRNewswire/ -- Waters Corporation (NYSE:WAT) will hold its Q4 2025 financial results conference call on Thursday, February 12th, 2026 at 8:00 a.m. Eastern Time. A live webcast of the presentation will be available on Waters Investor Relations website at...
Genentech More than Doubles Investment in Holly Springs, North Carolina Manufacturing Facility
Genentech
2026-01-20
car
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced an expansion of its initial investment in a new Holly Springs, North Car...
AstraZeneca Pays $630M for Complete Global Rights Over AbelZeta’s CAR T
BioSpace
2026-01-20
car t
cell therapy
The arrangement will boost AstraZeneca’s cell therapy portfolio as the pharma targets $80 billion in revenue by 2030.
What the Field of Cell and Gene Therapy Needs Most in the Current Moment
CGTLive
2026-01-20
gene therapy
Deborah Phippard, PhD, shared her thoughts with Renier Brentjens, MD, PhD, on the importance of big wins for those in research.
AstraZeneca pays $630m to get full control of AbelZeta CAR-T
PharmaPhorum
2026-01-20
car-t
AstraZeneca's push into CAR-T therapies for cancer has continued with an agreement to take full control of C-CAR031 from Chinese partner AbelZeta.
Biotech enGene secures $125M to advance bladder cancer treatment detalimogene
enGene Google
2026-01-20
bla
bladder cancer
Non-dilutive facility adds $100M in future borrowing plus $25M to refinance debt, supporting enGene’s 2026 FDA BLA plans and possible 2027 launch.
FDA Hits Atara Biotherapeutics’ T-Cell Immunotherapy Tabelecleucel for EBV+ PTLD With Second CRL
CGTLive
2026-01-20
tabelecleucel
The CRL indicates that the ALLELE clinical trial does not sufficiently show efficacy of the product.
Genezen and Atsena Therapeutics Announce Strategic Commercial Manufacturing Partnership
atsena therapeutics
2026-01-20
gene therapy
Lexington, MA and Durham, NC – January 20, 2026 – Genezen, a leading gene therapy contract development and manufacturing organization (CDMO), and Atsena Therapeutics (“Atsena”), ... The post Genezen and Atsena Therapeutics Announce Strategic Commercial Manufacturing Partnership appeared first on Atsena Therapeutics .
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA Following FDA Review of HOPE-3 Topline Data
Capricor Therapeutics
2026-01-20
bla
deramiocel
Subspecialist second opinions for cancer care can help patients save thousands in costs
Healio
2026-01-20
multiple myeloma
Second opinions on cancer treatment plans can save thousands of dollars in health care costs.A retrospective analysis showed subspecialists who changed care for newly diagnosed multiple myeloma , colorectal, head and neck, and lung cancers decreased costs an average of $15,000 per patient.
Top FDA Gene and Cell Therapy News: 2025 Year-End Recap
CGTLive
2026-01-20
cell therapy
gene therapy
Recapping some the FDA approvals and regulatory updates in cell and gene therapy that made waves in 2025.
Aspect Bio takes Novo castoff therapies; Takeda trims US jobs
Endpoints News
2026-01-20
cell therapies
Plus, news about AstraZeneca, AbelZeta, Novavax, Pfizer, Alteogen, Tesaro, Epidarex Capital and Boundless Bio: 🤝 Aspect Biosystems picks up Novo Nordisk’s castoff cell therapies: The Vancouver biotech has taken over stem cell-derived islet cell and ...
Vertex to Announce Fourth Quarter and Full Year 2025 Financial Results on February 12th
Vertex
2026-01-20
financial results
BOSTON --(BUSINESS WIRE)--Jan. 20, 2026-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its fourth quarter and full year 2025 financial results on Thursday, February 12, 2026 , after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .
FDA Decisions to Look For in 1H 2026
CGTLive
2026-01-20
gene therapies
We highlighted a few cell and gene therapies that are nearing or likely nearing key FDA decisions in the first half of 2026.
Research Assistant Analytics
coave therapeutics
2026-01-20
aav
To strengthen our analytical team involved in the development of assays and the characterization of innovative AAV conjugate products, we are looking for a research assistant, preferably with experience in biotechnology. Knowledge of AAV would be a plus.
Sangamo Starts Putting BLA for Fabry Disease Gene Therapy Isa-Vec in Front of FDA
CGTLive
2026-01-20
bla
phase 1/2
gene therapy
fabry disease
The rolling BLA is supported by data from the phase 1/2 STAAR clinical trial (NCT04046224).
enGene Secures Expanded $125 Million Debt Facility to Advance Bladder Cancer Gene Therapy
enGene Google
2026-01-20
gene therapy
bladder cancer
enGene Holdings ( ($ENGN) ) has provided an announcement. enGene has expanded its debt facility with Hercules Capital to up to $125 million, securing additional non...
Ocugen Posts Positive Preliminary Phase 2 ArMaDa Trial Data of OCU410
CGTLive
2026-01-20
gene therapy
Ocugen's OCU410 gene therapy shows promising results in reducing lesion growth for geographic atrophy, potentially transforming treatment for retinal health.
Nanoscope Secures Japan MHLW Sakigake and Orphan Drug Designations Across Inherited Retinal Diseases – A First for a Retinal Gene Therapy
Nanoscope Therapeutics
2026-01-20
mco-010
gene therapy
Japan’s Ministry of Health, Labor and Welfare (MHLW) grants MCO-010 Pioneering Regenerative Medical Product (Sakigake) and Orphan Drug designations, enabling an accelerated regulatory pathway to treat patients with severe vision loss from inherited retinal diseases Builds on strong global regulatory momentum, including five EMA Orphan designations and multiple FDA expedited programs across retinal indications FDA, EMA […] The post Nanoscope Secures Japan MHLW Sakigake and Orphan Drug Designations Across Inherited Retinal Diseases – A First for a Retinal Gene Therapy appeared first on Nanoscope Therapeutics .
QURE Investigation Notice: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2026-01-19
bla
amt-130
gene therapy
RADNOR, PA - January 19, 2026 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Novartis’ ianalumab receives FDA Breakthrough Therapy designation for Sjögren’s disease
PMLive Regulatory
2026-01-19
ianalumab
breakthrough therapy
Approximately half of those with Sjögren’s disease are thought to be undiagnosed
Steven W. Pipe, MD, on the Implications of the Final Data From Hemgenix’s HOPE-B Trial
CGTLive
2026-01-19
hemgenix
Researchers say multiple myeloma patients are living longer
Rare Cancer News
2026-01-19
multiple myeloma
Here’s a ray of hope from the American Cancer Society (ACS): Its recently released annual Cancer Statistics report noted that the five-year relative cancer survival rate for U.S. patients is now 70%. For multiple myeloma patients, that rate has nearly doubled since the 1990s, from 32% to 62%! So what does that mean? The U.S. […] The post Researchers say multiple myeloma patients are living longer appeared first on Rare Cancer News .
EU starts review of first-line Enhertu for breast cancer
PharmaPhorum
2026-01-19
breast cancer
EMA starts its review of first-line Enhertu in HER2+ breast cancer, which could dramatically increase the eligible patient population for the drug.
AGC Biologics Celebrates U.S. and EU Approval of Fondazione Telethon’s Waskyra™ Treatment for Wiskott-Aldrich Syndrome
Fondazione Telethon
2026-01-19
waskyra
wiskott-aldrich
wiskott-aldrich syndrome
NICE relents, ending postcode access to prostate cancer drug
PharmaPhorum
2026-01-19
prostate cancer
Men in England with high-risk prostate cancer will soon be able to get treatment with a drug available in Scotland and Wales for almost three years.
FDA Grants Regenerative Medicine Advanced Therapy (RMAT) Designation to iRegene's NouvNeu001, Making It the World's First iPSC Therapy with Both FTD and RMAT Recognitions
PR Newswire
2026-01-19
cell therapy
parkinson's disease
Designation underscores potential of chemically induced, off-the-shelf cell therapy to address high unmet need in Parkinson's disease, following compelling Phase I clinical data CHENGDU, China, Jan. 19, 2026 /PRNewswire/ -- iRegene Therapeutics Co., Ltd. ("iRegene" or the "Company"), a...
StockWatch: Sarepta CEO Defends Elevidys as Q4 Sales Fall Short
GenEng News
2026-01-18
dmd
sarepta
elevidys
gene therapy
duchenne muscular dystrophy
Sarepta Therapeutics saw its stock skid 11% after the company announced preliminary unaudited 2025 sales results that missed analyst forecasts for its marketed Elevidys® (delandistrogene moxeparvovec-rokl), which reignited the longtime issue of gene therapy safety following the deaths of three Duchenne muscular dystrophy (DMD) patients last year. The post StockWatch: Sarepta CEO Defends Elevidys as Q4 Sales Fall Short appeared first on GEN - Genetic Engineering and Biotechnology News .
Assessing CG Oncology (CGON) Valuation After Accelerated PIVOT-006 Phase 3 Trial Timeline
CG Oncology
2026-01-18
bladder cancer
CG Oncology (CGON) is back in focus after accelerating the topline data timeline for its Phase 3 PIVOT-006 trial, the first randomized study for intermediate-risk non muscle invasive bladder cancer patients. See our latest analysis for CG Oncology. The accelerated PIVOT-006 timeline sits against a strong recent run, with a 30 day share price return of 40.23% and a 1 year total shareholder return of 88.20%, suggesting momentum has been building around the story. If CG Oncology’s progress has...
Guess the Therapy Answer January 14, 2026
CGTLive
2026-01-17
gene therapy
Cell and Gene Therapy Live
Is BioMarin (BMRN) Elevating Digital Strategy To Sharpen Its Innovation And Efficiency Edge?
BioMarin Pharmaceutical
2026-01-17
bristol myers squibb
biomarin pharmaceutical
Earlier this week, BioMarin Pharmaceutical appointed Arpit Davé as Executive Vice President, Chief Digital and Information Officer, a new C-level role reporting directly to CEO Alexander Hardy and overseeing enterprise technology, data science, and digital transformation. By bringing in a biopharma technology veteran with extensive artificial intelligence experience from Amgen, Bristol Myers Squibb, and Merck, BioMarin is signaling a stronger emphasis on data-driven decision making across...
Krystal Biotech (KRYS) Is Up 9.0% After Positive Interim Data From Inhaled CF Gene Therapy Study
Krystal Biotech
2026-01-17
cf
gene therapy
U.S. stocks, Saudi stocks, stock trading and investment platforms
Krystal Biotech (KRYS) Is Up 9.0% After Positive Interim Data From Inhaled CF Gene Therapy Study
Krystal Biotech
2026-01-17
cf
gene therapy
cystic fibrosis
Earlier this month, Krystal Biotech reported a positive interim update from the highest-dose cohort of its Phase 1 CORAL-1 study, showing successful lung delivery and expression of wild-type CFTR protein with inhaled gene therapy candidate KB407 in cystic fibrosis patients. This progress in validating in-lung gene delivery with KB407 adds a new potential pillar to Krystal’s HSV-1-based platform beyond its existing dermatology franchise. We’ll now examine how KB407’s early success in...
ASH: Financial Difficulties Common During Pediatric Leukemia Treatment
Hematology Advisor News
2026-01-16
leukemia
Cumulative incidence of any new household material hardship was 30.0 percent at 24 months.
New Minimally Invasive Therapy at MemorialCare Heart & Vascular Institute Reduces Cardiovascular Mortality by Nearly 30% in Patients with Resistant Hypertension
PR Newswire
2026-01-16
heart failure
MemorialCare introduces the Symplicity™ Renal Denervation procedure across its health system, targeting patients with uncontrolled high blood pressure—a therapy shown in clinical trials to reduce major cardiovascular events by 20%, stroke by 27%, and heart failure by 28%. FOUNTAIN VALLEY,...
Ouroboros Announces Leading $60M Series A Investment in Cytotheryx to Advance Cell Therapy Platform for Liver Disease
PR Newswire
2026-01-16
cell therapy
NEW YORK and SAN FRANCISCO, Jan. 16, 2026 /PRNewswire/ -- Ouroboros Family Founders Fund I, LP announced this week it will lead a $60 million Series A financing in Cytotheryx, Inc., a preclinical cell therapy company developing transformative treatments for liver disease. The announcement...
AGC Biologics Celebrates U.S. and EU Approval of Fondazione Telethon’s Waskyra™ Treatment for Wiskot
Fondazione Telethon
2026-01-16
waskyra
This further establishes the global leadership of AGC Biologics' Milan Cell and Gene Center of Excellence in number of commercial products approvedMILAN--(BUSINESS WIRE)--#CGT--With the decision by the U.S. Food and Drug Administration and the European Commission to grant market authorization for...
BTK Inhibitor Therapy Makes Inroads Into First-Line MCL
Hematology Advisor Features
2026-01-16
mcl
Tycel J. Phillips, MD, elucidates the research landscape of BTKis in MCL, particularly its potential as a first-line treatment.
AbbVie Announces Topline Results for Epcoritamab (DuoBody® CD3xCD20) from Phase 3 EPCORE® DLBCL-1 Trial in Patients with Relapsed/Refractory Diffuse Large B-cell Lymphoma (DLBCL)
Abbvie
2026-01-16
dlbcl
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
Based on the topline results from the EPCORE® DLBCL-1 trial, AbbVie along with partner Genmab will engage global regulatory authorities to discuss next steps NORTH CHICAGO, Ill., Jan. 16, 2026...
HEMOSTEMIX RECEIVES FDA SUPPORT FOR ITS BASKET PROTOCOL APPROACH
Hemostemix
2026-01-16
ind
autologous
cardiomyopathy
Calgary, Alberta, January 16, 2026: Hemostemix Inc. (TSXV: HEM; OTCQB: HMTXF; FSE: 2VF0), the leading autologous stem cell company treating those who suffer in pain from peripheral arterial disease, chronic limb threatening ischemia, angina, ischemic cardiomyopathy, dilated cardiomyopathy, and vascular dementia, in Florida, Canada, The Bahamas, announces the successful completion of its pre-Investigational New Drug (pre-IND) meeting with the U.S. Food and Drug Administration (FDA).
Atossa Therapeutics Receives FDA Orphan Drug Designation for (Z)-Endoxifen for the Treatment of Duchenne Muscular Dystrophy
PR Newswire
2026-01-16
duchenne muscular dystrophy
Designation further supports (Z)-Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, Jan. 16, 2026 /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa" or the "Company"), a clinical-stage...
A Look At uniQure (QURE) Valuation After FDA Feedback On AMT-130 Delays BLA Timeline
uniQure
2026-01-16
bla
amt-130
gene therapy
The FDA has informed uniQure (QURE) that existing Phase I/II data for its Huntington’s disease gene therapy AMT-130 is not sufficient for a Biologics License Application, and the company now plans to hold a Type A meeting in Q1 2026. See our latest analysis for uniQure. At a share price of $21.995, uniQure has seen a 1 day share price return of 1.59% decline and a 7 day share price return of 4.99% decline, while its 90 day share price return of 62.08% decline contrasts with a 1 year total...
ImmunityBio Announces Durable Complete Response of 15 Months with a Chemotherapy-Free CD19 CAR-NK Cell Therapy in Waldenstrom Lymphoma
Immunity Bio
2026-01-16
cd19
car-nk
cell therapy
nk cell therapy
complete response
The post ImmunityBio Announces Durable Complete Response of 15 Months with a Chemotherapy-Free CD19 CAR-NK Cell Therapy in Waldenstrom Lymphoma appeared first on ImmunityBio .
Ocugen shares fall, despite positive eye disorder trial
PharmaPhorum
2026-01-16
gene therapy
A trial of Ocugen's gene therapy for sight-robbing disease geographic atrophy (GA) showed efficacy – but still sparked a selloff in its shares.
Novartis ianalumab receives FDA Breakthrough Therapy designation for Sjögren’s disease
Novartis
2026-01-16
ianalumab
breakthrough therapy
AbbVie Announces Topline Results for Epcoritamab (DuoBody® CD3xCD20) from Phase 3 EPCORE® DLBCL-1 Trial in Patients with Relapsed/Refractory Diffuse Large B-cell Lymphoma (DLBCL)
PR Newswire
2026-01-16
dlbcl
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
Based on the topline results from the EPCORE® DLBCL-1 trial, AbbVie along with partner Genmab will engage global regulatory authorities to discuss next steps NORTH CHICAGO, Ill., Jan. 16, 2026 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced topline results from the Phase 3 EPCORE®...
GLP-1s may reduce all-cause mortality, adverse events in sickle cell disease
Healio
2026-01-16
ischemic stroke
sickle cell disease
ORLANDO — GLP-1 receptor agonist use appeared linked to significantly reduced all-cause mortality among people with sickle cell disease, according to study results presented at ASH Annual Meeting and Exposition.People who used the medications also exhibited reduced risk for other adverse outcomes, including sickle cell crisis, venous thromboembolism, ischemic stroke or transient ischemic
Daily pill to stop pancreatic cancer spread granted FDA orphan drug status
Rare Cancer News
2026-01-16
orphan drug
pancreatic cancer
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to NP-G2-044, an experimental treatment for pancreatic cancer that’s currently in clinical testing. The FDA gives this designation to investigational medicines intended to treat conditions that affect fewer than 200,000 people in the U.S. The goal is to provide additional incentives to companies […] The post Daily pill to stop pancreatic cancer spread granted FDA orphan drug status appeared first on Rare Cancer News .
Speculation of Sanofi Takeover Sends Ocular’s Stock Soaring
BioSpace
2026-01-16
wet age-related macular degeneration
Ocular Therapeutix’s lead asset is Axpaxli, being developed for wet age-related macular degeneration. A Phase III study is underway, with data expected this quarter.
Iovance Biotherapeutics Reports Inducement Grants under NASDAQ Listing Rule 5635(c)(4)
Iovance Biotherapeutics
2026-01-16
til
SAN CARLOS, Calif. , Jan. 16, 2026 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA) ("Iovance" or the “Company”), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (“TIL”) therapies for patients with cancer,
Krystal Biotech: Beyond VYJUVEK Commercialization
Krystal Biotech
2026-01-16
vyjuvek
Krystal Biotech is upgraded to Strong Buy as Vyjuvek sales surge and catalysts build. Click here to know more.
EC authorization for Waskyra for Wiskott-Aldrich syndrome
Fondazione Telethon
2026-01-16
waskyra
gene therapy
wiskott-aldrich
ex vivo gene therapy
etuvetidigene autotemcel
wiskott-aldrich syndrome
Italian non-profit organization Fondazione Telethon today announced the European Commission’s decision of January 9, to grant marketing authorization for Waskyra (etuvetidigene autotemcel), an ex vivo gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS), a rare and severe genetic immunodeficiency.
ImmunityBio's cell therapy posts encouraging early data in rare blood cancer
Endpoints News
2026-01-16
cell therapy
ImmunityBio’s off-the-shelf cell therapy has shown early promise in a small trial in a rare form of lymphoma. The ongoing Phase 1 study, named QUILT-106, has so far signed up four patients with Waldenström non ...
AGC Biologics Celebrates U.S. and EU Approval of Fondazione Telethon’s Waskyra™ Treatment for Wiskott-Aldrich Syndrome
Fondazione Telethon
2026-01-16
waskyra
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
MILAN, January 16, 2026--Following U.S. and EU market authorization for Waskyra™, AGC Biologics will continue to support the commercial development of the gene therapy.
VIDEO: Accessibility, delays in diagnosis remain challenges in breast cancer
Healio
2026-01-15
breast cancer
Four Markers Capture Triple-Negative Breast Cancer Cells in Blood Samples
Inside Precision Medicine
2026-01-15
breast cancer
The identification of four new markers specific to triple-negative breast cancer (TNBC) cells could address a major limitation preventing the use of liquid biopsies in the diagnosis and monitoring of this type of cancer. The post Four Markers Capture Triple-Negative Breast Cancer Cells in Blood Samples appeared first on Inside Precision Medicine .
Rabi Hanna, MD, on Practical Considerations for Integrating Gene Therapy
CGTLive
2026-01-15
gene therapy
J&J reports positive Phase 3 data for Tecvayli in earlier blood cancer setting
Endpoints News
2026-01-15
multiple myeloma
Johnson & Johnson’s Tecvayli has achieved survival outcomes in a Phase 3 trial that bode well for its ambitions to expand its use to earlier lines of multiple myeloma. The MajesTEC-9 study enrolled patients with ...
Oxford Biomedica confirms takeover talks with biotech investor EQT
Biopharma Dive
2026-01-15
gene therapy
Oxford, major contract manufacturer to cell and gene therapy companies, has already rejected multiple, unsolicited take-private bids from EQT that it felt “undervalued” the company and its prospects.
Jazz Sells Priority Review Voucher for $200M as Program’s Renewal Languishes in Senate
BioSpace
2026-01-15
priority review
Half of the net proceeds will go to Jazz; it remains unclear who will get the remaining sum. Jazz did not reveal the buyer of the voucher.
Tecvayli tops standard drugs in early multiple myeloma, J&J says
Biopharma Dive
2026-01-15
multiple myeloma
The results build on evidence U.S. regulators found so compelling last year they proactively awarded J&J a new kind of drug review voucher.
FDA Activity Recap: December 2025 Features Multiple Approvals, RMAT Designation, and More
CGTLive
2026-01-15
rmat
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.
VIDEO: Questions remain as treatment improves in HER2-positive breast cancer
Healio
2026-01-15
her2
breast cancer
VIDEO: Questions remain as treatment improves in HER2-positive breast cancer
Delaying consolidation durvalumab a month for locally advanced NSCLC may benefit survival
Healio
2026-01-15
lung cancer
Timing of consolidation durvalumab significantly impacts survival for patients with locally advanced non-small cell lung cancer, according to findings from a real-world analysis.Patients who received durvalumab (Imfinzi, AstraZeneca) following concurrent chemoradiation survived more than 20 months longer than those who did not, but researchers found those who received durvalumab in the first
Acute Ischemic Stroke Pipeline 2025: Therapies, MOA Insights, and Key Clinical Trial Updates by DelveInsight | Genentech, NoNO, Biogen, Avilex Pharma, SanBio, ZZ Biotech, Revalesio, Stemedica Cell
Genentech
2026-01-15
ischemic stroke
(Las Vegas, Nevada, United States) As per DelveInsight’s assessment, globally, Acute Ischemic Stroke pipeline constitutes 20+ key companies continuously working towards developing 25+ Acute Ischemic Stroke treatment therapies, analysis of Clinical Tr...
Genomic Models Sharpen AML Risk and Survival Prediction in Chinese MDS
Hematology Advisor News
2026-01-15
aml
Bone marrow blasts and KRAS, STAG2, and TP53 mutations independently predict progression to AML.
Ocugen touts Phase 2 data for eye disease gene therapy
Endpoints News
2026-01-15
gene therapy
Eye disease gene therapy developer Ocugen reported positive interim Phase 2 data for its geographic atrophy program from a small number of patients. Researchers were testing two different dose levels of the experimental therapy, which ...
VIDEO: Socioeconomic factors, genetics influence diagnosis, outcomes in breast cancer
Healio
2026-01-15
breast cancer
VIDEO: Socioeconomic factors, genetics influence diagnosis, outcomes in breast cancer
JPM Day 3: Rare Disease in the Spotlight
BioSpace
2026-01-15
pivotal
2026 is shaping up to be a pivotal year for rare disease drugmakers, with key approvals, filings and readouts lined up for this year.
New cell therapy for glioblastoma doubles progression-free survival
Rare Cancer News
2026-01-15
glioma
cell therapy
glioblastoma
Treatment with DeltEx DRI, a cell therapy developed by In8bio, is prolonging survival outcomes among people with newly diagnosed glioblastoma in a clinical trial, according to new interim data shared by the company. Glioblastoma, a type of glioma, “is an extremely aggressive and devastating brain cancer, with a short median survival of only [about] 12 […] The post New cell therapy for glioblastoma doubles progression-free survival appeared first on Rare Cancer News .
J&J builds case for earlier Tecvayli use in multiple myeloma
PharmaPhorum
2026-01-15
multiple myeloma
Johnson & Johnson has added another piece to the data behind its effort to move Tecvayli into earlier-line therapy for multiple myeloma.
VIDEO: Further research needed on de-escalating, curative intent therapies in breast cancer
Healio
2026-01-15
breast cancer
VIDEO: Further research needed on de-escalating, curative intent therapies in breast cancer
Pittsford High School Hockey to Host 16th Annual Pink the Rink Night on January 31 at 5:30 PM in Support of the Breast Cancer Coalition of Rochester
PR Newswire
2026-01-15
breast cancer
PITTSFORD, N.Y., Jan. 15, 2026 /PRNewswire/ -- The Pittsford JV and Varsity Hockey teams are proud to announce the 16th Annual Pink the Rink, a longstanding community tradition in support of the Breast Cancer Coalition of Rochester. This year's event will take place on Saturday, January...
BGI, Roche roll out diagnostic tests for Alzheimer's in China
Roche Group
2026-01-15
alzheimer's disease
BGI Genomics and Roche Diagnostics have rolled out tests for Alzheimer's disease in China, the companies said, in an effort to expand access to easier-to-use diagnosis and monitoring choices for patients with the brain-wasting condition.
End-of-Study Results From Hemgenix’s HOPE-B Trial
CGTLive
2026-01-15
hemgenix
gene therapy
hemophilia b
Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, discussed the final results of the phase 3 study that evaluated the hemophilia B gene therapy.
Color Blindness Linked with Worse Bladder Cancer Survival
Inside Precision Medicine
2026-01-15
bladder cancer
People with color blindness may be more likely to die after being diagnosed with bladder cancer than those with full color vision because they miss a vital early warning sign of the disease. The post Color Blindness Linked with Worse Bladder Cancer Survival appeared first on Inside Precision Medicine .
VIDEO: Access to latest treatments, supportive care key in breast cancer
Healio
2026-01-15
breast cancer
VIDEO: Access to latest treatments, supportive care key in breast cancer
Scribe Therapeutics plans to test cholesterol-lowering therapy this summer
Endpoints News
2026-01-14
crispr
gene editing
Scribe Therapeutics, founded almost nine years ago to develop medicines based on novel CRISPR enzymes, is getting ready to test its first therapy in the clinic this summer, the gene editing startup told Endpoints News ...
High School Students Nationwide Kick off Philanthropic Competition to Help All Blood Cancer Patients Live Longer, Better Lives
PR Newswire
2026-01-14
leukemia
WASHINGTON, Jan. 14, 2026 /PRNewswire/ -- Blood Cancer United®, formerly The Leukemia & Lymphoma Society, is proud to kick-off of its annual iconic philanthropic competition, Student Visionaries of the Year. High school students nationwide will raise funds for patients and families...
Digital SHERLOCK: Rapid Detection and Resistance Profiling of Candida auris
GenEng News
2026-01-14
crispr
dSHERLOCK, a rapid CRISPR-based diagnostic that detects Candida auris from patient swabs and identifies antifungal resistance mutations, could enable faster treatment decisions and improved control of hospital outbreaks. The post Digital SHERLOCK: Rapid Detection and Resistance Profiling of Candida auris appeared first on GEN - Genetic Engineering and Biotechnology News .
ImmunityBio's Anktiva lung cancer data; Glaukos' glaucoma implant sales disappoint
Endpoints News
2026-01-14
lung cancer
🫁 ImmunityBio touts lung cancer data: The biotech said its drug Anktiva succeeded in restoring immune cells for lung cancer patients who received a checkpoint inhibitor. Researchers tested the ...
An Agile, Intelligent and Sustainable Future: Biopharma Trends to Watch
GenEng News
2026-01-14
pivotal
2026 will be a pivotal year for biopharma. From digital transformation to an AI revolution to more sustainable technologies, the forces driving change are redefining how therapies are discovered, developed, and delivered. The post An Agile, Intelligent and Sustainable Future: Biopharma Trends to Watch appeared first on GEN - Genetic Engineering and Biotechnology News .
Does Autolus Therapeutics (AUTL) Pairing AUCATZYL Approvals With Revenue Guidance Redefine Its Long-Term Story?
Autolus Therapeutics
2026-01-14
aucatzyl
Autolus Therapeutics recently reported preliminary unaudited 2025 net product revenue of about US$75,000,000, issued 2026 revenue guidance of US$120,000,000–US$135,000,000, and secured UK and EU approvals for AUCATZYL®, alongside advancing obe-cel across oncology and autoimmune indications. An interesting implication is how Autolus is coupling early commercial traction with manufacturing upgrades and broader obe-cel trial expansion to support its long-term product and pipeline...
BGI, Roche roll out diagnostic tests for Alzheimer’s in China
Roche Group
2026-01-14
alzheimer's disease
By Andrew Silver SHANGHAI, Jan 14 (Reuters) - BGI Genomics and Roche Diagnostics have rolled out tests for Alzheimer's disease in China, the companies said, in an effort to expand access to easier-to-use diagnosis and monitoring choices for patients ...
AstraZeneca’s Path to $80B by 2030 Paved With ADCs, Cell Therapies, Near-Term Product Launches
BioSpace
2026-01-14
breast cancer
cell therapies
myasthenia gravis
AstraZeneca is relying on several upcoming products to help hit its target of $80 billion in revenue by 2030, including drugs for hypertension, breast cancer and generalized myasthenia gravis, all of which are currently under FDA review.
Evaluating Gene Editing Therapy Reni-Cel for Severe Sickle Cell Disease
CGTLive
2026-01-14
phase 1/2
gene editing
sickle cell disease
Rabi Hanna, MD, the chairman of the Division of Pediatric Hematology & Oncology and BMT at Cleveland Clinic Children's, discussed results from the phase 1/2 RUBY trial.
Gene-Driven Obesity Influences Survival in Breast Cancer
Inside Precision Medicine
2026-01-14
breast cancer
More hours of walking per week, the researchers also found, could help improve survival in overweight breast cancer survivors. The post Gene-Driven Obesity Influences Survival in Breast Cancer appeared first on Inside Precision Medicine .
#JPM26: Q&A with Bristol Myers Squibb CEO Chris Boerner on 2026 outlook
Endpoints News
2026-01-14
pivotal
SAN FRANCISCO — In his third year as CEO of Bristol Myers Squibb, Chris Boerner kicked off the JP Morgan Healthcare Conference with plans for six pivotal readouts of new drugs ...
Beyond PSA Cutoffs: Long-Term Model Predicts Prostate Cancer Death Risk
Inside Precision Medicine
2026-01-14
prostate cancer
A new long-term risk model predicts prostate cancer mortality using PSA results, life expectancy, and competing health risks, offering a more precise framework for personalized screening decisions and reducing overtreatment. The post Beyond PSA Cutoffs: Long-Term Model Predicts Prostate Cancer Death Risk appeared first on Inside Precision Medicine .
PADCEV™ Plus Keytruda® Significantly Improves Survival for Patients with Muscle-Invasive Bladder Cancer Regardless of Cisplatin Eligibility
astellas
2026-01-14
bladder cancer
PADCEV plus Keytruda is the first and only regimen without platinum-based chemotherapy to improve event-free and overall survival when used before and after surgery in cisplatin-eligible patients...
Saudi FDA Grants Accelerated Approval to ImmunityBio’s ANKTIVA® In Combination with Checkpoint Inhibitors for Metastatic Non-Small Cell Lung Cancer
Immunity Bio
2026-01-14
lung cancer
accelerated approval
The post Saudi FDA Grants Accelerated Approval to ImmunityBio’s ANKTIVA® In Combination with Checkpoint Inhibitors for Metastatic Non-Small Cell Lung Cancer appeared first on ImmunityBio .
Study: One dose of HPV vaccine on par with two doses
Healio
2026-01-14
cervical cancer
A single dose of either bivalent or nonvalent HPV vaccine was just as protective as the standard two doses of either vaccine in preventing HPV16 or HPV18, according to a study published in he New England Journal of Medicine.“Persistent HPV infection can cause cervical cancer, and 77% of the global burden of cervical cancer is attributable to HPV types 16 and 18,” Aimée R. Kreimer,
Cholera Toxin Reshapes Tumor Immunity to Slow Colorectal Cancer Growth
Inside Precision Medicine
2026-01-14
colorectal cancer
In mouse models, systemic administration of the purified cytotoxin MakA reduced tumor growth, increased cancer cell death, and reshaped immune cell activity within tumors, while showing no measurable toxicity. The post Cholera Toxin Reshapes Tumor Immunity to Slow Colorectal Cancer Growth appeared first on Inside Precision Medicine .
JPM26: Paying cash for obesity drugs, renewed IPO optimism and pharma’s cell therapy view
Biopharma Dive
2026-01-14
cell therapy
Drugmaker CEOs see sales online channels as a budding opportunity, while some biotech investors told BioPharma Dive to expect a rebound in new stock offerings.
Blood Test Predicts Response to Newly Approved SCLC Immunotherapy
Inside Precision Medicine
2026-01-14
lung cancer
A liquid biopsy test could accurately stratify patients with small cell lung cancer (SCLC) based on their likelihood to positively respond to Amgen’s Imdelltra (tarlatamab), representing a major step forward in diagnosis and treatment. The post Blood Test Predicts Response to Newly Approved SCLC Immunotherapy appeared first on Inside Precision Medicine .
Saudi FDA Grants Accelerated Approval to ImmunityBio’s ANKTIVA® for Non-Muscle Invasive Bladder Cancer with Carcinoma In-Situ
Immunity Bio
2026-01-14
bladder cancer
accelerated approval
The post Saudi FDA Grants Accelerated Approval to ImmunityBio’s ANKTIVA® for Non-Muscle Invasive Bladder Cancer with Carcinoma In-Situ appeared first on ImmunityBio .
Fast-Tracked CGT Platform Eliminates Patient Preconditioning
GenEng News
2026-01-14
autologous
Immusoft’s autologous immune system programming platform is in the clinic for MPS 1, paving the way for use with additional genetic indications—sans patient preconditioning—while offering redosing capabilities. The post Fast-Tracked CGT Platform Eliminates Patient Preconditioning appeared first on GEN - Genetic Engineering and Biotechnology News .
JPM26: With Leqembi Launched, Biogen Looks To Expand Alzheimer’s Presence
BioSpace
2026-01-14
accelerated approval
Three years after the accelerated approval of its anti-amyloid Alzheimer’s therapy, Biogen—neck and neck in the market with Eli Lilly and its Kisunla offering—is focused on a near-term FDA decision for a subcutaneous induction dose of Leqembi, a presymptomatic readout in 2028 and a clutch of next-generation candidates.
Fixed-Duration Venetoclax Noninferior to Continuous Ibrutinib in Untreated CLL
Hematology Advisor News
2026-01-14
cll
Fixed-duration therapy achieved deeper remissions, with undetectable MRD in 73.3% of patients treated with venetoclax-obinutuzumab and 47.2% of those treated with venetoclax-ibrutinib.
AVEO Oncology, an LG Chem company, Announces First Patient Dosed in Front-Line AML Combination Study of Ficlatuzumab
PR Newswire
2026-01-14
aml
– Collaboration with Blood Cancer United® in its Beat AML® Master Clinical Trial – – AVEO's ficlatuzumab compound to be studied in combination with azacitidine and venetoclax in patients with AML in a Phase 1b/2 clinical trial – – First patient dosed into the Phase 1b/2 clinical trial –...
Genethon Signs Licensing Agreement with AskBio for the Development of an Investigational Gene Therapy for Pompe Disease
Genethon
2026-01-14
gene therapy
pompe disease
Genethon, a leading laboratory in gene therapy for rare diseases, today announced that it has entered into an exclusive, worldwide licensing agreement with AskBio, a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, for the use of a patented component of AB-1009 for the treatment of Pompe disease. Pompe … Continued
Heartstream lance ses activités en tant que société indépendante spécialisée dans les soins d'urgence
PR Newswire
2026-01-14
ind
L'ancienne division Emergency Care de Philips devient la première société plate-forme d'Emergency Care Holdings, poursuivant ainsi une mission centenaire visant à sauver des vies BOTHELL, Wash., 14 janvier 2026 /PRNewswire/ -- Emergency Care Holdings (ECH), une plateforme formée par...
Oral treatment for ovarian cancer put on FDA fast track
Rare Cancer News
2026-01-14
fast track
ovarian cancer
The U.S. Food and Drug Administration (FDA) has granted fast track designation to ETX-19477, an oral treatment for ovarian cancer currently in early clinical testing. The FDA grants this designation to investigational drugs intended to treat serious or life-threatening conditions and address unmet medical needs. The goal is to accelerate the development of important new […] The post Oral treatment for ovarian cancer put on FDA fast track appeared first on Rare Cancer News .
Jazz sells priority review voucher for $200M, scoring highest price in a decade
Endpoints News
2026-01-14
priority review
Jazz Pharmaceuticals said it sold a priority review voucher to an undisclosed company for $200 million, landing the highest price for such a voucher since 2016. Jazz will take home half ...
‘Clear’ data: Neutropenic diet reduces major infection risk during blood cancer treatment
Healio
2026-01-14
leukemia
hematopoietic stem cell
A long-standing practice through which people hospitalized for blood cancer treatment are restricted to eating cooked foods appears justified, study results suggest.Patients undergoing hematopoietic stem cell transplant or induction chemotherapy for acute leukemia demonstrated a lower risk for major infections with a neutropenic diet than a more liberalized diet, results of a randomized phase 3
Gilead Expects FDA Decision on Anito-Cel in First Quarter of 2026
Gilead Sciences
2026-01-13
lung cancer
The company is also anticipating new data on its antibody-drug conjugate Trodelvy in PD-L1-high non-small cell lung cancer later this year.
Autolus Therapeutics (AUTL) Valuation Check After Revenue Guidance And AUCATZYL UK And EU Approvals
Autolus Therapeutics
2026-01-13
aucatzyl
Autolus Therapeutics (AUTL) is in focus after reporting preliminary 2025 net product revenue of about US$75 million, issuing 2026 revenue guidance of US$120 million to US$135 million, and securing UK and EU approvals for AUCATZYL®. See our latest analysis for Autolus Therapeutics. Despite the revenue update and regulatory approvals, Autolus Therapeutics’ share price has been under pressure, with a 1-day share price return of a 17.13% decline and a 1-year total shareholder return of a 32.13%...
FDA grants fast track designation to vectorized antibody to treat ALS
Healio
2026-01-13
fast track
The FDA has granted fast track designation to a vectorized antibody that targets RNA-binding proteins characteristic of neurodegenerative diseases.According to a press release from VectorY Therapeutics, the therapy is specifically designed to address ALS.
Assessing uniQure (QURE) Valuation After Recent Volatility And Gene Therapy Progress
uniQure
2026-01-13
gene therapy
uniQure (QURE) is back on traders’ radar after a sharp 9% one day decline, prompting fresh attention on how its gene therapy pipeline and current financial profile might line up with recent share price swings. See our latest analysis for uniQure. That 9% one day share price drop sits against a mixed backdrop, with a 7 day share price return of 3.27% and 30 day share price return of 18.09% contrasting with a 90 day share price return decline of 63.33%. At the same time, the 1 year total...
Asimov and AGC Biologics Agree to Develop New Viral Vector Production Approach
GenEng News
2026-01-13
viral vector
Combining an engineered HEK293 cell line containing inducible viral genes and software for transfer plasmid design, the LV Edge Packaging system was created to minimize GMP plasmid cost and process complexity. The post Asimov and AGC Biologics Agree to Develop New Viral Vector Production Approach appeared first on GEN - Genetic Engineering and Biotechnology News .
J&J seeks approval in EU of Tecvayli-Darzalex for hard-to-treat myeloma
Rare Cancer News
2026-01-13
multiple myeloma
Johnson & Johnson is now seeking regulatory approval in the European Union for Tecvayli (teclistamab) plus Darzalex Faspro (daratumumab and hyaluronidase-fihj) for individuals with previously-treated multiple myeloma. The company has submitted an application to the European Medicines Agency (EMA) to allow the combo’s use as a second-line treatment. Specifically, the application seeks approval of Tecvayli-Darzalex […] The post J&J seeks approval in EU of Tecvayli-Darzalex for hard-to-treat myeloma appeared first on Rare Cancer News .
Autolus Therapeutics (AUTL) Is Down 25.0% After Raising 2026 Revenue Guidance And Advancing Obe-cel Trials
Autolus Therapeutics
2026-01-13
lupus
aucatzyl
Autolus Therapeutics recently reported preliminary unaudited 2025 net product revenue of about US$75 million and issued 2026 guidance of US$120 million to US$135 million, alongside progressing obe-cel into Phase 2 trials for lupus nephritis and other indications. The company also secured UK and EU approvals for AUCATZYL®, pairing early commercial traction with expanding clinical data in both pediatric oncology and autoimmune disease. Next, we’ll examine how this combination of higher revenue...
ImmunityBio Announces Positive Results Demonstrating ANKTIVA® as a Lymphocyte Stimulating Agent in Combination with Checkpoint Inhibitors in Non-Small Cell Lung Cancer
Immunity Bio
2026-01-13
lung cancer
The post ImmunityBio Announces Positive Results Demonstrating ANKTIVA® as a Lymphocyte Stimulating Agent in Combination with Checkpoint Inhibitors in Non-Small Cell Lung Cancer appeared first on ImmunityBio .
Lexeo Therapeutics Announces Positive Interim Phase I/II Data for LX2020 for the Treatment of PKP2-Associated Arrhythmogenic Cardiomyopathy
Lexeo Therapeutics
2026-01-13
cardiomyopathy
arrhythmogenic cardiomyopathy
January Issue of the Journal of Cardiac Failure Highlights Prevention, Innovation, and New Strategies to Improve Outcomes Across the Heart Failure Spectrum
PR Newswire
2026-01-13
heart failure
WASHINGTON, Jan. 13, 2026 /PRNewswire/ -- A novel approach to removing excess fluid through the skin and a new strategy to prevent right ventricular failure after LVAD implantation are among the highlights of the January issue of the Journal of Cardiac Failure (JCF). Together, these...
Lung Cancer Trial to Proceed After FDA Releases Hold on Merck, Daiichi’s ADC
BioSpace
2026-01-13
lung cancer
The FDA initially placed the Phase III IDeate-Lung02 study on hold due to a “higher than expected” number of deaths in patients treated with ifinatamab deruxtecan.
After Mortality-Marred Year, Sarepta Looks Ahead to Catalyst-Rich 2026
BioSpace
2026-01-13
sarepta
facioscapulohumeral muscular dystrophy
Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.
Patients With Hemophilia B Report Improvement in QoL After Gene Therapy
Hematology Advisor News
2026-01-13
gene therapy
hemophilia b
A majority of patients with hemophilia B reported that their impression of life with hemophilia was “greatly improved” after gene therapy with fidanacogene elaparvovec.
Balanced View on BioMarin: Solid Near-Term Guidance Offset by Voxzogo Competitive Risks and Roctavian Challenges, Supporting a Hold Rating
BioMarin Pharmaceutical
2026-01-13
roctavian
William Blair analyst Sami Corwin has maintained their neutral stance on BMRN stock, giving a Hold rating on January 8. Sami Corwin has given his Hold rating due to...
What Sanofi’s multiple sclerosis troubles could mean for the space
Biopharma Dive
2026-01-13
multiple sclerosis
Regulatory and clinical roadblocks have set back its oral BTK inhibitor tolebrutinib, but the drug class still has many opportunities to make an impact.
uniQure updates gene therapy pipeline and regulatory outlook
uniQure
2026-01-13
gene therapy
The latest update is out from uniQure ( ($QURE) ). In January 2026, uniQure N.V. updated its corporate presentation to highlight its positioning as a leader in gene...
AI Treatment Suggestions for Liver Cancer More Accurate at Early Stages
Inside Precision Medicine
2026-01-13
hepatocellular carcinoma
A large Korean study shows AI-based treatment suggestions are most effective in simple, earlier stage hepatocellular carcinoma cases but not in more complex, advanced cases. The post AI Treatment Suggestions for Liver Cancer More Accurate at Early Stages appeared first on Inside Precision Medicine .
uniQure N.V. Updates on Gene Therapy Developments
uniQure
2026-01-13
gene therapy
On January 13, 2026, uniQure N.V. provided updates on its gene therapy programs, highlighting significant progress in clinical trials. Key Highlights: Original SEC Filing: uniQure N.V. [ QURE ] - 8-K - Jan. 13, 2026This is an AI-powered summary. It may contain inaccuracies. Consider verifying impo…
Reflections on pivotal year in endocrinology: A conversation with Scott Isaacs, MD
Healio
2026-01-13
pivotal
The past year was a busy one for the American Association of Clinical Endocrinology and its 2025 president, Scott Isaacs, MD, FACP, FACE. Isaacs, who is also an adjunct associate professor of medicine at Emory University and a member of the Healio | Endocrine Today Editorial Board, described 2025 as a “record year” for AACE regarding the number of consensus statements the organization published.
Study identifies optimal hydroxychloroquine blood level range in lupus
Healio
2026-01-13
lupus
The optimal hydroxychloroquine blood level in lupus is between 750 ng/mL and 1,150 ng/mL, which can help prevent active disease but also reduce the risk for toxicity, according to data published in Arthritis & Rheumatology. The researchers additionally suggested that monitoring HCQ blood levels may be superior to dosing based on a patient’s body weight in order to avoid toxicities and
U.S. FDA Grants RMAT Designation to ECT204, an ARTEMIS® CAR (AbTCR) T-Cell Therapy for Advanced Hepatocellular Carcinoma
Eureka Therapeutics
2026-01-12
hcc
gpc3
rmat
car t
cell therapy
cell therapies
hepatocellular carcinoma
EMERYVILLE, Calif. – January 12, 2026 – Eureka Therapeutics, Inc., a clinical-stage biotechnology company advancing next-generation T-cell therapies for cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ECT204, a GPC3-targeting ARTEMIS® CAR T-cell therapy, for the treatment of patients with advanced hepatocellular carcinoma (HCC), The post U.S. FDA Grants RMAT Designation to ECT204, an ARTEMIS® CAR (AbTCR) T-Cell Therapy for Advanced Hepatocellular Carcinoma appeared first on Eureka .
CRISPR Therapeutics Highlights Strategic Priorities and Anticipated 2026 Milestones
CRISPR
2026-01-12
crispr
casgevy
2026 is poised to be a data- and milestone-rich year across the portfolio- -CASGEVY ® launch acceleration continues, supporting multi-billion-dollar potential- -Broad pipeline supported by leading gene-editing and siRNA platforms across cardiovascular, autoimmune, oncology and rare diseases-
8-K - Sarepta Therapeutics, Inc. (0000873303) (Filer)
SEC Filing (8-K)
2026-01-12
sarepta therapeutics
Filed: 2026-01-12 AccNo: 0001193125-26-010116 Size: 234 KB Item 2.02: Results of Operations and Financial Condition Item 9.01: Financial Statements and Exhibits
Nurix Therapeutics Outlines 2026 Goals and Objectives for Advancing Bexobrutideg and Its Pipeline of Novel Degrader-Based Medicines in Cancer and Autoimmune Diseases
nurix therapeutics
2026-01-12
cll
pivotal
leukemia
chronic lymphocytic leukemia
Execute a pivotal program for potential best-in-class BTK degrader, bexobrutideg, including Phase 2 and confirmatory Phase 3 studies to support global registration in relapsed/refractory chronic lymphocytic leukemia (r/r CLL) Expand bexobrutideg into autoimmune and inflammatory indications,
FDA again rejects tabelecleucel for EBV-driven post-transplant lymphoproliferative disease
Healio
2026-01-12
ebvallo
allogeneic
cell therapy
tabelecleucel
complete response
post-transplant lymphoproliferative disease
The FDA has issued a second complete response letter to Atara Biotherapeutics for its biologicals license application of tabelecleucel for adults and children with Epstein-Barr virus-positive post-transplant lymphoproliferative disease.The company had submitted the application for Ebvallo (tabelecleucel) — an off-the-shelf allogeneic Epstein-Barr virus-specific cytotoxic T-cell therapy
Krystal Biotech Reports ~$389M 2025 VYJUVEK Revenue, $955M Cash Reserves
Krystal Biotech
2026-01-12
vyjuvek
financial results
(RTTNews) - Krystal Biotech Inc. (KRYS) announced preliminary unaudited financial results for 2025, including strong performance from its lead product VYJUVEK, and shared its strategic vision for advancing rare disease therapies.
Autolus Posts $75 Million In First-Year AUCATZYL Sales, Guides Up To $135 Million For 2026
Autolus Therapeutics
2026-01-12
cd19
car t
aucatzyl
Autolus Therapeutics announced it expects preliminary unaudited net product revenue from AUCATZYL (obecabtagene autoleucel; obe-cel) of about $24 million for the fourth quarter of 2025 and about $75 million for the full year, marking its first year of commercial sales following the CD19 CAR T therapy’s U.S. approval in November 2024.
Gilead Sciences: From All-Time Highs To Higher Highs
Gilead Sciences
2026-01-12
car-t
Gilead’s Yeztugo launch, rising margins, and CAR-T pipeline progress (anito-cel, KITE-753/363) support a 2026 bull case—read the outlook now.
Nurix Therapeutics Presents New Data Demonstrating Durable, Deepening Responses in Phase 1 Trial of Bexobrutideg (NX-5948) in Patients with Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition
nurix therapeutics
2026-01-12
cll
leukemia
chronic lymphocytic leukemia
Objective response rate (ORR) of 83% including two complete responses in CLL patients in Phase 1a study with median progression free survival (PFS) of 22.1 months across all doses tested Emerging data from randomized Phase 1b cohorts points to higher ORR and longer progression free survival at the
ZR2 Regimen Achieves High Remission, Durable Survival in Older Adults With Newly Diagnosed DLBCL
Hematology Advisor News
2026-01-12
dlbcl
Phase 2 study results showed zanubrutinib combined with rituximab and lenalidomide produced high remission rates and durable survival in older adults with newly diagnosed DLBCL.
AbbVie buys into PD-1/VEGF sweepstakes; Alnylam and Vertex set expectations
Biopharma Dive
2026-01-12
cell therapy
AbbVie is paying $650 million for a dual-targeting immunotherapy from China’s RemeGen. Elsewhere, Alnylam missed an earnings target and the FDA flipped its position on a cell therapy from Atara.
How a new cell therapy is targeting leukemia, lupus and multiple sclerosis
Autolus Therapeutics
2026-01-12
lupus
aucatzyl
leukemia
cell therapy
multiple sclerosis
Autolus forecasts $24M Q4 and $75M 2025 AUCATZYL revenue, guides $120M-$135M for 2026 and advances obe-cel trials in leukemia, lupus and MS, with MS data expected in 2026.
Eisai licenses Nuvation lung cancer pill; Alnylam's stock dips after sales data report
Endpoints News
2026-01-12
uniqure
lung cancer
bristol myers squibb
Plus, news about Summit, Pfizer, Insmed, Bristol Myers Squibb, Lynk Pharmaceuticals, Stoke Therapeutics, UniQure, Teva, Royalty Pharma, Vibrant Therapeutics and Recludix Pharma: 💊 Nuvation Bio licenses lung cancer pill to Eisai: The Japanese company has ...
Press Release: Autolus Therapeutics Announces Preliminary Unaudited Fourth Quarter and Full Year 2025 Net Product Revenue, Pipeline Advancements and Outlook for 2026
Autolus Therapeutics
2026-01-12
aucatzyl
Company expects preliminary unaudited AUCATZYL(R) net product revenue of approximately $24 million for the fourth quarter of 2025 and approximately $75 million for the full year of 2
JPM26: US biotech’s ‘Sputnik moment,’ Pfizer’s obesity ambitions and Bristol Myers’ big year
Biopharma Dive
2026-01-12
sarepta
Four recent deals fueled more angst about China’s biotech progress, while Pfizer, Bristol Myers and Sarepta all worked to appease jittery investors.
UniQure Secures Type A Meeting with FDA: What This Means for AMT-130
uniQure
2026-01-12
amt-130
⏱️6 min read | UniQure secured a Type A FDA meeting, a high-priority discussion for urgent issues. Within 30 days, both sides will discuss what kind of data package might support the advancement of AMT-130 in the US.
Sarepta Reports Preliminary* Fourth Quarter and Full-Year 2025 Net Product Revenues
Sarepta Therapeutics
2026-01-12
sarepta
elevidys
Preliminary total net product revenue of $369.6 million for the fourth quarter and $1.86 billion for full-year 2025 Preliminary ELEVIDYS net product revenue totaled $110.4 million for the fourth quarter and $898.7 million for full-year 2025 Preliminary PMO net product revenues totaled $259.2
Autolus Therapeutics Announces Preliminary Unaudited Fourth Quarter and Full Year 2025 Net Product Revenue, Pipeline Advancements and Outlook for 2026
Autolus Therapeutics
2026-01-12
aucatzyl
Company expects preliminary unaudited AUCATZYL® net product revenue of approximately $24 million for the fourth quarter of 2025 and approximately $75 million for the full year of 2025Autolus...
FDA lifts partial hold on Merck, Daiichi's Phase 3 lung cancer study
Endpoints News
2026-01-12
lung cancer
clinical hold
partial clinical hold
The FDA on Monday lifted a partial clinical hold on a Phase 3 lung cancer study run by Merck and Daiichi Sankyo. Regulators had placed the hold just last month after seeing ...
Why Men Face a Higher Risk of Multiple Myeloma
Inside Precision Medicine
2026-01-12
multiple myeloma
A large clinical study sheds light on why men are more likely than women to develop multiple myeloma, showing that male patients present with more advanced disease and greater tumor burden. The post Why Men Face a Higher Risk of Multiple Myeloma appeared first on Inside Precision Medicine .
BCG May Be Less Effective for Treating NMIBC Following UTUC Surgery
Hematology Advisor News
2026-01-12
bladder cancer
By default, metachronous bladder recurrences after UTUC surgery have been managed similarly to primary non-muscle invasive bladder cancer.
Pfizer’s Braftovi combination therapy shows positive results for colorectal cancer
PMLive Research
2026-01-12
colorectal cancer
Colorectal cancer is the third most common cancer globally, with approximately 1.8 million people diagnosed in 2022
REGENXBIO Highlights Key 2026 Catalysts and Announces Positive Long-Term Functional Outcomes in Lead Duchenne Gene Therapy Program
REGENXBIO
2026-01-12
bla
pdufa
pivotal
gene therapy
New Phase I/II RGX-202 functional data demonstrates long-term, durable treatment effect at pivotal dose at 18 months Robust patient enrollment in confirmatory trial continues, expect majority of patients to be dosed by planned BLA filing, mid-year Expecting FDA PDUFA decision and multiple pivotal
Suvoda sets new standard for streamlined patient and site experience in clinical trials
PR Newswire
2026-01-12
pivotal
CONSHOHOCKEN, Pa., Jan. 12, 2026 /PRNewswire/ -- Suvoda, a clinical trial technology company, took decisive steps in 2025 toward a more connected and streamlined clinical trial experience for both patients and sites. The company's merger with Greenphire marked a pivotal moment in that...
Blood Test Could Predict Crohn’s Disease Years Before Symptoms Appear
Inside Precision Medicine
2026-01-12
crohn's disease
Testing for bacterial flagellin antibodies in the blood could help predict who will develop Crohn's disease years before symptoms start. The post Blood Test Could Predict Crohn’s Disease Years Before Symptoms Appear appeared first on Inside Precision Medicine .
Autolus reports $75 million in 2025 AUCATZYL revenue, projects growth
Autolus Therapeutics
2026-01-12
aucatzyl
Autolus Therapeutics plc (NASDAQ: AUTL) reported preliminary unaudited net product revenue of approximately $24 million for the fourth quarter of 2025 and approximately $75 million for the full year from sales of...
CG Oncology price target raised to $82 from $55 at Goldman Sachs
CG Oncology
2026-01-12
pivotal
Goldman Sachs raised the firm’s price target on CG Oncology (CGON) to $82 from $55 and keeps a Buy rating on the shares. CG Oncology’s advancement of the pivotal PI...
Biomarin Pharmaceutical Inc estimates $3.2 billion in total revenues for FY
BioMarin Pharmaceutical
2026-01-12
roctavian
SEC filing: Biomarin Pharmaceutical Inc estimates $3.2 billion in total revenues for FY - SEC filing Biomarin Pharmaceutical Inc expects $230-$260 million asset write-down for Roctavian in Q4 -...
Autolus Therapeutics sees cash runway into 4Q27
Autolus Therapeutics
2026-01-12
aucatzyl
Dr. Christian Itin concluded: “Our 2026 commercial focus for AUCATZYL is to build on the strong center presence and positive physician experience to drive top line ...
Neurogene Announces Recent Achievements and Anticipated 2026 Key Milestones for NGN-401, a Potential Best-in-Class Gene Therapy for Rett Syndrome
neurogene
2026-01-12
phase 1/2
gene therapy
rett syndrome
Dosed multiple participants in Embolden™ registrational trial in fourth quarter of 2025 Completion of dosing in Embolden expected in second quarter of 2026 Plan to present interim data on pediatric and adolescent/adult cohorts from Phase 1/2 trial in mid-2026 Early commercial-readiness activities
FDA shares eased manufacturing rules for cell and gene therapies
Endpoints News
2026-01-12
gene therapies
The FDA is taking steps to ease some manufacturing requirements for cell and gene therapies in an effort to expedite their development. Traditionally, the FDA’s Center for Biologics Evaluation and Research has used the same ...
Protara Therapeutics Highlights Recent Updates and Anticipated 2026 Milestones
protara therapeutics
2026-01-12
fast track
breakthrough therapy
On track to report interim results from approximately 25 six-month evaluable BCG-Unresponsive patients in ADVANCED-2 trial in Q1 2026 Received Breakthrough Therapy and Fast Track designations for TARA-002 in LMs; regulatory update expected in 1H 2026 Dosed first patient in THRIVE-3 registrational
Enasidenib Is a Safe and Effective Intervention in IDH2-Mutated AML and MDS
Hematology Advisor News
2026-01-12
aml
Researchers sought to determine whether combining enasidenib with venetoclax would be effective in patients with IDH2-mutated, relapsed or refractory AML.
8-K - BRISTOL MYERS SQUIBB CO (0000014272) (Filer)
SEC Filing (8-K)
2026-01-12
bristol myers squibb
Filed: 2026-01-12 AccNo: 0001140361-26-000929 Size: 3 MB Item 7.01: Regulation FD Disclosure Item 9.01: Financial Statements and Exhibits
FDA again rejects Atara, Pierre Fabre's cell therapy for Epstein-Barr virus
Endpoints News
2026-01-12
tab-cel
cell therapy
tabelecleucel
The FDA has once again rejected a T cell therapy for a rare and serious transplant complication related to Epstein-Barr virus. Atara Biotherapeutics' treatment, called tabelecleucel or tab-cel, is designed to treat patients with Epstein ...
8-K - PTC THERAPEUTICS, INC. (0001070081) (Filer)
SEC Filing (8-K)
2026-01-12
ptc therapeutics
Filed: 2026-01-12 AccNo: 0001104659-26-002799 Size: 4 MB Item 2.02: Results of Operations and Financial Condition Item 7.01: Regulation FD Disclosure Item 9.01: Financial Statements and Exhibits
FDA Carves Out Manufacturing Exemptions for CGTs To Accelerate Development
BioSpace
2026-01-12
gene therapies
FDA Commissioner Marty Makary called these changes “common-sense reforms” that could expedite the development of cell and gene therapies.
JPM 2026: Dyno Tx’s AI-Evolved AAVs Rewrite Rules of Brain Gene Therapy Delivery
Inside Precision Medicine
2026-01-12
gene therapy
Eric Kelsic, CEO and co-founder, unveils Dyno-yp2 and explains how adding strategic delivery agility to its portfolio and partnerships can overcome the modality’s safety concerns The post JPM 2026: Dyno Tx’s AI-Evolved AAVs Rewrite Rules of Brain Gene Therapy Delivery appeared first on Inside Precision Medicine .
Atara Biotherapeutics Provides Regulatory and Business Update on EBVALLO™ (tabelecleucel)
Atara Biotherapeutics
2026-01-12
ebvallo
tabelecleucel
1st 4 patients in AML treatment combo trial show response
Rare Cancer News
2026-01-12
aml
leukemia
phase 1/2
acute myeloid leukemia
All four adults with hard-to-treat acute myeloid leukemia (AML) who received treatment with a combination of Venclexta (venetoclax) and AB Science‘s experimental therapy AB8939 have responded to treatment, according to an update from AB. The company previously announced that the first three participants in the AB18001 Phase 1/2 (NCT05211570) had all responded to the treatment, […] The post 1st 4 patients in AML treatment combo trial show response appeared first on Rare Cancer News .
Startup Aurora Seeks To Replicate Baby KJ Success With $16M in Seed Money
BioSpace
2026-01-12
crispr
Aurora joins the clutch of companies linked to Nobel Prize winner and CRISPR trailblazer Jennifer Doudna.
FDA removes barriers to cell, gene therapy development
PharmaPhorum
2026-01-12
gene therapy
gene therapies
FDA wants to exert regulatory flexibility on cell and gene therapies in an effort to bring them to patients more quickly and efficiently.
Basecamp Research launches world-first AI models for programmable gene insertion
PR Newswire
2026-01-12
gene therapies
The breakthrough tackles a longstanding challenge in genetic medicine with the goal of developing a new generation of curative cell and gene therapies. Developed in collaboration with NVIDIA by training the largest evolutionary AI models on a new, globally collected dataset, accelerated...
Nvidia-partnered startup reveals AI-generated enzymes for precise gene insertion
Endpoints News
2026-01-12
gene editing
Scientists at Basecamp Research say they have used “evolutionary AI models,” developed with help from chipmaker Nvidia, to create a powerful new gene editing tool that can insert a new healthy gene in front of ...
Kyverna Therapeutics Provides Corporate Update and Outlines 2026 Strategic Priorities at the J.P. Morgan Healthcare Conference
Kyverna Therapeutics
2026-01-12
bla
myasthenia gravis
stiff person syndrome
Advancing valuable commercial opportunity in stiff person syndrome (SPS) following landmark registrational data; Biologics License Application (BLA) submission anticipated in 1H 2026 First patient enrolled in registrational Phase 3 trial in generalized myasthenia gravis (gMG) Completed follow-on
Johnson & Johnson’s Rybrevant shows positive effects in difficult-to-treat colorectal cancer
PMLive Research
2026-01-12
colorectal cancer
Colorectal cancer accounts for around 10% of all cancer cases globally, with an increase in diagnoses in younger people
THE THINK PINK GALA DEBUTS MARCH 14TH 2026 AT THE PLAZA WITH BERNADETTE PETERS AND A MICHELIN-STARRED CULINARY EXPERIENCE
PR Newswire
2026-01-12
breast cancer
A landmark night of glamour, generosity, and world-class artistry in support of breast cancer patients, survivors, and families NEW YORK, Jan. 12, 2026 /PRNewswire/ -- The Think Pink Gala will debut on Saturday, March 14th, 2026, at the iconic Plaza Hotel, marking a premier cultural...
Field Medical to Present at the 44th Annual J.P. Morgan Healthcare Conference
PR Newswire
2026-01-12
atrial fibrillation
CARDIFF-BY-THE-SEA, Calif., Jan. 12, 2026 /PRNewswire/ -- Field Medical Inc., a clinical-stage medical technology company advancing a versatile, next-generation pulsed field ablation (PFA) platform designed to support both ventricular tachycardia (VT) and atrial fibrillation (AF)...
Immune-Related Genes that Shape Pancreatic Cancer Risk and Outcomes Identified
Inside Precision Medicine
2026-01-12
pancreatic cancer
The findings show that mutations in two genes, FCN1 and PLAT , a part of the innate defense mechanism called the complement system, are associated with an increased risk of developing PDAC. The post Immune-Related Genes that Shape Pancreatic Cancer Risk and Outcomes Identified appeared first on Inside Precision Medicine .
Curasight Announces Encouraging Preliminary Data from Phase 1 Trial Evaluating First-in-Class Radiopharmaceutical uTREAT in High-Grade Gliomas®
PR Newswire
2026-01-12
glioblastoma
Clear uptake of uTREAT® in aggressive brain cancer (glioblastoma) Early data support the potential of first-in-class uPAR-targeted theranostic approach to enable more specific diagnosis and more targeted treatment of high-grade gliomas and other uPAR expressing aggressive solid tumors...
Why uniQure (QURE) Is Up 11.9% After FDA Type A Meeting On AMT-130 Accelerated Pathway
uniQure
2026-01-12
amt-130
gene therapy
accelerated approval
uniQure has already held a Type A meeting with the U.S. FDA to discuss the Biologics License Application data package supporting a potential accelerated approval pathway for AMT-130, its investigational gene therapy for Huntington’s disease. This interaction marks an important regulatory inflection point for AMT-130 after earlier FDA concerns about data sufficiency, potentially reshaping expectations around the program’s development path. We’ll now examine how this FDA Type A meeting on...
Field Medical se presentará en la 44ª Conferencia Anual de Salud de J.P. Morgan
PR Newswire
2026-01-12
til
CARDIFF-BY-THE-SEA, California, 12 de enero de 2026 /PRNewswire/ -- Field Medical Inc., una empresa de tecnología médica en fase clínica que desarrolla una plataforma versátil de ablación por campo pulsado (PFA) de última generación, diseñada para la ablación de taquicardia ventricular...
IN8bio Presents Updated Phase I/II Data Demonstrating Meaningful and Durable Survival Improvements in Newly Diagnosed Glioblastoma
In8Bio
2026-01-12
glioblastoma
Repeat-doses of DeltEx™ Drug-Resistant Immunotherapy gamma-delta (γδ) T cells (DRI) nearly doubled median progression-free survival (mPFS) to 13.0 months compared to only 6.6 months (+97%) in a control cohort treated with the standard-of-care (SOC) Stupp protocol Median overall survival (mOS)
Guess the Therapy Answer January 7, 2026
CGTLive
2026-01-11
gene therapy
Cell and Gene Therapy Live
Rpt-Krystal Biotech Expects VYJUVEK Net Revenue Of $106 Million To $107 Million For Q4 2025
Krystal Biotech
2026-01-11
vyjuvek
Krystal Biotech Inc NASDAQ:KRYS:
Krystal Biotech Announces Preliminary 4Q 2025 VYJUVEK Revenue of $106-$107 Million and Strategic Vision for Growth in Rare Disease Market
Krystal Biotech
2026-01-11
vyjuvek
Krystal Biotech reports preliminary Q4 2025 VYJUVEK revenue of $106-$107 million, emphasizing growth in rare disease treatments.Quiver AI SummaryKrystal Biotech, Inc. announced preliminary fourth quarter 2025 net revenue for its product VYJUVEK, estimated at $106 million to $107 million, contributing to an expected full year revenue of $388 million to $389 million. The company highlighted its robust clinical pipeline aimed at addressing rare dise
Steven W. Pipe, MD, on the End-of-Study Results From Hemgenix’s HOPE-B Trial
CGTLive
2026-01-11
hemgenix
Biotech's 2026 outlook: Six questions for the year ahead
Endpoints News
2026-01-11
pivotal
This feels like a truly pivotal year for the industry, for the markets and for Washington. It’s been one of the biggest periods of change since I started at Endpoints News three-plus years ago. So ...
Krystal Biotech expects Vyjuvek net revenue of $106 million to $107 million for Q4 2025
Krystal Biotech
2026-01-11
vyjuvek
Krystal Biotech Inc: Krystal Biotech Inc expects Vyjuvek net revenue of $106 million to $107 million for Q4 2025 Krystal Biotech provides business update at 44th annual J.P. Morgan Healthcare...
Around the Helix: Cell and Gene Therapy Company Updates – December 10, 2025
CGTLive
2026-01-11
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Krystal Biotech Provides Business Update at 44th Annual J.P. Morgan Healthcare Conference
Krystal Biotech
2026-01-11
vyjuvek
financial results
Preliminary 4Q 2025 VYJUVEK net revenue of $106 million to $107 million Robust clinical pipeline with multibillion dollar opportunities and strong balance sheet for sustained growth PITTSBURGH, Jan. 11, 2026 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) today announced selected preliminary unaudited 2025 financial results, including fourth quarter and full year 2025 VYJUVEK® net product revenue, and outlined the Company’s strategic vision to drive the next stage of gro
CG Oncology: 'Strong Buy' On Earlier Than Expected Completed Recruitment Cretostimogene
CG Oncology
2026-01-11
bla
CG Oncology develops cretostimogene via rolling BLA, phase 3 PIVOT-006 data in 1H 2026, and $680M cash into 2028. Find out why CGON stock is a strong buy.
Beam Therapeutics Sets Strategic Priorities for its Genetic Disease and Hematology Franchises to Drive Execution of Late-Stage Clinical Programs and Extends its Operating Runway through Commercial Transition
beam therapeutics
2026-01-11
bla
accelerated approval
alpha-1 antitrypsin deficiency
Alignment Reached with U.S. FDA on Potential Accelerated Approval Pathway for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) Based on Biomarker Endpoints U.S. B iologics Licensing Application (BLA) Submission for risto-cel (Previously Known as BEAM-101) Expected as Early as Year-End 2026
Inside one company's plan to reach 10,000 rare disease patients
Krystal Biotech
2026-01-11
vyjuvek
Krystal Biotech pegs preliminary Q4 VYJUVEK revenue at $106M-$107M and 2025 at $388M-$389M, with $955M in cash and 2026 non-GAAP spend of $175M-$195M.
FDA Increases Flexibility on Requirements for Cell and Gene Therapies to Advance Innovation
FDA Press
2026-01-11
gene therapies
The U.S. Food and Drug Administration today announced it is sharing information about the agency’s flexible approach to overseeing chemistry, manufacturing and control (CMC) requirements for cell and gene therapies (CGT).
Krystal Biotech Provides Business Update at 44th Annual J.P. Morgan Healthcare Conference
Krystal Biotech
2026-01-11
vyjuvek
Preliminary 4Q 2025 VYJUVEK net revenue of $106 million to $107 million Robust clinical pipeline with multibillion dollar opportunities and strong balance sheet for sustained growth PITTSBURGH,...
uniQure N.V. Investigation Notice: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2026-01-10
bla
amt-130
gene therapy
RADNOR, PA - January 10, 2026 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Krystal Biotech (KRYS) Is Up 5.9% After Positive KB407 CF Gene Therapy Data And FDA Study Filing
Krystal Biotech
2026-01-10
cf
gene therapy
cystic fibrosis
Earlier this week, Krystal Biotech reported positive interim data from the highest-dose cohort of its Phase 1 CORAL-1 trial, showing successful inhaled delivery of KB407 to the lungs of cystic fibrosis patients with confirmed expression of wild-type CFTR protein and a generally mild-to-moderate safety profile. The company also disclosed that it has submitted the design of its planned repeat-dosing CORAL-3 study to the FDA, signaling an accelerated path toward potentially registrational...
Assessing Krystal Biotech (KRYS) Valuation After Positive KB407 Cystic Fibrosis Trial Update
Krystal Biotech
2026-01-10
gene therapy
cystic fibrosis
Krystal Biotech (KRYS) has drawn fresh attention after reporting positive interim data from the highest dose cohort of its Phase 1 CORAL-1 study of inhaled gene therapy KB407 in cystic fibrosis. See our latest analysis for Krystal Biotech. The KB407 update comes after a run of company events in early January, including an investor call, a planned appearance at the J.P. Morgan Healthcare Conference, and recent research coverage changes. The 90 day share price return of 44.08% suggests building...
CG Oncology (CGON) Is Up 29.7% After Accelerating PIVOT-006 Phase 3 Timeline - Has The Bull Case Changed?
CG Oncology
2026-01-10
bladder cancer
cretostimogene grenadenorepvec
CG Oncology has announced that topline data from its Phase 3 PIVOT-006 trial of intravesical cretostimogene grenadenorepvec in intermediate-risk non-muscle invasive bladder cancer will now be available in the first half of 2026, nearly a year earlier than originally planned due to rapid enrollment across more than 90 sites. This is the first randomized Phase 3 registrational study conducted in this under-treated patient group, highlighting both the scale of unmet medical need and the medical...
RYBREVANT® (amivantamab-vmjw) longer-term results show promising and durable responses in difficult-to-treat colorectal cancer
PR Newswire
2026-01-10
colorectal cancer
Over 70 percent of patients in the first-line subgroup responded to amivantamab plus chemotherapy with most responses lasting beyond 16 months Notable responses were also seen in patients with liver metastases, who often face poorer outcomes with this disease RARITAN, N.J., Jan. 10, 2026...
Is It Time To Reassess uniQure (QURE) After 83% One Year Share Price Gain?
uniQure
2026-01-10
gene therapy
If you are wondering whether uniQure's share price lines up with its underlying value, this article walks through the key numbers so you can judge for yourself. Over the past year, uniQure's stock has returned 82.8%, with shorter term moves of 8.3% over 7 days and 25.7% over 30 days from a last close of US$25.32. These shifts can affect how investors think about both upside and risk. Recent news around uniQure has focused on its gene therapy pipeline and partnerships, which helps explain why...
Ribo Life Science (06938.HK) cotiza con éxito en la Bolsa de Valores de Hong Kong
PR Newswire
2026-01-10
til
HONG KONG, 10 de enero de 2026 /PRNewswire/ -- Ribo Life Science Co., Ltd. ("Ribo" o la "compañía", código bursátil: 06938.HK), empresa líder en terapias con ARN de interferencia pequeño (ARNip), ha empezado hoy a cotizar con éxito en la Bolsa de Hong Kong, lo que marca un hito...
Krystal Biotech (KRYS) Is Up 5.9% After Positive KB407 CF Gene Therapy Update and CORAL-3 Plans
Krystal Biotech
2026-01-10
cf
gene therapy
cystic fibrosis
In early January 2026, Krystal Biotech reported a positive interim update from the highest-dose cohort of its Phase 1 CORAL-1 study of inhaled gene therapy KB407 in cystic fibrosis, confirming successful lung delivery and expression of wild-type CFTR protein with generally mild to moderate side effects and one procedure-related serious adverse event that resolved. The company also disclosed that it has submitted the design of its repeat-dosing CORAL-3 study to the US FDA, aiming to advance...
Krystal Biotech (KRYS) Is Up 5.9% After Positive KB407 CF Gene Therapy Update and CORAL-3 Plans
Krystal Biotech
2026-01-10
cf
gene therapy
U.S. stocks, Saudi stocks, stock trading and investment platforms
Cellares and City of Hope Sign Deal to Automate Solid Tumor CAR T Cell Therapy Manufacturing
GenEng News
2026-01-09
car t
solid tumor
cell therapy
City of Hope will evaluate Cellares’ Cell Shuttle™ automated manufacturing platform and Cell Q™ automated quality control system to enable high-throughput manufacturing and quality control of its CARpool program. The post Cellares and City of Hope Sign Deal to Automate Solid Tumor CAR T Cell Therapy Manufacturing appeared first on GEN - Genetic Engineering and Biotechnology News .
FDA to review data on experimental Huntington's gene therapy at uniQure
uniQure
2026-01-09
bla
amt-130
gene therapy
uniQure schedules a Type A FDA meeting to discuss the BLA data for AMT-130, an investigational gene therapy for Huntington's disease.
CG Oncology rises after expediting a late-stage trial readout for lead asset
CG Oncology
2026-01-09
cretostimogene grenadenorepvec
CG Oncology (CGON) shares climbed ~31% on Friday after the company said that a late-stage trial for its lead asset, cretostimogene grenadenorepvec, is now expected to generate topline data in H1 2026, nearly a year earlier than its prior plan. The Phase 3 PIVOT-006 clinical trial is designed to evaluate adjuvant intravesical cretostimogene versus s...
Krystal Biotech says it can deliver native protein to cystic fibrosis patients
Krystal Biotech
2026-01-09
pivotal
gene therapy
cystic fibrosis
Krystal Biotech disclosed promising early findings on a ‘mutation-agnostic’ treatment for cystic fibrosis, and it wants to head directly into a pivotal trial for the gene therapy.
uniQure N.V. Investigation: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2026-01-09
bla
amt-130
gene therapy
RADNOR, PA - January 9, 2026 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Continued Zenocutuzumab Treatment Beyond Progression Shows Benefit in Patients with NRG1+ Pancreatic Cancer and Cholangiocarcinoma: New Results from the eNRGy Trial Presented at ASCO GI
PR Newswire
2026-01-09
nrg1
pancreatic cancer
cholangiocarcinoma
LEXINGTON, Mass., Jan. 9, 2026 /PRNewswire/ -- Partner Therapeutics, Inc. (PTx), a private, fully integrated biotechnology company, announced new data from a post hoc analysis of the eNRGy trial (NCT02912949), evaluating zenocutuzumab in patients with advanced neuregulin 1 fusion-positive...
8-K - uniQure N.V. (0001590560) (Filer)
SEC Filing (8-K)
2026-01-09
uniqure
Filed: 2026-01-09 AccNo: 0001104659-26-002317 Size: 169 KB Item 7.01: Regulation FD Disclosure Item 9.01: Financial Statements and Exhibits
Camurus announces FDA acceptance of NDA resubmission for Oclaiz™ for the treatment of acromegaly
PR Newswire
2026-01-09
pdufa
PDUFA target action date set to 10 June 2026 LUND, Sweden, Jan. 9, 2026 /PRNewswire/ -- Camurus (NASDAQ STO: CAMX) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company's resubmission of the New Drug Application (NDA) for Oclaiz™ (CAM2029),...
CG Oncology to Share Data From Phase 3 Bladder Cancer Trial a Year Ahead of Schedule; Shares Up
CG Oncology
2026-01-09
bladder cancer
CG Oncology shares were up 26% in recent Friday trading after the company said it expects to deliver topline data from its phase 3 clinical trial evaluating cretostimogene monotherapy in patients...
Medipost gets $140M to take Korean stem cell therapy into US
Endpoints News
2026-01-09
allogeneic
cell therapy
A Seoul-area biotech has raised $140 million to bring its allogeneic cell therapy, approved in South Korea since 2012, into the US and Japan. Medipost Co. raised the funding for its Cambridge, MA subsidiary Medipost ...
8-K - Intellia Therapeutics, Inc. (0001652130) (Filer)
SEC Filing (8-K)
2026-01-09
intellia therapeutics
inc.
Filed: 2026-01-09 AccNo: 0001193125-26-008307 Size: 5 MB Item 7.01: Regulation FD Disclosure Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
Patients With SCD Need Better Psychosocial Care
Hematology Advisor Features
2026-01-09
scd
Sickle cell patients are likelier to experience mental health disorders compared to the general population with similar demographics.
CG Oncology rises after expediting a late-stage trial readout for lead asset
CG Oncology
2026-01-09
cretostimogene grenadenorepvec
CG Oncology (CGON) shares climbed ~31% on Friday after the company said that a late-stage trial for its lead asset, cretostimogene grenadenorepvec, is now expected to generate topline data in H1 2026, nearly a year earlier than its prior plan. The Phase 3 PIVOT-006 clinical trial is designed to evaluate adjuvant intravesical cretostimogene versus s...
uniQure Announces Type A Meeting Scheduled with FDA
uniQure
2026-01-09
bla
amt-130
gene therapy
accelerated approval
LEXINGTON, Mass. and AMSTERDAM, Jan. 09, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced a Type A meeting with the U.S. Food and Drug Administration (FDA) has been scheduled to discuss the Biologics License Application (BLA) data package to support accelerated approval of AMT-130, the Company’s investigational gene therapy for the treatment of Huntington’s disease. “We
8-K - CG Oncology, Inc. (0001991792) (Filer)
SEC Filing (8-K)
2026-01-09
cg oncology
Filed: 2026-01-09 AccNo: 0001193125-26-008329 Size: 15 MB Item 7.01: Regulation FD Disclosure Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
uniQure gains as FDA sets up meeting on gene therapy
uniQure
2026-01-09
amt-130
gene therapy
accelerated approval
uniQure (QURE) stock jumps after FDA schedules a Type A meeting to discuss a potential accelerated approval for its AMT-130 gene therapy. Read more here.
Is Accelerated PIVOT-006 Data for Cretostimogene Reshaping the Investment Case for CG Oncology (CGON)?
CG Oncology
2026-01-09
bladder cancer
cretostimogene grenadenorepvec
CG Oncology has announced an expedited timeline, with topline data from its Phase 3 PIVOT-006 trial of intravesical cretostimogene grenadenorepvec in intermediate-risk non-muscle invasive bladder cancer now expected in the first half of 2026, alongside ongoing Phase 2 and Phase 3 studies and an Expanded Access Program in North America. This is the first randomized Phase 3 trial in this intermediate-risk bladder cancer population under AUA/SUO guideline definitions, positioning cretostimogene...
New Approach to Treating ‘Cold’ Neuroblastoma Developed
Inside Precision Medicine
2026-01-09
neuroblastoma
The approach centers on an engineered protein system called HLA-Shuttle, designed to manipulate class I human leukocyte antigen (HLA-I) processing so that tumor cells become visible to T cells and to immunotherapies. The post New Approach to Treating ‘Cold’ Neuroblastoma Developed appeared first on Inside Precision Medicine .
Orca adds $250M in financing to back launch of T cell therapy
Endpoints News
2026-01-09
allogeneic
cell therapy
Orca Bio has pulled together $250 million in new financing for the commercial launch of its treatment for complications from allogeneic stem cell transplants. The money comes in part from two of Orca’s most recent ...
8-K - IOVANCE BIOTHERAPEUTICS, INC. (0001425205) (Filer)
SEC Filing (8-K)
2026-01-09
iovance biotherapeutics
Filed: 2026-01-09 AccNo: 0001104659-26-002323 Size: 8 MB Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
Bone Marrow Immune Cell Map Boosts Survival, Relapse Prediction in Multiple Myeloma
GenEng News
2026-01-09
multiple myeloma
A comprehensive map of the immune cells in bone marrow from multiple myeloma patients links immune composition to disease aggressiveness, patient outcomes, and treatment development strategies. The post Bone Marrow Immune Cell Map Boosts Survival, Relapse Prediction in Multiple Myeloma appeared first on GEN - Genetic Engineering and Biotechnology News .
CG Oncology stock jumps 25% after pulling Phase 3 PIVOT-006 bladder cancer data into 1H 2026
CG Oncology
2026-01-09
bladder cancer
CG Oncology stock jumps 25% after pulling Phase 3 PIVOT-006 bladder cancer data into 1H 2026 - TechStock²
FDA fast tracks CTx001 for geographic atrophy
Healio
2026-01-09
fast track
gene therapy
adeno-associated virus
The FDA granted fast track designation for CTx001, an investigational gene therapy candidate for geographic atrophy secondary to age-related macular degeneration. According to a press release from Complement Therapeutics, CTx001, an adeno-associated virus-based therapy, is designed to treat GA by delivering a truncated version of complement receptor 1, with the aim of yielding “long-term potent
Krystal Biotech rises on new phase 1 data for cystic fibrosis asset
Krystal Biotech
2026-01-09
cystic fibrosis
Krystal Biotech (KRYS) is up ~5% in Friday afternoon trading after reporting additional positive phase 1 data for KB407 for cystic fibrosis. The CORAL-1 dose escalation study is examining KB407 at one, two, or four daily administrations via inhalation. The latest data is on the highest dose cohort. Prior data on the other cohorts was reported in De...
uniQure N.V. Schedules FDA Meeting for AMT-130 Approval
uniQure
2026-01-09
amt-130
On January 9, 2026, uniQure N.V. announced a scheduled Type A meeting with the FDA to discuss AMT-130 approval. Key Highlights: Original SEC Filing: uniQure N.V. [ QURE ] - 8-K - Jan. 09, 2026This is an AI-powered summary. It may contain inaccuracies. Consider verifying important information with…
Acepodia and Biocytogen Expand Collaboration Through Option-based Evaluation Framework for First-in-Class Bispecific and Dual-Payload ADCs (BsAD2C)-Acepodia® | POWERFUL, ACCESSIBLE CELL THERAPIES FOR PATIENTS WITH CANCER
Acepodia
2026-01-09
cell therapies
Acepodia and Biocytogen Expand Collaboration Through Option-based Evaluation Framework for First-in-Class Bispecific and Dual-Payload ADCs (BsAD2C)Expanded collaboration builds on Acepodia and Biocytogen’s recent co-development efforts to evaluate selected bispecific antibody and dual-payload ADC programsBEIJING & ALAMEDA, Calif. & TAIPEI, January 09, 2026, Biocytogen Pharm..
CG Oncology accelerates timeline for bladder cancer trial data By Investing.com
CG Oncology
2026-01-09
bladder cancer
CG Oncology accelerates timeline for bladder cancer trial data
Gene editing pioneers launch first startup focused on custom CRISPR therapies
Endpoints News
2026-01-09
crispr
gene editing
Last spring, gene editing scientists stunned the world when they created a bespoke CRISPR therapy to fix a unique genetic mutation that left a boy, known as Baby KJ, hospitalized since birth. The treatment stabilized ...
Adding Megestrol to Letrozole Improves Antiproliferative Activity in Early-Stage, ER+ Breast Cancer
Hematology Advisor News
2026-01-09
breast cancer
In the PIONEER trial, researchers aimed to assess the change in tumor proliferation when megestrol was added to letrozole in the treatment of early, ER-positive breast cancer.
CG Oncology Accelerates Phase 3 Bladder Cancer Trial Timeline
CG Oncology
2026-01-09
bladder cancer
The latest announcement is out from CG Oncology, Inc. ( ($CGON) ). On January 9, 2026, CG Oncology announced that topline data from its Phase 3 PIVOT-006 trial of a...
Bladder cancer trial to deliver key results nearly a year early
CG Oncology
2026-01-09
bladder cancer
Phase 3 PIVOT-006 topline data now expected in 1H 2026 after rapid enrollment of 360+ patients across 90+ sites in intermediate-risk NMIBC.
Now Iktos teams up with Pierre Fabre in oncology
PharmaPhorum
2026-01-09
pierre fabre
As Servier announces its partnership with Paris, France-based Iktos, now Pierre Fabre Laboratories has revealed an integrated collaboration with Iktos
TCP-25 gel may accelerate wound healing in RDEB: Phase 1 trial
Epidermolysis Bullosa News
2026-01-09
dystrophic epidermolysis bullosa
recessive dystrophic epidermolysis bullosa
TCP-25 gel is well tolerated and may accelerate wound healing in people with recessive dystrophic epidermolysis bullosa, per a Phase 1 trial.
APOE Gene May Be Responsible for Most Alzheimer’s Disease
Inside Precision Medicine
2026-01-09
alzheimer's disease
Research suggests both the ε3 and ε4 variants of the APOE gene should be treated as risk factors for Alzheimer's disease and that together they account for most cases of the condition. The post APOE Gene May Be Responsible for Most Alzheimer’s Disease appeared first on Inside Precision Medicine .
UniQure Announces Type A Meeting Scheduled with FDA
uniQure
2026-01-09
gene therapy
LEXINGTON, Mass. and AMSTERDAM, Jan. 09, 2026 -- uniQure N.V. , a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced a Type A...
Enliven’s Phase 1 leukemia pill data look competitive with Novartis and Terns
Endpoints News
2026-01-09
leukemia
solid tumor
It appears that Enliven Therapeutics’ decision to pivot away from a solid tumor drug now seems like a wise one. Enliven said on Thursday that its tyrosine kinase ...
FDA grants orphan drug status to ARB1002 for pancreatic cancer
Rare Cancer News
2026-01-09
orphan drug
pancreatic cancer
A new experimental treatment for pancreatic cancer, ARB1002, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA) and is expected to enter early clinical testing later this year. The FDA reserves this status for treatments targeting rare diseases, or those affecting fewer than 200,000 people in the U.S. For […] The post FDA grants orphan drug status to ARB1002 for pancreatic cancer appeared first on Rare Cancer News .
uniQure Announces Type A Meeting Scheduled with FDA
uniQure
2026-01-09
gene therapy
LEXINGTON, Mass. and AMSTERDAM, Jan. 09, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative...
CG Oncology’s Bladder Cancer Bet Heads Toward A Big 2026 Test
CG Oncology
2026-01-09
bladder cancer
With its lead therapy posting strong durability signals and key Phase 3 data due in 1H 2026, CGON’s lofty valuation hinges on execution and the FDA path.
Bionova Scientific and Syenex Enter Plasmid DNA Alliance
GenEng News
2026-01-09
plasmid dna
Bionova Scientific, an Asahi Kasei CDMO, engineered its genetically stable cell line platform to eliminate insertion sequence elements (ISEs), cryptic prophages, and nonessential bacterial genomic regions. The post Bionova Scientific and Syenex Enter Plasmid DNA Alliance appeared first on GEN - Genetic Engineering and Biotechnology News .
Assessing uniQure (QURE) Valuation After FDA Setback On AMT-130 Approval Path
uniQure
2026-01-09
bla
amt-130
Regulatory setback reshapes the AMT-130 story uniQure (QURE) is in focus after the FDA reversed its position on using external controls for AMT-130’s approval. This decision has delayed the company’s BLA submission and increased uncertainty around its Huntington’s disease program. See our latest analysis for uniQure. At a share price of $23.15, uniQure has seen a 62.39% decline in its 3 month share price return, even though the 1 year total shareholder return is 45.41%. This suggests that...
Assessing FDA-Approved CAR-T in Patients With Multiple Myeloma and Renal Failure
CGTLive
2026-01-09
car-t
multiple myeloma
Deevyashali Parekh, MBBS, an internal medicine resident at SUNY Upstate Medical University Hospital, discussed findings from a patient population traditionally excluded from clinical trials for CAR-T.
Lexeo Therapeutics Announces Research Collaboration to Explore Targeted Cardiac Delivery of AAV Gene Therapy
Lexeo Therapeutics
2026-01-09
aav
gene therapy
Rampart Closes Down Less Than 2 Years After Launch
BioSpace
2026-01-09
gene therapies
Rampart Bioscience was working on a platform to deliver gene therapies without the need for viral vectors.
Krystal Biotech Touts Positive KB407 CF Gene Therapy Data, Eyes Repeat Dosing and Registrational Path
Krystal Biotech
2026-01-09
cf
gene therapy
cystic fibrosis
Krystal Biotech (NASDAQ:KRYS) said it has generated positive interim clinical results for its inhaled cystic fibrosis (CF) gene therapy candidate KB407, including molecular confirmation of wild-type CFTR protein expression in patients’ lungs. The company discussed the data on a clinical update call
Cause for Progressive Blindness Identified
GenEng News
2026-01-09
retinitis pigmentosa
A large-scale DNA analysis of 5,000 retinitis pigmentosa patients with an unknown genetic cause found both inherited and de novo mutations in multiple variants of RNU genes. The post Cause for Progressive Blindness Identified appeared first on GEN - Genetic Engineering and Biotechnology News .
ASH: Sustained Benefit Seen for Etranacogene Dezaparvovec in Hemophilia B
Hematology Advisor News
2026-01-09
gene therapy
hemophilia b
Gene therapy results in sustained endogenous factor IX expression and low annualized bleeding rates over five years.
CG Oncology Announces Expedited Timeline for Phase 3 PIVOT-006 Trial Data on Cretostimogene for Intermediate-Risk NMIBC
CG Oncology
2026-01-09
bladder cancer
CG Oncology expects PIVOT-006 Phase 3 data for cretostimogene in IR NMIBC in 1H 2026, nearly one year early.Quiver AI SummaryCG Oncology, Inc. has announced that topline data from its Phase 3 PIVOT-006 clinical trial evaluating cretostimogene monotherapy for intermediate-risk non-muscle invasive bladder cancer (IR NMIBC) is now expected in the first half of 2026, nearly a year earlier than originally planned. This trial is significant as it is th
UniQure rises as US FDA review talks revive hopes for Huntington's therapy
uniQure
2026-01-09
amt-130
gene therapy
accelerated approval
U.S.-listed shares of Dutch firm UniQure NASDAQ:QURE, NASDAQ:QURE rise 16% to $26.90 premarket** Co says it has scheduled Type A meeting with U.S. FDA to discuss data for potential marketing application for AMT-130** Co seeks accelerated approval for AMT-130, a gene therapy for Huntington's dise…
uniQure N.V. Investigation: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2026-01-09
bla
amt-130
gene therapy
RADNOR, PA - January 9, 2026 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
‘Reassuring’ data show most with NSCLC receive guideline-concordant biomarker testing
Healio
2026-01-09
lung cancer
Biomarker testing has become “increasingly critical” in the care of non-small cell lung cancer.A new investigation into testing rates proved “reassuring” to Kenneth L. Kehl, MD, MPH, assistant professor of medicine at Harvard Medical School and associate director of clinical research at Dana-Farber Cancer Institute, as researchers found more than 85% of patients treated at
CG Oncology accelerates timeline for bladder cancer trial data By Investing.com
CG Oncology
2026-01-09
bladder cancer
CG Oncology accelerates timeline for bladder cancer trial data
Roche returns to Chinese biotech MediLink for another ADC pact
Endpoints News
2026-01-09
b7-h3
Roche has signed another licensing deal with its China-based partner MediLink, this time focused on an antibody-drug conjugate for solid tumors that targets B7-H3. The Swiss drugmaker will obtain exclusive rights to MediLink’s drug, called ...
Aurora sets out to capitalize on FDA’s new framework for bespoke drug therapies
Biopharma Dive
2026-01-09
gene editing
Co-founded by Jennifer Doudna and Fyodor Urnov, the company intends to simultaneously develop many gene editing treatments for rare conditions by using the agency’s “plausible mechanism” pathway.
Largest Immune Cell Atlas of Multiple Myeloma Predicts Recurrence Risk
Inside Precision Medicine
2026-01-09
multiple myeloma
Using data from over a million cells, scientists have generated an immune cell atlas that could complement and improve current genetic prediction tools to identify multiple myeloma patients at high risk of early relapse. The post Largest Immune Cell Atlas of Multiple Myeloma Predicts Recurrence Risk appeared first on Inside Precision Medicine .
CVS Health to hold fourth quarter and full year 2025 earnings conference call
PR Newswire
2026-01-09
financial results
WOONSOCKET, R.I., Jan. 9, 2026 /PRNewswire/ -- CVS Health® (NYSE: CVS) will hold a conference call with analysts and investors on Wednesday, February 11th, 2026, at 8:00 a.m. ET to discuss fourth quarter and full year 2025 financial results. An audio webcast of the event will be broadcast...
MiNK Therapeutics and University of Wisconsin–Madison Announce Phase 1 Clinical Trial of Allo-iNKT Cell Therapy (AgenT-797) to Evaluate Prevention of Graft-Versus-Host Disease
Mink Therapeutics
2026-01-09
cell therapy
Non-dilutive public-private funded trial with NIAID STTR & Mary Gooze Clinical Trial and Translation Award Expands iNKT platform into transplantation with an off-the-shelf, HLA-independent, lymphodepletion-free experimental therapy in patients at risk for GvHD NEW YORK , Jan.
uniQure gains as FDA sets up meeting on gene therapy
uniQure
2026-01-09
amt-130
gene therapy
uniQure (QURE) added ~14% in the premarket on Friday after the Dutch biotech announced that the U.S. FDA has scheduled a meeting to discuss a marketing application for its gene therapy candidate, AMT-130. In November, the company disclosed that its plans to submit a Biologics License Application for AMT-130 against a neurodegenerative disorder call...
Stem Cell-Derived Neurons Navigate to Form Connections in the Injured Brain
GenEng News
2026-01-08
cell therapies
Study in mouse models of stroke showed how transplanted stem cell-derived neurons contain intrinsic codes for navigating and forming connections with the nervous system, which may help develop future cell therapies. The post Stem Cell-Derived Neurons Navigate to Form Connections in the Injured Brain appeared first on GEN - Genetic Engineering and Biotechnology News .
Early study delivers healthy gene to lungs of people with cystic fibrosis
Krystal Biotech
2026-01-08
cf
cystic fibrosis
KB407 reached 29%-42% of airway cells in 6 CF patients at the top dose, with mostly mild side effects, as Krystal advances the CORAL-3 repeat dosing trial.
Bionova Scientific and Syenex Forge Strategic Alliance to Expand Access to High-Quality Plasmid DNA for Advanced Therapies
PR Newswire
2026-01-08
plasmid dna
HOUSTON, Jan. 8, 2026 /PRNewswire/ -- Bionova Scientific LLC, an Asahi Kasei company and a bespoke contract development and manufacturing organization (CDMO), announced a strategic manufacturing alliance with Syenex, a genetic medicines platform company. This partnership will expand...
CREATE Medicines to Present at the 44th Annual J.P. Morgan Healthcare Conference
PR Newswire
2026-01-08
car-t
Providing interim updates on ongoing in vivo CAR clinical programs Highlighting non-human primate B cell depletion data with proprietary in vivo CAR-T CAMBRIDGE, Mass., Jan. 8, 2026 /PRNewswire/ -- CREATE Medicines, Inc. ("CREATE" or the "Company"), a clinical-stage biotechnology company...
Secretive Rampart Bioscience closes after pursuing non-viral gene therapy
Endpoints News
2026-01-08
gene therapy
Less than 15 months ago, Rampart Bioscience launched with $125 million to solve one of the toughest problems in genetic medicine: figuring out how to deliver genes into cells without a virus ...
FDA accepts IND application for frontotemporal dementia therapeutic
Healio
2026-01-08
ind
frontotemporal dementia
The FDA has accepted an investigational new drug application for an investigational and proprietary combination therapy to treat frontotemporal dementia, according to a release from the manufacturer.“Frontotemporal dementia (FTD) is a devastating progressive neurodegenerative disorder worsening over time, leading to severe changes in behavioral, personality, language and memory
FORMER NFL STARS TEAM UP WITH CDC TO TACKLE LUNG CANCER IN "GEAR UP AGAINST CANCER" CAMPAIGN
PR Newswire
2026-01-08
lung cancer
NFL Alumni expands efforts to prevent and support early detection in Dallas, Kansas City, New York/Northern New Jersey, and Pittsburgh MOUNT LAUREL, N.J., Jan. 8, 2026 /PRNewswire/ -- The National Football League Alumni Association (NFL Alumni) and the Centers for Disease Control and...
‘Mind-blowing’: GLP-1s may be superior to aspirin for colorectal cancer prevention
Healio
2026-01-08
colorectal cancer
GLP-1 receptor agonists may be superior to aspirin for primary prevention of colorectal cancer, according to study results presented at ASCO Gastrointestinal Cancers Symposium.A retrospective analysis of more than 281,000 people showed those who used GLP-1s exhibited a 36% reduced risk for colorectal cancer compared with those who used aspirin. The benefit with GLP-1s appeared even greater among
Tessera Therapeutics to lay off 90 employees, according to document
Endpoints News
2026-01-08
gene editing
Tessera Therapeutics, one of the biggest and most heavily funded gene editing startups, is planning sweeping layoffs that will impact 90 employees just as it begins its first clinical study, according to ...
ProQR Announces Encouraging AX-0810 Phase 1 Safety and PK Data, Development Candidate Selections, and 2026 Outlook
proqr therapeutics
2026-01-08
rett syndrome
Initial AX-0810 data show no safety signals after 4 weeks of dosing and pharmacokinetics consistent with non-clinical data; Phase 1 enrollment and dosing in healthy volunteers ongoing with target engagement data expected in H1 2026, followed by inclusion of a patient cohort Development Candidates selected for pipeline programs AX-2402 for Rett syndrome (MECP2, R270X) and AX-2911 for MASH (PNPLA3) Strategic collaboration with Eli Lilly achieved $4.5 million in milestones in 2025, contributing to strong financial position with runway into mid-2027 LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan. 08, 2026 (GLOBE NEWSWIRE) --
Akadeum Life Sciences to Highlight How Microbubbles Enable Rapid Cell Therapy Manufacturing for Larger Patient Populations at Biotech Showcase 2026
PR Newswire
2026-01-08
cell therapy
ANN ARBOR, Mich., Jan. 8, 2026 /PRNewswire/ -- Akadeum Life Sciences, the leader in buoyant microbubble cell separation, today announced that its Chief Executive Officer Brandon McNaughton, PhD, will present at Biotech Showcase™ 2026 in San Francisco, CA, on January 12, 2026. Dr....
Assessing Krystal Biotech (KRYS) Valuation As Investors Await CORAL-1 KB407 Interim Cystic Fibrosis Data
Krystal Biotech
2026-01-08
cystic fibrosis
U.S. stocks, Saudi stocks, stock trading and investment platforms
Florida Cancer Specialists & Research Institute Expand Global Impact in Gastrointestinal Cancer Research
PR Newswire
2026-01-08
gastrointestinal cancer
Collaborative Research Efforts Advance Treatment Strategies for GI Cancers FORT MYERS, Fla., Jan. 8, 2026 /PRNewswire/ -- Florida Cancer Specialists & Research Institute, LLC (FCS) advances gastrointestinal cancer treatment through participation at the global ASCO® 2025 Gastrointestinal...
uniQure: Now What?
uniQure
2026-01-08
bla
amt-130
phase 1/2
uniQure hits an FDA setback on AMT-130, delaying BLA plans despite strong Phase 1/2 data. Click here to read my latest analysis of QURE stock.
AskBio Announces FDA Acceptance of Investigational New Drug (IND) Application for AB-1009 Gene Therapy for Treatment of Late-Onset Pompe Disease (LOPD)
askbio
2026-01-08
aav
ind
fast track
orphan drug
gene therapy
pompe disease
priority review
accelerated approval
adeno-associated virus
Durham, N.C. – JANUARY 8, 2026 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the United States Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for AB-1009, an adeno-associated virus (AAV) gene therapy being developed for the treatment of late-onset Pompe disease (LOPD). With this announcement, the AB-1009 program advances to Phase 1/Phase 2, and AskBio has initiated a clinical trial in the United States to explore the safety of AB-1009. The company anticipates recruiting its first patient in early 2026. “This investigational gene therapy is being studied for its potential to address the underlying genetic defect and to explore whether it can increase production of the deficient enzyme in patients with Pompe disease,” said Tahseen Mozaffar, MD, Director of the UCI Health ALS & Neuromuscular Center, and Principal Investigator, AB-1009 Clinical Trial Program. “Patients receiving gene therapy may reduce reliance on exogenous enzyme replacement. AskBio’s approach leverages its experience in gene therapy development as it seeks to advance treatment…
Ryoncil Sales for the Quarter Increase 60% to US$35.1M
Mesoblast
2026-01-08
ryoncil
Cas12a3 CRISPR System Targets tRNA Without Destroying Host Cell
GenEng News
2026-01-08
crispr
Researchers report on a CRISPR system known as Cas12a3 that, on recognition of target DNA, is directed to cut the tail off specific tRNAs, halting virus production and spread, while sparing the host cell DNA. The post Cas12a3 CRISPR System Targets tRNA Without Destroying Host Cell appeared first on GEN - Genetic Engineering and Biotechnology News .
Krystal Biotech Reports Positive Early Results for KB407 in Cystic Fibrosis
Krystal Biotech
2026-01-08
gene therapy
Krystal Biotech reported a positive interim update from the highest-dose group in its Phase 1 trial of KB407, an inhaled gene therapy for cystic fibrosis. The update confirmed that the treatment...
Peptide Treatment Protects Brain from Post-Stroke Damage in Mice
Inside Precision Medicine
2026-01-08
ischemic stroke
Researchers have developed a peptide-based treatment that can noninvasively cross the blood-brain barrier and significantly reduce brain damage after an acute ischemic stroke. The post Peptide Treatment Protects Brain from Post-Stroke Damage in Mice appeared first on Inside Precision Medicine .
Preclinical study shows RNA therapy halts glioblastoma growth
Rare Cancer News
2026-01-08
glioblastoma
TTX-MC138, an RNA-based therapy from Transcode Therapeutics that’s in early clinical development, induced tumor cell death and prolonged survival in a mouse model of glioblastoma. That’s according to the study, “Nanotherapy Targeting miR-10b Improves Survival in Orthotopic Glioblastoma Models,” which was published in the Journal of Functional Biomaterials. The work was funded in part by a grant […] The post Preclinical study shows RNA therapy halts glioblastoma growth appeared first on Rare Cancer News .
A2 Biotherapeutics Announces FDA Clearance of IND for A2B543 for Treatment of Adults with Recurrent Unresectable, Locally Advanced, or Metastatic Solid Tumors that Express Mesothelin and Have Lost HLA-A*02 Expression
A2 Bio
2026-01-08
ind
mesothelin
The post A2 Biotherapeutics Announces FDA Clearance of IND for A2B543 for Treatment of Adults with Recurrent Unresectable, Locally Advanced, or Metastatic Solid Tumors that Express Mesothelin and Have Lost HLA-A*02 Expression appeared first on A2 Biotherapeutics .
Assessing Krystal Biotech (KRYS) Valuation As Investors Await CORAL-1 KB407 Interim Cystic Fibrosis Data
Krystal Biotech
2026-01-08
cystic fibrosis
Krystal Biotech (KRYS) is back in focus after the company scheduled a January 8 investor call to share interim data from the highest dose cohort of its Phase 1 CORAL-1 cystic fibrosis study of KB407. See our latest analysis for Krystal Biotech. Krystal Biotech’s recent product news and conference appearances come against a backdrop of a US$246.37 share price, a 30.52% 3 month share price return and a 57.25% 1 year total shareholder return. This suggests momentum has been building as investors...
Allogene Therapeutics Positions 2026 as a Program-Defining Year for Scalable, Real-World Allogeneic CAR T
Allogene
2026-01-08
car t
pivotal
allogeneic
b-cell lymphoma
large b-cell lymphoma
1H 2026 Catalyst Stack Anticipated to Validate Scalable, Off-the-Shelf CAR T in Oncology and Autoimmune Disease Interim Futility Analysis of MRD Clearance from the Pivotal Phase 2 ALPHA3 Trial with Cemacabtagene Ansegedleucel (Cema-Cel) in First-Line (1L) Consolidation Large B-Cell Lymphoma (LBCL)
Circuit Driving Aggressive Pancreatic Cancer Targeted by Splice-Switching ASO
Inside Precision Medicine
2026-01-08
pancreatic cancer
Researchers developed an antisense oligonucleotide (ASO) designed to disrupt alternative splicing of Aurora kinase A ( AURKA ), which essentially short circuits a three-component loop involving AURKA, the splicing factor SRSF1, and the transcription factor MYC. The post Circuit Driving Aggressive Pancreatic Cancer Targeted by Splice-Switching ASO appeared first on Inside Precision Medicine .
Cellectis Announces 2026 Strategy and Catalysts
cellectis
2026-01-08
cd19
cd22
b-all
car-t
pivotal
allogeneic
autologous
gene editing
gene therapies
New York, NY – January 8, 2026 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, today outlined its strategic priorities and key catalysts expected for 2026. “2025 was a transformational year for Cellectis, as we transitioned to a late-stage development allogeneic CAR-T company with the initiation of a pivotal Phase 2 trial for lasme-cel.” said André Choulika, Ph.D., Chief Executive Officer of Cellectis. “As we enter 2026, we remain fully committed to executing our pivotal Phase 2 BALLI-01 trial for lasme-cel in ALL, with interim data expected in Q4, presenting the full Phase 1 data of the NATHALI-01 trial for eti-cel in NHL, and leveraging the momentum of our strategic partnership with AstraZeneca.” Allogeneic CAR-T Pipeline Lasme-cel in r/r B-ALL (BALLI-01) Following the initiation of the pivotal Phase 2 BALLI-01 clinical trial in October 2025, Cellectis expects to complete the first interim analysis in Q4 2026. This upcoming milestone (n=40) builds upon the encouraging Phase 1 clinical data presented at the Cellectis’ R&D Day ,…
Atsena Completes Dosing in Part B of the Phase I/II/III LIGHTHOUSE Trial Evaluating ATSN-201 to Treat X-linked Retinoschisis and Announces Plans for Initiation of Pivotal Part C Cohort
atsena therapeutics
2026-01-08
pivotal
x-linked
x-linked retinoschisis
Atsena Completes Dosing in Part B of the Phase I/II/III LIGHTHOUSE Trial Evaluating ATSN-201 to Treat X-linked Retinoschisis and Announces Plans for Initiation of Pivotal ... The post Atsena Completes Dosing in Part B of the Phase I/II/III LIGHTHOUSE Trial Evaluating ATSN-201 to Treat X-linked Retinoschisis and Announces Plans for Initiation of Pivotal Part C Cohort appeared first on Atsena Therapeutics .
Novel Gene Therapy Approach Targets Pain Centers in the Brain
GenEng News
2026-01-08
gene therapy
A first-of-its-kind brain-targeted gene therapy mimics morphine’s analgesia by targeting pain signals, relieves pain in mice, and avoids addiction risk without harmful side effects. The post Novel Gene Therapy Approach Targets Pain Centers in the Brain appeared first on GEN - Genetic Engineering and Biotechnology News .
SU2C DREAM TEAM TARGETS EARLY DETECTION OF GASTRIC CANCER
PR Newswire
2026-01-08
gastric cancer
Global Team Will Launch Gastric Cancer Prevention Clinical Trial LOS ANGELES, Jan. 8, 2026 /PRNewswire/ -- Stand Up To Cancer® (SU2C) and Cancer Research UK announced today new support for a dream team focused on stopping gastric cancer in its earliest stages. The extended funding for the...
Hengrui gets first OK for a PD-L1/TGF-beta drug for cancer
PharmaPhorum
2026-01-08
gastric cancer
Hengrui has achieved a milestone in oncology as retlirafusp alfa is approved in China, its first world market, for gastric cancer.
Quest Diagnostics to Release Fourth Quarter and Full Year 2025 Financial Results on February 10, 2026
PR Newswire
2026-01-08
financial results
SECAUCUS, N.J., Jan. 8, 2026 /PRNewswire/ -- Quest Diagnostics Incorporated (NYSE: DGX), a leader in diagnostic information services, today announced that it will report fourth quarter and full year 2025 financial results on Tuesday, February 10, 2026, before the market opens. It will...
AstraZeneca’s Saphnelo demonstrates positive results in systemic lupus erythematosus
PMLive Research
2026-01-08
lupus
Over 3.4 million people globally are affected by SLE, and it is one of the leading causes of death in young women in the US
Rabi Hanna, MD, on Evaluating Gene Editing Therapy Reni-Cel for Severe Sickle Cell Disease
CGTLive
2026-01-08
gene editing
sickle cell disease
5 FDA decisions to watch in the first quarter of 2026
Biopharma Dive
2026-01-07
gene therapy
By the end of March, the agency could approve multiple “national priority” voucher winners, as well as a gene therapy it rejected two years ago.
Immuneering’s pancreatic cancer pill continues to beat standard care, updated first-line data show
Endpoints News
2026-01-07
pancreatic cancer
Immuneering’s oral pancreatic cancer therapy demonstrated an overall survival rate of 64% at one year when used as the initial treatment, the company said Wednesday. This is notably better than standard of care, and Immuneering ...
CCGT Sector Needs Purpose-Built Quality Management Systems
GenEng News
2026-01-07
gene therapies
The CGT therapy sector needs purpose-built quality management systems, suggesting technologies designed for large-scale, traditional biopharmaceutical manufacturing are not a good fit for the complex, smaller-scale processes used to make cell and gene therapies. The post CCGT Sector Needs Purpose-Built Quality Management Systems appeared first on GEN - Genetic Engineering and Biotechnology News .
Organ Swaps and Mass Preventative Gene Therapy to Extend Lifespan
Inside Precision Medicine
2026-01-07
gene therapy
Immortal Dragons' founder, Boyang Wang, backs "moonshot" initiatives that aim to improve health and longevity while simultaneously questioning conventional medical wisdom. The post Organ Swaps and Mass Preventative Gene Therapy to Extend Lifespan appeared first on Inside Precision Medicine .
Ernexa’s stem cell therapy could soon enter ovarian cancer trials
Rare Cancer News
2026-01-07
cell therapy
ovarian cancer
Ernexa Therapeutics is making headway on plans to start clinical testing of ERNA-101, its stem cell therapy designed to treat ovarian cancer. The U.S. Food and Drug Administration (FDA) at a recent meeting “provided strong regulatory alignment on Ernexa’s development approach” and outlined a clear pathway for what will be needed for the agency to […] The post Ernexa’s stem cell therapy could soon enter ovarian cancer trials appeared first on Rare Cancer News .
Hutchmed scores with drug for rare autoimmune disease
PharmaPhorum
2026-01-07
pivotal
phase 2/3
Hutchmed is preparing to file for approval of its drug for the rare disorder wAIHA in China, after chalking up a win in a pivotal phase 2/3 trial.
Sarepta Therapeutics Announces Submission of Clinical Trial Application for SRP-1005, its Investigational Treatment for Huntington’s Disease
Sarepta Therapeutics
2026-01-07
sarepta
– The first-in-human clinical study of SRP-1005, known as INSIGHTT, is expected to begin in the second quarter of 2026 CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jan. 7, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the
Autolus Therapeutics to Evaluate Automated Manufacturing of AUCATZYL® (obe-cel) on the Cellares Cell ShuttleTM Platform
Autolus Therapeutics
2026-01-07
aucatzyl
The State of Cell and Gene Therapy 2026
GenEng News
2026-01-07
gene therapy
In The State of Cell and Gene Therapy we present a group of outstanding researchers and thought-leaders to discuss the latest advances and challenges in delivering genetic therapies. Among the confirmed speakers are Jennifer Adair, PhD (UMass Chan Medical School); Cecilia Cotta-Ramusino, PhD (Tessera Therapeutics); and Eriona Hysolli, PhD (Manhattan Genomics). The post The State of Cell and Gene Therapy 2026 appeared first on GEN - Genetic Engineering and Biotechnology News .
Cellular Intelligence Unveils with “AlphaFold Vision” for AI-Driven Cell Therapy
GenEng News
2026-01-07
cell therapy
Formerly Somite AI, the rebranded company aims to broaden the regenerative medicine landscape by building the first universal virtual cell signaling model that predicts how sequences of signaling cues lead to cell fate. The post Cellular Intelligence Unveils with “AlphaFold Vision” for AI-Driven Cell Therapy appeared first on GEN - Genetic Engineering and Biotechnology News .
Adicet Bio Provides Corporate Update and Highlights Expected 2026 Milestones
Adicet Bio
2026-01-07
lupus
Successfully completed meeting with FDA to align on and enable outpatient dosing of systemic lupus erythematosus (SLE) and lupus nephritis (LN) patients with prulacabtagene leucel (prula-cel, formerly ADI-001) in ongoing and future clinical studies All seven cohorts now actively enrolling in Phase
Bacterial Abundance Linked to Immunotherapy Response in Head and Neck Cancer
Inside Precision Medicine
2026-01-07
head and neck cancer
High intratumoral bacterial burden drives resistance to immunotherapy in head and neck cancer through increased neutrophil levels. Antibiotics show potential to improve response in some patients. The post Bacterial Abundance Linked to Immunotherapy Response in Head and Neck Cancer appeared first on Inside Precision Medicine .
PSMA-Targeting Ligand Reduces Salivary Gland Damage in Advanced Prostate Cancer
Inside Precision Medicine
2026-01-07
psma
prostate cancer
A newly engineered prostate-specific membrane antigen (PSMA)-targeting molecule called PSMA-1-DOTA binds more tightly to prostate cancer cells than existing therapeutic ligands and shows significantly lower uptake in salivary tissue. The post PSMA-Targeting Ligand Reduces Salivary Gland Damage in Advanced Prostate Cancer appeared first on Inside Precision Medicine .
Machine Learning Algorithm Effectively Distinguishes Alpha Thalassemia Subtypes
Hematology Advisor News
2026-01-07
beta thalassemia
Researchers determined a machine learning algorithm may be the optimal method for distinguishing beta thalassemia subtypes.
iECURE Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ECUR-506 for Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency
Iecure
2026-01-07
rmat
ornithine transcarbamylase
January 7, 2026 The post iECURE Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ECUR-506 for Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency first appeared on iECURE .
Genprex Provides Clinical Update on Diabetes Gene Therapy Program
Genprex
2026-01-07
gene therapy
Company Achieves Clinical Development Milestones in 2025, Advances Program for Future Scale Up and Studies AUSTIN, Texas — (Jan. 7, 2026) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced a clinical program update on its diabetes gene therapy program […]
CorePlus Collaborates with AIxMED Providing 100% QC on Bladder Cancer Detection with AI
PR Newswire
2026-01-07
bladder cancer
SAN JUAN, Puerto Rico, Jan. 7, 2026 /PRNewswire/ -- CorePlus announced the implementation of AIxURO, a state-of-the-art software solution that utilizes artificial intelligence to significantly improve the accuracy and efficiency of bladder cancer detection. This innovative platform...
Telitacicept shows efficacy vs placebo in adults with systemic lupus erythematosus
Healio
2026-01-07
lupus
Telitacicept, a dual inhibitor of the cytokines B-lymphocyte stimulator and a proliferation-inducing ligand, showed efficacy in systemic lupus erythematosus vs. placebo, according to data published in the New England Journal of Medicine.“Despite several advances in recent years, patients with SLE continue to experience persistent disease activity, frequent flares and the side effects of
FDA grants breakthrough therapy designation to privosegtor for optic neuritis
Healio
2026-01-07
breakthrough therapy
The FDA granted breakthrough therapy designation to privosegtor, a novel peptoid small molecule for the treatment of optic neuritis, according to a press release from Oculis.If approved, privosegtor would become the first neuroprotective therapy for optic neuropathies.
Pediatric Patients with Low-Risk AML Can Receive Fewer Cycles of Chemotherapy
Hematology Advisor News
2026-01-07
aml
leukemia
acute myeloid leukemia
Some pediatric patients with low-risk acute myeloid leukemia can receive 4 cycles of chemotherapy, rather than 5, without affecting outcomes.
Nonprofits partner to bring therapy for Wiskott-Aldrich syndrome to US
AADC News/BioNews
2026-01-07
waskyra
gene therapy
wiskott-aldrich
etuvetidigene autotemcel
wiskott-aldrich syndrome
Fondazione Telethon and Orphan Therapeutics Accelerator, both nonprofit biotech organizations, have signed a Memorandum of Understanding (MoU) to facilitate commercial access in the U.S. to the first gene therapy approved in the nation for the treatment of Wiskott-Aldrich syndrome. The gene therapy, Waskyra (etuvetidigene autotemcel), was recently approved by the U.S. Food and Drug Administration […] The post Nonprofits partner to bring therapy for Wiskott-Aldrich syndrome to US appeared first on AADC News .
American Society of Gene & Cell Therapy and Orphan Therapeutics Accelerator Partner to Advance and Commercialize Promising Rare Disease Treatments
PR Newswire
2026-01-07
cell therapy
gene therapies
First-of-its-kind venture to facilitate and catalyze development of deprioritized cell and gene therapies, supported by AI-enabled platform MILWAUKEE and CAMBRIDGE, Mass., Jan. 7, 2026 /PRNewswire/ -- The American Society of Gene & Cell Therapy (ASGCT), the leading professional...
Inimmune Commences Pivotal Phase 2 Allergy Trial of INI-2004 in Allergic Rhinitis Patients
PR Newswire
2026-01-07
pivotal
MISSOULA, Mont., Jan. 7, 2026 /PRNewswire/ -- Inimmune Corporation ("Inimmune"), a clinical-stage biotechnology company focused on developing innovative immunotherapies, today announced the start of its pivotal Phase 2 clinical trial evaluating INI-2004 in patients with allergic rhinitis....
Autolus tests Cellares’ automated CAR T manufacturing tech
Autolus Therapeutics
2026-01-07
car t
Autolus Therapeutics is working with Cellares to assess automated CAR T manufacturing to support rising demand for its FDA-approved therapy.
Arbele Receives FDA Orphan Drug Designation for ARB1002, an Anti-CDH17 Antibody-Drug Conjugate for the Treatment of Pancreatic Cancer
PR Newswire
2026-01-07
orphan drug
pancreatic cancer
SEATTLE, Jan. 7, 2026 /PRNewswire/ -- Arbele, a biotechnology company focused on developing innovative targeted cancer therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ARB1002, its investigational antibody-drug...
CSA Medical Submits PMA for RejuvenAir® System Following Positive Results from Pivotal SPRAY-CB Trial
PR Newswire
2026-01-07
pivotal
SPRAY-CB met its primary endpoint and multiple secondary endpoints with statistical significance, FDA review underway BOSTON, Jan. 7, 2026 /PRNewswire/ -- CSA Medical, Inc., a medical device company focused on interventional pulmonary therapies, today announced the submission of a...
Krystal Biotech to Announce Interim Clinical Update from Highest Dose Cohort of Phase 1 CORAL-1 Study Evaluating KB407 in Patients with Cystic Fibrosis
Krystal Biotech
2026-01-07
cystic fibrosis
Krystal Biotech, Inc. announced that it will host an investor conference call and webcast on January 8, 2026, to disclose an interim clinical update from CORAL-1, the Company's multi-center, dose...
ICI‑Resistant Melanoma Targeted with Engineered Bispecific Exosome
Inside Precision Medicine
2026-01-07
melanoma
The therapy, called bispecific exosome activator of T cells, or BEAT, delivers two therapeutic proteins at the same time to lung metastases, the most common non-skin site of melanoma metastasis. The post ICI‑Resistant Melanoma Targeted with Engineered Bispecific Exosome appeared first on Inside Precision Medicine .
Amgen Makes Up To $840M Cancer Play With Dark Blue Takeover
BioSpace
2026-01-07
leukemia
acute myeloid leukemia
The acquisition is centered on Dark Blue Therapeutics' small-molecule degrader of the MLLT1 and MLLT3 proteins, which is being tested for acute myeloid leukemia.
Crinetics Advances Potential Neurocrine Competitor With Fresh Data and Money
BioSpace
2026-01-07
congenital adrenal hyperplasia
The company announced a $350 million public offering on Monday shortly after revealing positive Phase II results for its investigational congenital adrenal hyperplasia drug, with hopes to one day compete with Neurocrine’s Crenessity.
Ernexa Therapeutics Announces Successful Pre-IND FDA Meeting and Continues Progress Toward First-in-Human Trial in Ovarian Cancer
Ernexa Therapeutics
2026-01-07
ind
ovarian cancer
8-K - 4D Molecular Therapeutics, Inc. (0001650648) (Filer)
SEC Filing (8-K)
2026-01-07
4d molecular therapeutics
Filed: 2026-01-07 AccNo: 0001193125-26-005470 Size: 164 KB Item 2.02: Results of Operations and Financial Condition Item 5.02: Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers: Compensatory Arrangements of Certain Officers
Ascidian Therapeutics and Forge Biologics Announce Manufacturing Partnership to Advance RNA Editing Therapy for Stargardt Disease
Forge Biologics
2026-01-07
stargardt disease
Ascidian and Forge announce a strategic partnership to advance the development and manufacturing of ACDN-01, Ascidian’s lead retinal program for the treatment of Stargardt disease. The post Ascidian Therapeutics and Forge Biologics Announce Manufacturing Partnership to Advance RNA Editing Therapy for Stargardt Disease appeared first on Forge Biologics .
LENZ Therapeutics Reports Fourth Quarter 2025 Preliminary Unaudited Financial Results and Recent Corporate Updates
LENZ Therapeutics
2026-01-07
financial results
Krystal Biotech to share interim results from cystic fibrosis trial By Investing.com
Krystal Biotech
2026-01-07
cystic fibrosis
Krystal Biotech to share interim results from cystic fibrosis trial
Krystal Biotech Announces Investor Conference Call for Interim Clinical Update on KB407 in Cystic Fibrosis Study
Krystal Biotech
2026-01-07
cystic fibrosis
Krystal Biotech will host an investor call on January 8, 2026, to discuss interim results from its KB407 cystic fibrosis study.Quiver AI SummaryKrystal Biotech, Inc. announced it will hold an investor conference call and webcast on January 8, 2026, at 4:30 pm ET to provide an interim clinical update from its Phase 1 CORAL-1 study, which is assessing the efficacy of KB407 in cystic fibrosis patients. The update will highlight results from the high
Nonprofits partner to bring Wiskott-Aldrich syndrome therapy to US
Fondazione Telethon
2026-01-07
wiskott-aldrich
wiskott-aldrich syndrome
A nonprofit partnership aims to expand market access to therapies for ultra-rare diseases, starting with Wiskott-Aldrich syndrome.
AbbVie to Host Full-Year and Fourth-Quarter 2025 Earnings Conference Call
Abbvie Investors
2026-01-07
financial results
NORTH CHICAGO, Ill. , Jan. 7, 2026 /PRNewswire/ -- AbbVie (NYSE: ABBV) will announce its full-year and fourth-quarter 2025 financial results on Wednesday, February 4, 2026 , before the market opens. AbbVie will host a live webcast of the earnings conference call at 8 a.m. Central time .
Intellia Therapeutics to Present at the 44th Annual J.P. Morgan Healthcare Conference
Intellia Therapeutics Inc.
2026-01-07
crispr
gene editing
CAMBRIDGE, Mass. , Jan. 07, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that the company will present at the 44 th Annual J.P.
Roche's patent deal with Structure; AstraZeneca reports lupus data
Roche Group
2026-01-06
lupus
Plus, news about Instil Bio, Gilead and OncoNano, MetaVia, Variant and Boehringer.
Live Cell Phenotyping Stratifies CLL and Predicts Response to Targeted Therapy
Hematology Advisor News
2026-01-06
cll
Machine-learning analysis of untreated CLL samples identified 5 stable patient clusters associated with distinct therapeutic sensitivities.
CG Oncology Announces Participation in 44th Annual J.P. Morgan Healthcare Conference
CG Oncology
2026-01-06
bladder cancer
CG Oncology executives will present at the J.P. Morgan Healthcare Conference on January 15, 2026, discussing bladder cancer therapies.Quiver AI SummaryCG Oncology, Inc., a biopharmaceutical company focused on developing treatments for bladder cancer, announced that its leadership, Chairman and CEO Arthur Kuan and President and COO Ambaw Bellete, will present at the 44th Annual J.P. Morgan Healthcare Conference on January 15, 2026. The presentatio
Jazz and Zymeworks spell out HER2 bispecific win
Endpoints News
2026-01-06
her2
Jazz Pharmaceuticals and Zymeworks detailed successful results from a late-stage trial of Ziihera that they say support the potential of replacing Herceptin in a form of advanced stomach and esophagus cancer. The drugmakers
New Study Finds Millions of Americans With Heart Failure Are Not Receiving Proven, Life-Saving Therapy
PR Newswire
2026-01-06
heart failure
Nationwide analysis from Motive Medical Intelligence reveals widespread underuse of low-cost, evidence-based medications proven to decrease the risk of death SAN FRANCISCO, Jan. 6, 2026 /PRNewswire/ -- Millions of Americans living with heart failure are not receiving medications that have...
CG Oncology to Present at the 44th Annual J.P. Morgan Healthcare Conference
CG Oncology
2026-01-06
bladder cancer
IRVINE, Calif., Jan. 06, 2026 (GLOBE NEWSWIRE) -- CG Oncology, Inc. (NASDAQ: CGON), a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients with bladder cancer, today announced that Arthur Kuan, Chairman & Chief Executive Officer, and Ambaw Bellete, President & Chief Operating Officer, will present at the 44th Annual J.P. Morgan Healthcare Conference on January 15, 2026, at 8:15 am PT / 11:15 am ET. The conference is taking place at the Westin St. Francis San Francisco, California.
Dark Blue Therapeutics acquired by Amgen for up to $840 million
PR Newswire
2026-01-06
leukemia
acute myeloid leukemia
Lead candidate in the MLLT1/3 programme, DBT 3757, is a differentiated investigational molecule designed to treat Acute Myeloid Leukemia Acquisition brings a new therapeutic mechanism to Amgen's extensive cancer portfolio – Dark Blue has pioneered the biology of targeting MLLT1/3, and DBT...
Deevyashali Parekh, MBBS, on Evaluating CAR-T in Patients With Multiple Myeloma and Renal Failure
CGTLive
2026-01-06
car-t
multiple myeloma
Ziihera® (zanidatamab-hrii) Combinations Achieve Unprecedented Results in First-Line HER2+ Locally Advanced or Metastatic GEA Including More Than Two Years Median Overall Survival Benefit
PR Newswire
2026-01-06
her2
Positive Phase 3 HERIZON-GEA-01 results support Ziihera as the HER2-targeted agent-of-choice in HER2+ first-line metastatic GEA and Ziihera plus chemotherapy to replace trastuzumab as the new standard of care, with or without tislelizumab regardless of PD-L1 status Late-breaking results...
CRISPR Screen Uncovers Genes Driving Brain Cell Development, Neurodevelopmental Disorder
GenEng News
2026-01-06
crispr
gene editing
With the help of gene editing tools, scientists uncovered hundreds of genes required for brain cell differentiation and implicated one gene, PEDS1, in a severe neurodevelopmental disorder. The post CRISPR Screen Uncovers Genes Driving Brain Cell Development, Neurodevelopmental Disorder appeared first on GEN - Genetic Engineering and Biotechnology News .
Automated Manufacturing Will Meet CAR T Demand
BioSpace
2026-01-06
car t
Next-generation automation is closing the gap between curative science and real-world demand, enabling faster development, global consistency and broader patient access to CAR T therapies.
Genprex Announces Positive Preliminary Preclinical Data from Study of GPX-002 in Type 2 Diabetic Animal Studies
Genprex
2026-01-06
gene therapy
Preliminary Preclinical Data Demonstrates In Vivo Proof-of-Concept for Novel Diabetes Gene Therapy in Type 2 Diabetic Non-Human Primates GPX-002 Rejuvenated Exhausted Beta Cells and Normalized Glucose Levels in Type 2 Diabetic Mice AUSTIN, Texas — (Jan. 6, 2026) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for […]
Saphnelo self-administration TULIP-SC trial demonstrates statistically significant and clinically meaningful reduction in systemic lupus erythematosus disease activity 06 January 2026
AstraZeneca
2026-01-06
lupus
Lilly Doubles Down With Nimbus, Paying $55M for Preclinical Obesity Drug
BioSpace
2026-01-06
pivotal
Eli Lilly kicked off a pivotal 2026 campaign with a multi-year collaboration with current partner Nimbus Therapeutics worth a potential $1.3 billion for a preclinical obesity therapeutic. The deal follows a 2022 partnership struck by the companies to target the AMPK protein in cardiometabolic diseases.
Tiger BioSciences Announces Acquisition of Advanced Wound Care Technology, Expanding Its Autologous Wound Dressing Therapy Capabilities
PR Newswire
2026-01-06
autologous
CONSHOHOCKEN, Pa., Jan. 6, 2026 /PRNewswire/ -- Tiger BioSciences, a leader in regenerative medicine and tissue innovation, today announced the acquisition of Platelet-Rich Fibrin Matrix (PRFM) technology for advanced wound care, including its proprietary PRFM tubes and unique wound...
FDA Approves Waskyra For Treatment of Wiskott-Aldrich Syndrome
Fondazione Telethon
2026-01-06
gene therapy
wiskott-aldrich
ex vivo gene therapy
wiskott-aldrich syndrome
Waskyra’s FDA approval marks a milestone for rare disease care and for Fondazione Telethon, establishing the first nonprofit-led ex vivo gene therapy to reach market after decades of research.
Lineage Takes Delivery of Gene-edited Hypoimmune Cell Line Under Partnership With Factor Bioscience
lineage cell therapeutics
2026-01-06
allogeneic
cell therapies
CARLSBAD, Calif. --(BUSINESS WIRE)--Jan. 6, 2026-- Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel allogeneic, or “off the shelf,” cell therapies for serious medical conditions, today announced the receipt of a novel, induced
Taysha Gene Therapies Announces Progress Across TSHA-102 Pivotal Gene Therapy Program in Rett Syndrome
Taysha Gene Therapies
2026-01-06
pivotal
gene therapy
rett syndrome
gene therapies
First patient dosed in REVEAL pivotal trial evaluating TSHA-102 (N=15, aged 6 to 22 years) in Q4 2025, with enrollment advancing across multiple sites Reached written alignment with FDA on inclusion of ≥3 months of safety data from ASPIRE trial evaluating TSHA-102 (N=3, aged 2 to 4 years) in
Lung Cancer Research Foundation Announces Additional 2025 Scientific Research Grant Awards
PR Newswire
2026-01-06
lung cancer
Foundation funds five new research projects, bringing 2025 total to $5.2 million NEW YORK, Jan. 6, 2026 /PRNewswire/ -- The Lung Cancer Research Foundation® (LCRF) recently awarded five new research grants in the following areas: three LCRF Leading-Edge Research Awards, one LCRF Research...
Autolus Therapeutics to evaluate automated manufacturing of AUCATZYL
Autolus Therapeutics
2026-01-06
aucatzyl
Autolus Therapeutics (AUTL) announced that Autolus will assess the feasibility of Cellares’ Cell Shuttle platform to complement its commercial manufacturing operati...
Autolus Therapeutics to Evaluate Automated Manufacturing of AUCATZYL® (obe-cel) on the Cellares Cell Shuttle™ Platform
syncona
2026-01-06
aucatzyl
Expanding Healthcare Access for Women's Health -- Noul Unveils AI-Automated Cervical Cancer Diagnostics Solution at CES 2026
PR Newswire
2026-01-06
cervical cancer
Dramatically cuts diagnostic workflows by 80%…delivering results in under 20 minutes Decentralized, fully automated diagnostic model helps reduce healthcare infrastructure gaps and supports WHO's 2030 cervical cancer screening goals Eco-friendly, sustainable AI diagnostics with NGSI...
Autolus Therapeutics to Evaluate Automated Manufacturing of AUCATZYL® (obe-cel) on the Cellares Cell ShuttleTM Platform
Autolus Therapeutics
2026-01-06
aucatzyl
Autolus Therapeutics plc (Nasdaq: AUTL), a commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T c...
Integrating Gene Therapy into Clinical Care for Sickle Cell Disease
CGTLive
2026-01-06
gene therapy
sickle cell disease
Crawford Strunk, MD, an associate staff member at the Cleveland Clinic, discussed the institution's experience with integrating use of exa-cel and lovo-cel.
First Patients Enrolled in Groundbreaking Brain Cancer Study at Nationally Recognized Academic Medical Center
PR Newswire
2026-01-06
glioblastoma
Trial investigates the FDA Breakthrough device "Tumor Monorail," a paradigm-shifting approach for tracking and treating recurring glioblastoma, one of the deadliest cancers, in real time - DURHAM, N.C., Jan. 6, 2026 /PRNewswire/ -- Exvade Bioscience™, a clinical-stage neuro-oncology...
Roche's patent deal with Structure; AstraZeneca reports lupus data
Endpoints News
2026-01-06
lupus
Plus, news about Instil Bio, Gilead and OncoNano, MetaVia, Variant and Boehringer. 🤝 Structure Therapeutics reaches licensing deal with Roche: The California biotech granted what’s known as a “freedom to operate ...
Bladder cancer therapy company to speak at J.P. Morgan event
CG Oncology
2026-01-06
bladder cancer
CG Oncology leaders present Jan. 15 at 8:15 am PT at the 44th J.P. Morgan Healthcare Conference, with a live webcast and 90-day replay.
Instil Bio’s Subsidiary Discontinues Clinical Development of AXN-2510 and Terminates License and Collaboration Agreement with ImmuneOnco
Instil Bio
2026-01-06
til
DALLAS, Jan. 06, 2026 (GLOBE NEWSWIRE) -- Instil Bio, Inc. (Nasdaq: TIL) (“Instil”) today announced that Axion Bio, Inc. (“Axion”), a wholly-owned subsidiary of Instil, has decided to discontinue clinical development of AXN-2510 and that Axion and ImmuneOnco Biopharmaceuticals (Shanghai) Inc.
J&J plans late-stage trial for lupus candidate after Phase 2 success
Endpoints News
2026-01-06
lupus
Johnson & Johnson’s monoclonal antibody Imaavy succeeded in a mid-stage lupus trial, bringing the company one step closer to its goal of expanding the drug in other autoimmune diseases. Imaavy met its primary endpoint of ...
Amgen buys protein-degrading startup Dark Blue for up to $840M
Biopharma Dive
2026-01-06
leukemia
acute myeloid leukemia
The deal hands Amgen a drug that destroys two proteins known to drive certain types of acute myeloid leukemia, a profile that could set it apart from current medications.
Sanofi's MS Drug Tripped Up By Toxicities, Unclear Benefit, Rejection Letter Reveals
BioSpace
2026-01-06
complete response
multiple sclerosis
Last month, the FDA declined to approve Sanofi’s tolebrutinib for a specific form of multiple sclerosis. In a recently published complete response letter, the agency detailed its reasoning behind the rejection.
New Potential MS Biomarker Could Inform Drug Response, Disease Progression
GenEng News
2026-01-05
multiple sclerosis
Researchers identified a possible biomarker linked to multiple sclerosis disease progression. Their findings, validated in both mice and humans, could help to identify patients most likely to benefit from new drugs that target leptomeningeal inflammation. The post New Potential MS Biomarker Could Inform Drug Response, Disease Progression appeared first on GEN - Genetic Engineering and Biotechnology News .
Sarepta Therapeutics to Present at the 44th Annual J.P. Morgan Healthcare Conference
Sarepta Therapeutics
2026-01-05
sarepta
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jan. 5, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will present at the 44th Annual J.P. Morgan Healthcare Conference in San Francisco, Calif. on Monday, Jan.
Phase 3 blood cancer vaccine trial moves toward final analysis
Rare Cancer News
2026-01-05
aml
leukemia
acute myeloid leukemia
A Phase 3 clinical trial testing the experimental cancer vaccine galinpepimut-S (GPS) in people with acute myeloid leukemia (AML), a type of blood cancer, is nearing its end, according to an update from the therapy’s developer, Sellas Life Sciences. The Phase 3 trial, dubbed REGAL (NCT04229979), enrolled more than 100 people with AML who were […] The post Phase 3 blood cancer vaccine trial moves toward final analysis appeared first on Rare Cancer News .
How Investors Are Reacting To BioMarin (BMRN) Halting BMN349 And Refocusing Its Rare Disease Pipeline
BioMarin Pharmaceutical
2026-01-05
alpha-1 antitrypsin deficiency
On December 19, 2025, BioMarin Pharmaceutical Inc. announced it would discontinue development of BMN349, an oral therapy for Alpha-1 antitrypsin deficiency-associated liver disease. This move trims BioMarin’s rare disease pipeline and may sharpen management’s focus on other late-stage programs and recently acquired assets. We’ll now explore how discontinuing BMN349 could reshape BioMarin’s investment narrative built around pipeline progression and rare disease focus. Trump has pledged to...
Lineage Cell Therapeutics Issues Letter to Stockholders
lineage cell therapeutics
2026-01-05
allogeneic
cell therapies
CARLSBAD, Calif. --(BUSINESS WIRE)--Jan. 5, 2026-- Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel allogeneic, or “off the shelf,” cell therapies for serious medical conditions, today published a letter to shareholders
Fate Therapeutics Reports New Employee Inducement Awards Under Nasdaq Listing Rule 5635(c)(4)
Fate Therapeutics
2026-01-05
ipsc
SAN DIEGO , Jan. 05, 2026 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases,
Pirtobrutinib Approved for R/R CLL/SLL After Covalent BTK Therapy
Hematology Advisor News
2026-01-05
cll
The approval, which was based on results from the BRUIN CLL-321 trial, expands the treatment options for patients earlier in their disease course.
Guideline-Concordant Care Rate Roughly 60 Percent After Positive Lung Cancer Screen in Seniors
Hematology Advisor News
2026-01-05
lung cancer
Among persons with a first positive lung cancer screening result at age 65 years or older, the rate of guideline-concordant care is 59.7%.
5 Clinical Readouts to Watch in H1 2026
BioSpace
2026-01-05
pivotal
As 2026 begins, a slate of high-stakes clinical readouts—from a pivotal study of Novartis’ cardiovascular candidate pelacarsen to a Phase III test of Eli Lilly’s next-gen Alzheimer's drug—are poised to reshape therapeutic landscapes.
AbbVie Challenges Amgen with $100M Upfront for Trispecific Lung Cancer Drug
BioSpace
2026-01-05
lung cancer
The Illinois-based pharma has committed more than $1 billion in milestones to secure rights to ZG006 and join a who’s who of drugmakers targeting the DLL3 protein.
Veligrotug Receives FDA Priority Review for Thyroid Eye Disease
Hematology Advisor News
2026-01-05
priority review
The FDA has accepted for Priority Review the Biologics License Application for veligrotug for the treatment of thyroid eye disease.
Protara Therapeutics Receives Both FDA Breakthrough Therapy and Fast Track Designations for TARA-002 in Pediatric Patients with Lymphatic Malformations
protara therapeutics
2026-01-05
fast track
breakthrough therapy
TARA-002 selected for FDA manufacturing development and readiness pilot program NEW YORK , Jan. 05, 2026 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced that
Aqvesme Approved for Anemia in Alpha- or Beta-Thalassemia
Hematology Advisor News
2026-01-05
beta-thalassemia
The FDA has approved Aqvesme (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia.
Esophageal pH Monitoring Capsule Recall: Medtronic and Given Imaging Remove Bravo CF Capsule Delivery Devices
FDA MedWatch
2026-01-05
cf
This communication is part of the Communications Pilot to Enhance the Medical Device Recall Program
FDA raises new questions with Sanofi's MS drug in release of rejection letter
Endpoints News
2026-01-05
multiple sclerosis
The FDA offered new details on why it rejected Sanofi's potential new multiple sclerosis drug late last year — a peek behind the veil that goes beyond what Sanofi said in public statements at the time ...
GenEditBio Receives FDA Clearance of IND Application for Its Lead In Vivo Genome-Editing Program GEB-101 for TGFBI Corneal Dystrophy
PR Newswire
2026-01-05
ind
HONG KONG, Jan. 5, 2026 /PRNewswire/ -- GenEditBio Limited ("GenEditBio"), a clinical-stage biotechnology startup focusing on genome-editing therapeutic solutions, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company's Investigational New Drug (IND)...
Sanofi’s Tzield accepted for priority review in the US for young children with stage 2 type 1 diabetes
sanofi
2026-01-05
priority review
CRISPR Therapeutics to Present at the 44th Annual J.P. Morgan Healthcare Conference
CRISPR
2026-01-05
crispr
ZUG , Switzerland and BOSTON, Jan. 05, 2026 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that members of its senior management team will present at the 44th Annual
Dried Blood Spot Testing Accurately Measures Alzheimer’s Biomarkers
Inside Precision Medicine
2026-01-05
alzheimer's disease
Less invasive dried blood spot testing is accurate for detection of a key Alzheimer's disease biomarker, phosphorylated tau‑217, although it does not yet match the performance of standard blood tests for the same marker. The post Dried Blood Spot Testing Accurately Measures Alzheimer’s Biomarkers appeared first on Inside Precision Medicine .
Zenas Bio Inflammation Drug Meets Phase 3 Goals, But Is It Enough to Compete With Amgen?
MedCity News
2026-01-05
pivotal
Zenas Biopharma’s obexelimab achieved a statistically significant and clinically meaningful reduction in signs and symptoms of immunoglobulin G4-related disease, but not to the extent shown by Amgen’s Uplizna in its pivotal study. Still, Zenas points to features of its drug that could make it competitive as a maintenance therapy for the rare autoimmune disorder. The post Zenas Bio Inflammation Drug Meets Phase 3 Goals, But Is It Enough to Compete With Amgen? appeared first on MedCity News .
Zenas BioPharma’s Phase 3 autoimmune disease readout disappoints
Endpoints News
2026-01-05
pivotal
Zenas BioPharma said its experimental antibody succeeded in a pivotal study for IgG4-related disease, an autoimmune condition, and that it plans to seek FDA approval. But the topline findings compare poorly to an on-the-market rival ...
Synthetic Progesterone Enhances Anti-Estrogen Therapy in Early Breast Cancer
Inside Precision Medicine
2026-01-05
breast cancer
The findings provide added evidence that low doses of the drug megestrol acetate could alter how ER-positive breast cancer is treated in the future. The post Synthetic Progesterone Enhances Anti-Estrogen Therapy in Early Breast Cancer appeared first on Inside Precision Medicine .
Kyverna Therapeutics to Present at the J.P. Morgan 2026 Healthcare Conference
Kyverna Therapeutics
2026-01-05
cell therapies
EMERYVILLE, Calif., Jan. 05, 2026 (GLOBE NEWSWIRE) -- Kyverna Therapeutics, Inc. (Kyverna, Nasdaq: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, today announced that Warner Biddle, Chief Executive Officer of Kyverna,
Federal guidelines for cervical cancer screening now include self-collection option
Healio
2026-01-05
cervical cancer
HHS’s Health Resources and Services Administration released updated guidance for cervical cancer screening, which includes a new option for average-risk women to self-collect vaginal samples for HPV testing.The guidance, published in JAMA, “is designed to help close the screening gap by expanding access and reducing cost barriers,” HRSA told Healio.
Starting a new year with hope for myeloma treatment
Rare Cancer News
2026-01-05
multiple myeloma
I’m writing this a few days after the end of ASH 2025, the annual meeting of the American Society of Hematology. As I mentioned in a previous column, more than 8,000 abstracts were presented at the gathering, held Dec. 6-9 in Orlando, Florida. Those who cover multiple myeloma news are still sorting through a lot […] The post Starting a new year with hope for myeloma treatment appeared first on Rare Cancer News .
CAR T’s Decade of Growing Pains Just Got Personal
GenEng News
2026-01-05
car t
Hana El‑Samad, PhD, Editor in Chief of GEN Biotechnology , examines the uneven progress of CAR T therapy—its successes, failures, scientific bottlenecks, and systemic barriers—and outlines what must change for the field to deliver broader, safer impact. The post CAR T’s Decade of Growing Pains Just Got Personal appeared first on GEN - Genetic Engineering and Biotechnology News .
HEMOSTEMIX CLOSES $480,000 PRIVATE PLACEMENT AT $0.12 PER SHARE
Hemostemix
2026-01-05
autologous
cardiomyopathy
Calgary, Alberta – December 31, 2025 – Hemostemix Inc. (TSXV: HEM | OTCQB: HMTXF | FSE: 2VF0) (the “ Company ” or “ Hemostemix ”), an autologous stem cell company treating those who suffer in pain from peripheral arterial disease, chronic limb threatening ischemia, angina, ischemic cardiomyopathy, dilated cardiomyopathy, total body ischemia, and vascular dementia, is pleased to announce that it has closed a non-brokered private placement for aggregate gross proceeds of CDN$480,000, through the issuance of 4,000,000 common shares of the Company at a price of $0.12 per common share.
Crawford Strunk, MD, on Integrating Gene Therapy into Clinical Care for Sickle Cell Disease
CGTLive
2026-01-05
gene therapy
sickle cell disease
QURE Investigation: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2026-01-04
bla
amt-130
gene therapy
RADNOR, PA - January 4, 2026 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Downfall of BioMarin's Kyndrisa raises more doubts on Sarepta's eteplirsen
BioMarin Pharmaceutical
2026-01-04
sarepta
Downfall of BioMarin's Kyndrisa raises more doubts on Sarepta's eteplirsen
Announced FDA approval of a Gene Therapy for Wiskott-Aldrich Syndrome
Fondazione Telethon
2026-01-04
gene therapy
wiskott-aldrich
ex vivo gene therapy
wiskott-aldrich syndrome
Fondazione Telethon announced that the U.S. Food and Drug Administration (FDA) has approved an ex vivo gene therapy for Wiskott-Aldrich syndrome (WAS).
UniQure attempts to keep comeback on track with showcase
uniQure
2026-01-03
gene therapy
Dutch biotech uniQure, the company which failed with its attempts to market Glybera (alipogene tiparvovec), the world’s first approved gene therapy, could have
The Importance of Palliative Care in Sickle Cell Disease
CGTLive
2026-01-03
sickle cell disease
Crawford Strunk, MD, an associate staff member at the Cleveland Clinic, discussed a study he presented at ASH’s 2025 Annual Meeting.
Crawford Strunk, MD, on the Importance of Palliative Care in Sickle Cell Disease
CGTLive
2026-01-03
sickle cell disease
Intellia Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Intellia Therapeutics Inc.
2026-01-02
crispr
gene editing
CAMBRIDGE, Mass. , Jan. 02, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that on January 1, 2026 , it awarded inducement grants to two new employees
NICE recommends wider use of Biogen MS drug via NHS
PharmaPhorum
2026-01-02
multiple sclerosis
NICE has backed NHS use of natalizumab, a key treatment option that is safe for use in pregnancy, for more patients with multiple sclerosis.
Rare Inherited Genetic Variant Protects Against Blood Cancer, Reduces Leukemia Risk
GenEng News
2026-01-02
leukemia
A rare inherited genetic variant slows mutant blood stem cell growth, reducing clonal hematopoiesis, leukemia risk, and blood cancer progression by dampening MSI2-driven stem cell expansion. The post Rare Inherited Genetic Variant Protects Against Blood Cancer, Reduces Leukemia Risk appeared first on GEN - Genetic Engineering and Biotechnology News .
Phase 3 trial testing if oral pill can stop pancreatic cancer from returning
Rare Cancer News
2026-01-02
pancreatic cancer
A new Phase 3 clinical trial has officially begun dosing patients to see if an experimental oral therapy can prevent the recurrence of pancreatic cancer following surgery. The study, known as RASolute 304 (NCT07252232), is testing daraxonrasib, a targeted therapy designed to block the genetic “on-switch” that drives tumor growth in the majority of pancreatic […] The post Phase 3 trial testing if oral pill can stop pancreatic cancer from returning appeared first on Rare Cancer News .
QURE Investigation Alert: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2026-01-02
bla
amt-130
gene therapy
RADNOR, PA - January 2, 2026 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Genmab ends lung cancer drug development
PharmaPhorum
2026-01-02
lung cancer
Genmab has decided to stop development of a drug for cancer, acasunlimab for non-small cell lung cancer, following a review of its pipeline.
Deucrictibant shortens time to onset of symptom relief after hereditary angioedema attack
Healio
2026-01-02
hereditary angioedema
Patients with hereditary angioedema using deucrictibant as an on-demand treatment for attacks achieved onset of symptom relief and complete symptom resolution faster than those using placebo, according to RAPIDe-3 study topline results.“Bradykinin B2 receptor antagonism is a clinically proven and effective mechanism for treatment of bradykinin-mediated angioedema attacks,” Peng Lu,
Gene Therapy: 5 Biotech Stocks Chasing the $36B Prize
PR Newswire
2026-01-02
gene therapy
Issued on behalf of Avant Technologies Inc. VANCOUVER, BC, Jan. 2, 2026 /PRNewswire/ -- Equity Insider News Commentary – The gene therapy market is surging toward $36.55 billion by 2032, powered by rising demand for curative treatments that target genetic diseases at their source[1]. The...
Ultragenyx Loses $1B in Market Value as Bone Drug Fails To Reduce Fractures
BioSpace
2026-01-02
angelman syndrome
With a pair of Phase III trial flops, Ultragenyx will explore cost reductions as analysts turn attention to an upcoming Angelman syndrome readout.
AML Therapy May Benefit from Breast Cancer Drug
GenEng News
2026-01-02
aml
leukemia
breast cancer
Preclinical studies in human AML samples and mouse models showed that adding palbociclib to venetoclax produced stronger, more durable anti‑leukemia activity than venetoclax alone and may help overcome treatment resistance. The post AML Therapy May Benefit from Breast Cancer Drug appeared first on GEN - Genetic Engineering and Biotechnology News .
FasTCAR Manufactured BCMAxCD19 CAR T-cell Therapy Shows Promise for Multiple Myeloma
CGTLive
2026-01-01
car t
cell therapy
multiple myeloma
AZD0120, a dual-targeted CAR T-cell therapy, shows high efficacy in treating relapsed multiple myeloma, achieving a 96% response rate.
FDA Approves BMS’s Liso-Cel for R/R Marginal Zone Lymphoma
CGTLive
2026-01-01
marginal zone lymphoma
Sarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Sarepta Therapeutics
2025-12-31
sarepta
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Dec. 31, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, granted equity awards on Dec. 31, 2025 that were previously approved by the Compensation Committee of its Board of Directors under Sarepta’s 2024
FDA declines to approve relacorilant for hypertension linked to hypercortisolism
Healio
2025-12-31
complete response
The FDA has issued a complete response letter for an oral selective glucocorticoid receptor antagonist under investigation for the treatment of hypertension secondary to hypercortisolism, according to an industry press release.Corcept Therapeutics announced the FDA issued a complete response letter for relacorilant (Corcept Therapeutics). The drug is under investigation for the treatment of
Janssen Pharmaceutica patents new SMARCA2 inhibitors for NSCLC
Janssen Global Services
2025-12-31
lung cancer
Janssen Pharmaceutica NV has synthesized new 1,6-naphthridine compounds acting as probable global transcription activator SNF2L2 (SMARCA2; BAF190B; SNF2-α) inhibitors reported to be useful for the treatment of non-small-cell lung cancer (NSCLC).
Autolus presents CARLYSLE trial data for lupus treatment at ASH meeting
Autolus Therapeutics
2025-12-31
lupus
Autolus Therapeutics (NASDAQ: AUTL) presented updated clinical data from its Phase 1 CARLYSLE trial of obecabtagene autoleucel (obe-cel) in patients with severe refractory systemic lupus erythematosus at the American Society of...
Driven Brands Collision Group Celebrates $6M Cystic Fibrosis Fundraising Milestone
PR Newswire
2025-12-30
cf
cystic fibrosis
CHARLOTTE, N.C., Dec. 30, 2025 /PRNewswire/ -- The Driven Brands Collision Group, the parent company of leading collision repair brands including Abra, CARSTAR, and Fix Auto USA announced that it has surpassed $6 million raised for cystic fibrosis (CF) research, care, and advocacy. Cystic...
Needham ups Autolus target, names top pick for 2026
Autolus Therapeutics
2025-12-30
aucatzyl
Needham raised the firm’s price target on Autolus Therapeutics (AUTL) to $11 from $10 and keeps a Buy rating on the shares. The firm also names Autolus a top pick for 2026. While Aucatzyl’s launch has been impressive with $76M in sales, investors remain skeptical of Autolus, questioning if the company can reach breakeven, the analyst tells investors in a research note. Needham disagrees with skepticism, saying the shares offers an “attractive valuation” and a “strong opportunity for value creati
ASH: Late Effects Uncommon for Patients With Sickle Cell Disease After Allogeneic HCT
Hematology Advisor News
2025-12-30
allogeneic
sickle cell disease
Most patients had no sickle cell complications post-HCT; complications included osteonecrosis, vaso-occlusive crisis, nephropathy, stroke.
Trial Scorecard: DECAF
Healio
2025-12-30
atrial fibrillation
The DECAF trial evaluated the effects of caffeinated coffee on risk for recurrent atrial fibrillation episodes in patients with persistent AF or atrial flutter undergoing cardioversion.Read Healio’s in-depth coverage of the DECAF trial.
Enhertu Plus Pertuzumab Approved for First-Line HER2+ Breast Cancer
Hematology Advisor News
2025-12-30
her2
breast cancer
Patients should be selected for treatment with Enhertu in combination with pertuzumab based on confirmed HER2-positive status or HER2 gene amplification (IHC 3+ or ISH+).
Ovarian Cancer Talc Claims Advance Nationwide as Scientific Evidence Mounts
PR Newswire
2025-12-30
ovarian cancer
After multiple failed attempts by Johnson & Johnson to move its talc litigation into bankruptcy, ovarian cancer claims are once again advancing in courts across the country. LOS ANGELES, Dec. 30, 2025 /PRNewswire/ -- Wisner Baum, a nationally recognized plaintiffs' law firm, continues to...
Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-12-30
aav
bla
dtx401
gene therapy
glycogen storage disease type ia
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Verastem axes Phase 1/2 lung cancer trial of Avmapki, pivots to KRAS blocker
Endpoints News
2025-12-30
phase 1/2
lung cancer
Verastem Oncology is ending an early-stage trial of its MEK inhibitor Avmapki in lung cancer and pivoting to another program that it says could have a greater impact on patients. The biotech
8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
SEC Filing (8-K)
2025-12-29
ultragenyx
Filed: 2025-12-29 AccNo: 0001193125-25-332422 Size: 157 KB Item 8.01: Other Events
Genmab terminates Phase 3 lung cancer asset that BioNTech ditched
Endpoints News
2025-12-29
lung cancer
Genmab is ending clinical development of acasunlimab, a bispecific antibody that the biotech had worked on with BioNTech until that partner walked away from the asset last year. The Danish drug developer ...
Biosimilars unlock use of bevacizumab in bowel cancer by NHS
PharmaPhorum
2025-12-29
colorectal cancer
More than 20 years after its approval, NICE has backed the use of bevacizumab as a treatment for colorectal cancer for the first time.
Needham raises Autolus Therapeutics stock price target on Aucatzyl launch success By Investing.com
Autolus Therapeutics
2025-12-29
aucatzyl
Needham raises Autolus Therapeutics stock price target on Aucatzyl launch success
FDA Accepts for Review INOVIO's BLA for INO-3107 for the Treatment of Adults with Recurrent Respiratory Papillomatosis (RRP)
PR Newswire
2025-12-29
bla
PLYMOUTH MEETING, Pa., Dec. 29, 2025 /PRNewswire/ -- INOVIO (NASDAQ: INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases, today announced that the U.S. Food and...
Patients with hematologic cancers value blood transfusions most in hospice services
Healio
2025-12-29
leukemia
myelodysplastic
myelodysplastic syndrome
Individuals with hematologic malignancies enroll in hospice care less than patients with other cancers, and lack of access to blood transfusions is likely a key reason why.New survey data indicate patients with lymphoma, leukemia, myeloma and myelodysplastic syndrome value access to blood transfusions more than any other potential hospice service. However, hospice care generally does not include
No Christmas present for Sanofi, as FDA turns down MS drug
PharmaPhorum
2025-12-29
multiple sclerosis
FDA has turned down Sanofi's oral BTK inhibitor tolebrutinib for multiple sclerosis, despite suggesting earlier it would delay a decision until 2026.
Fenfluramine linked to significantly improved seizure outcomes vs. placebo at 14 weeks
Healio
2025-12-29
x-linked
Individuals with a rare form of epilepsy treated with fenfluramine experienced significantly greater seizure reduction as well as clinically meaningful global functioning improvement compared with placebo at 14 weeks, according to a poster.“CDKL5 deficiency disorder (CDD) is an ultra-rare, X-linked developmental and epileptic encephalopathy characterized by very early-onset, highly
Updated Analysis Supports Damoctocog Alfa Pegol for Patients With Hemophilia A
Hematology Advisor News
2025-12-29
hemophilia a
Researchers determined damoctocog alfa pegol is safe and effective in patients with previously treated severe or nonsevere hemophilia A.
Cancer incidence varies among kidney transplant recipients in disadvantaged neighborhoods
Healio
2025-12-29
kidney transplant recipients
Neighborhood-level social determinants of health were associated with varying cancer risks and greater mortality among kidney transplant recipients, according to study results. Cancer is a leading cause of death for kidney transplant recipients, particularly those living in disadvantaged neighborhoods, according to Yue-Harn Ng, MD, MPH, clinical professor of medicine and director of the
Why Ultragenyx Pharmaceutical Shares Are Trading Lower By Around 42%? Here Are Other Stocks Moving In Monday's Mid-Day Session - Autolus Therapeutics (NASDAQ:AUTL), Brand Engagement Network (NASDAQ:BNAI)
Autolus Therapeutics
2025-12-29
ultragenyx
autolus therapeutics
FDA Accepts for Review INOVIO's BLA for INO-3107 for the Treatment of Adults with Recurrent Respiratory Papillomatosis (RRP)
Inovio Pharmaceuticals
2025-12-29
bla
29 Dec 2025 CARsgen Submits Dual IND Applications for Allogeneic BCMA CAR-T Product CT0596 Dec 29, 2025
Carsgen Therapeutics
2025-12-29
ind
bcma
car-t
allogeneic
Lunsumio Velo Approved for SC Administration in R/R Follicular Lymphoma
Hematology Advisor News
2025-12-29
follicular lymphoma
accelerated approval
FDA grants accelerated approval to subcutaneous mosunetuzumab-axgb for adults with relapsed or refractory follicular lymphoma after 2 therapy lines.
As gene editing tools multiply, the hunt for the final technology continues
Endpoints News
2025-12-29
gene editing
John Finn still remembers the twinge of jealousy when he heard someone else had invented a long-sought form of gene editing, one that might finally cure thousands of genetic diseases. At Intellia Therapeutics, Finn had ...
Adicet Bio Announces Reverse Stock Split
Adicet Bio
2025-12-26
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. --(BUSINESS WIRE)--Dec. 26, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced that its board of directors has determined to effect
QURE INVESTIGATION REMINDER: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2025-12-26
bla
amt-130
gene therapy
RADNOR, PA - December 26, 2025 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Fatal Complication of Stem Cell Transplants Gets Its First FDA-Approved Therapy
MedCity News
2025-12-26
hematopoietic stem cell
Omeros’s complement system drug Yartemlea is now FDA approved for treating a severe and potentially fatal complication of hematopoietic stem cell transplants. Our recap of other regulatory actions includes drug approvals in cancer, rare disease, and metabolic disorders. The post Fatal Complication of Stem Cell Transplants Gets Its First FDA-Approved Therapy appeared first on MedCity News .
Challenge the ‘status quo’ of serial prescription of proven HFpEF therapies
Healio
2025-12-26
heart failure
Simultaneous or rapid initiation of evidence-based therapies significantly improves quality of life and outcomes in patients with heart failure with moderately reduced or preserved ejection fraction, a speaker reported.The “status quo” serial or selective approach to prescribing medical therapy for HF with EF more than 40% may be needless, introducing risk for disease progression and
Ultra-processed foods associated with precursor condition to early-onset colorectal cancer
Healio
2025-12-26
colorectal cancer
Individuals who consume higher amounts of ultra-processed foods may have a significantly higher likelihood of developing a precursor condition to early-onset colorectal cancer.A study of nearly 30,000 nurses showed those who ate the most ultra-processed foods had a 45% greater chance of being diagnosed with conventional adenomas than those who ingested the least amount.
Nemluvio relieves itch, sleep loss ‘as early as 2 days’ in eczema, prurigo nodularis
Healio
2025-12-26
pivotal
Nemolizumab, which targets the interleukin-31 pathway, rapidly improves itch and sleep in people with atopic dermatitis and prurigo nodularis, according to a post hoc analysis of four pivotal trials. Itch is the most burdensome symptom in atopic dermatitis and prurigo nodularis (PN), according to the study published in the Journal of the European Academy of Dermatology & Venerology.
Autologous Stem Cell Therapy for Alzheimer Disease Advances to Phase 2 After Positive Early Findings
CGTLive
2025-12-25
autologous
cell therapy
gene therapy
Cell and Gene Therapy Live
uniQure (QURE) Is Up 9.4% After FDA Deems AMT-130 Phase I/II Data Inadequate for Filing
uniQure
2025-12-25
amt-130
gene therapy
In December 2025, uniQure reported that final minutes from its pre-Biologics License Application meeting showed the FDA views existing Phase I/II data for AMT-130 as insufficient to support a filing, pushing the company to seek further regulatory discussions in fiscal Q1 2026. This feedback directly affects the timeline and evidence required for AMT-130 in Huntington’s disease, potentially reshaping how investors assess uniQure’s lead gene therapy program within its broader pipeline. We’ll...
Caliway's Fat Reduction Drug Candidate CBL-514 Completes U.S. FDA IND Submission for Phase 2 Clinical Study in Weight Management, Expanding Development Combined with GLP-1RA-Based Weight-Loss Therapies
PR Newswire
2025-12-25
ind
Caliway has completed submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a Phase 2 clinical study of CBL-514 for weight management (CBL-0201WR Phase 2). The study will evaluate CBL-514 in combination with Zepbound® (Eli Lilly) in...
Will FDA’s Rapid-Delivery Lunsumio VELO Approval Reshape Roche Holding's (SWX:ROG) High-Value Biologics Narrative?
Roche Group
2025-12-25
follicular lymphoma
accelerated approval
Earlier this month, Genentech, a member of the Roche Group, received U.S. FDA accelerated approval for Lunsumio VELO, a subcutaneous formulation of mosunetuzumab for adults with relapsed or refractory follicular lymphoma after at least two prior systemic therapies, cutting administration time from hours to about one minute. This approval not only broadens Roche’s oncology offering but also highlights how treatment convenience and clinic efficiency are becoming core differentiators in cancer...
United Breast Cancer Foundation's Free Mattress Giveaway Delivers Rest and Relief
PR Newswire
2025-12-24
breast cancer
160 Therapeutic Mattresses Distributed to Breast Cancer Patients and Survivors in Kentucky LOUISVILLE, Ky., Dec. 23, 2025 /PRNewswire/ -- United Breast Cancer Foundation (UBCF) proudly celebrates the remarkable success of its recent Free Mattress Giveaway in Kentucky last month. This was...
VIDEO: Practice-changing CAR T-cell therapy studies presented at ASH 2025
Healio
2025-12-24
car t
cell therapy
cell therapies
multiple myeloma
In this interview Shahzad Raza, MD, highlights data on CAR T-cell therapies and the use of bispecifics in earlier lines of multiple myeloma as presented at ASH 2025.
Sanofi to Acquire Dynavax, Adding a Marketed Adult Hepatitis B Vaccine and Phase 1/2 Shingles Candidate to the Pipeline
PR Newswire
2025-12-24
phase 1/2
Dynavax stockholders to receive $15.50 in cash, a 39% premium to Dynavax closing share price on December 23, 2025 EMERYVILLE, Calif., Dec. 24, 2025 /PRNewswire/ -- Sanofi announced today that it has entered into an agreement to acquire Dynavax Technologies Corporation (Nasdaq: DVAX), a...
Co-PSMA abstract accepted for oral presentation at EAU Annual Congress 2026
PR Newswire
2025-12-24
psma
SYDNEY, Dec. 24, 2025 /PRNewswire/ -- Clarity Pharmaceuticals (ASX: CU6) ("Clarity" or "Company"), a clinical-stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for patients with cancer, is pleased to announce the...
VIDEO: In vivo CAR T-cell therapy used across hematologic malignancies
Healio
2025-12-24
car t
cell therapy
multiple myeloma
In this interview, Ann S. LaCasce, MD, highlights a late-breaking abstract on in vivo CAR T-cell therapy use in multiple myeloma, as presented at ASH 2025.
Edgewise Therapeutics Announces Completion of the EDG-7500 CIRRUS-HCM Phase 2 Parts B and C and Favorable Interim Safety Results from the Ongoing Part D Study in Hypertrophic Cardiomyopathy
PR Newswire
2025-12-24
cardiomyopathy
– In the CIRRUS-HCM trial, including interim safety results from Part D, EDG-7500 was generally well tolerated; no clinically meaningful reductions in LVEF or LVEF
Reaffirming Buy on enGene: Strengthened LEGEND Phase 2 Data, Robust Cash Runway to 2028, and Path to 2026 BLA for Detalimogene
enGene Google
2025-12-24
bla
Guggenheim analyst Michael Schmidt maintained a Buy rating on enGene Holdings yesterday and set a price target of $30.00. Michael Schmidt has given his Buy rating d...
Leads Biolabs And Dianthus Therapeutics Announce Initiation of Phase 1 Trial Of LBL-047 (DNTH212) In Healthy Volunteers and Patients With Systemic Lupus Erythematosus (SLE)PDF VersionRead More
Dianthus Therapeutics
2025-12-24
lupus
top-line results
LBL-047 (DNTH212) is a bifunctional fusion protein targeting plasmacytoid dendritic cell (pDC) BDCA2 to reduce Type 1 interferon production, while simultaneously inhibiting BAFF/APRIL to suppress B cell function Top-line results in healthy volunteers anticipated in 2H’26 Dianthus to provide update PDF Version Read More
Is FDA’s Fast Subcutaneous Lunsumio VELO Approval Reshaping the Investment Case For Roche (SWX:ROG)?
Roche Group
2025-12-24
follicular lymphoma
accelerated approval
In December 2025, Genentech, part of the Roche Group, received U.S. FDA accelerated approval for Lunsumio VELO, a subcutaneous formulation of mosunetuzumab for adults with relapsed or refractory follicular lymphoma after at least two prior systemic therapies, cutting administration time from hours to about one minute. This approval underscores Roche’s push toward more patient‑centric oncology treatments that ease clinic resource constraints while expanding options in later-line blood cancer...
Cystic fibrosis triple therapy linked to less chronic respiratory therapy use
Healio
2025-12-24
cf
cystic fibrosis
Among pediatric and adult patients with cystic fibrosis, the average number of chronic respiratory therapies used fell after starting triple therapy, according to results published in Journal of Cystic Fibrosis.“A shared goal among clinicians and people with cystic fibrosis (CF) is to maintain long-term health while minimizing treatment burden,” Scott D. Sagel, MD, PhD, professor of
VIDEO: Real-world multiple myeloma study reflects population, success of bispecifics
Healio
2025-12-24
multiple myeloma
In this interview Ira Zackon, MD, highlights data on bispecific antibody use in patients with multiple myeloma in the community setting as presented at ASH 2025.
‘Potential disconnect’: Cardiovascular risk calculators not working within prostate cancer
Healio
2025-12-24
prostate cancer
Assessments used to calculate cardiovascular disease risk in the general population may not have the same predictive power among men with prostate cancer treated with hormone therapy.Using common risk calculators, such as the American Heart Association/American College of Cardiology atherosclerotic cardiovascular disease (ASCVD) score, researchers found a “disconnect” among patients
Mabwell Receives IND Clearance for Novel Anti-ST2 Monoclonal Antibody 9MW1911 to Initiate Phase IIa Study
PR Newswire
2025-12-24
ind
SHANGHAI, Dec. 24, 2025 /PRNewswire/ -- Mabwell (688062.SH), an innovation-driven biopharmaceutical company with a fully integrated industry chain, announced that it has received IND clearance from the U.S. Food and Drug Administration (FDA) for its self-developed anti-ST2 monoclonal...
Genentech's subcutaneous Lunsumio gains FDA approval
Genentech
2025-12-23
accelerated approval
The US FDA has approved a subcutaneous version of Roche (RHHBY)-owned Genentech's follicular lymphoma treatment, Lunsumio (mosunetuzumab). Lunsumio VELO takes about one minute to administer, compared to roughly two to four hours with the infusion version. Lunsumio VELO received accelerated approval based on results of the phase I/II GO29781 study t...
Thyroid eye disease treatment receives priority review, PDUFA date
Healio
2025-12-23
pdufa
priority review
The FDA granted priority review to veligrotug for the treatment of thyroid eye disease, according to a press release from Viridian Therapeutics.
Citius Pharmaceuticals, Inc. Reports Fiscal Year 2025 Financial Results and Provides Business Update
PR Newswire
2025-12-23
financial results
Subsidiary, Citius Oncology, launches cancer immunotherapy, LYMPHIR™, in the U.S. in December 2025 CRANFORD, N.J., Dec. 23, 2025 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius Pharma" or the "Company") (Nasdaq: CTXR), a biopharmaceutical company dedicated to the development and...
Galaxy Therapeutics Completes Enrollment in Pivotal SEAL IT IDE Trial
PR Newswire
2025-12-23
pivotal
MILPITAS, Calif., Dec. 23, 2025 /PRNewswire/ -- Galaxy Therapeutics, a privately held, clinical-stage medical device company focused on treating brain aneurysms, today announced it has successfully completed enrollment in SEAL IT, its U.S. IDE trial evaluating the SEAL device for brain...
GenSight Biologics Announces Regulatory Authorization for Early Access Treatment with GS010/LUMEVOQ® in Israel
GenSight Biologics
2025-12-23
lumevoq
SN BioScience received US FDA Orphan Drug Designation for its Nano Anti-Cancer Drug 'SNB-101' on Gastric Cancer.
PR Newswire
2025-12-23
orphan drug
gastric cancer
SEONGNAM, South Korea, Dec. 23, 2025 /PRNewswire/ -- SN Bioscience Inc. (CEO Young Hwan PARK) announced that the FDA had granted Orphan Drug Designation (ODD) on December 10 for gastric cancer (including gastroesophageal junction cancer) to SNB-101 (API: SN-38) which is a polymer...
A Slice of Humble Pie: How One Farmer's Harvest-Time Tradition Is Lifting Up Her Community
PR Newswire
2025-12-23
pivotal
Pivot Bio's "Pivotal People Inspired by Real Farm Heroes" Series Spotlights Marilyn Backman and her world-famous pies MINNEAPOLIS, Dec. 23, 2025 /PRNewswire/ -- Marilyn Backman isn't your ordinary farmer. Part entrepreneur and farmer with her family's seed business, part esteemed advocate...
Pfizer Reports Patient Death in Long-Term Trial of Hympavzi for Hemophilia A
BioSpace
2025-12-23
hemophilia a
The patient, who died on December 14, was originally enrolled in a Phase III study in 2022 and transitioned into an extension phase in 2023.
Citius Oncology, Inc. Reports Fiscal Year 2025 Financial Results and Provides Business Update
PR Newswire
2025-12-23
financial results
Cancer Immunotherapy, LYMPHIR™, launched in the U.S. in December 2025 Completed $36 million in strategic financings, of which $18 million was via private placement and concurrent registered direct offering on December 10, 2025, to strengthen cash position and support continued...
Vyriad targets 2026 clinical trial for new experimental cell therapy VV169
Rare Cancer News
2025-12-23
cell therapy
VV169, an experimental cellular therapy being developed by Vyriad to treat myeloma, is expected to enter clinical testing in 2026, the company announced. Earlier this month at the 67th annual meeting of the American Society of Hematology (ASH), Vyriad presented preclinical data showing that VV169 completely cleared myeloma tumors in 100% of treated animals in laboratory studies. […] The post Vyriad targets 2026 clinical trial for new experimental cell therapy VV169 appeared first on Rare Cancer News .
Analysts grow more bullish on BioMarin Pharmaceuticals Inc. (BMRN) after Amicus acquisition
BioMarin Pharmaceutical
2025-12-23
gene editing
We recently compiled a list of the 7 Most Promising Gene Editing Stocks According to Analysts. BioMarin Pharmaceuticals Inc. stands second among the most promising stocks. TheFly reported on December 19 that BMRN announced it will acquire Amicus Therapeutics in an all‑cash transaction valued at approximately $4.8 billion, paying $14.50 per share. The deal strengthens BMRN’s […]
Neurocrine’s Ingrezza flunks pivotal study in dyskinetic cerebral palsy
Endpoints News
2025-12-23
pivotal
Neurocrine Biosciences’ Ingrezza has failed a late-stage trial in a type of cerebral palsy, adding to a spate of clinical disappointments for the biotech in recent years. Neurocrine did not immediately respond to an ...
Atrial Fibrillation Linked With Increased Thrombosis Risk in Hospitalized Patients With MF
Hematology Advisor News
2025-12-23
atrial fibrillation
Atrial fibrillation was not significantly linked with major bleeding events; however, older age, alcohol use, greater comorbidity burden, and anticoagulant exposure all increased this risk.
Lunit SCOPE Demonstrates High Concordance with Pathologists in HER2 Interpretation for Advanced Biliary Tract Cancer
PR Newswire
2025-12-23
her2
Study published in Laboratory Investigation, the official journal of the United States and Canadian Academy of Pathology SEOUL, South Korea, Dec. 23, 2025 /PRNewswire/ -- Lunit (KRX: 328130) , a leading provider of AI for cancer diagnostics and precision oncology, today announced that a...
8-K - Krystal Biotech, Inc. (0001711279) (Filer)
SEC Filing (8-K)
2025-12-23
krystal biotech
Filed: 2025-12-23 AccNo: 0001711279-25-000092 Size: 138 KB Item 5.02: Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers: Compensatory Arrangements of Certain Officers Item 9.01: Financial Statements and Exhibits
Zydus Partners with Bioeq for U.S. Commercialisation rights for NUFYMCO®, an Interchangeable Biosimilar to Lucentis®
PR Newswire
2025-12-23
bla
NUFYMCO® BLA has been approved by the USFDA AHMEDABAD, India and ZUG, Switzerland, Dec. 23, 2025 /PRNewswire/ -- Bioeq AG ("Bioeq"), a Swiss biopharmaceutical company, and Zydus Lifesciences Limited ("Zydus"), an innovation-led life-sciences company with an international presence, today...
Analysts Grow More Bullish on BioMarin Pharmaceuticals Inc. (BMRN) After Amicus Acquisition
BioMarin Pharmaceutical
2025-12-23
gene editing
We recently compiled a list of the 7 Most Promising Gene Editing Stocks According to Analysts. BioMarin Pharmaceuticals Inc. stands second among the most promising stocks. TheFly reported on December 19 that BMRN announced it will acquire Amicus Therapeutics in an all‑cash transaction valued at approximately $4.8 billion, paying $14.50 per share. The deal strengthens BMRN’s […]
Labcorp backs Cellens in developing physics test aimed at bladder cancer
Endpoints News
2025-12-23
bladder cancer
Boston-based company Cellens secured $6.5 million to develop its platform that detects bladder cancer through an approach that combines physics and AI, Endpoints News learned exclusively. Its technology is a departure from the norm in ...
enGene Reports Full Year 2025 Financial Results and Provides Business Update
enGene Google
2025-12-23
financial results
Genentech secures FDA approval for Lunsumio VELO SC for follicular lymphoma
Genentech
2025-12-23
follicular lymphoma
accelerated approval
Genentech, a subsidiary of the Roche Group, has received the US FDA accelerated approval for Lunsumio VELO SC formulation to treat adult patients with R/R FL after two or more lines of systemic therapy.
Genentech's subcutaneous Lunsumio gains FDA approval
Genentech
2025-12-23
follicular lymphoma
The FDA approved Genentech's Lunsumio VELO for follicular lymphoma, offering administration in one minute. Read more here.
Tigerlily Foundation Launches RACE Alliance - Putting Community First
PR Newswire
2025-12-23
breast cancer
WASHINGTON, Dec. 23, 2025 /PRNewswire/ -- Tigerlily Foundation is proud to announce the launch of the RACE ALLIANCE. The Rallying Around Community and Equity Alliance, an exciting and innovative initiative designed to expand expertise and awareness about breast cancer education, care and...
Payers play an evolving role in small cell lung cancer treatment
Prime Therapeutics
2025-12-23
lung cancer
Abby Kim, PharmD, BCOP, is a senior director, clinical strategy and oncology specialty solutions, shares key takeaways from the American Journal of Managed Care (AJMC) forum, “Bridging evidence and access in advanced small cell lung cancer.”
Precision BioSciences Announces Grant of Inducement Awards Under Nasdaq Listing Rule 5635(c)(4)
precision biosciences
2025-12-23
gene editing
DURHAM, N.C. --(BUSINESS WIRE)--Dec. 23, 2025-- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced that, on December 9, 2025 ,
FDA Approves Genentech’s Lunsumio VELO™ for Subcutaneous Use in Relapsed or Refractory Follicular Ly
Genentech
2025-12-22
complete response
– Lunsumio VELO reduces administration time from 2-4 hours to approximately one minute –– Availability of Lunsumio VELO allows treatment aligned to people’s clinical needs and personal preferences –– Approval supported by data demonstrating compelling complete response rate in third-line or later...
AHA Invests in AI that Tackles Cardiology’s Gender Bias Problem
MedCity News
2025-12-22
heart failure
The American Heart Association is backing an AI startup aimed at improving detection of hard-to-diagnose conditions like cardiac amyloidosis and heart failure with preserved ejection fraction (HFpEF). The company, named Ultromics, offers a platform that analyzes routine echocardiograms to help clinicians catch disease earlier, particularly for women who are more likely to be missed. The post AHA Invests in AI that Tackles Cardiology’s Gender Bias Problem appeared first on MedCity News .
Update on LATIFY Phase III trial of ceralasertib plus Imfinzi in previously treated advanced non-small cell lung cancer 22 December 2025
AstraZeneca
2025-12-22
lung cancer
Sangamo Therapeutics Reports Recent Business Highlights and Second Quarter 2025 Financial Results
Sangamo BioSciences
2025-12-22
financial results
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Bioretec Ltd's schedule for financial reporting and Annual General Meeting in 2026
PR Newswire
2025-12-22
financial statements
Bioretec Ltd Company announcement 22 December 2025 at 5:30 p.m. EET TAMPERE, Finland, Dec. 22, 2025 /PRNewswire/ -- In 2025, Bioretec will publish the following financial reports: financial statements bulletin for January-December 2025 on Friday 13 February 2025 annual report, Board of...
Olverembatinib and chemo show deep response in trial for Ph-positive ALL
Rare Cancer News
2025-12-22
leukemia
acute lymphoblastic leukemia
Olverembatinib (HQP1351), used in combination with low-intensity chemotherapy, has shown encouraging therapeutic potential — along with a favorable safety profile — in individuals with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph-positive ALL), a type of fast-growing blood cancer. These new findings come from an initial dataset of the Phase 3 POLARIS-1 trial (NCT06051409), underway […] The post Olverembatinib and chemo show deep response in trial for Ph-positive ALL appeared first on Rare Cancer News .
Abbott's Volt™ Pulsed Field Ablation System Receives FDA Approval to Treat Patients with Atrial Fibrillation
PR Newswire
2025-12-22
atrial fibrillation
Abbott's Volt™ PFA System, the latest generation of cardiac ablation technology, is designed for people battling heart rhythm disorders such as atrial fibrillation (AFib) Pulsed field ablation – or PFA – is a minimally invasive procedure that uses high-energy electrical pulses in targeted...
Enhertu granted Breakthrough Therapy Designation in the US as post-neoadjuvant therapy for patients with HER2-positive early breast cancer 22 December 2025
AstraZeneca
2025-12-22
her2
breast cancer
breakthrough therapy
BLOG: Adoptive cell therapies offer ‘new hope’ for patients with limited treatment options
Healio
2025-12-22
solid tumor
cell therapies
The cellular therapy landscape continues to evolve beyond hematologic malignancies, now making significant progress in solid tumor treatment.Certain characteristics seen in solid tumors — such as antigenic heterogeneity and the immunosuppressive tumor microenvironment — have dampened the efficacy of antigen-directed therapies for these patients, leading to the emergence of other
FDA Approval Puts New Cytokinetics Heart Drug in Competition With Fast-Growing BMS Med
MedCity News
2025-12-22
cardiomyopathy
Cytokinetics drug Myqorzo won FDA approval for treating obstructive hypertrophic cardiomyopathy, or oHCM. Compared to Bristol Myers Squibb’s projected blockbuster drug Camzyos, the Cytokinetics daily pill has less restrictive safety and monitoring requirements, giving it a competitive advantage, according to analysts. The post FDA Approval Puts New Cytokinetics Heart Drug in Competition With Fast-Growing BMS Med appeared first on MedCity News .
US charges six people with insider trading in case including Olema Oncology
Endpoints News
2025-12-22
breast cancer
The US Justice Department charged several people with insider trading and market manipulation in a case that centers on California biotech Olema Oncology and its lead candidate for breast cancer. Brothers Muhammad Saad Shoukat and ...
Bladder cancer trial hits 62% response as enGene extends cash runway
enGene Google
2025-12-22
bladder cancer
complete response
enGene ends 2025 with $342.4M in cash and funding into 2H 2028, and reports a 62% 6-month complete response rate in its LEGEND bladder cancer study.
enGene (NASDAQ:ENGN) Announces Earnings Results
enGene Google
2025-12-22
quarterly
enGene (NASDAQ:ENGN - Get Free Report) issued its quarterly earnings results on Monday. The company reported ($0.73) earnings per share (EPS) for the quarter, missing analysts' consensus estimates of ($0.55) by ($0.18).
PTC Therapeutics Announces Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU) in Japan
ptc biotherapeutics
2025-12-22
phenylketonuria
– Indication includes all ages and the full spectrum of disease severity – – First Japan product approval for PTC – WARREN, N.J. , Dec. 22, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that Sephience™ (sepiapterin) has been approved by the Japanese Ministry of Health,
China's new commercial insurance model could broaden access to Alzheimer's drugs
Endpoints News
2025-12-22
alzheimer's disease
China's new commercial insurance formulary looks to expand patient access to Alzheimer's disease medicines from Eli Lilly and Eisai while potentially boosting revenue for the companies. Announced in December, the Commercial Health Insurance Innovative Drug ...
CRISPR Therapeutics Provides Broad Update on Zugocaptagene Geleucel (Zugo-cel; formerly CTX112™) in Autoimmune Diseases and Hematologic Malignancies
CRISPR
2025-12-22
lupus
crispr
Four autoimmune patients treated to date demonstrate deep B-cell depletion sustained for at least 28 days; initial efficacy data suggest significant clinical improvement in patients dosed at the 100 million cell dose, with the first systemic lupus erythematosus (SLE) patient achieving Definitions
BioSpace Editors’ Top 5 Biggest News Stories of the Year
BioSpace
2025-12-22
sarepta
Pfizer and Metsera, Sarepta and uniQure made the list with dramatic tales. The other two spots went to the regulatory challenges facing biopharma under the new administration, especially in the vaccines sector.
enGene completes enrollment in bladder cancer treatment trial By Investing.com
enGene Google
2025-12-22
bladder cancer
enGene completes enrollment in bladder cancer treatment trial
AZ's ATR inhibitor ceralasertib flunks lung cancer test
PharmaPhorum
2025-12-22
lung cancer
AstraZeneca has had a setback in its oncology pipeline, as AKT inhibitor ceralasertib fails to improve survival in a phase 3 lung cancer trial.
Prognosis Worsens Once Patients Exhaust BTKi and BCL2i in CLL
Hematology Advisor News
2025-12-22
cll
Longitudinal data reveal consistent initial responsiveness to targeted agents but limited durability once patients cycle through both BTKi and BCL2i classes.
enGene completes enrollment in bladder cancer treatment trial By Investing.com
enGene Google
2025-12-22
bladder cancer
enGene completes enrollment in bladder cancer treatment trial
8-K - BIOMARIN PHARMACEUTICAL INC (0001048477) (Filer)
SEC Filing (8-K)
2025-12-22
biomarin pharmaceutical
Filed: 2025-12-22 AccNo: 0001193125-25-327828 Size: 131 KB Item 8.01: Other Events
A lymphoma treatment that used to take hours can now be given in 1 minute
Genentech
2025-12-22
complete response
follicular lymphoma
FDA clears subcutaneous Lunsumio VELO for relapsed follicular lymphoma after 2+ therapies, with 75% response and 59% complete response rates.
AstraZeneca’s ATR Inhibitor Fails to Improve Survival in Phase III Lung Cancer Trial
BioSpace
2025-12-22
lung cancer
Ceralasertib is part of AstraZeneca’s ambitious plan to hit $80 billion in revenue by 2030.
HHS adds neuromuscular conditions to newborn screening protocol
Healio
2025-12-22
duchenne muscular dystrophy
metachromatic leukodystrophy
HHS has announced the addition of Duchenne muscular dystrophy and metachromatic leukodystrophy to the Recommended Uniform Screening Panel following scientific review and public comment. The decision was made because early detection of both rare, pediatric neuromuscular conditions will allow this patient population to receive FDA-approved therapies at a crucial time to slow disease progression and
GenSight Biologics Announces the Granting of Compassionate Use Authorization (CUA/AAC) for GS010/LUMEVOQ® in France
GenSight Biologics
2025-12-22
lumevoq
New one-minute lymphoma shot slashes time patients spend in clinic
Roche Group
2025-12-22
follicular lymphoma
FDA approves Roche’s Lunsumio VELO as a 1-minute SC option for adults with R/R follicular lymphoma after 2+ therapies, with 75% response and 59% CR.
Sangamo Therapeutics Reports Recent Business Highlights and First Quarter 2025 Financial Results
Sangamo BioSciences
2025-12-22
financial results
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
enGene completes enrollment in bladder cancer treatment trial By Investing.com
enGene Google
2025-12-22
bladder cancer
enGene completes enrollment in bladder cancer treatment trial
Platelet-rich plasma, mesenchymal stem cells yield similar functional outcomes in knee OA
Healio
2025-12-22
mesenchymal stem cells
Published results showed umbilical cord-derived platelet-rich plasma provided better short-term pain relief but similar functional outcomes compared with adipose tissue-derived mesenchymal stem cells in early knee osteoarthritis.“We were expecting the adipose tissue to yield superior results, and we were hoping to show that PRP was not as good but still a valid alternative,” Simone
Trump to test ‘most favored nation’ pricing for Medicare; Shionogi pays $2.5B for ALS drug
Biopharma Dive
2025-12-22
cell therapy
Two pilot programs would tie Medicare costs and rebates in the U.S. to what’s paid in comparable countries. Elsewhere, AstraZeneca reported a setback and an “off the shelf” cell therapy showed promise.
7 Most Promising Gene Editing Stocks According to Analysts
BioMarin Pharmaceutical
2025-12-22
gene editing
Gene editing has rapidly evolved into a transformative class of technologies that allow scientists to precisely insert, delete, or modify DNA within living cells. - Part 5
VIDEO: Durable response of epcoritamab ‘very encouraging’
Healio
2025-12-22
b-cell lymphoma
large b-cell lymphoma
ORLANDO —In this video, Henry Chi Hang Fung, MD, FACP, FRCPE, discusses long-term follow-up results from the phase 2 EPCORE- NHL-1 trial, presented at the ASH Annual Meeting and Exposition. The research assessed the impact of epcoritamab (Epkinly; Genmab, AbbVie) monotherapy after 4 years of treatment in patients with relapsed or refractory large B-cell lymphoma.
Colorectal Cancer Detection in Nigeria Gets Boost with Community Campaign
Inside Precision Medicine
2025-12-22
colorectal cancer
A six-month community outreach program in Osun State in Nigeria helped boost knowledge of colorectal cancer among residents and improve early diagnosis of the disease in the region. The post Colorectal Cancer Detection in Nigeria Gets Boost with Community Campaign appeared first on Inside Precision Medicine .
Lunsumio Velo Approved for SC Administration in R/R Follicular Lymphoma
Hematology Advisor News
2025-12-22
follicular lymphoma
Lunsumio Velo is supplied as a ready-to-use, preservative-free solution in single-dose vials containing 5mg/0.5mL and 45mg/mL.
FDA Approves Genentech’s Lunsumio VELO™ for Subcutaneous Use in Relapsed or Refractory Follicular Lymphoma
Genentech
2025-12-22
follicular lymphoma
SOUTH SAN FRANCISCO, Calif., December 22, 2025--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved CD20xCD3 bispecific Lunsumio VELO™ (mosunetuzumab-axgb), as a subcutaneous (SC) formulation, for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy, based on results from the Phase I/II GO29781 study. Based on the study r
Around the Helix: Cell and Gene Therapy Company Updates – December 3, 2025
CGTLive
2025-12-21
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
T-MAXIMUM Pharmaceutical's Allogeneic CAR-T Therapy MT027 Receives FDA IND Clearance to Proceed to Phase II clinical Trial for Recurrent Glioblastoma
PR Newswire
2025-12-21
ind
b7-h3
car-t
allogeneic
glioblastoma
BEIJING, Dec. 21, 2025 /PRNewswire/ -- T-MAXIMUM Pharmaceutical announced that its proprietary allogeneic, B7-H3-targeted CAR-T therapy, MT027, has received IND Clearance from the U.S. Food and Drug Administration (FDA) to initiate a Phase II clinical trial for the treatment of recurrent...
Sangamo Therapeutics Reports Recent Business Highlights and Third Quarter 2025 Financial Results
Sangamo BioSciences
2025-12-21
financial results
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Is KOMZIFTI’s Early AML Data and CEO Review Altering The Investment Case For Kyowa Kirin (TSE:4151)?
Kyowa Kirin
2025-12-21
aml
leukemia
acute myeloid leukemia
Earlier in December 2025, Kura Oncology and Kyowa Kirin reported Phase 1a/1b data showing a favorable safety profile and encouraging antileukemic activity for KOMZIFTI (ziftomenib) combined with venetoclax and azacitidine in difficult-to-treat acute myeloid leukemia, alongside Kyowa Kirin’s board meeting to consider a change in CEO. The ASH presentations positioned KOMZIFTI as a potential backbone menin inhibitor therapy in both newly diagnosed and relapsed or refractory AML, underlining...
Guess the Therapy Answer December 17, 2025
CGTLive
2025-12-21
gene therapy
Cell and Gene Therapy Live
VIDEO: Questions remain regarding use of giredestrant, oral SERDs for breast cancer
Healio
2025-12-21
breast cancer
SAN ANTONIO — In this video, Eleonora Teplinsky, MD, discusses the lidERA trial, presented at San Antonio Breast Cancer Symposium, and what questions and research remain for use of giredestrant in breast cancer.The phase 3 lidERA trial compared giredestrant (Genentech, Roche), an oral selective estrogen receptor degrader, with standard-of-care chemotherapy among patients with early-stage,
Zemcelpro® (UM171 Cell Therapy) improved outcomes compared with common graft sources: European real-world evidence
Excellthera
2025-12-20
cell therapy
MONTREAL, December 19, 2025 – ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and metabolic fitness, and its wholly owned subsidiary Cordex Biologics, announced the publication in the journal Transplantation and Cell Therapy of results from a retrospective effectiveness analysis comparing outcomes of adult patients with blood cancers who received Zemcelpro® (UM171 Cell […]
Kyowa Kirin (TSE:4151) Valuation Check After New KOMZIFTI Menin Inhibitor Data in AML Trial
Kyowa Kirin
2025-12-20
aml
leukemia
acute myeloid leukemia
Kyowa Kirin (TSE:4151) just gave investors a fresh data point to chew on, with new KOMZIFTI combination trial results in acute myeloid leukemia highlighting encouraging activity alongside a clean looking safety profile. See our latest analysis for Kyowa Kirin. Those KOMZIFTI data land as Kyowa Kirin’s 90 day share price return of 7.9% and roughly 9% year to date share price gain suggest sentiment is quietly improving, even though the three year total shareholder return is still in negative...
VIDEO: Adding tucatinib to maintenance therapy a ‘great option’ in metastatic breast cancer
Healio
2025-12-20
her2
breast cancer
SAN ANTONIO — In this video, Eleonora Teplinsky, MD, discusses results of the HER2CLIMB-05 study, showing that adding tucatinib to first-line maintenance therapy improved patient outcomes in HER2-positive metastatic breast cancer.Results of the trial, which compared tucatinib (Tukysa, Seagen) with placebo in combination with trastuzumab (Herceptin, Genentech) and pertuzumab (Perjeta,
Double Vyjuvek dose heals extensive wounds in woman with RDEB
Epidermolysis Bullosa News
2025-12-19
vyjuvek
Six months of treatment with a high dose of Vyjuvek helped resolve new wounds covering about half the body of a woman with recessive DEB.
Iovance Biotherapeutics Reports Inducement Grants under NASDAQ Listing Rule 5635(c)(4)
Iovance Biotherapeutics
2025-12-19
til
SAN CARLOS, Calif. , Dec. 19, 2025 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA) ("Iovance" or the “Company”), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (“TIL”) therapies for patients with cancer,
BioMarin to Acquire Amicus Therapeutics for $4.8 Billion, Expanding Position as a Leader in Rare Diseases, Accelerating Revenue Growth and Strengthening Financial Outlook
BioMarin Pharmaceutical
2025-12-19
fabry disease
pompe disease
BioMarin to Gain Galafold ® for Fabry Disease and Pombiliti ®+ Opfolda ® for Pompe Disease, Adding Two Marketed, High-Growth Products with $599 Million in Revenue Over Past Four Quarters. Provides Opportunity to Expand Access to Galafold and Pombiliti+ Opfolda to Patients in New Markets Across BioMarin's Global Footprint; Pending U.S. Galafold Patent...
Tokyo Lifestyle Co., Ltd. Reports First Six Months of Fiscal Year 2026 Financial Results
PR Newswire
2025-12-19
financial results
Revenue Increased by 94.3% YoY; Gross Profit Increased by 29.8% YoY TOKYO, Dec. 19, 2025 /PRNewswire/ -- Tokyo Lifestyle Co., Ltd. ("Tokyo Lifestyle" or the "Company") (Nasdaq: TKLF), a retailer and wholesaler of Japanese beauty and health products, sundry products, luxury products,...
Pancreatic cancer treatment atebimetinib set for Phase 3 trial
Rare Cancer News
2025-12-19
pancreatic cancer
Immuneering is preparing to launch a Phase 3 clinical trial to test its experimental therapy, atebimetinib, in people with pancreatic ductal adenocarcinoma (PDAC), the most common form of pancreatic cancer. The company recently received positive feedback from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), with both agencies agreeing on key aspects […] The post Pancreatic cancer treatment atebimetinib set for Phase 3 trial appeared first on Rare Cancer News .
Weekly Rundown: Genentech partners with Caris Life Sciences in $1.1B deal
Genentech
2025-12-19
car t
solid tumor
cell therapy
stiff person syndrome
Investments in solid tumor targets and autoimmune disease, success for a peanut allergy patch, a promising CAR T cell therapy for stiff person syndrome, and more led the news this week.
Study shifts understanding of lupus, Epstein-Barr link ‘from correlation to mechanism’
Healio
2025-12-19
lupus
Activation of certain memory B cells may be driving systemic lupus erythematosus incidence in individuals who had Epstein-Barr virus, according to recent data published in Science Translational Medicine.“The study provides a clearer mechanistic explanation for why SLE can be so persistent,” Shady Younis, PhD, an instructor at the division of immunology and rheumatology at Stanford
CG Oncology (CGON) Is Down 8.7% After Strong Cretostimogene NMIBC Data
CG Oncology
2025-12-19
bladder cancer
complete response
CG Oncology recently reported topline data from its BOND-003 and CORE-004 trials of investigational therapy cretostimogene in non-muscle invasive bladder cancer, showing encouraging efficacy measures, a consistent safety profile, and largely localized, low-grade bladder-related adverse events in patients across multiple high-risk settings. The data, which will be featured as late-breaking abstracts at the Society of Urologic Oncology meeting, highlight high complete response rates in...
VIDEO: ‘Proof-of-concept’ shows deescalation in chemo due to targeted therapy in DLBCL
Healio
2025-12-19
dlbcl
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
ORLANDO —Henry Chi Hang Fung, MD, FACP, FRCPE, said the Smart Stop trial showed many patients with newly diagnosed diffuse large B-cell lymphoma who receive a targeted combination therapy may “avoid or substantially reduce CHOP exposure.”Presented at the ASH Annual Meeting and Exposition, the research examined the impact of lenalidomide (Revlimid, Bristol Myers Squibb),
BTK Inhibitors Drive Durable CNS Responses in CLL/SLL
Hematology Advisor News
2025-12-19
cll
Secondary CNS involvement in CLL/SLL was rare at 1.1% to 1.7% across 15 years.
GLP-1s show benefit in heart failure, but monitor, manage heart rate
Healio
2025-12-19
heart failure
GLP-1s improved outcomes in patients with heart failure with preserved ejection fraction, with or without obesity, but those with HF with reduced EF may require special considerations, a speaker reported.Due to the heart rate-raising adverse events of GLP-1s, physicians should consider monitoring changes among patients with HFrEF and consider interventions to help lower their heart rates to the
Direct image-guided convective perfusion of the bilateral thalami for gene therapy in frontotemporal dementia: technical note
aviadobio
2025-12-19
gene therapy
frontotemporal dementia
The post Direct image-guided convective perfusion of the bilateral thalami for gene therapy in frontotemporal dementia: technical note appeared first on AviadoBio .
Matrix-assisted autologous chondrocyte implantation may yield high return to sports rate
Healio
2025-12-19
autologous
Results showed chondral defects of the knee treated with matrix-assisted autologous chondrocyte implantation may yield a high rate of patient satisfaction and return to sports in a community practice.“[Matrix-assisted autologous chondrocyte implantation (MACI)] surgery is exceptional technology to treat chondral defects in the knee,” Alexander K. Meininger, MD, FAAOS, of Steamboat
Engineered Dendritic Cells Harness Tumor EVs to Boost Cancer Immunotherapy
GenEng News
2025-12-19
melanoma
Engineered dendritic cells with EV‑internalizing and chimeric antigen receptors capture tumor vesicles, activate T cells, and delay melanoma growth in preclinical models, advancing next‑generation cancer immunotherapy strategies. The post Engineered Dendritic Cells Harness Tumor EVs to Boost Cancer Immunotherapy appeared first on GEN - Genetic Engineering and Biotechnology News .
Patient deaths put Merck, Daiichi’s ADC trial on partial hold
Biopharma Dive
2025-12-19
lung cancer
The FDA placed the hold after researchers recorded a “higher than anticipated” incidence of deaths in a study testing “I-DXd” in people with small cell lung cancer.
IN8bio Announces Pricing of Private Placement of up to $40.2 Million to Advance Novel Gamma-Delta T Cell Engager
In8Bio
2025-12-19
ind
gamma-delta t cell
$20.1 million upfront with a milestone-driven additional $20.1 million to advance INB-619, IN8bio’s novel gamma-delta (“γδ”) T cell engager through an Investigational New Drug (“IND”) application Financing led by Coastlands Capital with participation from new and existing biotechnology investors
VIDEO: ‘Stellar’ response rates seen with bispecific antibody combinations in lymphoma
Healio
2025-12-19
follicular lymphoma
mantle cell lymphoma
ORLANDO — In this video, Stephen M. Ansell, MD, PhD, discusses data from ASH Annual Meeting and Exposition on the use of bispecific antibody combinations in follicular lymphoma and mantle cell lymphoma.
HAE research continues to swell post-CSL Andembry win
CSL Behring
2025-12-19
clearance
hereditary angioedema
The U.S. FDA clearance June 16 of CSL Behring LLC’s humanized anti-factor XIIa monoclonal antibody garadacimab (CSL-312) to prevent hereditary angioedema (HAE) attacks juiced up the space in which – although other therapies are available as well – developers continue working on new prospects.
8-K - BIOMARIN PHARMACEUTICAL INC (0001048477) (Filer)
SEC Filing (8-K)
2025-12-19
biomarin pharmaceutical
Filed: 2025-12-19 AccNo: 0001193125-25-325856 Size: 1 MB Item 1.01: Entry into a Material Definitive Agreement Item 7.01: Regulation FD Disclosure Item 9.01: Financial Statements and Exhibits
Galapagos TYK2 drug hits goal in one trial, misses in another
Biopharma Dive
2025-12-19
lupus
The Belgian biotech is still on the hunt for partners after reporting that its drug succeeded against an inflammatory skin and muscle disorder but missed in a lupus study.
Hansa Biopharma Submits BLA to FDA for Imlifidase in desensitization for Kidney Transplantation
PR Newswire
2025-12-19
bla
pivotal
The application is based on the successful outcome of the pivotal US Phase 3 ConfIdeS trial in highly sensitized patients Imlifidase is an enzyme that rapidly and efficiently cleaves all types of immunoglobulin G (IgG) antibodies both intra- and extra-vascularly to inhibit IgG-mediated...
Cytokinetics’ Moment of Truth: Can Aficamten Take On BMS in Hypertrophic Cardiomyopathy?
BioSpace
2025-12-19
pdufa
cardiomyopathy
After 27 years in business, Cytokinetics hopes to pit its own cardiac myosin inhibitor against one it initially developed—now owned by Bristol Myers Squibb—in a market worth billions. Aficamten has a PDUFA date of Dec. 26.
VIDEO: No ‘major improvement’ in PFS with sacituzumab-govitecan in breast cancer subset
Healio
2025-12-19
her2
breast cancer
SAN ANTONIO — In this video, Sarat Chandarlapaty, MD, PhD, discusses results from the ASCENT-07 trial, which evaluated sacituzumab-govitecan after endocrine therapy in patients with hormone receptor-positive, HER2-negative breast cancer.The findings, presented at San Antonio Breast Cancer Symposium, showed earlier treatment with sacituzumab-govitecan (Trodelvy, Gilead Sciences) did not
VIDEO: ‘Very good outcomes’ in follicular lymphoma subset up to 2 decades after diagnosis
Healio
2025-12-19
follicular lymphoma
ORLANDO — In this video, Stephen M. Ansell, MD, PhD, discusses long-term outcomes from a large prospective cohort study of patients with follicular lymphoma, presented at ASH Annual Meeting and Exposition.
BioMarin’s Presence in Rare Enzyme Disorders Grows With $4.8B Amicus Therapeutics Acquisition
BioMarin Pharmaceutical
2025-12-19
fabry disease
pompe disease
BioMarin Pharmaceutical’s acquisition of Amicus Therapeutics is a marriage of two rare disease biotech companies. Amicus’s two commercialized products, for the enzyme deficiencies Fabry disease and Pompe disease, are each projected to become blockbuster sellers.
Tolmar Announces Expanded Indication for Rubraca® (rucaparib) First and Only PARP Inhibitor to Outperform Docetaxel in a Head-to-Head Phase 3 Trial
PR Newswire
2025-12-19
prostate cancer
FDA approval is based on the positive results of TRITON3, a Phase 3, multicenter, open-label, randomized clinical trial of Rubraca® (rucaparib) in chemotherapy-naïve metastatic castration-resistant prostate cancer (mCRPC) patients This approval makes rucaparib an earlier treatment option...
BioMarin Stock Jumps After Nearly $5 Billion Amicus Therapeutics Deal
BioMarin Pharmaceutical
2025-12-19
gene therapy
hemophilia a
It’s the second acquisition for BioMarin in 2025, as the company seeks to diversify its product portfolio after the commercial failure of a hemophilia A gene therapy.
VIDEO: Bispecific antibody ‘potentially the future’ front-line option for DLBCL
Healio
2025-12-19
dlbcl
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
ORLANDO — In this video, Henry Chi Hang Fung, MD, FACP, FRCPE, discusses data from part 1a of the phase 3 OLYMPIA-3 study of odronextamab plus chemotherapy in patients with previously untreated diffuse large B-cell lymphoma.Results presented at the ASH Annual Meeting and Exposition showed odronextamab (Regeneron Pharmaceuticals) plus chemotherapy had “strong, early signals of high
Key Unknowns Remain in Cell and Gene Therapy
CGTLive
2025-12-19
gene therapy
Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, spoke about how our understanding of advanced therapeutics is still evolving.
VIDEO: Abemaciclib promising for advanced breast cancer if CDK4/6 inhibitor fails
Healio
2025-12-19
breast cancer
SAN ANTONIO — In this video, Halle C. F. Moore, MD, discusses the rAMBER study looking at abemaciclib monotherapy as a treatment option for women with metastatic breast cancer, presented at the 2025 San Antonio Breast Cancer Symposium.“We have further evidence supporting with our modern types of more aggressive endocrine therapy, that includes the [cyclin-dependent kinase 4/6
VIDEO: Epcoritamab monotherapy has potential to replace standard R-CHOP in DLBCL
Healio
2025-12-19
dlbcl
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
ORLANDO — In this video, Henry Chi Hang Fung, MD, FACP, FRCPE, discusses results from the phase 2 EPCORE-DLBCL-3 trial, presented at the ASH Annual Meeting and Exposition. In stage 2 of the trial, researchers assessed the efficacy and safety of fixed-duration epcoritamab (Epkinly; Genmab, AbbVie) monotherapy in patients with newly diagnosed diffuse large B-cell lymphoma with comorbidities
UI Health performs first islet cell transplant with Lantidra
CellTrans Inc.
2025-12-19
lantidra
Doctors at UI Health performed the first islet transplant with Lantidra, the only therapy approved by the U.S. Food and Drug Administration to treat brittle type 1 diabetes. A 69-year-old man from Illinois received the therapy on Aug. 26 and subsequently was able to stop taking daily, life-saving insulin injections. Lantidra became available exclusively at UI Health last November.
Kyowa Kirin Could Get Windfall From Updated Newborn Screening Recommendations
Kyowa Kirin
2025-12-19
gene therapy
duchenne muscular dystrophy
metachromatic leukodystrophy
A report from analysts at Jefferies suggested that new screenings for metachromatic leukodystrophy and Duchenne muscular dystrophy could bump sales of the gene therapy Libmeldy by more than $100 million.
FDA approves aficamten for adults with obstructive HCM
Healio
2025-12-19
cardiomyopathy
Cytokinetics announced the FDA approval of aficamten to improve functional capacity and symptoms in adults with symptomatic obstructive hypertrophic cardiomyopathy.The agency approved 5 mg, 10 mg, 15 mg and 20 mg dose tablets of aficamten (Myqorzo), designed to inhibit cardiac myosin motor activity, which results in reducing cardiac contractility and left ventricular outflow tract obstruction,
Catalent handed two Form 483s, including for Sarepta gene therapy site
Endpoints News
2025-12-19
sarepta
gene therapy
Prior to last month’s series of mass layoffs in Maryland, Catalent was issued two Form 483s for its gene therapy facilities in the state, which make products for Sarepta. The FDA handed both forms to ...
Novo files for CagriSema approval; Merck and Pfizer’s trial win
Biopharma Dive
2025-12-19
bladder cancer
Novo Nordisk's application for CagriSema comes at the same time as competitor Eli Lilly's ask for its obesity drug orforglipron. Elsewhere, Merck & Co. and Pfizer succeeded in a bladder cancer study.
FDA grants novel agent accelerated approval for reducing proteinuria in IgA nephropathy
Healio
2025-12-19
accelerated approval
The FDA granted accelerated approval for sibeprenlimab-szsi to reduce proteinuria for adults with primary immunoglobulin A nephropathy at risk for disease progression, according to an industry press release. Sibeprenlimab-szsi (Voyxact, Otsuka Pharmaceutical) is the first A-proliferation-inducing-ligand (APRIL) blocker to be approved for treating immunoglobulin A nephropathy (IgAN), according to
Lilly announces new study data on Inluriyo in metastatic breast cancer
PMLive Research
2025-12-19
breast cancer
The five-year survival rate for metastatic breast cancer is 30%
Editors’ Choice: Top Stories of 2025
GenEng News
2025-12-19
sarepta
In this episode of GEN 's Touching Base , editors reflect on the stories from 2025 that they each felt were impactful. Stories include AI in drug discovery, CGT successes, the Sarepta saga, biotech in Africa, and NIH cuts. The post Editors’ Choice: Top Stories of 2025 appeared first on GEN - Genetic Engineering and Biotechnology News .
Lessons Learned From the Past 25 Years of Cell and Gene Therapy Research
CGTLive
2025-12-19
gene therapy
Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, discussed lessons that should guide the next generation of developers and regulators entering the field.
Hansa Biopharma Submits BLA to FDA for Imlifidase in desensitization for Kidney Transplantation
PR Newswire
2025-12-19
bla
pivotal
The application is based on the successful outcome of the pivotal US Phase 3 ConfIdeS trial in highly sensitized patients Imlifidase is an enzyme that rapidly and efficiently cleaves all types of immunoglobulin G (IgG) antibodies both intra- and extra-vascularly to inhibit IgG-mediated...
Senators point fingers over who blocked the rare disease voucher reauthorization
Endpoints News
2025-12-19
priority review
Sens. Bernie Sanders (I-VT) and Markwayne Mullin (R-OK) are not seeing eye to eye on why the Senate failed to pass legislation reauthorizing the FDA's rare pediatric priority review voucher program. Mullin took to the ...
uniQure: Why Waiting For Clarity Beats Chasing The Next Step Jump (NASDAQ:QURE)
uniQure
2025-12-18
amt-130
Get the latest on UniQure (QURE): FDA delays impact AMT-130 approval outlook.
Dr. William R. Shankle to Advance ACP-01 as a Treatment for Vascular Dementia
Hemostemix
2025-12-18
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta--(Newsfile Corp. - November 20, 2025) - Hemostemix Inc. (TSXV: HEM) (OTCQB: HMTXF) (FSE: 2VF0), the leading autologous (patient's own) stem cell therapy company offering VesCell™ (ACP-01) to no-option patients suffering from pain, angina, peripheral arterial disease, chronic limb threatening ischemia, ischemic cardiomyopathy, non-ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, is pleased to announce Dr. William R. Shankle, MS, MD, FACP will lead the Phase I clinical trial of ACP-01 as a treatment for vascular dementia (VaD).
ASH: Guidelines Updated for Management of Acute Myeloid Leukemia in Older Adults
Hematology Advisor News
2025-12-18
aml
leukemia
acute myeloid leukemia
Guidelines recommend older adults with newly diagnosed AML should be offered antileukemic therapy over best supportive care.
Lumicell Announces Collaboration with Learn Look Locate to Empower Breast Cancer Patients through Lumpectomy Education
PR Newswire
2025-12-18
breast cancer
NEWTON, Mass., Dec. 18, 2025 /PRNewswire/ -- Lumicell, Inc., a leader in developing innovative fluorescence-guided imaging technologies for cancer detection, today announced a new collaboration with Learn Look Locate, a global breast cancer education platform dedicated to empowering...
Hemostemix to Showcase Breakthrough Wound Healing Therapy at Innovations in Wound Care Conference Florida
Hemostemix
2025-12-18
cell therapy
Calgary, Alberta--(Newsfile Corp. - November 18, 2025) - Hemostemix Inc. (TSXV: HEM) (OTCQB: HMTXF) (FSE: 2VF0), a leader in regenerative medicine, will attend the Innovations in Wound Care Conference from December 11-14, 2025 in Key West, Florida. The company will highlight its world-leading angiogenic cell therapy, ACP-01 , which regenerates circulation, heals ulcers, alleviates pain, and improves quality of life for patients with advanced wound care needs including Peripheral Arterial Disease, Critical Limb-Threatening Ischemia (CLTI) .
VIDEO: Sacituzumab govitecan fails to extend PFS in advanced breast cancer
Healio
2025-12-18
her2
breast cancer
SAN ANTONIO — In this video, Jennifer A. Ligibel, MD, discusses results from the ASCENT-07 trial, in which sacituzumab govitecan did not meet its primary endpoint of PFS for patients with endocrine therapy-refractory advanced breast cancer.Results of the trial, which compared PFS among patients with hormone receptor-positive, HER2-negative breast cancer assigned either sacituzumab govitecan
VIDEO: Preliminary data support further study of combo therapy in older patients with DLBCL
Healio
2025-12-18
dlbcl
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
ORLANDO — In this video, Ann S. LaCasce, MD, MMSc, highlights preliminary results of a study of a chemotherapy-light triple combination regimen in older patients with diffuse large B-cell lymphoma.
VIDEO: Pirtobrutinib makes inroads in CLL, SLL as potential first-line therapy
Healio
2025-12-18
cll
leukemia
small lymphocytic lymphoma
chronic lymphocytic leukemia
ORLANDO — In this video, Matthew S. Davids, MD, said data from the BRUIN-313 trial supports front-line approval of pirtobrutinib in patients with previously untreated chronic lymphocytic leukemia or small lymphocytic lymphoma.The research presented at the ASH Annual Meeting and Exposition compared pirtobrutinib (Jaypirca, Eli Lilly & Co.) vs. the chemoimmunotherapy regimen, bendamustine
Healio Minute Podcast, Lung Cancer Edition: Top Headlines – Week of December 15, 2025
Healio
2025-12-18
lung cancer
SUO-CTC and CG Oncology Announce the Second Annual Non-Muscle Invasive Bladder Cancer (NMIBC) Research Fellowship Award
CG Oncology
2025-12-18
bladder cancer
Two applicants will each receive a Grant of $50,000 to support the development of outstanding clinical cancer research to improve the treatment of NMIBCApplications for the fellowship are due on February 27, 2026 SCHAUMBURG, Ill. and IRVINE, Calif., Dec. 18, 2025 (GLOBE NEWSWIRE) -- The Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC), a national alliance of leading academic and community based uro-oncologists, and CG Oncology, Inc. (NASDAQ: CGON), a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients with bladder cancer, today announced the second annual CG-SUO-CTC NMIBC Research Fellowship designed to support the development of outstanding clinical cancer research investigators who have demonstrated a commitment to improving the understanding and treatment of Non-Muscle Invasive Bladder Cancer (NMIBC).
New bladder cancer fellowships offer $50,000 grants for research talent
CG Oncology
2025-12-18
bladder cancer
Two NMIBC researchers will each receive $50,000, with projects due to be presented at the SUO session of the AUA meeting. Apply by Feb. 27, 2026.
Hemostemix Announces Its Webinar for Florida Vascular Surgeons: Autologous Stem Cell Therapy for CLTI
Hemostemix
2025-12-18
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta--(Newsfile Corp. - October 28, 2025) - Hemostemix Inc. (TSXV: HEM) (OTCQB: HMTXF) (FSE: 2VF0), the leading autologous (patient's own) stem cell therapy company offering VesCell™ (ACP-01) to no-option individuals suffering from pain related to angina, peripheral arterial disease, chronic limb threatening ischemia, ischemic cardiomyopathy, non-ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, is pleased to announce a one-hour educational webinar for Florida vascular surgeons and healthcare professionals. The session will introduce ACP-01 , Hemostemix's innovative autologous stem cell therapy designed for patients who suffer in pain with chronic limb-threatening ischemia (CLTI) who have no revascularization options.
Hemostemix Granted Trademark 'KNOW YOUR HEALTH' in Japan.
Hemostemix
2025-12-18
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta--(Newsfile Corp. - October 22, 2025) - Hemostemix Inc. (TSXV: HEM) (OTCQB: HMTXF) (FSE: 2VF0) , the leading autologous (patient's own) stem cell therapy company offering VesCell™ (ACP-01) to no-option individuals suffering from angina, peripheral arterial disease, chronic limb threatening ischemia, ischemic cardiomyopathy, non-ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, is pleased to announce that the Government of Japan has granted registration of its trademark "KNOW YOUR HEALTH" , under International Registration No. 1744775 , covering Classes 1, 5, and 42 .
Astellas Receives Advanced Manufacturing Technology Designation by U.S. FDA for Maholo Robotic Cell Culture Automation System
astellas
2025-12-18
cell therapy
Pioneering cell therapy innovation through robotics and AI - - Among the first innovator pharmaceutical companies granted AMT designation - - Enabling early, priority engagement with FDA to...
Minaris and Cell and Gene Therapy Catapult Announce Collaboration to Advance Delivery Methods for Cell and Gene Therapies
PR Newswire
2025-12-18
gene therapy
gene therapies
PHILADELPHIA and LONDON, Dec. 18, 2025 /PRNewswire/ -- Minaris, a global cell and gene therapy (CGT) contract development and manufacturing organization (CDMO) and multimodality biosafety testing provider and Cell and Gene Therapy Catapult (CGT Catapult), an independent technology and...
J&J's subcutaneous Rybrevant cleared by FDA
PharmaPhorum
2025-12-18
lung cancer
J&J has claimed FDA approval for Rybrevant Faspro, a subcutaneous version of the lung cancer therapy central to its blockbuster hopes for the drug.
VIDEO: ‘Significant benefit’ with epcoritamab regimen in patients with follicular lymphoma
Healio
2025-12-18
follicular lymphoma
ORLANDO — In this video, Ann S. LaCasce, MD, MMSc, discusses results from the phase 3 EPCORE FL-1 trial, presented at ASH Annual Meeting and Exposition.
Does uniQure (QURE) FDA Feedback on AMT-130 Reframe Its Risk, Funding and Strategy Profile?
uniQure
2025-12-18
amt-130
gene therapy
In early December 2025, uniQure reported that final FDA meeting minutes indicated Phase I/II data for its Huntington's gene therapy AMT-130 are currently unlikely to provide the primary evidence needed for a Biologics License Application, prompting the company to seek a follow-up meeting in early 2026. This feedback raises questions about trial design, endpoint robustness, and the potential need for additional studies, sharpening focus on uniQure’s regulatory pathway and funding capacity for...
Intravesical Oncolytic Adenovirus Delivers High Complete Response Rates in BCG-Naive, High-Risk, Non–Muscle-Invasive Bladder Cancer With Carcinoma in Situ
CG Oncology
2025-12-18
adenovirus
bladder cancer
complete response
Findings from a phase II trial highlight a promising new treatment option for non–muscle-invasive bladder cancer in a disease space challenged by bacillus Calmette-Guérin (BCG) shortages and the need ...
Nektar Therapeutics announces top-line results from alopecia areata study
PMLive Research
2025-12-18
top-line results
This autoimmune disease causes hair loss in both women and men, affecting 160 million people worldwide
Hemostemix Launches Enhanced HubSpot-Powered Websites to Support Global Marketing & Sales of ACP-01 for No-Option Patients
Hemostemix
2025-12-18
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta--(Newsfile Corp. - September 9, 2025) - Hemostemix Inc. (TSXV: HEM) (OTCQB: HMTXF) (FSE: 2VF0), the leading autologous (patient's own) stem cell therapy company offering VesCell™ (ACP-01) to individuals suffering from peripheral arterial disease, chronic limb threatening ischemia, angina, ischemic cardiomyopathy, non ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, in Florida under Florida's SB 1768, announces the launch of a newly upgraded corporate website, fully integrated by HubSpot, representing a significant improvement in regulatory compliant marketing and sales functionalities.
SUO-CTC and CG Oncology Announce the Second Annual Non-Muscle Invasive Bladder Cancer (NMIBC) Research Fellowship Award
CG Oncology
2025-12-18
bladder cancer
Two applicants will each receive a Grant of $50,000 to support the development of outstanding clinical cancer research to improve the treatment of...
Hemostemix Inc. Announces FDA Pre-IND Meeting and NBPP of $960,000
Hemostemix
2025-12-18
ind
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta--(Newsfile Corp. - December 10, 2025) - Hemostemix Inc. (TSXV: HEM) (OTCQB: HMTXF) (FSE: 2VF0) ("Hemostemix" or the "Company"), the leading autologous (patient's own) stem cell therapy company offering VesCell™ (ACP-01) to individuals suffering from vascular dementia, angina, ischemic cardiomyopathy, congestive heart failure, non-ischemic dilated cardiomyopathy, pain, total body ischemia, peripheral arterial disease and chronic limb threatening ischemia, with ACP-01 at Doctors Hospital, Nassau, The Bahamas, is pleased to announce two major corporate developments:
Beam Therapeutics to Present at 44th Annual J.P. Morgan Healthcare Conference
beam therapeutics
2025-12-18
base editing
CAMBRIDGE, Mass., Dec. 18, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that John Evans, chief executive officer of Beam, will present at the 44 th Annual J.P.
VIDEO: Highlights in mantle cell lymphoma from ASH
Healio
2025-12-18
mantle cell lymphoma
ORLANDO — In this video, Ann S. LaCasce, MD, MMSc, discusses highlights in mantle cell lymphoma presented at ASH Annual Meeting and Exposition.
Asked and Answered: Prime Medicine CEO on First Prime Editing Patient Data
Inside Precision Medicine
2025-12-18
prime editing
In this interview, Allan Reine, MD, dives into this monumental prime editing report, explaining how the clinical results affect the company’s position on its drug development pipeline, including the CGD program. The post Asked and Answered: Prime Medicine CEO on First Prime Editing Patient Data appeared first on Inside Precision Medicine .
Curasight Announces Successful Dosing of First Patient in Phase 1 trial with uTREAT® in Brain Cancer
PR Newswire
2025-12-18
glioblastoma
Phase 1 trial in aggressive brain cancer (Glioblastoma) is the first clinical trial of uTREAT® belonging to Curasight's treatment platform No patient safety issues were reported The dosing marks a key milestone with Curasight now in clinical development with both its therapeutic (uTREAT®)...
Brain’s Viral Defense Mechanism Linked to Tau Pathology in Alzheimer’s
GenEng News
2025-12-18
alzheimer's disease
The pathological features of Alzheimer's disease might once have been part of an orchestrated defense system against widespread infections in the brain when human lifespans were much shorter, scientists say. The post Brain’s Viral Defense Mechanism Linked to Tau Pathology in Alzheimer’s appeared first on GEN - Genetic Engineering and Biotechnology News .
Sangamo Therapeutics Initiates Rolling Submission of BLA to U.S. FDA for ST-920 in Fabry Disease
Sangamo BioSciences
2025-12-18
bla
st-920
fabry disease
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
CEPI steps in to fund Moderna's pandemic flu jab
PharmaPhorum
2025-12-18
pivotal
Thanks to CEPI, Moderna's H5 bird flu vaccine can start a pivotal trial shelved after funding for the jab was pulled in the US by HHS Sec Kennedy.
Datroway filed for TNBC in EU after data delay
PharmaPhorum
2025-12-18
breast cancer
Daiichi Sankyo and AstraZeneca have filed their first marketing application for TROP2-targeting Datroway in triple-negative breast cancer.
‘Passion project’: Study confirms link between Agent Orange, myelodysplastic syndrome
Healio
2025-12-18
myelodysplastic
myelodysplastic syndrome
ORLANDO — A national prospective study has confirmed a long-suspected link between Agent Orange and a rare but serious form of blood cancer.Individuals exposed to the powerful Vietnam War-era herbicide exhibited significantly elevated risk for myelodysplastic syndrome (MDS), findings presented at ASH Annual Meeting and Exposition showed.
Daiichi and Merck's I-Dxd lung cancer study faces partial hold due to deaths
Endpoints News
2025-12-18
lung cancer
The FDA placed a partial hold on Daiichi Sankyo and Merck’s Phase 3 study of a lung cancer drug after the companies paused enrollment for what they called a "higher than anticipated incidence" of deaths ...
Relief Cardiovascular Appoints Harry Rowland as Chairman of the Board
PR Newswire
2025-12-18
heart failure
IRVINE, Calif., Dec. 18, 2025 /PRNewswire/ -- Relief Cardiovascular, a private medical technology company developing the Relief System, a transcatheter implant designed to intelligently monitor and modulate cardiac preload to relieve congestion in heart failure, today announced the...
Voyager Throws 30 Staff Overboard After Novartis Sinks Two Programs
BioSpace
2025-12-18
spinal muscular atrophy
Novartis has discontinued two undisclosed programs under its current partership with Voyager, the biotech announced last month. Projects under the deal for spinal muscular atrophy and Huntington’s disease continue to advance.
Takeda Drug From $4B Deal Succeeds in Two Pivotal Plaque Psoriasis Trials
MedCity News
2025-12-18
pivotal
Takeda said more than half of plaque psoriasis patients treated with zasocitinib achieved clear or almost clear skin in two Phase 3 studies. Regulatory submissions for the TYK2 inhibitor are planned for 2026 and clinical trials are ongoing in other indications. The post Takeda Drug From $4B Deal Succeeds in Two Pivotal Plaque Psoriasis Trials appeared first on MedCity News .
Gemcitabine Intravesical System Shows High Disease-Free Survival Rates in BCG-Unresponsive Papillary-Only NMIBC
CG Oncology
2025-12-18
bla
Monotherapy with a gemcitabine intravesical system demonstrated high disease-free survival rates in patients with bacillus Calmette-Guérin-unresponsive papillary-only high-risk non–muscle-invasive bla...
Karlla W. Brigatti, MS, CGC, on Accelerating SMA Diagnosis and Treatment
CGTLive
2025-12-18
sma
VIDEO: Imlunestrant improves survival for women with advanced breast cancer
Healio
2025-12-18
her2
breast cancer
SAN ANTONIO — In this video, Halle C. F. Moore, MD, discusses the EMBER-3 study update evaluating imlunestrant for women with hormone receptor-positive, HER2-negative breast cancer, presented at San Antonio Breast Cancer Symposium.Moore said patients who received a combination of imlunestrant (Eli Lilly & Co.) and abemaciclib (Verzenio, Eli Lilly & Co.) had a trend toward improved
1st trial participant receives Alpha DaRT radiation therapy for glioblastoma
Rare Cancer News
2025-12-18
glioma
glioblastoma
The first participant has been dosed in an early clinical trial testing the experimental radiation therapy Alpha DaRT in people with hard-to-treat glioma. The clinical trial (NCT06910306), sponsored by Alpha DaRT’s developer, Alpha Tau Medical, is expected to enroll 10 adults with glioblastoma, an especially aggressive type of glioma. The study is open to patients […] The post 1st trial participant receives Alpha DaRT radiation therapy for glioblastoma appeared first on Rare Cancer News .
VIDEO: SABCS ‘moving the needle forward‘ in metastatic breast cancer treatment
Healio
2025-12-17
breast cancer
SAN ANTONIO — In this video, Megan Kruse, MD, discusses breast cancer treatment updates at the 2025 San Antonio Breast Cancer Symposium, including anti-estrogen therapies, targeted therapies, antibody-drug conjugates and biomarker research.“I would say the overall atmosphere is one of positivity, enthusiasm and encouragement that the clinical research that we are doing is really
VIDEO: New data on ‘exciting’ targeted agents a highlight of this year’s SABCS
Healio
2025-12-17
breast cancer
SAN ANTONIO — In this video, Erica L. Mayer, MD, MPH, discusses highlights from San Antonio Breast Cancer Symposium, from networking opportunities to the latest research on emerging therapies.“Now that we have so many exciting targeted agents that are in development for breast cancer, every session we have is presenting new data on targeted therapies from the research laboratory into
Patient-centered thyroid cancer guideline marks a shift in care
Healio
2025-12-17
thyroid cancer
The past decade has seen a major shift in how thyroid cancer is diagnosed and managed. R. Michael Tuttle, MD, chief of endocrinology service at Memorial Sloan Kettering Cancer Center and professor of medicine at Weill Cornell Medical University in New York, said thyroid cancer management was much more rigid in 2015 than it is today, with most diagnosed patients undergoing surgery, even those
Elacestrant combos confer ‘clinically meaningful’ outcomes for advanced breast cancer
Healio
2025-12-17
her2
breast cancer
SAN ANTONIO — Combining elacestrant with targeted agents produced “clinically meaningful” outcomes for patients with locally advanced or metastatic ER-positive/HER2-negative breast cancer who have received at least one line of treatment.The data, presented at San Antonio Breast Cancer Symposium, showed patients who received elacestrant (Orserdu, Stemline Therapeutics) plus
AstraZeneca’s Enhertu combination approved by FDA for HER2-positive breast cancer
PMLive Regulatory
2025-12-17
her2
breast cancer
Around one in five breast cancer cases are HER2-positive
RA Capital backs drug for rare kind of chronic pain
Biopharma Dive
2025-12-17
pivotal
The investment firm co-led a $125 million Series A round for Ambros Therapeutics, a Vivek Ramaswamy co-founded company with a medicine nearing pivotal testing.
Breast Cancer Drugs Market to Reach USD 57.1 Billion by 2031 as Precision Oncology and Targeted Therapies Redefine Treatment Outcomes
PR Newswire
2025-12-17
breast cancer
AUSTIN, Texas and TOKYO, Dec. 17, 2025 /PRNewswire/ -- According to DataM Intelligence, the global Breast Cancer Drugs Market reached USD 32.4 billion in 2024 and is projected to expand to USD 57.1 billion by 2031, growing at a robust CAGR of 9.2% during the forecast period 2025–2031....
Padcev/Keytruda scores again in early bladder cancer
PharmaPhorum
2025-12-17
bladder cancer
Pfizer and Astellas now have two studies supporting the use of Padcev with MSD's Keytruda as perioperative therapy for muscle-invasive bladder cancer.
ALZpath-Powered Blood Test Enables Landmark Study on Growing Alzheimer's Disease Prevalence
PR Newswire
2025-12-17
alzheimer's disease
First Study to Use a pTau217 Blood Test to Detect Alzheimer's Disease Early and at Scale CARLSBAD, Calif., Dec. 17, 2025 /PRNewswire/ -- ALZpath, Inc., a leading developer of innovative diagnostic tools for Alzheimer's and related diseases, today announced that the Simoa® ALZpath pTau217...
VIDEO: AAO 2025 includes ‘amazing talk’ on treatment durability in wet AMD
Healio
2025-12-17
wet age-related macular degeneration
ORLANDO — In this video, Carolyn K. Pan, MD, a clinical professor of ophthalmology at Stanford University, discussed highlights in wet age-related macular degeneration from the American Academy of Ophthalmology meeting.“Dr. Jennifer Lim gave an amazing talk at Retina Subspecialty Day on the first day on what’s new in wet AMD, and she touched on several case studies that looked
Teal Health Teams Up With Thatch to Expand Access to Cervical Cancer Screening
MedCity News
2025-12-17
cervical cancer
Through a new partnership, Thatch’s employer customers can now offer Teal Health’s at-home cervical cancer screening as an employee benefit through an ICHRA. The post Teal Health Teams Up With Thatch to Expand Access to Cervical Cancer Screening appeared first on MedCity News .
QURE INVESTIGATION: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2025-12-17
bla
amt-130
gene therapy
RADNOR, PA - December 17, 2025 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Kyverna Therapeutics Announces Pricing of $100 Million Public Offering of Common Stock
Kyverna Therapeutics
2025-12-17
cell therapies
EMERYVILLE, Calif., Dec. 17, 2025 (GLOBE NEWSWIRE) -- Kyverna Therapeutics, Inc. (Nasdaq: KYTX) (“Kyverna”), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, today announced the pricing of an underwritten public offering of
Novel AI model improves recurrence risk stratification in breast cancer subset
Healio
2025-12-17
her2
breast cancer
SAN ANTONIO — An AI model significantly improved recurrence risk stratification vs. recurrence score among patients with hormone receptor-positive, HER2-negative breast cancer, according to study results.Findings were presented at San Antonio Breast Cancer Symposium.
Ferring Sees Financial Success with Bladder Cancer Gene Therapy, Announces Plans for New Manufacturing Facility
Ferring Pharmaceuticals
2025-12-17
adstiladrin
gene therapy
bladder cancer
The company will be adding a new manufacturing site to its NJ campus for the drug Adstiladrin.
Roche Makes Good on Dealmaking Promise With $1.1B Caris Cancer Collab
Roche Group
2025-12-17
solid tumor
Roche CEO Thomas Schinecker said during the company's Q3 call that it is “not done” with deals—a promise he delivered on with Tuesday's solid tumor pact with AI-focused Caris Life Sciences.
Is active surveillance a good option for people with small thyroid cancers?
Healio
2025-12-17
thyroid cancer
Active surveillance is generally a good option because a lot of small papillary thyroid cancers are present in people for a long time. We know that people can live their whole lives, die of another cause and be found to have thyroid cancer in their body that never became clinically active while they were alive. Imaging now is so sophisticated that we can find small tumors, and it’s not
VIDEO: Pirtobrutinib could be front-line option for older, frail patients with CLL
Healio
2025-12-17
cll
ORLANDO — In this video, Matthew S. Davids, MD, spoke about the BRUIN-314 trial, one of the first head-to-head studies to compare pirtobrutinib, a noncovalent Bruton tyrosine kinase inhibitor, with ibrutinib, a covalent BTK inhibitor.Davids highlighted data presented at the ASH Annual Meeting and Exposition that showed the overall response rate with pirtobrutinib (Jaypirca, Eli Lilly &
VIDEO: Time-limited therapy for CLL may offer flexibility, minimize side effects
Healio
2025-12-17
cll
leukemia
chronic lymphocytic leukemia
ORLANDO — Healio spoke with Matthew S. Davids, MD, about a recent study comparing continuous treatment with a Bruton tyrosine kinase inhibitor with time-limited venetoclax-based therapy for chronic lymphocytic leukemia.Davids highlighted data presented at the ASH Annual meeting that showed “similar outcomes” after 3 years in patients with CLL, regardless if they received
FDA Activity Recap: November 2025 Features SMA Approval, Boxed Warning for Elevidys, and More
CGTLive
2025-12-17
sma
elevidys
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.
Cerapedics Announces Publication in Spine of 24-Month ASPIRE Study Results of PearlMatrix™ P-15 Peptide Enhanced Bone Graft
PR Newswire
2025-12-17
pivotal
ASPIRE pivotal IDE study results demonstrated PearlMatrix achieved superior clinical success and superior time-to-fusion at 24 months compared to local autograft. The rigor of the ASPIRE study reflects unmatched clinical design and results including: - More than twice as many patients...
Addition emerges with $100M to make gene therapies for chronic and rare diseases
Biopharma Dive
2025-12-17
gene therapy
gene therapies
Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes can sidestep issues seen with traditional gene therapy technology.
Padcev-Keytruda combination notches another bladder cancer win
Endpoints News
2025-12-17
bladder cancer
Pfizer, Astellas and Merck announced Wednesday that the combination of Padcev and Keytruda helped certain bladder cancer patients live longer in a Phase 3 trial, and plan to take the regimen to regulators to discuss ...
Orchard Therapeutics Celebrates Addition of Metachromatic Leukodystrophy to the U.S. Recommended Uniform Screening Panel
Kyowa Kirin Co.
2025-12-17
metachromatic leukodystrophy
Breast Cancer Genetic Screening Stratifies Risk, Biopsy Rates Stay Unchanged
Inside Precision Medicine
2025-12-17
breast cancer
Two new studies suggest there should be change to current guidelines that all women of average risk have a mammogram every year, starting at 40 and lasting until age 74. The post Breast Cancer Genetic Screening Stratifies Risk, Biopsy Rates Stay Unchanged appeared first on Inside Precision Medicine .
VIDEO: Epcoritamab triplet regimen potential second-line therapy in follicular lymphoma
Healio
2025-12-17
follicular lymphoma
ORLANDO — In this video, Brian T. Hill, MD, PhD, discusses data from the phase 3 EPCORE FL-1 trial, presented at the ASH Annual Meeting and Exposition.The research compared a triple drug combination therapy of epcoritamab (Epkinly; Genmab, AbbVie) plus rituximab (Rituxan; Genentech, Biogen) and lenalidomide (Revlimid, Bristol Myers Squibb) with standard therapy (rituximab-lenalidomide
ASH: Agent Orange Exposure Linked to Increased Risk for Myelodysplastic Syndrome
Hematology Advisor News
2025-12-17
myelodysplastic
myelodysplastic syndrome
In multivariate analyses, higher risk for AO exposure seen for 1945 to 1954 birth cohort, those with military service, Blacks versus Whites.
Sarepta Therapeutics Applauds Addition of Duchenne Muscular Dystrophy to the U.S. Recommended Uniform Screening Panel (RUSP)
Sarepta Therapeutics
2025-12-17
sarepta
duchenne muscular dystrophy
U.S. Federal recommendation for Duchenne on newborn screening panel signals benefits of early detection - Early diagnosis in Duchenne is now more essential than ever, as available treatments can help slow disease progression and preserve mobility CAMBRIDGE, Mass. --(BUSINESS WIRE)--Nov.
Orchard Therapeutics Celebrates Addition of Metachromatic Leukodystrophy to the U.S. Recommended Uniform Screening Panel
orchard therapeutics
2025-12-16
metachromatic leukodystrophy
Newborn screening is the only practical means of diagnosing MLD prior to the onset of symptoms, which is key to achieving optimal outcomes for children and their families with this rapidly progressive disease As of today, 14 states—representing more than 50 percent of U.S.
INCOG BioPharma Announces $200 Million Expansion, Growing the Team to Nearly 1,000 Employees
PR Newswire
2025-12-16
ind
FISHERS, Ind., Dec. 16, 2025 /PRNewswire/ -- INCOG BioPharma Services, a leading contract development and manufacturing organization (CDMO), today announced another major expansion with an investment of approximately $200 million in its manufacturing facility in Fishers, Indiana. This...
Capricor Therapeutics and Parent Project Muscular Dystrophy to Host Webinar Highlighting Positive Phase 3 HOPE-3 Topline Results in Duchenne Muscular Dystrophy
Capricor Therapeutics
2025-12-16
duchenne muscular dystrophy
ANKTIVA® with BCG Demonstrates 96% Survival from Bladder Cancer at Three Years with Median Survival Not Yet Reached in BCG-Unresponsive High-Grade Papillary-Only Non-Muscle Invasive Bladder Cancer
Immunity Bio
2025-12-16
bladder cancer
The post ANKTIVA® with BCG Demonstrates 96% Survival from Bladder Cancer at Three Years with Median Survival Not Yet Reached in BCG-Unresponsive High-Grade Papillary-Only Non-Muscle Invasive Bladder Cancer appeared first on ImmunityBio .
Our holiday trip is a symbol of our daughter’s journey after gene therapy
AADC News/BioNews
2025-12-16
gene therapy
Our family is packing our suitcases once again, but this time it feels different. Instead of bracing ourselves for a long journey ahead, Judy, my wife, and I find our excitement rising each day as we prepare for a short 10-day trip back to the U.S. We’ve been looking forward to this visit for months, […] The post Our holiday trip is a symbol of our daughter’s journey after gene therapy appeared first on AADC News .
FDA Greenlights First CAR T-Cell Therapy for Marginal Zone Lymphoma
Hematology Advisor News
2025-12-16
car t
breyanzi
cell therapy
marginal zone lymphoma
The FDA has approved Breyanzi for the treatment of adult patients with R/R MZL who have received at least 2 prior lines of systemic therapy.
Colorectal Cancer Outcomes Explained by Distinct Regulatory T-Cell Subsets
GenEng News
2025-12-16
colorectal cancer
Two distinct tumoral T-reg subsets with differential Il10 expression were identified in colorectal cancer, informing immunotherapy treatment for patients with cancers that affect the skin, stomach lining, mouth, and throat. The post Colorectal Cancer Outcomes Explained by Distinct Regulatory T-Cell Subsets appeared first on GEN - Genetic Engineering and Biotechnology News .
J&J Wins Proactive FDA National Priority Voucher for Multiple Myeloma Combo
BioSpace
2025-12-16
multiple myeloma
Johnson & Johnson, which did not apply for the national priority voucher, was granted the ticket based on results from a Phase III study testing Tecvayli plus Darzalex in patients with relapsed or refractory multiple myeloma.
AstraZeneca, Daiichi Sankyo ADC Enhertu Moves Up to First-Line Use in Breast Cancer
MedCity News
2025-12-16
her2
pivotal
breast cancer
FDA approval for Enhertu as a first-line breast cancer treatment is important because many patients with HER2-positive cancer do not live long enough to receive a second line of therapy. In Enhertu’s pivotal study in this setting, the HER2-targeting ADC helped patients live more than three years without disease progression or death. The post AstraZeneca, Daiichi Sankyo ADC Enhertu Moves Up to First-Line Use in Breast Cancer appeared first on MedCity News .
Kyverna’s CAR T Therapy Delivers ‘Impressive’ Mobility Benefits in Stiff Person Syndrome
BioSpace
2025-12-16
car t
stiff person syndrome
Kyverna plans to submit mivocabtagene autoleucel to the FDA for approval in the first half of 2026. If approved, it would be the first CAR T therapy for an autoimmune disease.
AstraZeneca, Daiichi Sankyo Move Enhertu to First-Line Metastatic Breast Cancer
BioSpace
2025-12-16
breast cancer
Analysts at Jefferies called the approval "highly significant," estimating it could add $2 billion to $3 billion to peak Enhertu sales.
FDA ‘proactively’ hands J&J a voucher; Pfizer issues 2026 forecasts
Biopharma Dive
2025-12-16
cell therapy
multiple myeloma
The FDA contacted J&J right after ASH to discuss using a special fast pass on a multiple myeloma regimen. Elsewhere, a new cell therapy player emerged and Sanofi cut deals with two startups.
ANKTIVA® with BCG Demonstrates 96% Survival from Bladder Cancer at Three Years with Median Survival Not Yet Reached in BCG-Unresponsive High-Grade Papillary-Only Non-Muscle Invasive Bladder Cancer
Immunity Bio
2025-12-16
bladder cancer
The post ANKTIVA® with BCG Demonstrates 96% Survival from Bladder Cancer at Three Years with Median Survival Not Yet Reached in BCG-Unresponsive High-Grade Papillary-Only Non-Muscle Invasive Bladder Cancer appeared first on ImmunityBio .
UniQure (QURE) Stock Drop Looms as FDA Concern Over Huntington’s Data Spurs Mizuho Downgrade and Delayed Launch
uniQure
2025-12-16
amt-130
UniQure (NASDAQ:QURE) is one of the stocks that will double in 2026. On December 9, Mizuho analyst Uy Ear lowered the firm’s price target on uniQure to $33 from $60, while keeping an Outperform rating on the shares. The firm adjusted its financial model for AMT-130 after the company announced that the FDA communicated concerns […]
VIDEO: ‘Overall excellent’ outcomes with acalabrutinib prior to chemotherapy in DLBCL
Healio
2025-12-16
dlbcl
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
ORLANDO — In this video, Brian T. Hill, MD, PhD, discusses promising results from a study examining the impact of acalabrutinib prior to frontline chemotherapy in patients with untreated diffuse large B-cell lymphoma.In the study presented at the ASH Annual Meeting and Exposition, patients with DLBCL received acalabrutinib (Calquence, AstraZeneca) as a monotherapy prior to chemotherapy.
ImmunoLogic, Episode 7: "IL-9 and CAR-T Therapy” With Anusha Kalbasi, MD
CGTLive
2025-12-16
car-t
Nektar's immunology drug misses in Phase 2b, execs cite enrollment errors
Endpoints News
2025-12-16
pivotal
Nektar Therapeutics said a Phase 2b study for its lead immunology drug failed in severe alopecia, but executives blamed the miss on trial enrollment errors and plan to advance the program into pivotal testing. ...
OrsoBio obesity data; Monte Rosa's prostate cancer pill; Development pacts for Adaptive and SandboxAQ
Endpoints News
2025-12-16
prostate cancer
Plus, news about: Hansoh, Caris-Genentech, Royalty, LIB, Greenwich LifeSciences, Gubra, J&J, Sprout, Tiziana and Vor. 💊 OrsoBio’s early obesity data: When combined with Lilly’s tirzepatide, OrsoBio’s oral obesity drug TLC-6740 led to an additional ...
J&J claims FDA's latest national priority voucher
PharmaPhorum
2025-12-16
multiple myeloma
The FDA has awarded its latest national priority voucher to Johnson & Johnson for a combination regimen based on Tecvayli for multiple myeloma.
Beacon Therapeutics Treats First Patient in LANDSCAPE Trial of laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP)
Beacon Therapeutics
2025-12-16
x-linked
retinitis pigmentosa
x-linked retinitis pigmentosa
The post Beacon Therapeutics Treats First Patient in LANDSCAPE Trial of laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) appeared first on Beacon Therapeutics .
U.S. Department of Health and Human Services Adds Duchenne to Recommended Uniform Screening Panel
PR Newswire
2025-12-16
duchenne muscular dystrophy
Parent Project Muscular Dystrophy and Muscular Dystrophy Association Celebrate Monumental Victory for Duchenne Muscular Dystrophy WASHINGTON, Dec. 16, 2025 /PRNewswire/ -- Today, the U.S. Department of Health and Human Services (HHS) officially added Duchenne muscular dystrophy (Duchenne)...
FDA to Review Nivolumab Combo for First-Line Classical Hodgkin Lymphoma
Hematology Advisor News
2025-12-16
priority review
hodgkin lymphoma
The FDA has granted Priority Review to nivolumab in combination with AVD for patients aged 12 years and older with untreated stage III or IV cHL.
AI Model Helps Diagnose Under-Recognized Heart Condition
Inside Precision Medicine
2025-12-16
coronary microvascular dysfunction
A self-supervised AI model can accurately predict if people have coronary microvascular dysfunction, a relatively common but underdiagnosed heart condition, using data from a simple ECG test. The post AI Model Helps Diagnose Under-Recognized Heart Condition appeared first on Inside Precision Medicine .
VIDEO: Combination ribociclib, fulvestrant beneficial for lobular breast cancer
Healio
2025-12-16
breast cancer
SAN ANTONIO — In this video, Megan Kruse, MD, discusses poster results from the MONALEESA-3 trial evaluating ribociclib plus fulvestrant for advanced breast cancer, presented at San Antonio Breast Cancer Symposium.“This is encouraging data to have out there for our patients and for providers who take care of [patients with metastatic lobular breast cancer] just to know that this
J&J backs financing round for Stanford CAR-T cell therapy spinout Link Cell
Endpoints News
2025-12-16
car-t
cell therapy
cell therapies
Link Cell Therapies launched Monday with a $60 million Series A round to fund the creation of the next wave of CAR-T therapies. The startup plans to go after the treatment of solid tumors, where ...
Beacon Therapeutics Treats First Patient in LANDSCAPE Trial of laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP)
syncona
2025-12-16
x-linked
retinitis pigmentosa
x-linked retinitis pigmentosa
Ambros Launches With $125M To Bring Italian Non-Opioid Pain Drug to America
BioSpace
2025-12-16
pivotal
Ambros Therapeutics’ non-opioid bisphosphonate analgesic, already approved in Italy, will soon begin a pivotal test in the U.S.
‘Zombie’ biotech buyer Xoma to acquire Generation Bio
Biopharma Dive
2025-12-16
gene therapy
The gene therapy developer has lost most of its value since going public in 2020 and saw a long road ahead for a delivery technology that had shown promise in preclinical tests.
Fondazione Telethon Announces Historic FDA Approval Of Waskyra
Fondazione Telethon
2025-12-16
waskyra
Goodwin congratulates Fondazione Telethon on receiving approval from the U.S. Food and Drug Administration (FDA) of its Biologics License Application for Waskyra™...
Enhertu combo cleared for use in frontline breast cancer
Biopharma Dive
2025-12-16
her2
breast cancer
The approval could help Enhertu, which is already a multibillion-dollar seller, supplant a regimen that’s been the standard of care for HER2-positive breast tumors for more than a decade.
Saphnelo approved in the EU for subcutaneous self-administration as a new pre-filled pen for systemic lupus erythematosus 16 December 2025
AstraZeneca
2025-12-16
lupus
GenSight Biologics shares efficacy and safety findings from REFLECT Phase III gene therapy trial
GenSight Biologics
2025-12-16
gene therapy
leber hereditary optic neuropathy
The company shared 5-year results for lenadogene nolparvovec (Lumevoq), administered unilaterally and bilaterally to patients with Leber Hereditary Optic Neuropathy
Bespoke ctDNA Test Detects MRD in Lung Cancer Patients Years Before Imaging
Inside Precision Medicine
2025-12-16
lung cancer
New data from Personalis suggest that the company’s ultra-sensitive NeXT Personal test can detect recurrence in non-small cell lung cancer (NSCLC) far before radiographic imaging. The post Bespoke ctDNA Test Detects MRD in Lung Cancer Patients Years Before Imaging appeared first on Inside Precision Medicine .
uniQure (QURE) Valuation Update After FDA Feedback Clouds AMT-130’s Current Path to a Biologics Filing
uniQure
2025-12-16
amt-130
gene therapy
uniQure (NasdaqGS:QURE) just received final meeting minutes from the FDA on its AMT-130 gene therapy for Huntington's disease, and regulators signaled that current Phase I/II data likely will not anchor a Biologics License Application. See our latest analysis for uniQure. The cautious FDA feedback lands after a volatile stretch, with uniQure’s share price at $20.43, a 30 day share price return of negative 31.63 percent, a 90 day share price return of 44.28 percent, and a 1 year total...
Beyond Insulin Pumps: Cell Therapies Unlock a $403B Cure Economy
PR Newswire
2025-12-16
pivotal
cell therapy
cell therapies
Issued on behalf of Avant Technologies Inc. Equity Insider News Commentary VANCOUVER, BC, Dec. 16, 2025 /PRNewswire/ -- The cell therapy manufacturing sector reached a pivotal inflection point in late 2025, with contract development and manufacturing organizations capturing 67.5% market...
Tecvayli-Darzalex Faspro combo up for approval in 2 nations for treating RRMM
Rare Cancer News
2025-12-16
multiple myeloma
Johnson & Johnson (J&J) is asking the U.S. Food and Drug Administration (FDA) to approve a combination of two medications, Tecvayli (teclistamab) and Darzalex Faspro (daratumumab and hyaluronidase-fihj), for people with relapsed or refractory, or treatment-resistant, multiple myeloma (RRMM). The regulatory submission was based on what the developer, in a company press release, called “unprecedented […] The post Tecvayli-Darzalex Faspro combo up for approval in 2 nations for treating RRMM appeared first on Rare Cancer News .
FDA approves first gene therapy for Wiskott-Aldrich syndrome
PMLive Regulatory
2025-12-16
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
Symptoms of the rare disease include bleeding, eczema and frequent infections
Enhertu moves into frontline breast cancer treatment
PharmaPhorum
2025-12-16
her2
breast cancer
Daiichi Sankyo and AstraZeneca's Enhertu has become the first new first-line option in the US for HER2-positive breast cancer in more than a decade.
VIDEO: Niraparib plus dostarlimab shows promise in BRCA , PALB2 mutated breast cancer
Healio
2025-12-16
breast cancer
SAN ANTONIO — In this video, Erica L. Mayer, MD, MPH, discusses results of the phase 2 TBCRC 056 trial evaluating neoadjuvant niraparib plus dostarlimab in patients with germline BRCA- or PALB2-mutated triple-negative breast cancer.Results of the trial, which assessed efficacy of niraparib (Zejula, GSK) with dostarlimab (Jemperli, GSK) in this breast cancer subset, were presented at San
Adagene Announces FDA Fast Track Designation for Muzastotug (ADG126)
AdaGene
2025-12-16
fast track
colorectal cancer
Designation underscores the potential of SAFEbody®-enabled anti-CTLA-4 therapy to address unmet need in MSS colorectal cancer SAN DIEGO and SUZHOU, China , Dec. 16, 2025 (GLOBE NEWSWIRE) -- Adagene Inc. (“Adagene”) (Nasdaq: ADAG), a company transforming the discovery and development of novel
What's Going On With uniQure Stock On Tuesday?
uniQure
2025-12-16
amt-130
phase 1/2
uniQure shares slid nearly 31% in a month as the FDA raised concerns over whether AMT-130 Phase 1/2 data can support approval.
Caris Life Sciences Announces Collaboration with Genentech to Discover Novel Therapeutic Targets in Cancers with High Unmet Clinical Need
Genentech
2025-12-16
solid tumor
Caris Life Sciences® (NASDAQ: CAI), a leading, patient-centric, next-generation AI TechBio company and precision medicine pioneer, today announced that its therapeutic research arm, Caris Discovery, has entered into a multi-year collaboration and license agreement with Genentech, a member of the Roche Group. In this collaboration, Caris will work to identify and validate novel oncology targets in solid tumor tissue.
PCPs have concerns about certain risk-based breast cancer screening recommendations
Healio
2025-12-15
breast cancer
SAN ANTONIO — Women who receive risk-based breast cancer screening recommendations discussed those recommendations with their primary care providers more frequently than those who undergo annual screening.The data from the Women Informed to Screen Depending On Measures of risk (WISDOM) study, presented at San Antonio Breast Cancer Symposium, showed PCPs also had a higher likelihood of
Roche receives FDA approval for first diagnostic tests to identify HER2-positive metastatic breast cancer patients eligible for ENHERTU
Roche Group
2025-12-15
her2
breast cancer
FDA expands labels for Roche's PATHWAY HER2 (4B5) test* and VENTANA HER2 Dual ISH DNA Probe Cocktail, which can now be used to identify HER2-positive metastatic breast cancer patients that
AstraZeneca's Enhertu and Genentech's Perjeta combo gains breast cancer approval
Genentech
2025-12-15
her2
breast cancer
FDA approves Enhertu and Perjeta combo as first-line HER2-positive breast cancer treatment. Read more here.
Enhertu plus pertuzumab approved in the US as first new treatment in a decade for the 1st-line treatment of patients with HER2-positive metastatic breast cancer 15 December 2025
AstraZeneca
2025-12-15
her2
breast cancer
Immunome Heads to the FDA With Desmoid Tumor Drug After Phase III Sweep
BioSpace
2025-12-15
pivotal
Varegacestat, a gamma secretase inhibitor, significantly improved progression-free survival while also meeting all key secondary endpoints in the pivotal RINGSIDE trial. Immunome is planning an FDA application for the second quarter of 2026.
Argenyx’s Vyvgart Flops Phase III Thyroid Eye Disease Test, Raising Risk for Ongoing Trials
BioSpace
2025-12-15
myasthenia gravis
Vyvgart, an FcRn inhibitor already approved for generalized myasthenia gravis, is also being tested in myositis, Sjögren’s disease and the “clinically related” Graves disease.
Cannabinoids Halt Ovarian Cancer Growth via Cell Cycle Pathway
Inside Precision Medicine
2025-12-15
ovarian cancer
A combination of CBD and THC has been found to synergistically regulate the PI3K/AKT/mTOR pathway, which is known to drive cancer growth and treatment resistance in ovarian cancer cells. The post Cannabinoids Halt Ovarian Cancer Growth via Cell Cycle Pathway appeared first on Inside Precision Medicine .
FDA Proactively Awards National Priority Voucher Based on Strong Phase 3 Study Results
FDA Press
2025-12-15
multiple myeloma
The U.S. Food and Drug Administration today awarded a national priority voucher to teclistamab in combination with daratumumab for relapsed/refractory multiple myeloma.
CGT Catapult establishes industry safety consortium for cell therapies
PMLive Research
2025-12-15
cell therapies
A universal safety test for CGTs could reduce both costs and wait times for treatment
Fondazione Telethon and Orphan Therapeutics Accelerator Sign Memorandum of Understanding to Pioneer Non-Profit Commercial Access Model for Ultra-Rare Disease Gene Therapy in the US
Fondazione Telethon
2025-12-15
gene therapy
/PRNewswire/ -- Orphan Therapeutics Accelerator (OTXL), a patient-centered non-profit biotech focused on bringing promising clinical-stage treatments for...
Roche receives FDA approval for first diagnostic tests to identify HER2-positive metastatic breast cancer patients eligible for ENHERTU
Roche Group
2025-12-15
her2
breast cancer
FDA expands labels for Roche's PATHWAY HER2 (4B5) test* and VENTANA HER2 Dual ISH DNA Probe Cocktail, which can now be used to identify HER2-positive metastatic breast cancer patients that
VIDEO: Triplet regimen ’paves the road’ for chemotherapy-free DLBCL treatment
Healio
2025-12-15
dlbcl
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
ORLANDO — In this video, Brian T. Hill, MD, PhD, discusses a study presented at ASH Annual Meeting and Exposition which assessed a triplet nonchemotherapy regimen in older and medically unfit patients with diffuse large B-cell lymphoma.In the study, patients with DLBCL who were considered unfit to receive R-CHOP chemotherapy received rituximab (Rituxan; Genentech, Biogen), polatuzumab
FDA backs faster development of new therapy for hard-to-treat AML
Rare Cancer News
2025-12-15
aml
rmat
leukemia
cell therapy
acute myeloid leukemia
The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to SENTI-202, Senti Biosciences‘ experimental cell therapy being developed for adults with hard-to-treat blood cancers, including acute myeloid leukemia (AML). RMAT status aims to speed the development of cellular and engineered therapies that show early potential to address unmet needs in […] The post FDA backs faster development of new therapy for hard-to-treat AML appeared first on Rare Cancer News .
Kyverna to seek first clearance of a CAR-T therapy for autoimmune disease
Biopharma Dive
2025-12-15
car-t
stiff person syndrome
The company intends to file a U.S. application in the first half of 2026 following positive study results in a condition called stiff person syndrome that has no approved therapies.
Immusoft Secures FDA Orphan Drug Designation for ISP-002 in MPS II, a Progressive Lysosomal Storage Disease
Immusoft Corporation
2025-12-15
mps ii
orphan drug
cell therapies
/PRNewswire/ -- Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, today announced that the U.S. Food and Drug...
Fertility-Linked Gene Helps Glioblastoma Cells Resist Chemotherapy
GenEng News
2025-12-15
glioblastoma
A subset of glioblastoma cells appear to survive chemotherapy by turning to an unexpected ally that reshapes their metabolism, the researchers report. The post Fertility-Linked Gene Helps Glioblastoma Cells Resist Chemotherapy appeared first on GEN - Genetic Engineering and Biotechnology News .
AstraZeneca's Enhertu and Genentech's Perjeta combo gains breast cancer approval
Genentech
2025-12-15
her2
breast cancer
The US FDA has approved a combination of AstraZeneca (AZN)/Daiichi Sankyo's (DSKYF) Enhertu and Roche (RHHBY)-owned Genentech's Perjeta (pertuzumab) as a first-line treatment for HER2-positive breast cancer. Approval was based on phase 3 data that showed the combo reduced the risk of disease progression or death by 44% vs. a regimen of a taxane, He...
FDA’s ‘Conundrum’: What To Do With Approved Drugs That Fail Pivotal or Confirmatory Trials
BioSpace
2025-12-15
pivotal
sarepta
With notable therapies from Biogen, Sarepta and MacroGenics failing to show efficacy in pivotal or confirmatory trials, experts question the use of biomarker evidence for approval while one former regulator insists that a “failed trial is not a failed drug.”
In a first, a gene therapy seems to slow Huntington disease
uniQure
2025-12-15
gene therapy
huntington disease
Small study suggests uniQure drug could be first successful treatment for devastating brain disorder
Sanofi MS drug hits two setbacks
Biopharma Dive
2025-12-15
multiple sclerosis
Tolebrutinib, which Sanofi acquired in a $3.7 billion buyout, failed a Phase 3 study in “primary progressive” multiple sclerosis and is facing a delayed U.S. approval decision in another form of the disease.
Sanofi’s multiple sclerosis drug flunks Phase 3 trial, faces another PDUFA delay
Endpoints News
2025-12-15
pdufa
multiple sclerosis
Sanofi reported that its BTK inhibitor which it said has blockbuster potential in multiple sclerosis hit setbacks in two different forms of the disease. The French drugmaker said tolebrutinib failed a ...
VIDEO: Giredestrant improves disease-free survival for early-stage breast cancer
Healio
2025-12-15
her2
breast cancer
SAN ANTONIO — In this video, Erica L. Mayer, MD, MPH, discusses data from the phase 3 lidERA trial demonstrating that giredestrant improved disease-free survival in early-stage, hormone receptor-positive, HER2-negative breast cancer.Results of the trial — which compared giredestrant (Genetech, Roche), an oral selective estrogen receptor degrader, with standard-of-care endocrine
Roche receives FDA approval for first diagnostic tests to identify HER2-positive metastatic breast cancer patients eligible for ENHERTU
Roche Group
2025-12-15
her2
breast cancer
FDA expands labels for Roche's PATHWAY HER2 (4B5) test* and VENTANA HER2 Dual ISH DNA Probe Cocktail, which can now be used to identify HER2-positive...
Kyverna to take autoimmune CAR-T to FDA after success in stiff person syndrome trial
Endpoints News
2025-12-15
car-t
stiff person syndrome
Kyverna Therapeutics' CAR-T therapy succeeded in a key study in stiff person syndrome, a rare autoimmune disorder that leads to painful muscle spasms and stiffness. The California biotech said Monday that ...
Enasidenib Is a Safe and Effective Intervention in IDH2-Mutated AML and MDS
Hematology Advisor News
2025-12-15
aml
Researchers determined Adding enasidenib to venetoclax appears to be safe and effective in patients with IDH2-mutated, relapsed or refractory AML.
Cellectis Announces Arbitral Decision in Dispute with Servier
cellectis
2025-12-15
gene therapies
New York (NY) - December 16, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, a nnounces that the Arbitral Tribunal has issued its decision in the arbitration proceedings against Les Laboratoires Servier and Institut de Recherches Internationales Servier IRIS SARL (“Servier”), relating to the License, Development and Commercialization Agreement entered into between Servier and Cellectis on March 6, 2019, as amended (the “License Agreement”). The Tribunal ruled on a partial termination of the License Agreement with respect to product UCART19 V1 (also referred to as “ALLO-501” by Allogene) and provided that Cellectis shall, at Allogene’s request, engage in good-faith discussions regarding the granting of a direct license to product UCART19 V1. All other claims brought by the parties were dismissed.
Kyverna Therapeutics Announces Positive Topline Data from Registrational KYSA-8 Trial of Miv-cel (KYV-101) in Stiff Person Syndrome
Kyverna Therapeutics
2025-12-15
bla
car t
kyv-101
cell therapy
stiff person syndrome
Landmark results could pave the way for miv-cel to become the first FDA-approved CAR T-cell therapy for autoimmune disease; Company on track to submit BLA for stiff person syndrome in 1H 2026 Miv-cel achieved statistically significant clinical benefit across all primary and secondary endpoints,
VIDEO: Tucatinib plus maintenance therapy delays metastatic breast cancer progression
Healio
2025-12-15
her2
breast cancer
SAN ANTONIO — In this video, Erika Hamilton, MD, discusses HER2CLIMB-05 trial results demonstrating that the addition of tucatinib to maintenance therapy delayed metastatic breast cancer progression for HER2-positive patients.The trial compared tucatinib (Tukysa, Seagen) with placebo in combination with trastuzumab (Herceptin, Genentech) and pertuzumab (Perjeta, Genentech).
Point of Prevention
Inside Precision Medicine
2025-12-15
breast cancer
prostate cancer
colorectal cancer
Although point-of-care is ubiquitous for many conditions, available options for cancer diagnosis or screening are limited but recent advances could soon make an impact. Risk screening for prostate cancer, breast cancer, and colorectal cancer are readily available in the developed world, but are not viable in a POC setting. The post Point of Prevention appeared first on Inside Precision Medicine .
FDA approves monthly PCSK9 inhibitor for high cholesterol
Healio
2025-12-15
familial hypercholesterolemia
The FDA has approved lerodalcibep-liga, a third-generation injectable PCSK9 inhibitor, to reduce LDL cholesterol in adults with hypercholesterolemia, including heterozygous familial hypercholesterolemia, LIB Therapeutics announced.Lerodalcibep-liga (Lerochol) is a once-monthly self-administered single small-volume subcutaneous injection with room temperature stability for up to 3 months, which
AstraZeneca's Enhertu and Genentech's Perjeta combo gains breast cancer approval
Genentech
2025-12-15
her2
breast cancer
The US FDA has approved a combination of AstraZeneca (AZN)/Daiichi Sankyo's (DSKYF) Enhertu and Roche (RHHBY)-owned Genentech's Perjeta (pertuzumab) as a first-line treatment for HER2-positive breast cancer. Approval was based on phase 3 data that showed the combo reduced the risk of disease progression or death by 44% vs. a regimen of a taxane, He...
FDA speeds up review of J&J's multiple myeloma combo treatment following positive data
Endpoints News
2025-12-15
multiple myeloma
Johnson & Johnson's Tecvayli and Darzalex combination treatment scored another win in second-line multiple myeloma, with the FDA moving with unprecedented speed after the latest data to issue a new voucher that expedites the combo' ...
Kyverna rises on stiff person syndrome data
PharmaPhorum
2025-12-15
car-t
stiff person syndrome
Kyverna hopes to file CAR-T therapy miv-cell for approval as the first therapy for stiff person syndrome, a rare and debilitating autoimmune disease.
Mortality risk elevated in patients with Lennox Gastaut, Dravet syndromes
Healio
2025-12-15
dravet syndrome
Mortality rates for Lennox Gastaut syndrome and Dravet syndrome, both developmental epileptic encephalopathies, were significantly higher than other forms of epilepsy, according to a poster at the American Epilepsy Society annual meeting. “Developmental epileptic encephalopathies (DEEs) are among the most severe epilepsies, characterized by drug resistant seizures and significant
Sanofi’s MS Drug Fails Phase III Trial, While FDA Again Delays Approval Application
BioSpace
2025-12-15
multiple sclerosis
Sanofi’s multiple sclerosis hopeful tolebrutinib faced dual setbacks on Monday, with a late-stage failure in one form of the disease and yet another regulatory setback in another.
Supportive care services ease stress, boost quality of life for patients with breast cancer
Healio
2025-12-15
breast cancer
SAN ANTONIO — Delivering essential supportive care services during breast cancer treatment lowered stress and improved quality of life for patients with breast cancer, according to Amy Comander, MD, DipABLM.“A diagnosis of breast cancer is life altering, affecting not only physical health but also generating considerable stress and psychosocial burdens,” Comander, director of
InnoCare says Zenas-partnered drug succeeds in China study in form of lupus
Endpoints News
2025-12-15
lupus
InnoCare Pharma’s experimental oral BTK inhibitor, called orelabrutinib, succeeded in a mid-stage trial in systemic lupus erythematosus (SLE), the Chinese biotech said Sunday. After nearly a year’s worth treatment with the ...
CRISPR and Precision Cancer Therapy Efficacy Influenced by Common Drugs
Inside Precision Medicine
2025-12-15
crispr
Research shows many commonly used drugs can change how human cells repair CRISPR-induced DNA breaks, sometimes boosting precise edits or skewing repair toward errors. It also identifies existing noncancer drugs that could be repurposed to selectively kill tumors with specific DNA repair defects. The post CRISPR and Precision Cancer Therapy Efficacy Influenced by Common Drugs appeared first on Inside Precision Medicine .
In vivo CAR-T exhibits clinical activity, manageable toxicity in advanced multiple myeloma
Healio
2025-12-15
car-t
cell therapy
multiple myeloma
chimeric antigen receptor
ORLANDO — An in vivo chimeric antigen receptor T-cell therapy administered without lymphodepletion exhibited activity among patients with advanced multiple myeloma, according to findings presented at ASH Annual Meeting and Exposition.The first four patients treated with KLN-1010 (Kelonia Therapeutics) achieved early minimal residual disease-negative response by 1 month and responses
Kyverna Therapeutics Announces Proposed Public Offering of Common Stock
Kyverna Therapeutics
2025-12-15
cell therapies
EMERYVILLE, Calif., Dec. 15, 2025 (GLOBE NEWSWIRE) -- Kyverna Therapeutics, Inc. (Nasdaq: KYTX) (“Kyverna”), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, today announced that it has commenced an underwritten public offering
Western genetic medicine makers turn to Chinese investigator-initiated trials
Endpoints News
2025-12-15
cell therapy
Earlier this year, the Belgian cell therapy startup EsoBiotec had only 12 employees and had raised €22 million to develop a cutting-edge infusion that turns a patient's immune cells into destroyers of disease. But despite ...
Anixa Biosciences Announces Transfer of IND for Breast Cancer Vaccine from Cleveland Clinic
anixa biosciences
2025-12-15
ind
car-t
breast cancer
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
SAN JOSE, Calif. , Dec. 15, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that it has completed the transfer of the Investigational New Drug application ("IND") for its breast cancer vaccine from Cleveland Clinic. Anixa is now the trial sponsor for future development of its breast cancer vaccine. The transfer of the IND is a natural and planned step in the progresson of the vaccine's development. With enrollment completed and encouraging immune response and safety data observed in the Phase 1 trial, Anixa plans to advance the vaccine into a Phase 2 clinical trial and has assumed full sponsorship of the IND. Anixa plans to utilize multiple clinical sites, including Cleveland Clinic, for the Phase 2 and other clinical trials. Anixa's breast cancer vaccine, developed in collaboration with Cleveland Clinic, targets α-lactalbumin—a lactation-associated protein that is typically expressed only in breast tissue during lactation, but which re-emerges in many forms of breast cancer. By establising an immune response against α-lactalbumin-expressing cells, the…
Non-profit deal to bring US commercial access to gene therapy
Fondazione Telethon
2025-12-15
gene therapy
Italian non-profit rare and complex genetic diseases specialist Fondazione Telethon (FT) and Orphan Therapeutics Accelerator (OTXL), a non-profit biotech focused on bringing promising clinical-stage treatments for ultra-rare conditions to patients, have announced the signing of a Memorandum of Understanding (MoU).
Enhertu combo wins FDA approval for first-line use in metastatic breast cancer
Endpoints News
2025-12-15
breast cancer
J&J's run of talc lawsuit setbacks continues
PharmaPhorum
2025-12-15
ovarian cancer
A California jury has awarded $40m in damages to two women who sued Johnson & Johnson, claiming that its talc products caused ovarian cancer.
8-K - Kyverna Therapeutics, Inc. (0001994702) (Filer)
SEC Filing (8-K)
2025-12-15
kyverna therapeutics
Filed: 2025-12-15 AccNo: 0001193125-25-318469 Size: 10 MB Item 7.01: Regulation FD Disclosure Item 9.01: Financial Statements and Exhibits
Sanofi provides update on tolebrutinib regulatory submission in non-relapsing secondary progressive multiple sclerosis
sanofi
2025-12-15
multiple sclerosis
• Ongoing discussions with the FDA regarding nrSPMS application have indicated regulatory decision for tolebrutinib is anticipated to be delayed
Sanofi gets double blow for MS drug tolebrutinib
PharmaPhorum
2025-12-15
multiple sclerosis
Shares in Sanofi have slid on a double dose of bad news for its oral BTK inhibitor tolebrutinib, in development as a therapy for multiple sclerosis.
FDA Approves Itvisma, a New Intrathecal Route for Novartis’ SMA Gene Therapy
CGTLive
2025-12-14
sma
gene therapy
Sarepta's Elevidys Gets Boxed Warning for ALI and ALF
CGTLive
2025-12-14
sarepta
elevidys
The FDA also added a new Warnings & Precaution for heightened susceptibility to serious infections caused by immunosuppression.
Around the Helix: Cell and Gene Therapy Company Updates – November 26, 2025
CGTLive
2025-12-14
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Should BioMarin’s Cost Cuts, Roctavian Exit, and Pipeline Shifts Require Action From BMRN Investors?
BioMarin Pharmaceutical
2025-12-14
roctavian
Earlier this month, BioMarin Pharmaceutical’s CEO reported that the company’s US$500 million cost transformation program is about two-thirds complete and reiterated an ambition to reach a 40% non-GAAP operating margin next year, while also reshaping the portfolio through workforce reductions and discontinuation of the Roctavian program. At the same time, analysts have raised concerns about rising competition in achondroplasia and potential gaps in BioMarin’s pipeline, even as they...
Kyverna Therapeutics to Report Topline Results from Registrational Phase 2 KYSA-8 Trial of KYV-101 in Stiff Person Syndrome
Kyverna Therapeutics
2025-12-14
kyv-101
cell therapies
stiff person syndrome
– Company to host live webcast and conference call Monday, December 15, 2025 at 8:00 am ET – EMERYVILLE, Calif., Dec. 14, 2025 (GLOBE NEWSWIRE) -- Kyverna Therapeutics, Inc. (Nasdaq: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune
Should BioMarin’s Cost Cuts, Roctavian Exit, and Pipeline Shifts Require Action From BMRN Investors?
BioMarin Pharmaceutical
2025-12-14
roctavian
U.S. stocks, Saudi stocks, stock trading and investment platforms
Is Earlier Yescarta Use in Lymphoma Altering The Investment Case For Gilead Sciences (GILD)?
Gilead Sciences
2025-12-13
car t
yescarta
b-cell lymphoma
large b-cell lymphoma
Kite, a Gilead Sciences company, recently reported new analysis showing that its CAR T therapy Yescarta delivers consistent benefits as a second-line treatment for patients with relapsed or refractory large B-cell lymphoma, including those previously considered ineligible for standard high-dose chemotherapy and stem cell transplant. This finding could encourage oncologists to use Yescarta earlier in the treatment pathway, potentially strengthening Gilead’s position in the fast-growing cell...
Gilead (GILD) Valuation Check as New Yescarta Data Reinforces CAR T Role in Second-Line Lymphoma
Gilead Sciences
2025-12-13
car t
yescarta
Kite, a Gilead Sciences (GILD) company, just released new analysis showing its CAR T therapy Yescarta delivers consistent benefits as a second line option for difficult large B cell lymphoma cases. See our latest analysis for Gilead Sciences. That stronger oncology story is quietly reshaping how investors see Gilead, with the share price at $120.4 after a softer recent patch but a robust year to date share price return of 31.04 percent, backed by a 1 year total shareholder return of 33.86...
Bladder Cancer Gene Therapy Detalimogene Effects 62% CRR at 6 Months Posttreatment
CGTLive
2025-12-13
phase 1/2
gene therapy
bladder cancer
This finding came from an analysis of 37 patients treated in the phase 1/2 LEGEND trial.
Notice of Investigation of QURE: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2025-12-13
bla
amt-130
gene therapy
RADNOR, PA - December 13, 2025 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
VIDEO: Pirtobrutinib ‘on the map’ as first-line, second-line CLL, SLL option
Healio
2025-12-12
cll
leukemia
small lymphocytic lymphoma
chronic lymphocytic leukemia
ORLANDO — In this video, Shazia K. Nakhoda, MD, discussed “exciting data” from the BRUIN CLL-314 and BRUIN CLL-313 trials, which assessed pirtobrutinib in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma.In the BRUIN CLL-313 trial, researchers compared pirtobrutinib (Jaypirca, Eli Lilly & Co.) vs. bendamustine plus rituximab (Rituxan; Genentech,
P&F secures FDA approval for TRICAV II pivotal clinical trial for severe tricuspid regurgitation
PR Newswire
2025-12-12
pivotal
TRICAV II will evaluate the safety and effectiveness of the TricValve® Transcatheter Bicaval Valve System WILMINGTON, Del., Dec. 12, 2025 /PRNewswire/ -- P&F USA, Inc., the U.S. subsidiary of heart valve manufacturer P&F Products and Features GmbH, today announced that the U.S. Food and...
VIDEO: Ixo-vec gene therapy shows safe results throughout 4-year study period
Healio
2025-12-12
ixo-vec
gene therapy
ixoberogene soroparvovec
ORLANDO — In this video from the American Academy of Ophthalmology meeting, Michael W. Stewart, MD, professor and chairman of ophthalmology at the Mayo Clinic, talks about findings from the phase 2 LUNA trial. Stewart told Healio that Ixo-vec (ixoberogene soroparvovec, Adverum Biotechnologies) in neovascular age-related macular degeneration “showed excellent safety signals” throughout the 4-year
Socioeconomic Factors, Comorbidities Tied to Late-Onset Depression in Long-Term Cancer Survivors
Hematology Advisor News
2025-12-12
colorectal cancer
Researchers aimed to identify risk factors for late-onset depression in 5-year survivors of breast, prostate, and colorectal cancer.
SGLT2 inhibitors may offer ‘survival benefits’ for patients with lupus, diabetes
Healio
2025-12-12
lupus
CHICAGO — Patients with systemic lupus erythematosus and diabetes who received sodium-glucose cotransporter 2 inhibitors exhibited decreased mortality rates but increased rates of nephritis, according to a speaker at ACR Convergence 2025.“SLE increases the risk for cardiovascular disease, chronic kidney involvement and metabolic complications; and diabetes further amplifies these
NanOlogy Unveils Drug Development Program for Rare Pediatric Brainstem Tumor
PR Newswire
2025-12-12
glioma
diffuse intrinsic pontine glioma
Next Generation Intratumoral Technology Targets Diffuse Intrinsic Pontine Glioma Investigational New Drug Enabling Studies are Underway with Planned Clinical Trial to Begin Late 2026 FORT WORTH, Texas, Dec. 11, 2025 /PRNewswire/ -- NanOlogy, LLC, a private clinical-stage oncology company,...
Actinium Pharmaceuticals Presents New Preclinical Data Demonstrating Potent Anti-Tumor Activity of ATNM-400 Across Multiple Breast Cancer Subtypes Including Hormone Receptor-Positive, Triple-Negative, and Tamoxifen- and HER2 Therapy-Resistant Breast Cancer Models at SABCS 2025
Actinium Pharma
2025-12-12
her2
lung cancer
solid tumor
breast cancer
prostate cancer
ATNM-400's target antigen is overexpressed in breast cancer and its expression is further increased in breast cancer cells resistant to standard of care endocrine therapy, tamoxifen and the HER2 therapy trastuzumab - The Actinium-225 alpha-emitter payload of ATNM-400 induced irreversible double-strand DNA breaks and has the potential to produce potent localized tumor killing with reduced off-target lung toxicity that limits the use of antibody drug conjugates - Data supports the continued development of ATNM-400 as a monotherapy or combination therapy to address multiple breast cancer subtypes with limited treatment options NEW YORK , Dec. 12, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE American: ATNM), a leader in the development of differentiated targeted radiotherapies, today announced the presentation of new preclinical data for ATNM-400, its first-in-class Actinium-225 (Ac-225) based antibody radioconjugate, at the 2025 San Antonio Breast Cancer Symposium (SABCS). The data highlight robust anti-tumor activity and a favorable tolerability profile across a broad panel of preclinical breast cancer models, including hormone receptor–positive (HR+), triple-negative…
QURE Investigation: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2025-12-12
bla
amt-130
gene therapy
RADNOR, PA - December 12, 2025 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Menopausal hormone therapy does not raise breast cancer risk for women with BRCA mutations
Healio
2025-12-12
breast cancer
SAN ANTONIO — Menopausal hormone therapy did not elevate risk for breast cancer among women with inherited BRCA mutations, according to observational study results presented at San Antonio Breast Cancer Symposium.Risk did not vary based on the type of hormone therapy formulation used, nor whether women had BRCA1 or BRCA2 mutations.
Tenaya targets $60M offering after heart disease pipeline updates
Endpoints News
2025-12-12
gene therapies
Tenaya Therapeutics, a company focused on developing gene therapies for heart conditions, made a series of announcements on Thursday. The biotech said the FDA has lifted a partial hold on one of its assets, reported ...
ImmunityBio Receives Conditional Marketing Authorization Recommendation from the European Medicines Agency for ANKTIVA® with BCG for Non-Muscle Invasive Bladder Cancer Carcinoma in Situ—A First in Europe
Immunity Bio
2025-12-12
bladder cancer
The post ImmunityBio Receives Conditional Marketing Authorization Recommendation from the European Medicines Agency for ANKTIVA® with BCG for Non-Muscle Invasive Bladder Cancer Carcinoma in Situ—A First in Europe appeared first on ImmunityBio .
‘Give this a try’: Digital toolkit aims to improve risk-informed breast cancer screening
Healio
2025-12-12
breast cancer
SAN ANTONIO — A digital toolkit with patient scripts, risk calculator comparisons and more could help primary care and OB/GYN providers increase risk-informed breast cancer screening.Reviewers of the toolkit said it had high usability, demonstrated clinical relevance, improved communication and could help with decision making, according to Amanda M. Woodworth, MD, FACS, FSSO, director of
Only 6% of FDA Pivotal Trials Reflect U.S. Racial Makeup
Inside Precision Medicine
2025-12-12
pivotal
There is a trend of declining enrollment of Black and Hispanic participants beginning in 2021 due to structural features of where and how trials are conducted. The post Only 6% of FDA Pivotal Trials Reflect U.S. Racial Makeup appeared first on Inside Precision Medicine .
Updated data for Lilly's Inluriyo™ (imlunestrant) reinforce efficacy results as monotherapy and in combination with Verzenio® (abemaciclib) in ER+, HER2- advanced breast cancer
PR Newswire
2025-12-12
her2
breast cancer
As monotherapy, imlunestrant demonstrated an 11.4-month numeric improvement in median OS versus endocrine therapy in patients with ESR1-mutated disease In all patients, the imlunestrant plus abemaciclib combination achieved a median PFS of 10.9 months, demonstrated a favorable OS trend,...
Preventive TNBC Vaccine Triggers Immune Responses in Phase I Study
Inside Precision Medicine
2025-12-12
breast cancer
A Phase I clinical study found that an investigational vaccine targeting α-lactalbumin was safe and induced immune responses in a majority of participants at high risk for triple-negative breast cancer, informing the design of a planned Phase II trial. The post Preventive TNBC Vaccine Triggers Immune Responses in Phase I Study appeared first on Inside Precision Medicine .
‘Scalable’ digital health tool improves quality of life for young women with breast cancer
Healio
2025-12-12
breast cancer
SAN ANTONIO — A mobile health tool used to serially monitor patients through patient-reported outcomes could significantly improve quality of life for adolescent and young adult women with breast cancer.The data, presented at San Antonio Breast Cancer Symposium, showed the Young, Empowered & Strong (YES) intervention significantly improved general and cancer-specific quality of life
Menopausal Hormone Therapy May Not Increase Cancer Risk in BRCA1/2 Carriers
Inside Precision Medicine
2025-12-12
breast cancer
Women with BRCA1/2 genetic variants do not have higher risks for breast cancer after taking hormone replacement therapy during menopause, suggest results from a new study. The post Menopausal Hormone Therapy May Not Increase Cancer Risk in BRCA1/2 Carriers appeared first on Inside Precision Medicine .
Michelle Alabek, MS, CGC, on the Role of Genetic Counselors in Supporting Patients Receiving Gene Therapy
CGTLive
2025-12-12
gene therapy
ImmunityBio Receives Conditional Marketing Authorization Recommendation from the European Medicines Agency for ANKTIVA® with BCG for Non-Muscle Invasive Bladder Cancer Carcinoma in Situ—A First in Europe
Immunity Bio
2025-12-12
bladder cancer
The post ImmunityBio Receives Conditional Marketing Authorization Recommendation from the European Medicines Agency for ANKTIVA® with BCG for Non-Muscle Invasive Bladder Cancer Carcinoma in Situ—A First in Europe appeared first on ImmunityBio .
Abbott increases quarterly dividend for 54th consecutive year
PR Newswire
2025-12-12
quarterly
Quarterly dividend increased by 6.8% Quarterly dividend payout has increased more than 70% since 2020 408th consecutive quarterly dividend to be paid by Abbott since 1924 ABBOTT PARK, Ill., Dec. 12, 2025 /PRNewswire/ -- Abbott (NYSE: ABT) today announced that its board of directors has...
‘This works’: Acupuncture improves cognitive impairment from breast cancer treatment
Healio
2025-12-12
breast cancer
SAN ANTONIO — Acupuncture conferred significant, clinically meaningful improvements in both perceived and objective cognitive function for breast cancer survivors, according to findings from a randomized phase 2 trial.The data, presented at San Antonio Breast Cancer Symposium, showed the process of receiving acupuncture, not the needling itself, improved a patient’s subjective
FDA approves berotralstat for children aged 2 to 12 years with hereditary angioedema
Healio
2025-12-12
hereditary angioedema
The FDA has approved berotralstat oral pellets for prophylactic use in patients aged 2 to than 12 years with hereditary angioedema, according to a press release from BioCryst Pharmaceuticals.“Until now, the only treatment options for patients under 12 years of age, whether long-term prophylaxis or on-demand therapy, were delivered intravenously or through subcutaneous injection,”
Carrie Haverty, MS, CGC, on Exploring Genetic Counseling's Growth and Challenges
CGTLive
2025-12-12
gene therapy
The NSGC president-elect spoke on the evolving role of genetic counselors, emphasizing personalized care, gene therapy advancements, and the need for multidisciplinary integration in healthcare.
Actinium Pharmaceuticals Presents New Preclinical Data Demonstrating Potent Anti-Tumor Activity of ATNM-400 Across Multiple Breast Cancer Subtypes Including Hormone Receptor-Positive, Triple-Negative, and Tamoxifen- and HER2 Therapy-Resistant Breast Cancer Models at SABCS 2025
PR Newswire
2025-12-12
her2
breast cancer
ATNM-400's target antigen is overexpressed in breast cancer and its expression is further increased in breast cancer cells resistant to standard of care endocrine therapy, tamoxifen and the HER2 therapy trastuzumab - The Actinium-225 alpha-emitter payload of ATNM-400 induced...
#SABCS25: Celcuity further details Phase 3 win; Relay’s PI3Kα inhibitor’s consistent performance; and more
Endpoints News
2025-12-12
breast cancer
Roche and BioNTech stole the show at this year’s San Antonio Breast Cancer Symposium. The Swiss drugmaker unveiled Phase 3 data for its oral SERD giredestrant, showing the drug slashed the risk ...
Pancreatic Cancer Survival Gains Accelerate as Novel Therapies Advance
PR Newswire
2025-12-12
pancreatic cancer
Issued on behalf of Oncolytics Biotech Inc. VANCOUVER, BC, Dec. 12, 2025 /PRNewswire/ -- USANewsGroup.com News Commentary – Scientists developed an antibody treatment that reawakens the immune system to attack pancreatic tumors by blocking sugar-coated disguises cancer cells use to evade...
VIDEO: Pirtobrutinib promising as monotherapy, part of combination regimens in CLL
Healio
2025-12-12
cll
leukemia
chronic lymphocytic leukemia
ORLANDO — In this video, Shazia K. Nakhoda, MD, discusses a study that showed time-limited triple combination therapy with pirtobrutinib yielded ‘favorable’ results in patients with chronic lymphocytic leukemia. In the study presented at the ASH Annual Meeting and Exposition, patients with CLL received a combination of pirtobrutinib (Jaypirca, Eli Lilly & Co.) with
‘Broader and more timely’ biomarker testing needed in advanced HER2-negative breast cancer
Healio
2025-12-12
her2
breast cancer
SAN ANTONIO — More than one-third of U.S. adults diagnosed with metastatic HER2-negative breast cancer in 2023 did not undergo testing for BRCA mutations, according to a retrospective analysis.Less than half underwent testing for three other potentially actionable mutations, findings presented at San Antonio Breast Cancer Symposium showed.
FDA approves Waskyra as first gene therapy for Wiskott-Aldrich syndrome
Fondazione Telethon
2025-12-12
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
Waskyra becomes the first FDA-approved gene therapy for Wiskott-Aldrich syndrome, offering hope to patients with this rare immunodeficiency.
Amgen Scores Uplizna Expansion Into Generalized Myasthenia Gravis
BioSpace
2025-12-12
cd19
myasthenia gravis
Generalized myasthenia gravis is Uplizna’s second new indication this year, after the FDA cleared the anti-CD19 antibody for IgG4-related disease in April.
Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing saRNA Therapeutic for Duchenne Muscular Dystrophy
PR Newswire
2025-12-12
duchenne muscular dystrophy
NANTONG, China, Dec. 12, 2025 /PRNewswire/ -- Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the successful dosing of the first patient in its investigator-initiated trial (IIT) of RAG-18 at Peking Union Medical College Hospital...
CRISPR Streamlines Goldenberry Growth to Enable Large-Scale Farming
GenEng News
2025-12-12
crispr
High-quality genome assemblies and CRISPR editing were applied to the goldenberry to produce compact, more tractable plants that advance domestication efforts and support this underutilized species’ potential as a scalable global crop. The post CRISPR Streamlines Goldenberry Growth to Enable Large-Scale Farming appeared first on GEN - Genetic Engineering and Biotechnology News .
Safe to consider bypassing sentinel lymph node biopsy in early-stage breast cancer
Healio
2025-12-12
her2
breast cancer
SAN ANTONIO — “Is omission of the sentinel lymph node biopsy safe in early breast cancer in patients treated with breast conserving therapy and whole-breast radiotherapy?”Bypassing sentinel lymph node biopsy in patients with clinically node-negative, hormone receptor-positive, HER2-negative early-stage breast cancer did not increase regional recurrence or survival after a median
FDA approves Uplizna for generalized myasthenia gravis
Healio
2025-12-12
myasthenia gravis
The FDA has approved inebilizumab-cdon (Uplizna, Amgen) for adults with anti-acetylcholine receptor and anti-muscle specific tyrosine kinase antibody positive generalized myasthenia gravis, according to a press release.The approval came in the wake of the phase 3, double-blind, randomized, placebo-controlled MINT trial, published by Richard Nowak, MD, MS, director of the Myasthenia Gravis Clinic
ISHI Health Launches Virtual Heart Failure Clinics in California and Arizona
PR Newswire
2025-12-12
heart failure
SAN DIEGO, Dec. 12, 2025 /PRNewswire/ -- ISHI Health, an innovator in AI-enabled virtual cardiac care, today announced the launch of its Virtual Heart Failure (HF) Clinics in California and Arizona. The program expands access to HF specialist-driven, guideline-aligned care by partnering...
Sara M. Tolaney, MD, MPH, to receive Outstanding Investigator Award for Breast Cancer Research
Healio
2025-12-11
breast cancer
Sara M. Tolaney, MD, MPH, will receive the 2025 American Association for Cancer Research’s Outstanding Investigator Award for Breast Cancer Research, given in partnership with Breast Cancer Research Foundation.Tolaney, chief of the division of breast oncology at Dana-Farber Cancer Institute, associate professor of medicine at Harvard Medical School and a member of Healio’s Women
Approval of Roche/Genentech’s Gazyvaro Set to Reshape the EU5 Lupus Nephritis Treatment Landscape, According to Spherix Global Insights
Genentech
2025-12-11
lupus
Strong physician readiness, high efficacy expectations, and anticipated displacement of off-label rituximab position Gazyvaro for rapid adoption across EuropeEXTON, PA, Dec. 11, 2025 (GLOBE NEWSWIRE) -- Following this week’s European Commission approval of Roche/Genentech’s Gazyvaro (obinutuzumab) for the treatment of lupus nephritis (LN), Spherix Global Insights’ latest RealTime Dynamix™: Lupus Nephritis (EU) study indicates that the newly approved biologic is positioned to drive significant th
Pancreatic Cancer Drug Treatments Improved by Targeting Collagen Signaling
Inside Precision Medicine
2025-12-11
pancreatic cancer
A new method inhibiting discoidin domain receptor 1 (DDR1), a collagen-activated receptor that helps fuels fibrosis, reduces collagen I production and increases the permeability of fibrotic tumor tissue to macromolecular drugs. The post Pancreatic Cancer Drug Treatments Improved by Targeting Collagen Signaling appeared first on Inside Precision Medicine .
Roche’s Oral SERD Cuts Death Risk by 30% in Early-Stage Breast Cancer
BioSpace
2025-12-11
breast cancer
While overall survival remains immature, results so far show a clear trend in favor of Roche’s giredestrant.
Olema Pharmaceuticals (OLMA): Reassessing Valuation After Roche’s Phase 3 SERD Success Boosts Palazestrant Expectations
Roche Group
2025-12-11
breast cancer
Roche’s upbeat Phase 3 data for its breast cancer SERD giredestrant has lit a fire under Olema Pharmaceuticals (OLMA), with investors recalibrating expectations around the company’s own SERD candidate, palazestrant. See our latest analysis for Olema Pharmaceuticals. The latest pop, including a 15.43% 1 day share price return and a 252.64% 30 day share price return, builds on a powerful backdrop of a 450.81% year to date share price return and a 1152.24% three year total shareholder return...
Roivant Highlights Continued Pipeline Progress and Outlook for Company’s Next Phase of Growth at 2025 Investor Day
roivant
2025-12-11
bla
pivotal
Roivant enters new phase of corporate journey with 3+ launches, 4+ NDA/BLA filings, 8+ pivotal and 3+ proof-of-concept study readouts expected over the next three years Highlighting clinical execution, Roivant announces positive updates to timing guidance across four key programs: Brepocitinib NDA
Preoperative radiation strategy heightens immune response in breast cancer subset
Healio
2025-12-11
her2
breast cancer
SAN ANTONIO — Preoperative radiation improved T-cell infiltration among patients with hormone receptor-positive, HER2-negative breast cancer when administered in combination with pembrolizumab and chemotherapy, according to study results.Findings from the randomized, phase 2 P-RAD clinical trial, presented at San Antonio Breast Cancer Symposium, also showed preoperative radiation improved
8-K - Sarepta Therapeutics, Inc. (0000873303) (Filer)
SEC Filing (8-K)
2025-12-11
sarepta therapeutics
Filed: 2025-12-11 AccNo: 0001193125-25-315032 Size: 166 KB Item 3.02: Unregistered Sales of Equity Securities Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
Mucopolysaccharidosis Type I Market Dynamics Indicate Upward Trajectory Through 2034, Reports DelveInsight | BioMarin Pharma, Sanofi, Orchard Therapeutics, Kyowa Kirin, JCR Pharma, Nippon Shinyaku
BioMarin Pharmaceutical
2025-12-11
regenxbio
biomarin pharmaceutical
mucopolysaccharidosis type i
The Key Mucopolysaccharidosis Type I Companies in the market include BioMarin Pharmaceutical Sanofi Orchard Therapeutics Kyowa Kirin JCR Pharmaceuticals Nippon Shinyaku Immusoft Genevector Biotechnology EdiGene Inc REGENXBIO Sigilon Therapeutics Paradigm Biopharma Jupiter Neurosciences and others DelveInsight s Mucopolysaccharidosis Type I ...
Next-Gen CAR T Cells Destroy Cancer Cells with Fewer Toxic Effects in Mice
GenEng News
2025-12-11
car t
Researchers developed a new generation of CAR T cells that effectively kill cancer cells in animal models, including those that typically escape detection, while causing fewer toxic side effects and persisting for longer. The post Next-Gen CAR T Cells Destroy Cancer Cells with Fewer Toxic Effects in Mice appeared first on GEN - Genetic Engineering and Biotechnology News .
Will Yescarta’s Stronger Second-Line Lymphoma Role Change Gilead Sciences’ (GILD) Cell Therapy Narrative?
Gilead Sciences
2025-12-11
yescarta
cell therapy
b-cell lymphoma
large b-cell lymphoma
Kite, a Gilead company, recently presented new analyses showing that Yescarta delivers durable efficacy and a consistent safety profile as a one-time second-line treatment for relapsed or refractory large B-cell lymphoma, including patients who were previously ineligible for intensive chemotherapy and stem cell transplant. By strengthening the case for Yescarta as a standard second-line option, these data highlight how Gilead’s cell therapy portfolio could shape future cancer care...
The PHERGuide study demonstrates the utility of circulating tumor DNA for the early detection and real-time monitoring of localized HER2+ breast cancer
PR Newswire
2025-12-11
her2
breast cancer
The results of the PHERGuide study, presented during the 2025 San Antonio Breast Cancer Symposium (SABCS), demonstrate that the presence of circulating tumor DNA (ctDNA) is associated with disease stage. This research paves the way for the use of blood-based ctDNA as a non-invasive tool...
Tenaya Therapeutics Announces Rapid Resolution and Lifting of Clinical Hold for MyPEAK-1™ Phase 1b/2a Clinical Trial of TN-201 Gene Therapy
Tenaya Therapeutics
2025-12-11
gene therapy
clinical hold
MyPEAK-1 Protocol Amendments Agreed Upon with FDA; Tenaya Implementing Changes with Sites SOUTH SAN FRANCISCO, Calif., Dec. 11, 2025 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially
Pfizer Builds Case for Tukysa as ‘New Standard Regimen’ in First-Line Metastatic Breast Cancer
BioSpace
2025-12-11
breast cancer
While new late-stage data point to some liver toxicity signals, analysts at BMO Capital Markets said Tukysa’s efficacy outcomes “appear to more than make up for any safety concerns.”
Lilly's obesity triple pulls off 29% weight loss in trial
PharmaPhorum
2025-12-11
pivotal
Eli Lilly has ramped up the competition in weight-loss therapies by reporting the first pivotal data with its triple therapy retatrutide.
Gilead Foundation Awards Over $3 Million to Support People Living With Metastatic Breast Cancer
Gilead Sciences
2025-12-11
breast cancer
Gilead Foundation Awards Over $3 Million to Support People Living With Metastatic Breast Cancer / Breast Cancer, cancer, foundation, Gilead Foundation,
AC Immune says Parkinson's drug slows progression
PharmaPhorum
2025-12-11
parkinson's disease
AC Immune's active immunotherapy study backs up the alpha-synuclein hypothesis in Parkinson's disease, after disappointments in earlier trials.
VIDEO: Fixed-duration therapy overcomes long-term side effects, financial toxicity in CLL
Healio
2025-12-11
cll
leukemia
chronic lymphocytic leukemia
ORLANDO — In this video, Shazia K. Nakhoda, MD, discusses “exciting” data from the CLL-17 trial that showed fixed-duration therapy produces similar results inpreviously untreated chronic lymphocytic leukemia.Results presented at ASH Annual Meeting and Exposition showed similar 3-year progression-free survival rates between continuous Bruton tyrosine kinase (BTK) inhibitor
Roche Completes Acquisition of Cell Therapy Pioneer Poseida Therapeutics
Roche Group
2025-12-11
cell therapy
Poseida Therapeutics US73730P1084
Prolynx banks $70M for longer-lasting obesity drugs
Biopharma Dive
2025-12-11
quarterly
Using a kind of novel linker technology, the startup is developing versions of incretin and non-incretin therapies that can be administered monthly or quarterly.
Umoja delays first readout for in vivo CAR-T therapy
Endpoints News
2025-12-11
car-t
Umoja Biopharma, one of the most well-capitalized developers of in vivo CAR-T therapy, is delaying its first clinical readout from its China-based first trial. The startup told Endpoints News that it now expects to share ...
Genentech’s Giredestrant Reduced Risk of Invasive Disease Recurrence or Death by 30% in ER-positive Early-Stage Breast Cancer
Genentech
2025-12-11
breast cancer
Immatics Presents IMA203CD8 PRAME Cell Therapy Data from Ongoing Dose Escalation and Shows Promising Initial Anti-tumor Activity in PRAME-Positive Tumors at ESMO-IO 2025 Congress
Immatics Biotechnologies
2025-12-11
cell therapy
IMA203CD8 is a second-generation PRAME cell therapy with enhanced pharmacology in ongoing Phase 1a dose escalation Manageable tolerability across all dose levels Encouraging early clinical anti-tumor activity in advanced solid tumors after one-time infusion of IMA203CD8 at a low median dose during
Preoperative MRI offers ‘no benefit’ for recurrence in early HR-negative breast cancer
Healio
2025-12-11
breast cancer
SAN ANTONIO — Patients with stage I or II hormone receptor-negative breast cancer do not benefit from preoperative breast MRI, according to data from San Antonio Breast Cancer Symposium.Given the comparable 5-year locoregional recurrence rates between those who underwent breast MRI in addition to diagnostic mammography and those who did not, researchers noted that “clinical benefit of
Sacituzumab govitecan falls short on survival gains in refractory breast cancer subset
Healio
2025-12-11
her2
breast cancer
SAN ANTONIO — Sacituzumab govitecan failed to extend PFS in patients with hormone receptor-positive, HER2-negative breast cancer following endocrine therapy, according to data from San Antonio Breast Cancer Symposium.Researchers did note a potential early OS benefit with sacituzumab govitecan (Trodelvy, Gilead Sciences) at the 15-month mark, but emphasized that this observation is based on
GLP-1s may reduce risk for cataracts, dry AMD
Healio
2025-12-11
dry age-related macular degeneration
GLP-1 receptor agonists are not associated with increased risks for dry age-related macular degeneration or cataract development, according to two recently published studies. The data instead showed significantly reduced risk for cataracts, as well as lower risk for dry AMD, linked with the use of GLP-1s, according to Abhimanyu Ahuja, MD, an ophthalmology resident at the Oregon Health &
Pfizer makes first-line case for Tukysa in breast cancer
PharmaPhorum
2025-12-11
her2
breast cancer
Pfizer's Tukysa looks poised to find a new role as first-line maintenance therapy for HER2-positive metastatic breast cancer.
Sarepta Therapeutics Announces Refinancing of Approximately $291 Million of 1.25% Convertible Senior Notes due 2027
Sarepta Therapeutics
2025-12-11
sarepta
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Dec. 11, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has entered into separate, privately negotiated exchange agreements with a limited number of holders of its 1.25%
Abeona Therapeutics® Announces New Qualified Treatment Center for ZEVASKYN® in Texas
Abeona Therapeutics
2025-12-11
zevaskyn
Roche’s Oral Breast Cancer Drug Cuts Recurrence Risk By 30% In Trial
Roche Group
2025-12-11
breast cancer
Roche’s oral therapy, giredestrant, has demonstrated a 30 per cent reduction in the risk of breast cancer recurrence compared with current standard endocrine treatments, as per report by reuters. The findings, from the Phase III lidERA trial, mark what the company describes as the most significant leap in hormonal therapy for breast cancer in more than two decades. According to the detailed data, after three years 92.4 per cent of patients treated with giredestrant remained alive and disease-free, compared with 89.6 per cent of those receiving standard care, reports said. A preliminary summary released last month had confirmed that the study met its primary endpoints, but the newly shared results provide a more comprehensive view of the drug’s impact. Levi Garraway, Roche’s Chief Medical Officer highlighted that when you talk about a 30 per cent increase in disease-free survival, you're basically saying that among patients who would have otherwise experienced a recurrence on standard therapy, you are seeing 30 per cent fewer recurrences. The results target a major unmet need in estrogen receptor–positive (ER-positive) breast cancer, which represents roughly 70 per cent of all…
Tenaya Therapeutics Reports Positive Interim Data from Cohort 1 of RIDGE™-1 Phase 1b/2 Clinical Trial of TN-401 Gene Therapy for Adults with PKP2-associated ARVC
Tenaya Therapeutics
2025-12-11
gene therapy
TN-401 was Well Tolerated at 3E13 vg/kg dose Robust Transduction and Demonstrated Increases in PKP2 Protein Levels in First Two Patients at Week 8 Clinically Meaningful Reductions in Arrhythmia Burden Observed in First Two Patients with More Than Six Months of Follow-Up Tenaya Management to Host a
Guess the Therapy Answer December 10, 2025
CGTLive
2025-12-11
gene therapy
Cell and Gene Therapy Live
FDA Flexibility Leads to First Approved Gene Therapy for Rare Blood & Immune System Disorder
Fondazione Telethon
2025-12-11
pivotal
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
Waskyra is the first FDA-approved gene therapy for the rare disease Wiskott-Aldrich syndrome and the first such approved product from a non-profit applicant, Fondazione Telethon. Data from Waskyra’s pivotal studies were presented this week during the annual meeting of the American Society of Hematology.
Approval of Roche/Genentech’s Gazyvaro Set to Reshape the EU5 Lupus Nephritis Treatment Landscape, According to Spherix Global Insights
Genentech
2025-12-11
lupus
Strong physician readiness, high efficacy expectations, and anticipated displacement of off-label rituximab position Gazyvaro for rapid adoption across...
Wobble Genomics To Present New Data At SABCS Confirming Platform's Ability To Determine Detailed Profiling of HER2 Status in Tissue and Blood
PR Newswire
2025-12-11
her2
breast cancer
EDINBURGH, Scotland, Dec. 11, 2025 /PRNewswire/ -- Wobble Genomics, a biotechnology company focused on improving cancer outcomes through better diagnosis and therapy selection, will present new data at the 2025 San Antonio Breast Cancer Symposium (SABCS), taking place from December 9-12...
Anixa Biosciences Announces Positive Phase 1 Data for Investigational Breast Cancer Vaccine; Primary Endpoints Were Met and Immune Response Observed in 74% of Participants
anixa biosciences
2025-12-11
breast cancer
Vaccine Was Safe and Well Tolerated at the Maximum Tolerated Dose Results Support Advancement of the Program into Phase 2 Development Combination of Keytruda ® and the vaccine generated T cell responses and showed no major additional side effects, supporting plans for a Phase 2 neoadjuvant combination study in newly diagnosed breast cancer patients SAN JOSE, Calif. , Dec. 11, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced the presentation of final data from the Phase 1 clinical trial of its investigational breast cancer vaccine ( NCT04674306 ) at the 2025 San Antonio Breast Cancer Symposium (SABCS). The trial was conducted in collaboration with Cleveland Clinic and funded by a grant from the U.S. Department of Defense. Final Phase 1 findings showed the investigational vaccine met all major primary endpoints, was safe and well tolerated at the maximum tolerated dose (MTD), and elicited protocol-defined immune responses in 74% of participants. The presentation, titled "Final Results of a Phase I Trial of Alpha-lactalbumin (aLA) Vaccine for Breast…
Roche delivers its data reveal for adjuvant giredestrant
PharmaPhorum
2025-12-11
breast cancer
Roche reveals the data that has it excited about giredestrant in early-stage breast cancer, but how might it be positioned versus CDK4/6 inhibitors?
Nitinotes Announces First Patient Treated in Pivotal EASE™ Clinical Trial Evaluating the EndoZip™ Automated Suturing System for ESG
PR Newswire
2025-12-10
pivotal
EASE™ is a prospective, randomized IDE study comparing automated ESG to manual suturing, advancing Nitinotes toward U.S. regulatory submission CAESAREA, Israel, Dec. 10, 2025 /PRNewswire/ -- Nitinotes Ltd., an early commercial stage medical technology company developing fully automated...
Research Supports FDA Approval of Sevabertinib for HER2-Mutant Lung Cancer
ASCO Post FDA
2025-12-10
her2
lung cancer
The U.S. Food and Drug Administration (FDA) has approved sevabertinib, an oral targeted therapy, for adult patients with non-small-cell lung cancer (NSCLC) whose tumors harbor certain HER2 (ERBB2) mut...
FDA Greenlights First Gene Therapy for Rare Pediatric Disease
Fondazione Telethon
2025-12-10
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a non-profit sponsor to win FDA approval.
Telethon lands in the US: first time a non-profit organisation gets the OK to distribute a therapy
Fondazione Telethon
2025-12-10
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
The Food and drug administration has authorised a gene therapy for patients suffering from Wiskott-Aldrich syndrome, a rare and severe genetic immunodeficiency.
Genentech breast cancer drug shows 30% reduction in recurrence risk By Investing.com
Genentech
2025-12-10
breast cancer
Genentech breast cancer drug shows 30% reduction in recurrence risk
Genentech breast cancer drug shows 30% reduction in recurrence risk By Investing.com
Genentech
2025-12-10
breast cancer
Genentech breast cancer drug shows 30% reduction in recurrence risk
UPC: Genentech And Roche Gather Evidence From Organon In Allegation Of Infringement Of Breast Cancer Drug Patent
Genentech
2025-12-10
breast cancer
On 6 May 2025, Genentech and Roche petitioned the Brussels Local Division for ex-parte orders for inspection and the preservation of evidence against two pharmaceutical manufacturing...
KOMZIFTI Menin Inhibitor Data Might Change The Case For Investing In Kyowa Kirin (TSE:4151)
Kyowa Kirin
2025-12-10
leukemia
acute myeloid leukemia
Kura Oncology and Kyowa Kirin recently reported new Phase 1a/1b data showing that KOMZIFTI (ziftomenib), combined with venetoclax and azacitidine, produced encouraging antileukemic activity and a favorable safety profile in patients with acute myeloid leukemia harboring NPM1 mutations or KMT2A rearrangements, with results presented at the 67th ASH Annual Meeting. An important insight is that these findings support ongoing registrational trials that aim to position KOMZIFTI as a core menin...
Genentech’s Giredestrant Reduced Risk of Invasive Disease Recurrence or Death by 30% in ER-positive Early-Stage Breast Cancer
Genentech
2025-12-10
breast cancer
SOUTH SAN FRANCISCO, Calif., December 10, 2025--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today positive data from the Phase III lidERA Breast Cancer study evaluating investigational giredestrant as an adjuvant endocrine treatment for people with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2-negative, early-stage breast cancer. At the pre-specified interim analysis, adjuvant giredestrant significantly reduced the risk of invasive d
Cantor Fitzgerald Maintains a Buy Rating on Gilead Sciences (GILD)
Gilead Sciences
2025-12-10
yescarta
Gilead Sciences, Inc. (NASDAQ:GILD) is one of the best pharma stocks to invest in. Cantor Fitzgerald analyst Carter Gould maintained a Buy rating on Gilead Sciences, Inc. (NASDAQ:GILD) on December 7, setting a price target of $135. The same day, Kite, a Gilead Sciences, Inc. (NASDAQ:GILD) company, released a new analysis showing that second-line Yescarta therapy […]
Plug-and-Play Future for Personalized T-Cell Cancer Therapies
GenEng News
2025-12-10
tcr-t
A new automated TCR-T manufacturing system streamlines activation, engineering, and expansion of tumor-targeting T cells, reducing patient burden and labor while producing potent, long-lasting therapeutic cells from a simple blood draw. The post Plug-and-Play Future for Personalized T-Cell Cancer Therapies appeared first on GEN - Genetic Engineering and Biotechnology News .
In a first, FDA approves a gene therapy from a nonprofit
Fondazione Telethon
2025-12-10
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
The FDA on Tuesday approved Waskyra for a rare immune disease called Wiskott-Aldrich syndrome, for which patients have little options besides a bone marrow transplant.
Genentech breast cancer drug shows 30% reduction in recurrence risk By Investing.com
Genentech
2025-12-10
breast cancer
Genentech breast cancer drug shows 30% reduction in recurrence risk
Voranigo® (vorasidenib) de Servier recibe premios Prix Galien en varias regiones
PR Newswire
2025-12-10
glioma
Voranigo® ha sido galardonado con los premios Prix Galien USA, Prix Galien Polonia y Prix Galien Bridges al Mejor Producto para Enfermedades Huérfanas/Raras. Estos premios, otorgados en diversas regiones geográficas, reconocen el progreso de Voranigo® para los pacientes con glioma....
Media Choice Significantly Affects Commercial MSC Production
GenEng News
2025-12-10
mesenchymal stem cells
Five basal and serum-free media used to produce mesenchymal stem cells were analyzed and identified in terms of cell proliferation, viability, size uniformity, stability, and other factors relevant for multibatch production. The post Media Choice Significantly Affects Commercial MSC Production appeared first on GEN - Genetic Engineering and Biotechnology News .
First Commissioner's Priority Review Approval Goes to Decades-Old Antibiotic
BioSpace
2025-12-10
priority review
The Commissioner’s National Priority Voucher program was launched in June to shorten review times for companies that align with various national priorities, such as improving domestic drug production and supply.
APhA names Thomas E. Menighan as the 2026 Remington Honor Medal recipient
PR Newswire
2025-12-10
scd
WASHINGTON, Dec. 10, 2025 /PRNewswire/ -- The American Pharmacists Association (APhA) is proud to announce that Thomas E. Menighan, BSPharm, MBA, ScD, has been selected as the 2026 Remington Honor Medal recipient. This accolade is widely recognized as the highest honor in the field of...
Adverse Events to CAR T for Multiple Myeloma Linked to CCR5 Pathway
Inside Precision Medicine
2025-12-10
bcma
car t
cell therapy
multiple myeloma
The delayed side effects such as neurotoxicity and intestinal inflammation that can appear weeks or months after B-cell maturation antigen–directed (BCMA) CAR T cell therapy for multiple myeloma have a common root cause. The post Adverse Events to CAR T for Multiple Myeloma Linked to CCR5 Pathway appeared first on Inside Precision Medicine .
Roche’s Columvi improves survival in large B-cell lymphoma
PMLive Research
2025-12-10
b-cell lymphoma
large b-cell lymphoma
Approximately 160,000 people are diagnosed with this aggressive form of cancer each year
Geriatric Assessment–Guided Approach to Treatment Intensity in Older Adults With Acute Myeloid Leukemia
ASCO Post Journals
2025-12-10
leukemia
acute myeloid leukemia
“Pretreatment geriatric assessment in older adults with acute myeloid leukemia is feasible, can identify several functional impairments, and [can] guide the selection of treatment intensity,” resultin...
Giredestrant Outperforms Standard-of-Care Endocrine Therapy in Breast Cancer
Inside Precision Medicine
2025-12-10
her2
breast cancer
Giredestrant significantly reduced the risk of recurrence compared to standard endocrine therapies in early-stage HR+ HER2- breast cancer, marking the first major shift for this indication in over 25 years. The post Giredestrant Outperforms Standard-of-Care Endocrine Therapy in Breast Cancer appeared first on Inside Precision Medicine .
Adjuvant giredestrant improves invasive DFS in early breast cancer subset
Healio
2025-12-10
her2
breast cancer
SAN ANTONIO — Adjuvant giredestrant significantly improved invasive DFS vs. standard-of-care endocrine therapy in patients with early-stage, ER-positive, HER2-negative breast cancer.The findings were presented at San Antonio Breast Cancer Symposium.
Krystal Biotech’s Neurotrophic Keratitis Gene Therapy Viral Vector Secures FDA Platform Technology Designation
Krystal Biotech
2025-12-10
gene therapy
viral vector
Krystal is the second company to have received the new FDA designation.
Prime Editing Clinical Results Published for the First Time Ever
Inside Precision Medicine
2025-12-10
autologous
cell therapy
prime editing
hematopoietic stem cell
Both patients treated with the prime-edited autologous hematopoietic stem cell therapy remained free of new chronic granulomatous disease-related complications during the first few months of follow-up. The post Prime Editing Clinical Results Published for the First Time Ever appeared first on Inside Precision Medicine .
American Cancer Society updates guidelines for cervical cancer screening
Healio
2025-12-10
cervical cancer
The American Cancer Society updated its guidance on cervical cancer screening, including two key changes that the organization said will improve screening compliance and reduce cancer development.According to an American Cancer Society (ACS) press release, the guidance now includes the option of self-collection of vaginal samples for HPV testing.
Anixa Biosciences to Participate in Water Tower Research Fireside Chat on Monday, December 15, 2025
anixa biosciences
2025-12-10
car-t
breast cancer
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
SAN JOSE, Calif. , Dec. 10, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that its Chairman and CEO, Dr. Amit Kumar, will participate in the upcoming Water Tower Research Fireside Chat Series taking place on Monday, December 15, 2025, at 1:00pm ET. The Fireside Chat will be hosted by Robert Sassoon, Managing Director – Healthcare at Water Tower Research, and will cover several topics, including: The final data from Anixa's Phase 1 breast cancer vaccine trial—data which will have been presented on December 11, 2025, at the San Antonio Breast Cancer Symposium Implications for the application of the retired protein strategy for cancer vaccines Next steps for vaccine development This event is open access for all investors to participate. Interested parties can register for the event through Water Tower Research at: Webinar Registration - Zoom About Anixa Biosciences, Inc. Anixa is a clinical-stage biotechnology company focused on the treatment and prevention of cancer. Anixa's therapeutic portfolio consists of an ovarian cancer immunotherapy program…
Roche’s Giredestrant Data Could Set The Stage For A New Standard Of Care
Roche Group
2025-12-10
breast cancer
The selective estrogen receptor degrader reduced the risk of invasive disease recurrence or death by 30% in ER-positive early stage breast cancer.
Italian nonprofit lands FDA approval for first gene therapy to treat rare genetic disease
Fondazione Telethon
2025-12-10
gene therapy
wiskott-aldrich
ex vivo gene therapy
wiskott-aldrich syndrome
The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the first therapy of its kind for the rare disease and making Italy’s Fondazion | Italian nonprofit Fondazione Telethon's Waskyra is an ex vivo gene therapy that can address the root genetic cause of Wiskott-Aldrich syndrome in children and adults.
Roche’s Columvi improves survival in large B-cell lymphoma
Roche Group
2025-12-10
b-cell lymphoma
large b-cell lymphoma
Approximately 160,000 people are diagnosed with this aggressive form of cancer each year
Roche boosts Olema after breast cancer drug data (RHHBY)
Roche Group
2025-12-10
breast cancer
Roche (RHHBY) posts Phase 3 trial data for breast cancer drug giredestrant, boosting the shares of Olema (OLMA) which is advancing a similar drug. Read more here.
FDA clears first gene therapy for rare genetic disease WAS
Fondazione Telethon
2025-12-10
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
Fondazione Telethon's Waskyra has become the first gene therapy in the US for Wiskott-Aldrich syndrome, and the first taken to market by a non-profit
First Commissioner's Priority Review Approval Goes to GSK’s Decades-Old Antibiotic
BioSpace
2025-12-10
priority review
The Commissioner’s National Priority Voucher program was launched in June to shorten review times for companies that align with various national priorities, such as improving domestic drug production and supply.
Younger age does not affect survival among women with breast cancer, BRCA mutations
Healio
2025-12-10
breast cancer
SAN ANTONIO — Younger age at diagnosis did not appear to affect outcomes among women with breast cancer who harbor BRCA mutations, according to study results presented at San Antonio Breast Cancer Symposium.In addition, survival did not differ between very young women — defined as 30 years or younger — and those aged 31 to 40 years.
Roche Breast Cancer Pill Cuts Risk of Recurrence by 30% in Study
Roche Group
2025-12-10
breast cancer
Roche Holding AG’s experimental pill reduced the risk of an invasive return of a form of breast cancer in a study that underscores the drug’s blockbuster potential.
Roche’s breast cancer pill cuts post-surgery disease recurrence risk by 30%
Roche Group
2025-12-10
breast cancer
The data for giredestrant should allow Roche to seek approval in the adjuvant setting, setting it up to become the first in its class to be approved for postsurgical use.
La Fondazione Telethon annonce l'approbation par la FDA de Waskyra™ (etuvetidigene autotemcel), une thérapie génique pour le traitement du syndrome de Wiskott-Aldrich
PR Newswire
2025-12-10
wiskott-aldrich
etuvetidigene autotemcel
Le WAS touche presque exclusivement les hommes, avec une incidence estimée à 1 pour 250 000 de naissances vivantes chez les garçons. Cette thérapie représente une avancée scientifique et clinique majeure, offrant un nouvel espoir aux patients atteints de cette maladie. ROME, 10 décembre...
AZ-, Gilead-backed program reduced side effects, distress in Black breast cancer patients: study
Gilead Sciences
2025-12-10
breast cancer
A year after a clutch of major pharmas threw their weight behind | A year after a clutch of major pharmas threw their weight behind a new campaign devoted to addressing the serious health disparities facing Black breast cancer patients, “Care for HER” has been shown to have a tangible positive impact on patients’ lives.
#SABCS25: Roche’s breast cancer pill cuts post-surgery disease recurrence risk by 30%
Endpoints News
2025-12-10
breast cancer
Patients who were given Roche’s experimental breast cancer pill directly after having surgery were 30% less likely to have invasive disease recurrence than those who were given standard of care, according to interim data presented ...
Fondazione Telethon anuncia la aprobación de la FDA de Waskyra™ para el tratamiento del WAS
PR Newswire
2025-12-10
wiskott-aldrich
etuvetidigene autotemcel
Fondazione Telethon anuncia la aprobación de la FDA de Waskyra™ (etuvetidigene autotemcel), una terapia génica para el tratamiento del síndrome de Wiskott-Aldrich WAS afecta casi exclusivamente a varones, con una incidencia estimada de 1 por cada 250.000 nacidos vivos. La terapia...
Placental Growth Factor Shows Utility for Predicting Early-Onset Preeclampsia SCD
Hematology Advisor News
2025-12-10
scd
Researchers determined evaluation of levels of PlGF showed utility in prediction of early-onset preeclampsia in pregnant individuals with SCD.
GSK’227, a B7-H3-targeted antibody-drug conjugate, granted Orphan Drug Designation in small-cell lung cancer by the US FDA
GSK
2025-12-10
b7-h3
lung cancer
orphan drug
Designation supported by early clinical data showing durable responses in certain types of small-cell lung cancer (SCLC)
Roche breast cancer pill cuts risk of disease recurrence by 30% in trial
Roche Group
2025-12-10
breast cancer
The company announced today that its experimental oral drug giredestrant reduced the risk of breast cancer recurrence by 30% compared with standard endocrine therapy, in what it said marked the first big advance in hormonal treatment for the disease in over 20 years.
The Inner Circle acknowledges, Bradley Hammersley, DPM, MSHM as a Pinnacle Lifetime Member
PR Newswire
2025-12-10
ind
PERU, Ind., Dec. 10, 2025 /PRNewswire/ -- Prominently featured in The Inner Circle, Bradley Hammersley, DPM, MSHM is acknowledged as a Pinnacle Lifetime Member for his contributions as a Dedicated Leader and Specialist in Podiatric Medicine. Dr. Bradley Hammersley, DPM, MSHM, is a highly...
EQS-News: Fondazione Telethon Announces FDA approval of Waskyra™ (etuvetidigene autotemcel), a Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome
Fondazione Telethon
2025-12-10
gene therapy
wiskott-aldrich
etuvetidigene autotemcel
wiskott-aldrich syndrome
EQS-News: Fondazione Telethon / Key word(s): Miscellaneous/Miscellaneous Fondazione Telethon Announces FDA approval of...
Dosing begins in trial testing Enhertu for ovarian cancer
Rare Cancer News
2025-12-10
her2
ovarian cancer
Dosing has begun in a Phase 3 clinical trial testing Enhertu (trastuzumab deruxtecan) as a maintenance treatment for certain people with ovarian cancer. The DESTINY-Ovarian01 trial (NCT06819007) aims to enroll 582 women with advanced ovarian cancer whose tumors express a protein called HER2. To be eligible, patients must have already completed standard first-line treatment involving platinum-based chemotherapy […] The post Dosing begins in trial testing Enhertu for ovarian cancer appeared first on Rare Cancer News .
Tucatinib regimen delays disease progression in advanced HER2-positive breast cancer
Healio
2025-12-10
her2
breast cancer
SAN ANTONIO — The addition of tucatinib to first-line trastuzumab plus pertuzumab delayed disease progression among a cohort of women with HER2-positive metastatic breast cancer compared with standard of care, according to study findings.Findings from the randomized, double-blind, phase 3 HER2CLIMB-05 trial, presented at San Antonio Breast Cancer Symposium, were simultaneously published in
‘Person-to-person’ approach may reduce disparities in lupus clinical trial recruitment
Healio
2025-12-10
lupus
CHICAGO — Direct-to-patient strategies may be more effective than broad campaigns at minimizing racial and ethnic disparities in lupus clinical trial recruitment, according to data presented at ACR Convergence 2025. Brandon Jackson, MPH, clinical trial manager for Lupus Therapeutics in Miami, which oversees the Lupus Clinical Investigators Network (LuCIN), and colleagues conducted an
Roche (OTCQX: RHHBY) drug lifts 3-year iDFS to 92.4% in ER+ early breast cancer
Roche Group
2025-12-10
breast cancer
Phase III lidERA trial finds Roche’s oral SERD giredestrant cut invasive recurrence/death risk 30% and distant relapse 31%, backing a potential new standard in ER+ early breast cancer.
Astrin Launches Blood-Based Early Breast Cancer Detection Test
Inside Precision Medicine
2025-12-10
breast cancer
This test works well in women with dense breast tissue, whereas mammography frequently misses early-stage cancers in this group. The post Astrin Launches Blood-Based Early Breast Cancer Detection Test appeared first on Inside Precision Medicine .
ASH: FDA okays Gamida aplastic anaemia therapy as data drops
PharmaPhorum
2025-12-09
omisirge
cell therapy
The first cell therapy for severe aplastic anaemia – Gamida Cell's Omisirge – has been approved by the FDA.
AskBio’s AB-1005 and AB-1002 Receive Pioneering Regenerative Medical Product Designation in Japan
askbio
2025-12-09
aav
rmat
fast track
gene therapy
heart failure
gene therapies
Not intended for UK Media Berlin, Germany, and Durham, N.C., USA, December 9, 2025 – AskBio Inc., a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted the Pioneering Regenerative Medical Product designation (SAKIGAKE) for two of AskBio’s investigational gene therapy programs: AB-1005, for the treatment of Parkinson’s disease (PD), and AB-1002, for the treatment of non-ischemic heart failure with reduced left ventricular ejection fraction and New York Heart Association (NYHA) Class III heart failure despite appropriate medical therapy. The designation reflects Japan’s commitment to expediting the development and review of breakthrough therapies and is awarded to products demonstrating innovativeness (a new mode of action), prominent efficacy or safety data, and the potential to address severe diseases, especially when submitted first or simultaneously with other countries. This recognition offers significant advantages, including priority consultations and accelerated review timelines, thereby facilitating earlier participant access to transformative…
Base Edited CAR Ts Show Off-The-Shelf Promise for T-Cell Leukemia
Inside Precision Medicine
2025-12-09
car t
leukemia
A new generation of genome editing to create “off-the-shelf” CAR T cells has shown positive results in a first-in-class clinical trial, enabling leukemia patients for whom chemotherapy has not worked to go into remission and have a bone marrow transplant. The post Base Edited CAR Ts Show Off-The-Shelf Promise for T-Cell Leukemia appeared first on Inside Precision Medicine .
#ASH25: Eli Lilly's Jaypirca makes first-line case, but ceiling may be capped
Endpoints News
2025-12-09
leukemia
ORLANDO, Fla. — Fresh off an approval of Jaypirca last week in second-line leukemia patients, Eli Lilly is now looking to move up to the first-line setting with impressive data presented Tuesday. The results, revealed at ...
Platform Approaches and Industry Investment in Cell and Gene Therapy
CGTLive
2025-12-09
gene therapy
Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, spoke about strategies for streamlining development of novel therapies.
Verismo Therapeutics Shares Positive Preclinical Data at ASH 2025 Comparing SynKIR™-310 With Conventional CAR-T
Verismo Therapeutics
2025-12-09
car-t
The post Verismo Therapeutics Shares Positive Preclinical Data at ASH 2025 Comparing SynKIR™-310 With Conventional CAR-T appeared first on Verismo .
“Smart” NK Cells: Senti Bio’s Logic-Gated Immunotherapy Strikes R/R AML
Inside Precision Medicine
2025-12-09
aml
car nk
cell therapy
nk cell therapy
SENTI-202, an off-the-shelf CAR NK cell therapy, avoids targeting healthy bone marrow stem cells while eliminating both AML blasts and the elusive, quiescent leukemic stem cells that drive relapse. The post “Smart” NK Cells: Senti Bio’s Logic-Gated Immunotherapy Strikes R/R AML appeared first on Inside Precision Medicine .
Roche (OTCQX: RHHBY) secures EU approval for Gazyva in active lupus nephritis
Roche Group
2025-12-09
lupus
EU clears Gazyva/Gazyvaro plus MMF for active lupus nephritis after REGENCY data showed 46.4% complete renal response vs 33.1%, impacting up to 135,000 patients.
1 in 4 metastatic breast cancer patients treated with Novartis Kisqali® remain progression-free beyond 4 years
Novartis
2025-12-09
breast cancer
The ALPHA3 Trial: Next-Gen CAR T and MRD Testing in LBCL
Inside Precision Medicine
2025-12-09
car t
pivotal
b-cell lymphoma
large b-cell lymphoma
Sponsored content brought to you by IPM: What is the objective of and rationale behind Allogene Therapeutics’ ALPHA3 trial? Zach Roberts: The pivotal Phase II ALPHA3 trial is designed to prevent relapse in patients with large B-cell lymphoma (LBCL) who remain at high risk after completing first-line (1L) chemoimmunotherapy. Approximately 30% of LBCL patients who […] The post The ALPHA3 Trial: Next-Gen CAR T and MRD Testing in LBCL appeared first on Inside Precision Medicine .
Translating the Impact of the IMvigor011 Trial Readout: Reshaping Oncology Care
Inside Precision Medicine
2025-12-09
bladder cancer
IMvigor011 marks a turning point in how oncologists think about adjuvant therapy for muscle-invasive bladder cancer (MIBC). This trial provides a framework for precision decision-making in MIBC and opens the door to other solid tumors. The post Translating the Impact of the IMvigor011 Trial Readout: Reshaping Oncology Care appeared first on Inside Precision Medicine .
Ernexa Therapeutics Presents New Preclinical Data at American Society of Hematology (ASH) Annual Meeting on Lead Cell Therapy Candidate for Treatment of Ovarian Cancer
Ernexa Therapeutics
2025-12-09
cell therapy
ovarian cancer
Ianalumab regimen may provide ‘true disease modification’ in immune thrombocytopenia
Healio
2025-12-09
ianalumab
ORLANDO — The addition of ianalumab to eltrombopag significantly improved outcomes for patients with pretreated primary immune thrombocytopenia, according to randomized phase 3 study results.The combination prolonged time to treatment failure by more than 40% compared with eltrombopag alone, data presented during ASH Annual Meeting and Exposition’s late-breaking abstract session
Gilead Foundation Awards Over $3 Million to Support People Living With Metastatic Breast Cancer
Gilead Sciences
2025-12-09
breast cancer
FOSTER CITY, Calif., December 09, 2025--Gilead Foundation awards $3M in grants to close gaps in education, care, and support for people living with metastatic breast cancer.
Saol To Pitch Ultrarare Disease Drug as “Poster Child” for RDEP Program
BioSpace
2025-12-09
complete response
Saol Therapeutics received a complete response letter for its pyruvate dehydrogenase complex deficiency treatment a week after the FDA unveiled its Rare Disease Evidence Principles program. On Dec. 18, in a Type A meeting, the biotech will attempt to convince the agency that its drug fits perfectly into the framework.
New EC approval for Roche’s Gazyva/Gazyvaro
Roche Group
2025-12-09
lupus
The European Commission has approved Roche’s Gazyva/Gazyvaro (obinutuzumab) in combination with mycophenolate mofetil (MMF) for the treatment of adult patients with active Class III or IV, with or without concomitant Class V, lupus nephritis. These disease classifications describe the extent and nature of damage to the kidneys and renal function, a key characteristic of lupus nephritis.
At ASH, GSK’s Top Cancer Exec Discusses the FDA’s Different Take on Multiple Myeloma Drug Blenrep
MedCity News
2025-12-09
multiple myeloma
GSK submitted the same Blenrep clinical trial data to global regulators and the FDA, but received a slightly more limited regulatory approval in the U.S. In an interview during the annual meeting of the American Society of Hematology, GSK oncology executive Hesham Abdullah explained what else the FDA wants from Blenrep clinical trials. The post At ASH, GSK’s Top Cancer Exec Discusses the FDA’s Different Take on Multiple Myeloma Drug Blenrep appeared first on MedCity News .
#ASH25: New data from Gilead, J&J set up a bispecific, CAR-T rivalry
Endpoints News
2025-12-09
car-t
multiple myeloma
ORLANDO, Fla. — Clinical trials in multiple myeloma continue to rapidly progress, and nowhere was this more apparent than at the American Society of Hematology’s annual meeting. Companies like Johnson & Johnson, Gilead and Arcellx were ...
Around the Helix: Cell and Gene Therapy Company Updates – November 19, 2025
CGTLive
2025-12-09
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Novartis notches another win for drug acquired in MorphoSys deal
Biopharma Dive
2025-12-09
ianalumab
Ianalumab, which has already succeeded in testing against Sjogren’s syndrome, proved impactful in a condition that causes low platelet counts.
Insilico Details AI-Designed, Cancer-Fighting PROTAC Targeting PKMYT1
GenEng News
2025-12-09
breast cancer
cholangiocarcinoma
In preclinical studies, D16-M1P2 showed consistent potency in CCNE1 -amplified breast cancer cell lines and in an FBXW7 -mutant cholangiocarcinoma (bile duct cancer) line, as well as in a CCNE1 -amplified breast cancer xenograft model. The post Insilico Details AI-Designed, Cancer-Fighting PROTAC Targeting PKMYT1 appeared first on GEN - Genetic Engineering and Biotechnology News .
J&J-backed Cellular Origins raises $40M for cell therapy manufacturing
Endpoints News
2025-12-09
cell therapy
UK-based cell therapy manufacturer Cellular Origins has raised $40 million in an over-subscribed Series A to expand to the US and secure more company partnerships. The round was led by Johnson & Johnson’s venture capital ...
Opus Genetics Announces Positive Recommendation from Independent Data Monitoring Committee for Phase 1/2 Trial in Best Disease
opus genetics
2025-12-09
phase 1/2
New Monoclonal Antibody Shows Promise in Immune Thrombocytopenia
GenEng News
2025-12-09
ianalumab
In a Phase III clinical trial, over half of patients who received a limited course of an experimental antibody known as ianalumab were able to sustain safe platelet counts for at least one year without any serious bleeding episodes. The post New Monoclonal Antibody Shows Promise in Immune Thrombocytopenia appeared first on GEN - Genetic Engineering and Biotechnology News .
FDA set to raise bar for clinical trials of CAR-T therapies
PharmaPhorum
2025-12-09
car-t
The FDA could make it harder for developers of CAR-T therapies to bring them to market by requiring full randomised controlled trials.
Autolus Therapeutics Presents Updated Clinical Data from the CARLYSLE Trial in Patients with Severe Refractory Systemic Lupus Erythematosus at the American Society of Hematology (ASH) Annual Meeting 2025
syncona
2025-12-09
lupus
How Positive Cretostimogene Trial Updates And Leadership Moves At CG Oncology (CGON) Have Changed Its Investment Story
CG Oncology
2025-12-09
bladder cancer
complete response
CG Oncology has reported positive topline data from its BOND-003 and CORE-008 trials of investigational oncolytic immunotherapy cretostimogene in high-risk non-muscle invasive bladder cancer, showing encouraging high-grade event-free survival, strong complete response rates and a generally well-tolerated safety profile that will be presented at the 26th Society of Urologic Oncology meeting. With Phase 3 trials ongoing, an expanded access program underway, and a newly appointed director...
Gilead Foundation (GILD) grants over $3 million to 53 groups supporting metastatic breast cancer care
Gilead Sciences
2025-12-09
breast cancer
Gilead Foundation commits over $3 million in 2025 grants to 23 programs across 53 groups, expanding education, navigation and support for people with metastatic breast cancer.
Kura Oncology and Kyowa Kirin Report Combination Data for KOMZIFTI™ (Ziftomenib) with Venetoclax and Azacitidine in Newly Diagnosed and Relapsed/Refractory AML
Kyowa Kirin Co.
2025-12-09
aml
‘A knockout’: Pirtobrutinib superior to chemoimmunotherapy as first-line CLL, SLL option
Healio
2025-12-09
cll
leukemia
small lymphocytic lymphoma
chronic lymphocytic leukemia
ORLANDO — Pirtobrutinib significantly improved PFS in previously untreated patients with chronic lymphocytic leukemia and small lymphocytic lymphoma compared with a standard chemoimmunotherapy regimen in a randomized phase 3 trial.The data, presented at ASH Annual Meeting and Exposition and co-published in Journal of Clinical Oncology, showed pirtobrutinib (Jaypirca, Eli Lilly & Co.)
VIDEO: Gene therapies under study for treatment of AMD
Healio
2025-12-09
gene therapies
ORLANDO — In this video from the American Academy of Ophthalmology meeting, Jennifer I. Lim, MD, MBA, discusses three gene therapies currently being studied for the treatment of age-related macular degeneration.
Autolus Therapeutics Presents Initial Clinical Data in Pediatric r/r B-ALL Patients and Other Oncology Data at the American Society of Hematology (ASH) Annual Meeting 2025
syncona
2025-12-09
b-all
‘Profound’ data show azacitidine plus venetoclax significantly benefits patients with AML
Healio
2025-12-09
aml
leukemia
acute myeloid leukemia
ORLANDO — Azacitidine plus venetoclax significantly improved multiple outcomes for certain patients with acute myeloid leukemia compared with induction chemotherapy, according to data presented at the ASH Annual Meeting and Exposition.A randomized phase 2 trial produced “profound” results, as participants who received azacitidine plus venetoclax (Venclexta; AbbVie, Genentech)
Pirtobrutinib Outperforms Bendamustine Plus Rituximab for Previously Untreated CLL/SLL
Hematology.org
2025-12-09
cll
Pirtobrutinib Outperforms Bendamustine Plus Rituximab for Previously Untreated CLL/SLL
Novel Agent Ianalumab Added to Standard Therapy Extends Time to Treatment Failure in Patients with Previously Treated Immune Thrombocytopenia
Hematology.org
2025-12-09
ianalumab
Novel Agent Ianalumab Added to Standard Therapy Extends Time to Treatment Failure in Patients with Previously Treated Immune Thrombocytopenia
EpilepsyGTx gets $33M to test epilepsy gene therapy in Phase 1/2a
Endpoints News
2025-12-09
gene therapy
After more than two years of pitching investors, a London upstart with two current employees has pulled through with a $33 million Series A to see if it can crack into a relatively niche pocket ...
#SABCS25: BioNTech, BMS unwrap Phase 2 data for PD-L1xVEGF-A bispecific in breast cancer
Endpoints News
2025-12-09
breast cancer
BioNTech and Bristol Myers Squibb on Tuesday shared positive mid-stage breast cancer results for their PD-L1xVEGF-A bispecific antibody, which they claim has the potential to be a treatment backbone across many different cancers. Combined with ...
#ASH25: Early in vivo CAR-T results like Kelonia's elicit excitement across the field
Endpoints News
2025-12-09
car-t
multiple myeloma
ORLANDO, Fla. — A single, ready-made infusion got rid of any detectable cancer cells in four multiple myeloma patients in Australia, Kelonia Therapeutics reported as part of its first clinical trial readout. The infusion is designed ...
Genentech uncovers ‘Hidden GeMS’ in latest iteration of multiple sclerosis visibility drive
Genentech
2025-12-09
multiple sclerosis
With a new edition of a long-running awareness campaign, Genentech is drawing attention to the talents and passions that are helping people with multiple sclerosis (MS) thrive. | With a new edition of a long-running awareness campaign, Genentech is drawing attention to the talents and passions that are helping people with multiple sclerosis thrive.
CAR-T Developers Will Need Randomized Trials as FDA Eyes Tighter Approval Requirements
BioSpace
2025-12-09
car t
car-t
For traditional approval, CAR T therapies will need to establish superiority over current standard treatments, including already-approved CAR T products.
Sentinel node biopsy pioneer to receive William L. McGuire Memorial Lecture Award
Healio
2025-12-09
breast cancer
At this year’s San Antonio Breast Cancer Symposium, Armando E. Giuliano, MD, will receive the William L. McGuire Memorial Lecture Award.Giuliano will receive the award, which recognizes physicians who have made significant contributions to the field of breast oncology, on the second day of the symposium.
Gilead Foundation Awards Over $3 Million to Support People Living With Metastatic Breast Cancer
Gilead Sciences
2025-12-09
breast cancer
Gilead Foundation awards $3M in grants to close gaps in education, care, and support for people living with metastatic breast cancer.
Cell and gene therapy benefits company Aradigm raises $20M
Endpoints News
2025-12-09
gene therapy
gene therapies
As cell and gene therapies with high price tags become more prevalent, a new startup is aiming to help employers and health plans navigate those costs in a more predictable way. The New York-based startup ...
FDA raises CAR-T approval standards; Novartis bets on an AI startup
Biopharma Dive
2025-12-09
car-t
breast cancer
CAR-T cancer therapies now need to succeed in randomized, controlled trials to earn approvals. Elsewhere, a drug from Bristol Myers Squibb and BioNTech proved effective in breast cancer patients.
Adding EPO to hydroxyurea improves fatigue, pain for chronic anemia in sickle cell disease
Healio
2025-12-09
sickle cell disease
ORLANDO — Adding erythropoietin to hydroxyurea treatment could significantly improve quality of life for patients with sickle cell disease and chronic anemia, according to data presented at ASH Annual Meeting and Exposition.Patients who received 12 weeks of erythropoietin (EPO) treatment with hydroxyurea reported improvements in pain, physical functioning, emotional well-being, and energy
CRISPR-Based Liquid Biopsy Method Detects Low-Frequency Cancer Mutations
Inside Precision Medicine
2025-12-09
crispr
The method, called MUTE-Seq, uses an engineered high-fidelity CRISPR enzyme to deplete wild-type DNA in a sample before sequencing, allowing for circulating tumor DNA (ctDNA) detection below 0.1% of total cfDNA. The post CRISPR-Based Liquid Biopsy Method Detects Low-Frequency Cancer Mutations appeared first on Inside Precision Medicine .
#ASH25: Novartis says its antibody ianalumab helps prolong control of rare blood disorder
Endpoints News
2025-12-09
ianalumab
Novartis said its latest experimental approach for a rare blood disorder prolonged patients’ disease control by 45% compared to a placebo arm. The Phase 3 results, which follow a topline readout in ...
Terns Climbs 10% As ‘Unprecedented’ Leukemia Data Beats Novartis’ Scemblix
BioSpace
2025-12-09
leukemia
TERN-701 more than doubled the response rate of Novartis’ rival approved therapy in an early-stage trial, sending the biotech’s shares flying.
Tec-Dara Combination Offers Substantial Improvement Over Standard Second-Line Therapies for Relapsed or Refractory Multiple Myeloma
Hematology.org
2025-12-09
multiple myeloma
Tec-Dara Combination Offers Substantial Improvement Over Standard Second-Line Therapies for Relapsed or Refractory Multiple Myeloma
ASH: Jaypirca matches Imbruvica in first-line CLL trial
PharmaPhorum
2025-12-09
cll
Eli Lilly has revealed the data it hopes will take BTK inhibitor Jaypirca into frontline use for chronic lymphocytic leukaemia.
FDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome
FDA Press
2025-12-09
gene therapy
wiskott-aldrich
etuvetidigene autotemcel
wiskott-aldrich syndrome
The U.S. Food and Drug Administration today approved Waskyra (etuvetidigene autotemcel), the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS).
Anito-cel shows high 2-year survival rate in myeloma trial: Data
Rare Cancer News
2025-12-09
cell therapy
Most people with hard-to-treat myeloma were still alive and free from disease progression two years after treatment with anito-cel (anitocabtagene autoleucel), an experimental cell therapy being developed by Arcellx and Kite Pharma, That’s according to updated data from iMMagine-1 (NCT05396885), a Phase 2 trial testing anito-cel in people with myeloma whose disease has failed to respond […] The post Anito-cel shows high 2-year survival rate in myeloma trial: Data appeared first on Rare Cancer News .
Accelerated Cure Project Appoints First Director of Marketing and Communications
PR Newswire
2025-12-09
multiple sclerosis
Cristal Balis to lead brand strategy and audience engagement for national MS research nonprofit and its iConquerMS initiative WALTHAM, Mass., Dec. 9, 2025 /PRNewswire/ -- Accelerated Cure Project for Multiple Sclerosis (ACP), a national nonprofit dedicated to accelerating research and...
J&J’s ‘remarkable’ Tecvayli data support earlier use in multiple myeloma
Biopharma Dive
2025-12-09
multiple myeloma
In a step forward for bispecific antibodies, study results presented at ASH suggest a Tecvayli-based combination could have curative potential early in a patient’s disease course.
Bionews Releases Inaugural Rare Trust in AI Index, Finds Low Confidence in AI-Generated Health Information Among Rare Disease Patients
BioNews
2025-12-09
quarterly
With an initial index score of –0.87, the report highlights both the trust gap and the emerging opportunities for AI to support patients more effectively. PENSACOLA, Fla., Dec. 10, 2025 — Bionews, a leading rare disease news and community network, today announced the release of its inaugural Rare Trust in AI Index, a new quarterly […] The post Bionews Releases Inaugural Rare Trust in AI Index, Finds Low Confidence in AI-Generated Health Information Among Rare Disease Patients appeared first on Bionews .
Cullinan Therapeutics Showcases Compelling Clinical Data in AML for CLN-049, Novel FLT3xCD3 T Cell Engager, in Oral Presentation at the 67th ASH Meeting
Cullinan Therapeutics
2025-12-09
aml
Novartis ianalumab significantly extends disease control in patients with immune thrombocytopenia with only four once-monthly doses
Novartis
2025-12-09
ianalumab
FDA Approves First Cellular Therapy to Treat Patients with Severe Aplastic Anemia
FDA Press
2025-12-08
omisirge
hematopoietic stem cell
The U.S. Food and Drug Administration today announced it has approved Omisirge (omidubicel-onlv), the first hematopoietic stem cell transplant (HSCT) therapy to treat patients with severe aplastic anemia (SAA).
CG Oncology (CGON) Valuation Check After Strong Late-Breaking Cretostimogene Bladder Cancer Trial Results
CG Oncology
2025-12-08
bladder cancer
U.S. stocks, Saudi stocks, stock trading and investment platforms
Mortality risk rises with breathlessness at hospitalization in Africa
Healio
2025-12-08
heart failure
Adults admitted to medical wards in Malawi, Africa, faced an elevated risk for dying within 1 year if they had a breathlessness-related hospitalization, according to findings published in Thorax.Further, patients with breathlessness often had additional conditions such as heart failure, pneumonia and tuberculosis, according to the study.
Immunotherapy-Linked ADC Offers Hope for Hard-to-Treat Blood Cancers
Inside Precision Medicine
2025-12-08
aml
cd123
At ASH 2025, MD Anderson researchers reported promising data on pivekimab sunirine across two difficult blood cancers, CD123-positive AML and BPDCN—highlighting strong responses, MRD negativity, and the ability to bridge even high-risk patients to transplant. The post Immunotherapy-Linked ADC Offers Hope for Hard-to-Treat Blood Cancers appeared first on Inside Precision Medicine .
Kura Oncology, Kyowa Kirin reveal Komsifti combo data for AML
Kyowa Kirin
2025-12-08
aml
leukemia
acute myeloid leukemia
Kura Oncology (KURA) and Kyowa Kirin (KYKOF)(KYKOY) said that a combination of their drug Komsifti (ziftomenib) with Venclexta (venetoclax) and Vidaza (azacitidine) led to significant remission in patients with acute myeloid leukemia. The KOMET-007 Phase 1a/1b trial enrolled patients with newly diagnosed chemotherapy-ineligible AML. Data showed 86%...
#ASH25: Will a biotech’s pivot away from obesity let it compete with Novartis in leukemia?
Endpoints News
2025-12-08
leukemia
ORLANDO, Fla. — In October, Terns Pharmaceuticals made an announcement that seemed to run counter to every popular sentiment in biotech: It got out of obesity to focus on cancer. The 9.4% stock drop that day ...
Genetix Presents Recent Patient Experience Data from U.S. Commercial Gene Therapy Implementation at the 67th American Society of Hematology (ASH) Annual Meeting | Press Release
Genetix Biotherapeutics
2025-12-08
gene therapy
At the American Society of Hematology (ASH) Annual Meeting, Genetix shares new real-world commercial demonstrating the value proposition of one-time, durable gene therapy.
Roche’s Columvi combination shows sustained survival benefit at three-year follow up of pivotal phase III STARGLO study
Roche Group
2025-12-08
pivotal
Overall survival was twice as long for people treated with Columvi in combination with GemOx versus MabThera/Rituxan plus GemOx1This Columvi combination is...
Autolus reports positive early data from lupus treatment trial By Investing.com
Autolus Therapeutics
2025-12-08
lupus
Autolus reports positive early data from lupus treatment trial
UniQure Receives FDA Meeting Minutes on AMT-130 While Community Support Remains Strong
uniQure
2025-12-08
amt-130
UniQure received FDA’s meeting minutes on AMT-130. While there were no new updates, the community’s response has been powerful, with 34K+ petition signatures & unified advocacy from major HD orgs.
BioNTech, OncoC4’s antibody more than halves risk of death in Phase 3 lung cancer trial
Endpoints News
2025-12-08
lung cancer
BioNTech and OncoC4 shared positive survival data from a late-stage trial of their drug candidate in lung cancer that was previously hit with a partial hold by the FDA. The biotech’s treatment, called gotistobart, slashed ...
Terns drug shows potential to challenge Novartis’ blockbuster leukemia treatment
Biopharma Dive
2025-12-08
leukemia
chronic myeloid leukemia
While early, study results presented at ASH show Tern’s drug could surpass Novartis’ Scemblix and “raise the bar in efficacy” for chronic myeloid leukemia medicines, the company’s CEO said.
Autolus (Nasdaq: AUTL) moves obe-cel into Phase 2 LUMINA lupus nephritis trial
Autolus Therapeutics
2025-12-08
icans
lupus
Phase 1 CARLYSLE data show 50M obe-cel dose with 83% DORIS responses, deep B-cell depletion and no ICANS, backing an FDA-aligned Phase 2 LN study now enrolling.
Janssen Pharmaceutica describes new IL-17A/ IL-17RA interaction inhibitors
Janssen Global Services
2025-12-08
lupus
multiple sclerosis
Janssen Pharmaceutica NV has identified interleukin-17A (IL-17A)/IL-17 receptor A (IL-17RA) interaction inhibitors reported to be useful for the treatment of psoriasis, rheumatoid arthritis, radiographic axial spondyloarthritis (ankylosing spondylitis), multiple sclerosis, asthma, chronic obstructive pulmonary disease, atopic dermatitis and systemic lupus erythematosus, among others.
Genentech releases Lunsumio data in earlier treatment lines for lymphomas
Genentech
2025-12-08
complete response
follicular lymphoma
Genentech's Lunsumio shows 87% complete response in r/r follicular lymphoma in combination with Revlimid. Read more here.
Ayrmid Presents New Data on Motixafortide for Stem Cell Mobilization in Sickle Cell Disease
Gamida Cell
2025-12-08
gene therapies
sickle cell disease
Motixafortide alone and in combination with natalizumab can support the collection of the large number of stem cells required by gene therapies for sickle cell disease within a single apheresis cycle Key Highlights Motixafortide alone and in combination with natalizumab (VLA-4 inhibitor) led to robu...
Astellas To Present New Data on XOSPATA™ (gilteritinib) Across the FLT3m+ AML Disease Continuum at ASH 2025 Annual Meeting
astellas
2025-12-08
aml
Presentations include pooled post-hoc analysis of the Phase 3 ADMIRAL and COMMODORE data on post-transplant gilteritinib resumption in relapsed or refractory FLT3m+ AML - - Findings from the...
Autolus Therapeutics presents data from CATULUS Phase 1 trial of obe-cel
Autolus Therapeutics
2025-12-08
b-all
Autolus Therapeutics (AUTL) announces presentation of preliminary data from the CATULUS Phase 1 trial of obe-cel in pediatric relapsed or refractory B-ALL patients,...
Epcoritamab regimen sets ‘new benchmark’ in advanced follicular lymphoma
Healio
2025-12-08
follicular lymphoma
ORLANDO — The addition of epcoritamab to standard therapy significantly improved PFS for patients with relapsed or refractory follicular lymphoma, according to study results presented at ASH Annual Meeting and Exposition.Fixed-duration epcoritamab (Epkinly; Genmab, AbbVie) plus rituximab (Rituxan; Genentech, Biogen) and lenalidomide (Revlimid, Bristol Myers Squibb) also induced higher rates
Kura Oncology, Kyowa Kirin Report 'Encouraging' Drug Combo Data to Treat Acute Myeloid Leukemia
Kyowa Kirin
2025-12-08
leukemia
acute myeloid leukemia
Kura Oncology and Kyowa Kirin reported data on Monday showing a favorable safety profile and encouraging antileukemic activity from a phase 1a/1b trial of Komzifti with venetoclax and azacitidine...
ASH: Gilead preps filing for anito-cel on iMMagine-1 data
Gilead Sciences
2025-12-08
car-t
multiple myeloma
Gilead's Kite unit has showcased three new CAR-T therapies at ASH, headed by multiple myeloma candidate anito-cel, which could launch next year.
Gilead’s Yescarta shows benefits for patients with large B-cell lymphoma
PMLive Research
2025-12-08
yescarta
b-cell lymphoma
hodgkin lymphoma
non-hodgkin lymphoma
large b-cell lymphoma
This is the most common type of non-Hodgkin lymphoma worldwide
ASH 2025: Cellectis Presents Development Plan to Further Enhance High Response Rate Observed for Eti-cel in r/r NHL
cellectis
2025-12-08
cd20
cd22
car-t
allogeneic
gene therapies
hodgkin lymphoma
complete response
non-hodgkin lymphoma
Eti-cel showed an 88% ORR and 63% CR (n=8) at current dose level in r/r NHL after > 2 prior lines of therapy In vivo data suggest IL-2 may further enhance response rates and optimize eti-cel expansion and persistence IL-2 cohort enrollment to start in Q1 2026; full Phase 1 dataset expected in 2026 New York, NY – December 8, 2025 – Cellectis (the “Company”) (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced the presentation of encouraging updated data of patients treated in the Phase 1 NATHALI-01 clinical trial with eti-cel, at the 67 th Annual Meeting of the American Society of Hematology (ASH) in Orlando, FL. Eti-cel product candidate is the first allogeneic dual CAR-T targeting CD20 and CD22 simultaneously, being developed in Phase 1 of the NATHALI-01 clinical trial, for patients with relapsed/refractory non-Hodgkin lymphoma (r/r NHL), following at least two lines of therapy. Cellectis presented preliminary results on eti-cel, which demonstrated an encouraging overall response rate (ORR) of 88% and a complete response (CR) rate of 63% (n=8) at the…
Five-day azacitidine best among shorter hypomethylating agent regimens for low-risk MDS
Healio
2025-12-08
myelodysplastic
myelodysplastic syndrome
ORLANDO — A decade-long investigation of shorter regimens of hypomethylating agents for low-risk myelodysplastic syndrome revealed a 5-day course of azacitidine significantly improved survival compared with 3-day azacitidine and decitabine.The results, presented at ASH Annual Meeting and Exposition, showed 5-day azacitidine improved EFS and OS with no early deaths or new safety signals.
Taiho Oncology Presents Data on All-Oral Regimens Azacitidine and Cedazuridine, and Decitabine and Cedazuridine at the 2025 American Society of Hematology Annual Meeting and Exposition
PR Newswire
2025-12-08
leukemia
myelodysplastic
acute myeloid leukemia
Two oral presentations will highlight Phase 2 results from the ASTX030-01 trial of oral azacitidine and cedazuridine in patients with myelodysplastic syndromes, chronic myelomonocytic leukemia or acute myeloid leukemia and Phase 2 results from ASTX727-03 trial of low-dose oral decitabine...
Kura Oncology (KURA) Reports Promising AML Trial Results
Kyowa Kirin
2025-12-08
aml
Key Takeaways: Kura Oncology (KURA) and Kyowa Kirin report promising results from their KOMET-007 trial for AML treatment. The trial achieved an 86% comp
#ASH25: Kura, Syndax detail data for rival menin inhibitors as both eye earlier use in AML
Endpoints News
2025-12-08
aml
leukemia
complete response
acute myeloid leukemia
Kura Oncology and Kyowa Kirin said 73% of newly-diagnosed patients with certain forms of acute myeloid leukemia had a complete response after receiving their oral menin inhibitor. The early-stage results stoke their rivalry with Syndax ...
Biogen, Stoke Boost Epilepsy Drug’s Blockbuster Potential With Early Seizure Reduction Data
BioSpace
2025-12-08
dravet syndrome
Analysts at Jefferies see blockbuster potential in zorevunersen in Dravet syndrome, with sales potentially reaching $1 billion to $4 billion.
Zolgensma-Like Gene Therapy Benefits Older Spinal Muscular Atrophy Patients
Inside Precision Medicine
2025-12-08
zolgensma
gene therapy
spinal muscular atrophy
A new version of Novartis's gene therapy Zolgensma, known as Itvisma, can improve daily life in older children aged two to 17 years with spinal muscular atrophy. The post Zolgensma-Like Gene Therapy Benefits Older Spinal Muscular Atrophy Patients appeared first on Inside Precision Medicine .
Autolus reports positive early data from lupus treatment trial By Investing.com
Autolus Therapeutics
2025-12-08
lupus
Autolus reports positive early data from lupus treatment trial
Dyne Therapeutics’ Exon Skipping Duchenne Therapy Delivers Good Results
Inside Precision Medicine
2025-12-08
duchenne muscular dystrophy
Dyne Therapeutics' exon 51 skipping therapy z-rostudirsen achieved good results in a Phase I/II trial in Duchenne muscular dystrophy. The company plans to submit the therapy for approval next year. The post Dyne Therapeutics’ Exon Skipping Duchenne Therapy Delivers Good Results appeared first on Inside Precision Medicine .
Dyne to seek Duchenne drug approval; Kymera, Wave and Structure shares climb
Biopharma Dive
2025-12-08
sarepta
Dyne is readying a competitor to Sarepta’s Exondys 51. Elsewhere, a pair of obesity drugs showed promise and Kymera’s protein-degrading medicine displayed “biologic-like or better efficacy,” according to one analyst.
Autolus Therapeutics plc Presents Initial Clinical Data in Pediatric R/R B-All Patients and Other Oncology Data At the American Society of Hematology Annual Meeting 2025
Autolus Therapeutics
2025-12-08
b-all
Autolus Therapeutics plc announced presentation of preliminary data from the CATULUS Phase 1 trial of obe-cel in pediatric relapsed or refractory B-ALL patients, as well as further insights from the...
Memorial Healthcare System Reports Strong First-Half Financial Results, Reinforcing Commitment to Community Investment
PR Newswire
2025-12-08
financial results
HOLLYWOOD, Fla., Dec. 8, 2025 /PRNewswire/ -- Memorial Healthcare System reported strong financial results for the first half of fiscal year 2025, reflecting continued growth, disciplined management, and an enduring commitment to reinvesting in the community. Through the first six months...
Abeona Therapeutics® Announces First Patient Treatment with ZEVASKYN® Gene Therapy
Abeona Therapeutics
2025-12-08
zevaskyn
gene therapy
FDA sets new, higher bar for CAR-T cancer treatments to prove superiority
Endpoints News
2025-12-08
car-t
New CAR-T treatments for cancer will need to prove that they're better than existing therapies to win FDA approval, the agency's biologics chief Vinay Prasad said in a journal article Monday, raising the bar on ...
CLN-049 now has FDA fast track designation for hard-to-treat AML
Rare Cancer News
2025-12-08
aml
leukemia
fast track
acute myeloid leukemia
The U.S. Food and Drug Administration (FDA) has granted fast track designation to the experimental medicine FCLN-049, which is being developed as a potential treatment for relapsed or refractory acute myeloid leukemia (AML). This designation aims to expedite the development of therapies with the potential to address unmet needs in the management of serious health […] The post CLN-049 now has FDA fast track designation for hard-to-treat AML appeared first on Rare Cancer News .
CG Oncology (CGON) Valuation Check After Strong Late-Breaking Cretostimogene Bladder Cancer Trial Results
CG Oncology
2025-12-08
bladder cancer
CG Oncology (CGON) just released new topline data on its bladder cancer drug cretostimogene, with late-breaking BOND 003 and CORE 008 results highlighting strong responses and a clean-looking safety profile. See our latest analysis for CG Oncology. Those upbeat cretostimogene data land after a busy stretch for CG Oncology, including fresh SUO conference presentations and a new board appointment. The stock’s momentum reflects that, with a roughly 49 percent year to date share price return and...
Dyne’s Exon Skipper Delivers ‘Best Ever’ Functional Outcomes in Early DMD Study
BioSpace
2025-12-08
dmd
Dyne’s exon-skipping therapy zeleciment rostudirsen resulted in an approximately sevenfold increase in dystrophin levels at six months and elicited functional improvements that are the “best ever” for this treatment class, Stifel analysts said.
ASH: Regeneron makes frontline case for Lynozyfic in myeloma
PharmaPhorum
2025-12-08
multiple myeloma
Regeneron's BCMAxCD3 bispecific Lynozyfic is the first drug in the class to show efficacy as a monotherapy in first-line multiple myeloma.
Pirtobrutinib produces ‘excellent’ results vs. ibrutinib as first-line CLL, SLL treatment
Healio
2025-12-08
cll
leukemia
small lymphocytic lymphoma
chronic lymphocytic leukemia
ORLANDO — Noncovalent Bruton tyrosine kinase inhibitors like pirtobrutinib may be a more effective first-line therapy for chronic lymphocytic leukemia and small lymphocytic lymphoma than covalent inhibitors, according to a phase 3 study.The data, presented at ASH Annual Meeting and Exposition, showed patients with relapsed or refractory disease who received pirtobrutinib (Jaypirca, Eli
Autolus Therapeutics Presents Promising Obe-cel Data for Pediatric r/r B-ALL at ASH Annual Meeting
Autolus Therapeutics
2025-12-08
b-all
car t
leukemia
cell therapy
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
Obe-cel shows 95.5% response rate in pediatric B-ALL, with low severe side effects; promising Phase 1 trial results presented.Quiver AI SummaryAutolus Therapeutics announced promising preliminary data from the CATULUS Phase 1 trial regarding its CAR T-cell therapy, obe-cel, for pediatric patients with high-risk relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL). The therapy demonstrated a remarkable overall response rate of 95.5%
FDA Expands Jaypirca Indication for Relapsed/Refractory CLL/SLL
Hematology Advisor News
2025-12-08
cll
The FDA has approved Jaypirca (pirtobrutinib) for the treatment of adult patients with R/R CLL/SLL who have previously been treated with a covalent BTK inhibitor.
Dyne says Duchenne drug succeeds in pivotal study, heads to FDA for approval
Endpoints News
2025-12-08
pivotal
accelerated approval
duchenne muscular dystrophy
Dyne Therapeutics announced Monday that its Duchenne muscular dystrophy drug hit the primary endpoint in a key study and plans to take the experimental treatment to the FDA for accelerated approval. Dyne is one of ...
Roche presents Lunsumio data showing potential across earlier treatment lines in indolent and aggressive lymphomas
Roche Group
2025-12-08
follicular lymphoma
Lunsumio in combination with lenalidomide may offer an effective treatment in relapsed or refractory follicular lymphoma based on first data from single-arm US cohort of phase III CELESTIMO study1Data from subcutaneous Lunsumio plus Polivy reinforce its outpatient, chemotherapy-free potential in people with R/R large B-cell lymphoma2,3Results highlight the potential of innovative Lunsumio combination regimens to offer improved outcomes for more people with lymphoma earlier in their disease Basel
ASH25: CAR-T Leaders Gilead, J&J Show in Multiple Myeloma; Fulcrum Rises on Sickle Cell Data
Gilead Sciences
2025-12-08
car-t
multiple myeloma
The 2025 meeting of the American Society of Hematology features some of the newest developments in blood cancers and rare diseases.
Genentech (OTCQX: RHHBY) reports 87% CR, longer PFS with Lunsumio regimens in lymphoma
Genentech
2025-12-08
complete response
New ASH 2025 data show an 87.0% complete response rate in FL and 77.5% ORR vs 50.0% in LBCL with Lunsumio combos, plus 5-year OS of 78.5% in R/R FL.
Roche’s Columvi combination shows sustained survival benefit at three-year follow up of pivotal phase III STARGLO study
Roche Group
2025-12-08
dlbcl
pivotal
Overall survival was twice as long for people treated with Columvi in combination with GemOx versus MabThera/Rituxan plus GemOx1This Columvi combination is available off-the-shelf and could offer a potentially curative treatment option for people with R/R DLBCL who are not candidates for transplantColumvi in combination with GemOx has now been approved in more than 50 countries worldwide and recommended in international treatment guidelines2-4 Basel, 8 December 2025 - Roche (SIX: RO, ROG; OTCQX:
Fate Therapeutics Presents Updated Phase 1 Clinical Data of FT819 Off-the-shelf CAR T-cell Product Candidate for Systemic Lupus Erythematosus and Preclinical Advances in Next-Generation Off-the-Shelf CAR T-cell Programs
Fate Therapeutics
2025-12-08
car t
lupus
FT819 continues to demonstrate meaningful decrease in disease and favorable safety profile with twelve systemic lupus erythematosus (SLE) patients now treated; first systemic sclerosis (SSc) patient treated First ex- U.S. SLE patient treated with FT819 expands enrollment capacity and supports
CALDOLOR® (IBUPROFEN) INJECTION CMS ISSUED J-CODE NOW ASSOCIATED WITH REIMBURSEMENT PRICE SUPPORTING NON-OPIOID PAIN MANAGEMENT
PR Newswire
2025-12-08
j-code
NASHVILLE, Tenn., Dec. 8, 2025 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), today announced an important update regarding its Caldolor® (ibuprofen) Injection. The product's permanent J-code, J1741, is now officially associated with a reimbursement price, providing...
Data from uniQure’s Phase I/II AMT-130 not enough, FDA confirms
uniQure
2025-12-08
bla
amt-130
gene therapy
Gene therapy company uniQure last week received final minutes from the US Food and Drug Administration (FDA) regarding its pre-Biologics License Application (BLA) meeting held in late October to discuss the company’s application for AMT-130 in Huntington’s disease (HD).
CSL Behring's Hemophilia B Gene Therapy Provides Bleeding Protection Out to Five Years
CSL Behring
2025-12-08
gene therapy
hemophilia b
Researchers at ASH shared Phase III data comparing the pre- and five-year posttreatment adjusted annualized bleeding rates, and showed a 63 percent reduction.
Kura Oncology and Kyowa Kirin Report Combination Data for KOMZIFTI with Venetoclax and Azacitidine in Newly Diagnosed and Relapsed/Refractory AML
Kyowa Kirin
2025-12-08
aml
Kura Oncology, Inc. and Kyowa Kirin Co. Ltd. announced new data demonstrating a favorable safety profile and encouraging antileukemic activity for KOMZIFTI in combination with venetoclax and...
Port delivery system shows durable vision outcomes up to 7 years
Healio
2025-12-08
wet age-related macular degeneration
PARIS — Long-term 7-year follow-up data from the Portal extension trial support the port delivery system as an effective strategy to maintain long-term visual outcomes with reduced treatment burden.At the Euretina congress, Arshad M. Khanani, MD, MA, FASRS, said that the trial was the longest prospective study of patients with wet age-related macular degeneration ever performed.
FDA misses PDUFA review deadline for Agios' thalassemia drug
Endpoints News
2025-12-08
pdufa
The FDA missed its target date for an approval decision on Agios’ thalassemia drug, another delay for the company as it tries to expand its reach in blood disorders. A regulatory decision for Pyrukynd was ...
Measurable residual disease an early marker for treatment response in AML
Healio
2025-12-08
aml
leukemia
acute myeloid leukemia
ORLANDO — Measurable residual disease after induction therapy may serve as an individual-level predictor of survival among patients with acute myeloid leukemia, according to study results presented at ASH Annual Meeting and Exposition.“AML remains a difficult disease to cure. There have been recent advancements in the past couple of years with new drugs, FLT3 and IDH inhibitors, but
Chemotherapy-free regimen could be new standard in Ph+ acute lymphoblastic leukemia
Healio
2025-12-08
leukemia
acute lymphoblastic leukemia
ORLANDO — A chemotherapy-free regimen significantly improved outcomes compared with standard therapy for newly diagnosed Ph+ acute lymphoblastic leukemia, according to study results presented at ASH Annual Meeting and Exposition.Patients who received ponatinib (Iclusig, Takeda) and blinatumomab (Blincyto, Amgen) achieved higher rates of clinical hematologic remission and minimal residual
CAR-T regulatory cells well-tolerated, with ‘promising’ efficacy in rheumatoid arthritis
Healio
2025-12-08
car-t
autologous
chimeric antigen receptor
CHICAGO — Citrullinated protein-targeted chimeric antigen receptor regulatory T cells showed a promising safety profile in highly refractory patients with rheumatoid arthritis, according to phase 1 data presented at ACR Convergence 2025. This novel therapy known as SBT777101, an autologous regulatory T-cell (Treg) product transduced with a CAR specific for citrullinated proteins, also
Kura’s ziftomenib shows promising results in AML treatment trials By Investing.com
Kyowa Kirin
2025-12-08
aml
Kura’s ziftomenib shows promising results in AML treatment trials
Lower Immunotherapy Dose Boosts Response by 30% in Melanoma Patients
Inside Precision Medicine
2025-12-08
melanoma
Researchers in Sweden have found that a lower dose of a common immunotherapy drug for malignant melanoma can significantly improve outcomes while reducing side effects. The post Lower Immunotherapy Dose Boosts Response by 30% in Melanoma Patients appeared first on Inside Precision Medicine .
08 Dec 2025 CARsgen Announces Data of Allogeneic BCMA CAR-T Cell Therapy CT0596 for Relapsed/Refractory Multiple Myeloma at the 2025 ASH Annual Meeting Dec 08, 2025
Carsgen Therapeutics
2025-12-08
bcma
car-t
allogeneic
cell therapy
multiple myeloma
Kura Oncology, Kyowa Kirin reveal Komsifti combo data for AML
Kyowa Kirin
2025-12-08
aml
leukemia
acute myeloid leukemia
Kura Oncology Kyowa Kirin's Komsifti combo shows high remission rates in acute myeloid leukemia patients. Read more here.
GILD's Yescarta Shows Promising Results for Lymphoma Patients
Gilead Sciences
2025-12-07
yescarta
Key Takeaways: Kite, a subsidiary of Gilead Sciences (GILD), reports promising results from Yescarta therapy for R/R LBCL patients. Gilead Sciences is a
Sara Pirzadeh-Miller, MS, CGC, on AI and Gene Therapy Working in Tandem
CGTLive
2025-12-07
gene therapy
Stephen M. Ansell, MD, PhD, on Integrating Cell Therapy into Clinical Practice in Oncology
CGTLive
2025-12-07
cell therapy
cell therapies
The chair of the Division of Hematology at Mayo Clinic discussed practical considerations for the introduction of cell therapies for the treatment of hematologic malignancies.
Lyell Immunopharma Presents New Clinical Data from Ongoing Trial of Ronde-Cel Showing High Rates of Durable Complete Responses in Patients with Large B-cell Lymphoma at the 67th ASH Annual Meeting and Exposition
lyell immunopharma
2025-12-07
b-cell lymphoma
complete response
large b-cell lymphoma
93% overall response and 76% complete response rates with median progression-free survival of 18 months in patients with large B-cell lymphoma in the 3L+ setting 83% overall response and 61% complete response rates in cohort comprised predominantly of patients with primary refractory large B-cell
GILD's Kite Unveils Promising CAR T-Cell Therapy Results at ASH Meeting
Gilead Sciences
2025-12-07
car t
cell therapy
cell therapies
Key Takeaways: Gilead Sciences (GILD) reports promising Phase 1 results for its CAR T-cell therapies, KITE-753 and KITE-363. The company is a leader in t
ASH: CSL proves efficacy of hemophilia B gene therapy Hemgenix holds up after 5 years
CSL Behring
2025-12-07
hemgenix
gene therapy
hemophilia b
Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data proving the treatment’s benefits remain durable after five years. | After five years of follow-up on its hemophilia B gene therapy Hemgenix, CSL found that a one-time dose cut down on annualized bleeding rates by 90% by the fifth year in its Hope-B study.
07 Dec 2025 CARsgen’s CAR-T Product Zevor-Cel Included in China’s Commercial Health Insurance Innovative Drug Catalogue Dec 07, 2025
Carsgen Therapeutics
2025-12-07
car-t
Lyell Immunopharma Presents New Clinical Data from Ongoing Trial of Ronde-Cel Showing High Rates of Durable Complete Responses in Patients with Large B-cell Lymphoma at the 67th ASH Annual Meeting and Exposition
lyell immunopharma
2025-12-07
b-cell lymphoma
complete response
large b-cell lymphoma
93% overall response and 76% complete response rates with median progression-free survival of 18 months in patients with large B-cell lymphoma in the 3L+ setting 83% overall response and 61% complete response rates in cohort comprised predominantly of patients with primary refractory large B-cell
At ASH, Lilly makes case to widen Jaypirca use in leukemia, lymphoma
Biopharma Dive
2025-12-07
leukemia
In a head-to-head trial, Lilly’s drug was as effective as Imbruvica at inducing responses and displayed certain safety advantages that could make it a preferred treatment in early lines of care.
New data from Gilead, Arcellx multiple myeloma CAR-T suggest benefit over rival treatment
Gilead Sciences
2025-12-07
car-t
multiple myeloma
Gilead and Arcellx reported promising data for their CAR-T therapy anito-cel at the annual American Society of Hematology meeting.
Odronextamab Plus CHOP Promising in Previously Untreated DLBCL
Hematology Advisor News
2025-12-07
dlbcl
Researchers sought to determine whether combination odronextamab plus CHOP would be safe and effective in patients with DLBCL.
Weekly and Monthly Mim8 Deliver Durable Bleed Control Across 52 Weeks in Hemophilia A
Hematology Advisor News
2025-12-07
hemophilia a
Researchers assessed whether Mim8 would provide sustained bleed protection for adolescents and adults with hemophilia A.
‘Transformative’ data: Near-universal donor access may be possible for allogeneic HCT
Healio
2025-12-07
allogeneic
hematopoietic stem cell
ORLANDO — A specific graft-versus-host disease prophylaxis regimen may expand allogeneic hematopoietic stem cell transplant access to nearly all patients in need, according to study results presented at ASH Annual Meeting and Exposition. Post-transplant cyclophosphamide (PTCy) conferred favorable outcomes for patients who received peripheral blood stem cell grafts from mismatched
Gilead Eyes Anito-cel Approval in 2026 Riding on Latest Phase II Read Out at ASH
Gilead Sciences
2025-12-07
car t
cell therapy
In the most recent data presented at the meeting, the CAR T-cell therapy demonstrated a 96 percent overall response rate.
Orna Therapeutics Presents New Data Supporting its in vivo CAR Programs for Autoimmune Diseases and Oncology at the 67th American Society of Hematology Annual Meeting
orna therapeutics
2025-12-07
bcma
cd19
lupus
Anti-CD19 panCARTM program (ORN-252) demonstrated robust B cell depletion with significant reduction in anti-dsDNA titers in humanized lupus model Anti-BCMA panCARTM program showed robust and durable plasma cell depletion in preclinical models Orna expects to submit its first Clinical Trial Application for ORN-252 by the end of 2025 and anticipates initiating a first-in-human study in… The post Orna Therapeutics Presents New Data Supporting its in vivo CAR Programs for Autoimmune Diseases and Oncology at the 67th American Society of Hematology Annual Meeting appeared first on Orna Therapeutics .
Prime Medicine Announces The New England Journal of Medicine Publication of PM359 Clinical Data for the Treatment of Chronic Granulomatous Disease
prime medicine
2025-12-07
phase 1/2
CAMBRIDGE, Mass., Dec. 07, 2025 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today announced the publication of Phase 1/2 clinical data with PM359, the Company’s
Where are the Biggest Bottlenecks in Cell and Gene Therapy?
CGTLive
2025-12-07
gene therapy
Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, discussed major friction points in cell and gene therapy development and uptake in the current day.
ASH: Gilead’s Kite Plots 2026 Launch For Anito-Cel For Multiple Myeloma
Gilead Sciences
2025-12-07
multiple myeloma
The company presented positive efficacy and safety data from its registrational Phase II iMMagine-1 trial at the 2025 ASH meeting.
ASH: Anito-cel strengthens best-in-class potential as Gilead's Kite preps for launch
Gilead Sciences
2025-12-07
car-t
carvykti
multiple myeloma
ORLANDO, Fla. | The leader of Gilead’s Kite Pharma is sure that anito-cel, the biotech’s next-generation multiple myeloma CAR-T challenger to Carvykti, is ready for prime time—and that the company is ready to bring CAR-T to the masses.
Palliative care increasing for patients with DLBCL, but racial disparities ‘striking’
Healio
2025-12-07
dlbcl
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
ORLANDO — Palliative care for diffuse large B-cell lymphoma has significantly improved over the past several years, but disparities persist based on race, insurance and hospital characteristics.Study results, presented at ASH Annual Meeting and Exposition, showed white patients received palliative care consultations three times more often than Black patients and more than double the rate of
ASH: AstraZeneca's dual-wielding CAR-T shows promise
PharmaPhorum
2025-12-07
car-t
multiple myeloma
AstraZeneca has reported an impressive 96% response rate in the DURGA-1 of BCMAxCD19 CAR-T therapy AZD0120 in heavily pretreated multiple myeloma.
Black race prognostic for shorter survival in AML regardless of cytogenetic profile
Healio
2025-12-07
aml
leukemia
acute myeloid leukemia
ORLANDO — Black race is an independent prognostic factor for shorter survival in acute myeloid leukemia independent of cytogenetic profiles, according to study results presented at ASH Annual Meeting and Exposition.A review of nearly 4,000 clinical trial participants showed no significant difference in European LeukemiaNet (ELN) 2017 risk scores or prevalence of cytogenetic abnormalities
Luspatercept Appears Safe for Pediatric NTD Beta-Thalassemia, Early Study Finds
Hematology Advisor News
2025-12-07
beta-thalassemia
Researchers assessed whether a set dose of luspatercept could be safe in treating pediatric patients with non–transfusion dependent beta-thalassemia.
uniQure (QURE): Reassessing Valuation After FDA Sets Higher Bar for AMT-130 Approval Pathway
uniQure
2025-12-07
amt-130
uniQure (NasdaqGS:QURE) just hit a key regulatory speed bump, with the FDA signaling that current Phase I/II data for its AMT-130 Huntington’s program likely will not carry a Biologics License Application. See our latest analysis for uniQure. The setback has hit sentiment hard in the short term, with a 1 week share price return of minus 22.46 percent and a 30 day share price return of minus 23.25 percent. However, momentum is still positive versus earlier in the year given a 90 day share...
At ASH 2025 Oral Presentation, Immix Biopharma Reports Positive Phase 2 NXC-201 Results, Advancing Toward BLA Submission as a Potentially First- and Best-in-Class Therapy for relapsed/refractory AL Amyloidosis
Immix Biopharma
2025-12-07
bla
nxc-201
complete response
– NXC-201 demonstrated a complete response (CR) rate of 75% (15/20) (at s/u IFE(-) level) by independent review committee – – In four out of five pending patients, MRD negativity in bone marrow predicts future complete response, potentially increasing future CR rate to 95% – – NEXICART-2 final readout and BLA submission planned in 2026 […] The post At ASH 2025 Oral Presentation, Immix Biopharma Reports Positive Phase 2 NXC-201 Results, Advancing Toward BLA Submission as a Potentially First- and Best-in-Class Therapy for relapsed/refractory AL Amyloidosis first appeared on IMMIX BIOPHARMA INC .
Legend Biotech Highlights New CARVYKTI® Data in Multiple Myeloma and First-in-Human Results from Novel CAR-T Platform in Non-Hodgkin Lymphoma at ASH 2025
Legend Biotech
2025-12-06
car-t
carvykti
hodgkin lymphoma
multiple myeloma
non-hodgkin lymphoma
How Restructuring-Driven Margin Gains And Higher Guidance At BioMarin (BMRN) Have Changed Its Investment Story
BioMarin Pharmaceutical
2025-12-06
orphan drug
In recent months, BioMarin Pharmaceutical reported very large year-over-year EPS growth in Q2 2025, highlighted the margin benefits of its 2024 restructuring, and raised guidance for revenue, income, and operating margins while pointing to limited competition for its orphan drug portfolio. Management now expects the full impact of these cost and efficiency measures to emerge in 2026, with promising late-stage trials potentially adding to an already focused rare-disease franchise. Next, we’ll...
Ayrmid Reports Promising New Real-World Data on Motixafortide for Stem Cell Mobilization in Sickle Cell Disease
Gamida Cell
2025-12-06
gene therapy
gene therapies
sickle cell disease
Key Highlights Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease enabling accelerated access to gene therapies 90% (9 of 10) of patients were able to collect sufficient HSC to enable progress to gene therapy manufacturing 86% (6 of...
Arcellx Announces New Positive Data for Its iMMagine-1 Study in Patients with Relapsed and/or Refractory Multiple Myeloma
arcellx, inc
2025-12-06
icans
multiple myeloma
Anito-cel demonstrated 96% ORR and 74% CR/sCR at a median follow-up of 15.9 months; responses continue to deepen over time - - Overall MRD negativity was 95% and sustained MRD negativity for >6 months was 83%, both at 10-5 sensitivity level - - 12-month PFS and OS rates were 82.1% and 94.0%; 18-month PFS and OS rates were 67.4% and 88.0%; 24-month PFS and OS rates were 61.7% and 83.0% - - To date, no delayed or non-ICANS neurotoxicities, including no Parkinsonism, no cranial nerve palsies, no Guillain-Barré syndrome, and no immune effector cell-associated enterocolitis have been observed with anito-cel to date; all patients were dosed at least 12 months ago - - The company reiterates its planned 2026 commercial launch - - iMMagine-1 data to be presented during an oral presentation at the ASH Annual Meeting on Saturday, December 6, 2025 - - Company to host a live webcast event with an expert panel of clinicians during ASH - Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company
Vertex Presents New Data on CASGEVY®, Including First-Ever Data in Children Ages 5-11 Years, at the American Society of Hematology Annual Meeting and Announces Plan for Global Regulatory Submissions
Vertex
2025-12-06
casgevy
pivotal
beta thalassemia
sickle cell disease
Data from pivotal studies of CASGEVY in children ages 5-11 years with severe sickle cell disease or transfusion-dependent beta thalassemia demonstrates the transformative potential of the therapy in younger patients - - Efficacy and safety data in children 5-11 years are consistent with the
Asian Patients With AML May Have Superior Survival Outcomes to White Patients, Analysis Suggests
Hematology Advisor News
2025-12-06
aml
Researchers assessed survival outcomes for Asian patients with newly diagnosed AML vs White patients.
Favorable outcomes after HCT for sickle cell disease can guide shared decision-making
Healio
2025-12-06
allogeneic
sickle cell disease
ORLANDO — Most people with sickle cell disease who undergo allogeneic hematopoietic cell transplantation achieve favorable long-term outcomes, according to study results presented at ASH Annual Meeting and Exposition.An analysis of more than 1,000 patients who underwent initial HCT over the past few decades showed most survived 7 years, with a majority remaining free of complications and
‘Catastrophic’ income loss occurs in nearly a third of families of kids treated for ALL
Healio
2025-12-06
leukemia
ORLANDO — During his first year of fellowship, Daniel J. Zheng, MD, MHS, MSHP, sat in clinic waiting for a young child with leukemia scheduled for chemotherapy.As they waited, a nurse navigator messaged Zheng that the patient’s family would not be able to make it.
Genetix Participates in the American Society of Hematology’s (ASH) Exclusive Press Program & Shares Recent Patient Experience Data from U.S. Commercial Gene Therapy Implementation | Press Release
Genetix Biotherapeutics
2025-12-06
gene therapy
gene therapies
Selected for ASH’s exclusive press program, Genetix presents real-world data showing rising adoption and faster access for its one-time gene therapies.
uniQure (QURE): Reassessing Valuation After FDA Doubts on AMT-130’s Path to Approval
uniQure
2025-12-06
amt-130
uniQure (NasdaqGS:QURE) just hit a regulatory speed bump, with the FDA signaling that early AMT-130 data probably will not carry a Biologics License Application, and the stock quickly reflected that uncertainty. See our latest analysis for uniQure. That FDA update helps explain why the 1 day share price return was down 6.2 percent and the 7 day share price return slid 22.5 percent, even though the 1 year total shareholder return is still a hefty 186 percent. This suggests long term holders...
Anova Today Announced the Enrollment of 550+ Patients at 50+ Sites in Six Months for a Single Study
anova evidence
2025-12-06
ind
Anova completed enrollment of 550+ patients for an intermediate-sized, treatment IND in just six months, significantly reducing traditional site activation and patient enrolment timelines, and provided safety and efficacy data to support its' drug development efforts. The post Anova Today Announced the Enrollment of 550+ Patients at 50+ Sites in Six Months for a Single Study appeared first on Anova .
‘Very concerning’: Opioids for sickle cell pain often not administered fast enough in ED
Healio
2025-12-06
sickle cell disease
ORLANDO — Administration of opioid medications to people who present to the ED with acute sickle cell disease pain often does not happen as quickly as guidelines recommend, data presented at ASH Annual Meeting and Exposition showed.An analysis of nearly 400,000 ED visits showed fewer than one-third adhered to guidelines for timely administration of the first dose. Fewer than one in 10
‘Extremely encouraging’: Exa-cel benefits kids with sickle cell disease, beta thalassemia
Healio
2025-12-06
beta thalassemia
sickle cell disease
ORLANDO — Exagamglogene autotemcel helped children with beta thalassemia achieve transfusion independence and prevented vaso-occlusive crises among those with sickle cell disease in a pair of phase 3 trials.Early data, presented at ASH Annual Meeting and Exposition, showed 100% of evaluable patients in both trials met the investigations’ primary endpoints — freedom from
Hydroxyurea does not affect fertility among women with sickle cell disease
Healio
2025-12-06
sickle cell disease
ORLANDO — Hydroxyurea does not affect fertility among women with sickle cell disease, according to study results presented at ASH Annual Meeting and Exposition.The agent also does not appear to impact newborn health, findings showed.
ORIC’s lung cancer pill shows promise in some settings but is dropped for HER2 mutations
Endpoints News
2025-12-06
her2
lung cancer
ORIC Pharmaceuticals’ lung cancer pill showed encouraging efficacy as a first- and second-line treatment in lung cancer patients who have a specific genetic mutation targeted by the drug, part of a series of early trial ...
Iron Overload Remains Prevalent in Individuals with Sickle Cell Disease Despite Guidelines
Hematology.org
2025-12-06
sickle cell disease
Iron Overload Remains Prevalent in Individuals with Sickle Cell Disease Despite Guidelines
Revumenib Shows Promise in Treating NPM1-Mutated Relapsed/Refractory AML
Hematology Advisor News
2025-12-06
aml
Researchers determined revumenib showed promising efficacy in patients with R/R NPM1m AML.
Patient Treated in Trial for Intellia’s Transthyretin Amyloidosis Gene Editing Therapy Nex-Z Dies
CGTLive
2025-12-06
gene editing
According to Intellia, the FDA placed clinical holds on both MAGNITUDE and MAGNITUDE-2 on October 29, 2025.
#ASH25: AstraZeneca's dual-targeting CAR-T sees similar responses in Western patients as in China
Endpoints News
2025-12-06
car-t
cell therapy
ORLANDO — The first data in Western cancer patients for AstraZeneca's dual-targeting CAR-T cell therapy appear to hold up with impressive, earlier results from China-only studies. AstraZeneca presented preliminary results on Saturday at the American Society ...
Vertex CRISPR therapy hits early goal in children with blood disorders
Biopharma Dive
2025-12-06
crispr
casgevy
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake.
‘Practice-changing’ findings may spare some young patients with leukemia from radiation
Healio
2025-12-06
leukemia
allogeneic
hematopoietic stem cell
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
ORLANDO — Some younger patients with B-cell acute lymphoblastic leukemia may be able to avoid total body irradiation conditioning prior to allogeneic hematopoietic stem cell transplantation, according to study results.Children, adolescents and young adults who were deemed minimal residual disease (MRD) negative by next-generation sequencing prior to transplant and received non-total body
Beam Therapeutics Reports Updated Data from BEACON Phase 1/2 Trial of ristoglogene autogetemcel (risto-cel) Highlighting Durable, Differentiated Profile in Sickle Cell Disease (SCD) at American Society of Hematology (ASH) Annual Meeting
beam therapeutics
2025-12-06
scd
phase 1/2
sickle cell disease
Updated Data from 31 Adult and Adolescent SCD Patients Treated with risto-cel (Formerly BEAM-101) Show Mean He moglobin F (HbF) Induction of >60%, Hemoglobin S (HbS) Reduction to 40%, and Resolution of Anemia Durable for up to 20 Months Patients Required a Median of One Cell Collection Cycle and
BMS's Breyanzi is first CAR-T for marginal zone lymphoma
PharmaPhorum
2025-12-05
car-t
breyanzi
marginal zone lymphoma
Bristol Myers Squibb has taken CAR-T therapy into new territory, after getting FDA approval for Breyanzi in marginal zone lymphoma.
Neoadjuvant Therapy for Melanoma Is Associated With Promising Survival Benefits
Hematology Advisor News
2025-12-05
melanoma
Among patients with melanoma, NT was associated with acceptable CR without increasing the risk for severe AEs and may be a promising treatment option.
Resistant Ovarian Cancer Combination Therapy Shows Promise
GenEng News
2025-12-05
ovarian cancer
In multiple preclinical models of PARPi-resistant ovarian cancer a treatment strategy combining PARPi with the indirect WNT inhibitor SM08502 significantly reduced disease progression, remodeled the tumor immune microenvironment, and extended survival. The post Resistant Ovarian Cancer Combination Therapy Shows Promise appeared first on GEN - Genetic Engineering and Biotechnology News .
Transforming Breast Cancer Screening: Lessons from a Landmark Colombian Study
Inside Precision Medicine
2025-12-05
breast cancer
This podcast features Dr. George Busby discussing his work on polygenic risk scores (and their expanding role in precision medicine. The post Transforming Breast Cancer Screening: Lessons from a Landmark Colombian Study appeared first on Inside Precision Medicine .
Taysha Gene Therapies Announces Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Taysha Gene Therapies
2025-12-05
aav
gene therapies
adeno-associated virus
DALLAS , Dec. 05, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today
FDA approves Omisirge® as First Approved Cell Therapy to Treat Severe Aplastic Anemia
Gamida Cell
2025-12-05
omisirge
ayrmid ltd.
gamida cell
cell therapy
Study demonstrated early and sustained hematopoietic recovery and transfusion independence Omisirge is a new and novel transplant option for patients with SAA Additional manufacturing capacity for Omisirge will be established in the U.S. Represents a supplementary FDA approval for Omisirge LONDON – December 5, 2025 – Ayrmid Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc.
Hemophilia gene therapies are struggling on the market, even as innovation soars
Biopharma Dive
2025-12-05
gene therapy
gene therapies
The business case for hemophilia gene therapy still isn’t adding up due to persistent market barriers.
Cohort P Data from the BOND-003 Study in BCG-Unresponsive Papillary Bladder Cancer
CG Oncology
2025-12-05
adenovirus
bladder cancer
top line results
complete response
Zachary Klaassen: Hi, my name is Zach Klaassen, urologic oncologist at the Georgia Cancer Center in Augusta, Georgia. I'm delighted to be joined on UroToday by Dr. Mark Tyson, who is a urologic oncologist at Mayo Clinic, Arizona. We're going to be discussing SUO 2025 late-breaking abstracts and data that Mark presented at the recent SUO meeting. Mark, thanks so much for joining us at UroToday. Mark Tyson: Thanks for having me, Zach. Glad to be here. Zachary Klaassen: Why don't you roll through some slides? We'll cover the background and the top line results from Cohort P, and then we'll have a little Q&A after that. Mark Tyson: That sounds good. Yeah, as you mentioned, this is Cohort P of the BOND-003 study. It's a single-arm, open-label trial evaluating cretostimogene monotherapy in patients with BCG-unresponsive papillary-only non-muscle-invasive bladder cancer. Just for the sake of your viewers, I have no conflicts of interest with CG Oncology. Just maybe a brief second on mechanism of action. This is an oncolytic adenovirus. It enters the cell through the Coxsackie and adenovirus receptor, and it exploits the biology of Rb pathway-deficient tumor cells. So any mutation in Rb…
Bristol Myers expands Breyanzi approval to marginal zone lymphoma
Endpoints News
2025-12-05
car-t
breyanzi
hodgkin lymphoma
non-hodgkin lymphoma
marginal zone lymphoma
Bristol Myers Squibb’s CAR-T therapy Breyanzi has become the first such treatment approved for patients with a rare, slow-growing type of non-Hodgkin lymphoma. The drug was approved Thursday for third-line patients with marginal zone lymphoma ...
Heightened COVID-19 Mortality Persists in CLL and Other Indolent Cancers
Hematology Advisor News
2025-12-05
cll
mcl
Patients with CLL, MCL, MDS, and myeloma experienced the greatest cumulative COVID-19 mortality across 39 months of follow-up.
CG Oncology Reports Promising Efficacy and Safety Data for Cretostimogene in High-Risk Non-Muscle Invasive Bladder Cancer Trials
CG Oncology
2025-12-05
bladder cancer
Cretostimogene shows promising efficacy and safety in treating high-risk NMIBC, with significant clinical trial results presented.Quiver AI SummaryCG Oncology, Inc. announced promising topline data for cretostimogene, an investigational immunotherapy for bladder cancer, from two clinical trials: BOND-003 Cohort P and CORE-008 Cohort A. In the BOND-003 trial, which involved patients with high-risk, BCG-unresponsive NMIBC, cretostimogene displayed
Oncolytics to test immunotherapy against aggressive pancreatic cancer
Rare Cancer News
2025-12-05
pivotal
pancreatic cancer
Oncolytics Biotech has cleared a significant hurdle in the U.S. to advance its unique viral immunotherapy, pelareorep, to a pivotal Phase 3 trial for advanced pancreatic cancer. The company has received clearance from the U.S. Food and Drug Administration (FDA) to launch a trial for pelareorep, an oncolytic virus designed to prime the immune system […] The post Oncolytics to test immunotherapy against aggressive pancreatic cancer appeared first on Rare Cancer News .
New Cretostimogene Grenadenorepvec Data Highlight its Potential to Become the Backbone Therapy for High-Risk Non-Muscle Invasive Bladder Cancer
CG Oncology
2025-12-05
bladder cancer
cretostimogene grenadenorepvec
Cretostimogene demonstrated HG-EFS at 3- 6- and 9-months of 95.7%, 84.6% and 80.4%, respectively, in HR BCG UR Ta/T1 Disease in BOND-003 Cohort PCORE-008 Cohort A Data in HR BCG-Naïve NMIBC demonstrates 88% CR and favorable safety with optimized administration Robust clinical pipeline that spans multiple late-stage studies across intermediate- and high-risk NMIBC IRVINE, Calif., Dec. 05, 2025 (GLOBE NEWSWIRE) -- CG Oncology, Inc. (NASDAQ: CGON), a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients with bladder cancer, today announced topline data from BOND-003 Cohort P and first results from CORE-008 Cohort A which demonstrated promising efficacy, safety and tolerability. These data will be presented today as Late-Breaking Abstracts at the Society of Urologic Oncology (SUO) 26th Annual Meeting.
uniQure (QURE) Is Down 17.3% After FDA Questions AMT-130 Data Sufficiency For BLA - Has The Bull Case Changed?
uniQure
2025-12-05
bla
amt-130
In late 2025, uniQure N.V. reported that the FDA’s final minutes from an October pre-BLA meeting indicated Phase I/II data for AMT-130 in Huntington’s disease are currently unlikely to support a Biologics License Application, prompting the company to seek a follow-up meeting in early 2026. This feedback introduces additional uncertainty around AMT-130’s regulatory path and timing, raising questions about one of uniQure’s most important pipeline drivers. We’ll now examine how the FDA’s...
Praxis soars on rare childhood epilepsy trial data
PharmaPhorum
2025-12-05
pivotal
Praxis looks like it could be filing for approval of two products next year, after its childhood epilepsy drug delivered a win in a pivotal trial.
FDA Pauses Denali’s Pompe Plans Due to Preclinical Safety Signals
BioSpace
2025-12-05
pompe disease
Hypersensitivity reactions in a mouse model prompted the agency to suspend Denali’s planned Phase I development for DNL952 for Pompe disease.
Praxis stops pivotal trial of rare epilepsy drug early for efficacy
Endpoints News
2025-12-05
pivotal
phase 2/3
Praxis Precision Medicines has stopped a trial of its sodium channel modulator in a rare brain disease early for efficacy. An interim analysis of the Phase 2/3 EMBOLD study found the once-daily pill, called relutrigine ...
Antibody Blocks Triple-Negative Breast Cancer Growth and Metastasis in Mice
GenEng News
2025-12-05
breast cancer
Preclinical study in cells and mouse models showed that an antibody targeting SFRP2 blocks multiple ways that triple-negative breast cancer cells grow, metastasize, evade the immune system, and resist chemotherapy. The post Antibody Blocks Triple-Negative Breast Cancer Growth and Metastasis in Mice appeared first on GEN - Genetic Engineering and Biotechnology News .
Praxis soars higher; Bristol Myers’ CAR-T therapy wins broader use
Biopharma Dive
2025-12-05
car-t
breyanzi
Praxis shares climbed another 40% on a pair of positive updates on two different drugs. Elsewhere, Bristol Myers’ Breyanzi was cleared for its fifth cancer indication and AstraZeneca expanded a rare disease deal.
Oncolytic Virus Rewires Immune Defense in Brain Tumors
Inside Precision Medicine
2025-12-05
glioblastoma
An engineered, PET-traceable herpes virus reprogrammed the glioblastoma microenvironment in preclinical models, boosting T-cell and NK-cell activity and prolonging survival. The post Oncolytic Virus Rewires Immune Defense in Brain Tumors appeared first on Inside Precision Medicine .
Pembrolizumab Associated With Durable Long-Term Survival in Previously Treated Melanoma
Hematology Advisor News
2025-12-05
melanoma
In this study, researchers investigated long-term outcomes from a real-world cohort of patients with stage IV melanoma treated with pembrolizumab following progression on prior therapies.
In Change That Pushed Pazdur Out, FDA May Lower Clinical Trial Requirements
BioSpace
2025-12-05
pivotal
Commissioner Marty Makary said that the FDA will soon start requiring only one pivotal trial, instead of two, for companies seeking approval for new drugs.
MYC Gene Fragment in Urine May Aid Bladder Cancer Diagnosis, Staging
Inside Precision Medicine
2025-12-05
bladder cancer
These research describe how the small fragment of the MYC gene, which is implicated in the development and progression of many types of cancer, could indicate the presence of both non–muscle-invasive and muscle-invasive bladder cancer. The post MYC Gene Fragment in Urine May Aid Bladder Cancer Diagnosis, Staging appeared first on Inside Precision Medicine .
Liquid Biopsy Firm Freenome Finds a Way to the Public Markets via $330M SPAC Merger
MedCity News
2025-12-05
colorectal cancer
Freenome’s SPAC merger agreement comes as the liquid biopsy developer prepares for potential 2026 commercialization of SimpleScreen, a colorectal cancer test currently under FDA review. If approved, the Freenome blood test would compete against Guardant Health’s liquid biopsy, Shield. The post Liquid Biopsy Firm Freenome Finds a Way to the Public Markets via $330M SPAC Merger appeared first on MedCity News .
Wugen to Present Correlative Data and Long-Term Follow-Up Updates for Off-the-Shelf, Allogeneic CD7-Targeted CAR-T Cell therapy at the 2025 ASH Annual Meeting
Wugen Inc.
2025-12-05
cd7
car-t
pivotal
phase 1/2
allogeneic
cell therapy
cell therapies
hematological malignancies
Results from the Phase 1/2 Trial are a Foundation for the Ongoing Pivotal Study of Soficabtagene Geleucel for R/R T-Cell ALL/ LBL in Pediatric and Adult Patients ST. LOUIS, MO, Dec. 5, 2025 – Wugen, Inc., a clinical-stage U.S. biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological malignancies, today announced an... The post Wugen to Present Correlative Data and Long-Term Follow-Up Updates for Off-the-Shelf, Allogeneic CD7-Targeted CAR-T Cell therapy at the 2025 ASH Annual Meeting appeared first on Wugen .
Daily deucrictibant keeps hereditary angioedema attack rate low for up to 34 months
Healio
2025-12-05
hereditary angioedema
ORLANDO — Decreases in hereditary angioedema attack rates seen with daily oral deucrictibant at 13 weeks stayed at a low level for up to 34 months, according to open-label extension study results.These data on deucrictibant (Pharvaris) were presented at the American College of Allergy, Asthma & Immunology Annual Scientific Meeting.
Intellia Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Intellia Therapeutics Inc.
2025-12-05
crispr
gene editing
CAMBRIDGE, Mass. , Dec. 05, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that on December 1, 2025 , it awarded inducement grants to three new
UniQure slides further on outlook for Huntington’s gene therapy
Biopharma Dive
2025-12-04
gene therapy
Finalized minutes from a late-October meeting with the FDA confirm the agency doesn't see the data collected so far as enough to support an approval filing.
Lymphir Now Available for R/R Cutaneous T-Cell Lymphoma
Hematology Advisor News
2025-12-04
t-cell lymphoma
Lymphir (denileukin diftitox-cxdl) is now available for the treatment of relapsed or refractory Stage I-III cutaneous T-cell lymphoma.
UniQure Hammered, Again, On Bleak Prospects For Gene Therapy
uniQure
2025-12-04
gene therapy
UniqQre stock plunged, again, Thursday after the FDA said it doesn't have enough evidence for a Huntington's approval.
AstraZeneca advances haematology and cell therapy ambition with largest-ever presence at ASH 04 December 2025
AstraZeneca
2025-12-04
cell therapy
REGENXBIO Reports Second Quarter 2025 Financial Results and Operational Highlights
REGENXBIO
2025-12-04
financial results
REGENXBIO Presents Positive Twelve-Month Pivotal Data from Phase I/II/III CAMPSIITE® Trial of RGX-121 for Treatment of MPS II
REGENXBIO
2025-12-04
mps ii
pivotal
rgx-121
FDA fast-tracks zotiraciclib for treating aggressive brain cancer
Rare Cancer News
2025-12-04
glioma
fast track
The U.S. Food and Drug Administration (FDA) has granted fast track status to zotiraciclib (ZTR/TG02) — an oral medication that targets pathways in the body that are used by tumor cells — as a potential treatment for certain aggressive forms of glioma. The designation, which aims to expedite the development of therapies with the potential […] The post FDA fast-tracks zotiraciclib for treating aggressive brain cancer appeared first on Rare Cancer News .
FDA Approves First CAR T-Cell Therapy for Marginal Zone Lymphoma In the US
FDA Press
2025-12-04
car t
breyanzi
cell therapy
marginal zone lymphoma
chimeric antigen receptor
The U.S. Food and Drug Administration today approved a new indication for Breyanzi (Lisocabtagene maraleucel) as the first Chimeric Antigen Receptor (CAR) T-cell therapy in the U.S.
Fuzuloparib Extends PFS as Maintenance Therapy for Advanced Ovarian Cancer
Hematology Advisor News
2025-12-04
ovarian cancer
FZOCUS-1 assessed the safety and efficacy of fuzuloparib alone and in combination with apatinib for patients with newly diagnosed or advanced ovarian cancer.
American Academy of Orthopaedic Surgeons Releases Inaugural Musculoskeletal Tumor Registry Annual Report
PR Newswire
2025-12-04
sarcoma
New data resource helps transform sarcoma care through real-world evidence and clinical insights ROSEMONT, Ill., Dec. 4, 2025 /PRNewswire/ -- The American Academy of Orthopaedic Surgeons' (AAOS) Musculoskeletal Tumor Registry (MsTR) has released its inaugural Annual Report, marking the...
UniQure stock falls after FDA's notes on pre-application of AMT-130
uniQure
2025-12-04
amt-130
gene therapy
uniQure N.V. (QURE) shares dropped after receiving the final meeting notes from the FDA concerning its pre-Biologics License Application for AMT-130, a gene therapy being investigated for Huntington’s disease. The FDA indicated in these notes that the data from the phase I/II trials of AMT-130 are unlikely to serve as the main evidence needed for a...
Blood Cancer Therapeutics Reach Critical Commercial and Clinical Inflection Points
PR Newswire
2025-12-04
leukemia
Issued on behalf of GT Biopharma, Inc. USA News Group News Commentary VANCOUVER, BC, Dec. 4, 2025 /PRNewswire/ -- Triple-therapy immunotherapy approaches are demonstrating complete leukemia elimination in preclinical models by reprogramming how cancer cells die to trigger powerful immune...
Kura Oncology: A Cautious Buy (NASDAQ:KURA)
Kyowa Kirin
2025-12-04
aml
Kura Oncology (KURA) is rated a cautious Speculative Buy, driven by Komzifti's FDA approval and frontline AML potential.
Adicet Bio Announces Positive Preliminary Data from ADI-001 Phase 1 Study in Patients with Lupus Nephritis (LN) and Systemic Lupus Erythematosus (SLE)
Adicet Bio
2025-12-04
lupus
FDA panel votes against approving interatrial shunt for heart failure
Healio
2025-12-04
heart failure
The FDA’s Circulatory System Devices Panel voted not to recommend approving use of an interatrial shunt to improve heart failure symptoms and prognosis.In a 0-15 vote, the committee determined risk from the interatrial shunt system (V-Wave Ventura, Johnson & Johnson) outweighed the benefits in patients with NYHA class III HF with left ventricular ejection fraction of 40% or less who
Kura Oncology Secures FDA Approval of Leukemia Drug
Kyowa Kirin
2025-12-04
leukemia
SAN DIEGO - Patients with a specific form of fast-growing blood and bone marrow cancer will soon have a new therapeutic drug treatment option following the regu
REGENXBIO Announces Pivotal Program for Surabgene Lomparvovec in Diabetic Retinopathy
REGENXBIO
2025-12-04
pivotal
diabetic retinopathy
AML Deaths in MDS Decline, but Racial and Geographic Disparities Persist
Hematology Advisor News
2025-12-04
aml
The mortality-to-incidence ratio initially increased in the early 2000s, declined sharply between 2008 and 2011, and remained stable thereafter.
Ianalumab may help patients with CLL stop daily, indefinite treatment with ibrutinib
Healio
2025-12-04
cll
leukemia
ianalumab
chronic lymphocytic leukemia
Bruton’s tyrosine kinase inhibitors such as ibrutinib have significantly improved outcomes for chronic lymphocytic leukemia, but they can cause long-term toxicities, and patients must remain on them indefinitely.Adding ianalumab (VAY736, Novartis), a “potent” B cell-activating factor receptor (BAFF-R) antibody, to therapy could allow patients to stop daily ibrutinib, according
American Cancer Society Updates Cervical Cancer Screening Guideline: Major Changes Include Self-Collection for HPV Testing and Guidance on Exiting Screening
PR Newswire
2025-12-04
cervical cancer
ACS researchers aim to reduce cervical cancer deaths with new screening recommendations ATLANTA, Dec. 4, 2025 /PRNewswire/ -- The American Cancer Society (ACS) released updated guidelines today for cervical cancer screening, reflecting advances in disease detection and accessibility in...
Nationwide Children’s SLC6A1 gene therapy shows early promise
Endpoints News
2025-12-04
gene therapy
A pioneering gene therapy from Nationwide Children's Hospital is bringing rare optimism to a field that has long struggled to develop such treatments for certain neurodevelopment disorders. In September, 8-year-old Maxwell Freed became the first ...
REGENXBIO to Host Conference Call on August 7 to Discuss Second Quarter 2025 Financial Results and Operational Highlights
REGENXBIO
2025-12-04
financial results
8-K - uniQure N.V. (0001590560) (Filer)
SEC Filing (8-K)
2025-12-04
uniqure
Filed: 2025-12-04 AccNo: 0001104659-25-118292 Size: 180 KB Item 7.01: Regulation FD Disclosure Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
Will CG Oncology’s (CGON) Board Refresh and Cretostimogene Data Shape Its Bladder Cancer Strategy?
CG Oncology
2025-12-04
bladder cancer
CG Oncology announced that former Blueprint Medicines executive Christina Rossi has joined its board, replacing Simone Song, and confirmed that late-breaking clinical data on its investigational bladder cancer therapy cretostimogene were recently presented at the Society of Urologic Oncology Annual Meeting. The combination of leadership change and emerging Phase 3 and Phase 2 data for cretostimogene highlights CG Oncology’s focus on advancing its bladder cancer pipeline. Against this...
FDA Minutes Confirm Challenging Path Ahead for UniQure’s Huntington’s Gene Therapy
BioSpace
2025-12-04
bla
amt-130
gene therapy
Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s Phase I/II studies of AMT-130 are “unlikely” to provide the primary evidence to support a biologics license application.
REGENXBIO Announces FDA Review Extension of BLA for RGX-121 to Treat Patients with MPS II
REGENXBIO
2025-12-04
bla
mps ii
rgx-121
Acepodia Announces NMPA Approval of ACE1831 for IgG4-RD Phase Ib/IIa Clinical Trial,Marking a Major Step Forward in Its Global Multi-Country Development Strategy-Acepodia® | POWERFUL, ACCESSIBLE CELL THERAPIES FOR PATIENTS WITH CANCER
Acepodia
2025-12-04
allogeneic
cell therapy
cell therapies
Acepodia Announces NMPA Approval of ACE1831 for IgG4-RD Phase Ib/IIa Clinical Trial,Marking a Major Step Forward in Its Global Multi-Country Development StrategyAcepodia Inc. (6976.TT) today announced that its lead allogeneic cell therapy candidate ACE1831 has officially received approval from China’s National Medical Products Administration (NMPA) to initiate a Phase Ib/IIa clinical trial for IgG4-related diseas..
Financial Results of Astellas for Q2 YTD/FY2025
astellas
2025-12-04
financial results
REGENXBIO Announces Publication of Preclinical Results Demonstrating Functional Benefits of Novel Microdystrophin Construct in RGX-202 Investigational Gene Therapy for Duchenne Muscular Dystrophy
REGENXBIO
2025-12-04
gene therapy
duchenne muscular dystrophy
uniQure Faces Setback in Gene Therapy Approval
uniQure
2025-12-04
amt-130
gene therapy
uniQure ( ($QURE) ) has shared an announcement. On December 4, 2025, uniQure announced a regulatory update on its investigational gene therapy, AMT-130, for Hunting...
Adicet Bio Reports Second Quarter 2025 Financial Results and Provides Business Updates
Adicet Bio
2025-12-04
financial results
uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease
uniQure
2025-12-04
bla
amt-130
gene therapy
LEXINGTON, Mass. and AMSTERDAM, Dec. 04, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the company received final meeting minutes from the U.S. Food and Drug Administration (FDA) regarding a pre-Biologics License Application (BLA) meeting held on October 29, 2025 to discuss the application for AMT-130, an investigational gene therapy for Huntington’s disease (HD)
FDA Minutes Confirm Challenging Path Ahead for UniQure’s Huntington’s Gene Therapy
uniQure
2025-12-04
bla
amt-130
gene therapy
Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s Phase I/II studies of AMT-130 are “unlikely” to provide the primary evidence to support a biologics license application.
Guess the Therapy Answer December 3, 2025
CGTLive
2025-12-04
gene therapy
Cell and Gene Therapy Live
Lilly gets approval for earlier use of Jaypirca in CLL
PharmaPhorum
2025-12-04
cll
Eli Lilly's fast-growing Jaypirca has won a new FDA approval as an earlier treatment for relapsed/refractory chronic lymphocytic leukaemia.
UniQure slides further on outlook for Huntington’s gene therapy
uniQure
2025-12-04
gene therapy
Finalized minutes from a late-October meeting with the FDA confirm the agency doesn't see the data collected so far as enough to support an approval filing.
Adicet Bio Reports Third Quarter 2025 Financial Results and Provides Business Updates
Adicet Bio
2025-12-04
financial results
San Diego Biotech Kura Snags FDA Green Light For High-Stakes Leukemia Pill
Kyowa Kirin
2025-12-04
aml
leukemia
San Diego's Kura wins FDA approval for Komzifti, an oral AML therapy.
Ischemic heart disease-related mortality in adults with OSA increased from 1999 to 2022
Healio
2025-12-03
ischemic heart disease
Among U.S. adults with obstructive sleep apnea, there has been an increase in deaths related to ischemic heart disease from 1999 to 2022, according to results published in Respiratory Medicine.“These findings really emphasize that obstructive sleep apnea should be viewed as a serious cardiovascular risk multiplier, not just a sleep disorder,” Muhammad Ali Tariq, MBBS, physician at Dow
Hemophilia gene therapies are struggling on the market, even as innovation soars
BioMarin Pharmaceutical
2025-12-03
gene therapy
gene therapies
The business case for hemophilia gene therapy still isn’t adding up due to persistent market barriers.
Data at Work: How Advanced Analytics Are Transforming Cell and Gene Therapy Manufacturing
MedCity News
2025-12-03
gene therapy
The growing use of advanced analytics and artificial intelligence (AI) is reshaping this landscape by enabling data-driven process control, predictive manufacturing, and greater transparency across the development lifecycle. The post Data at Work: How Advanced Analytics Are Transforming Cell and Gene Therapy Manufacturing appeared first on MedCity News .
Pharvaris Drug for Rare Swelling Disease Meets Phase 3 Goals; FDA Filing Planned for 2026
MedCity News
2025-12-03
hereditary angioedema
Pharvaris’s deucrictibant met the main and secondary goals of its Phase 3 test as an on-demand treatment for swelling attacks caused by the rare disease hereditary angioedema. Pharvaris’s capsule could compete against Kalvista Pharmaceuticals’ Ekterly, an HAE pill approved by the FDA over the summer. The post Pharvaris Drug for Rare Swelling Disease Meets Phase 3 Goals; FDA Filing Planned for 2026 appeared first on MedCity News .
Longer Travel Time to Treatment Facility Linked to Improved Survival in Prostate Cancer
Hematology Advisor News
2025-12-03
prostate cancer
In this retrospective cohort study, researchers investigated the association between travel time and mortality in prostate cancer patients.
NKGen Biotech, Inc. Issues Statement on Recent Board Appointments at Majority-Owned Korean Affiliate NKGen Korea Co., Ltd.
nkgen biotech
2025-12-03
allogeneic
autologous
SANTA ANA, Calif., December 3, 2025 – NKGen Biotech, Inc. (OTC: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer (“NK”) cell therapeutics and the majority owner of NKGen Biotech Korea Co., Ltd. (“NKGen Korea”), today issued the following statement regarding recent […] The post NKGen Biotech, Inc. Issues Statement on Recent Board Appointments at Majority-Owned Korean Affiliate NKGen Korea Co., Ltd. first appeared on NKGen Biotech .
New therapy DT2216 tackles tough-to-treat ovarian cancer
Rare Cancer News
2025-12-03
ovarian cancer
A new experimental therapy, DT2216, has begun testing in a clinical trial for people with hard-to-treat ovarian cancer, a significant step forward announced by Dialectic Therapeutics. The Phase 1b/2 study (NCT06964009) is targeting patients whose disease is relapsed or refractory, meaning it has either returned after initial treatment or failed to respond to previous therapies, […] The post New therapy DT2216 tackles tough-to-treat ovarian cancer appeared first on Rare Cancer News .
One-time infusion well tolerated, lowers monthly hereditary angioedema attack rate
Healio
2025-12-03
ntla-2002
hereditary angioedema
ORLANDO — Among adults with hereditary angioedema, a one-time infusion of lonvoguran ziclumeran immediately lowered the average monthly attack rate and was well tolerated up to 3 years, according to pooled study results.These data on lonvoguran ziclumeran (lonvo-z, NTLA-2002; Intellia Therapeutics) were presented at the American College of Allergy, Asthma & Immunology Annual Scientific
Orion's €180M milestone payment; Iolyx partners dry eye candidate
Endpoints News
2025-12-03
prostate cancer
Plus, news about QurAlis, Clene, ADC Therapeutics and Junevity: 💶 Orion to receive €180M milestone payment: The cash will come from Bayer, on the back of sales of prostate cancer therapy Nubeqa. Subsequently, Orion also ...
Protara Therapeutics Announces Updated Interim Data from Phase 2 ADVANCED-2 Trial of TARA-002 in BCG-Naïve NMIBC Patients
protara therapeutics
2025-12-03
complete response
TARA-002 demonstrates 72% complete response rate at any time in BCG-Naïve patients TARA-002 demonstrates a 69% complete response rate at the 6-month landmark and a 50% complete response rate at the 12-month landmark in BCG-Naïve patients Favorable safety and tolerability profile observed with no
Cutaneous T-cell Lymphoma Market Expected to Experience Major Growth by 2032, According to DelveInsight | Innate Pharma, Galderma R&D, Kyowa Kirin, Citius Pharma, Soligenix, 4SC AG, oligenix, Ligand
Kyowa Kirin
2025-12-03
t-cell lymphoma
The Key Cutaneous T-cell Lymphoma Companies in the market include - Citius Pharmaceuticals, Soligenix, 4SC AG, Innate Pharma, Galderma R&D, Kyowa Kirin, Inc, oligenix, Ligand Pharmaceuticals, Bausch Health Americas, Inc., BioCryst Pharmaceuticals, No...
U.S. FDA approves expanded indication for Lilly's Jaypirca (pirtobrutinib), the first and only non-covalent (reversible) BTK inhibitor, for adults with relapsed or refractory CLL/SLL previously treated with a covalent BTK inhibitor
PR Newswire
2025-12-03
cll
Approval is based on results from the BRUIN CLL-321 trial, the only randomized Phase 3 study in CLL/SLL in which all patients were previously treated with a covalent BTK inhibitor This expanded indication represents a substantial increase in the number of CLL/SLL patients who may benefit...
Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy
Capricor Therapeutics
2025-12-03
pivotal
deramiocel
duchenne muscular dystrophy
Evaluating CAR-T KYV-101 for Generalized Myasthenia Gravis
CGTLive
2025-12-03
car-t
kyv-101
myasthenia gravis
Naji Gehchan, MD, MBA, the chief medical and development officer of Kyverna Therapeutics, went over new data the company presented at AANEM’s 2025 meeting.
Pharvaris’ pill succeeds in late-stage trial for rare swelling disorder
Biopharma Dive
2025-12-03
hereditary angioedema
Phase 3 data could make the Dutch biotech's drug competitive in the increasingly crowded market of hereditary angioedema treatments.
Breast Cancer Genomic Instability Appears Decades Before Diagnosis
Inside Precision Medicine
2025-12-03
breast cancer
Korean researchers have revealed that the first seeds of breast cancer could be sown decades before it is discovered, as far back as early puberty, after studying whole genome sequences from more than 1000 tumors. The post Breast Cancer Genomic Instability Appears Decades Before Diagnosis appeared first on Inside Precision Medicine .
What Were the Most Significant Inflection Points or Breakthroughs in Cell and Gene Therapy?
CGTLive
2025-12-03
gene therapy
Deborah Phippard, PhD, and Renier Brentjens, MD, PhD, discussed major events in the past quarter century of cell and gene therapy research.
AZ uses a priority review voucher for baxdrostat filing
PharmaPhorum
2025-12-03
priority review
Seeking a lead over rival Mineralys, AstraZeneca is using a priority review voucher for its US filing for uncontrolled hypertension drug baxdrostat.
Common Childhood Virus Causes DNA Damage Driving Bladder Cancer
Inside Precision Medicine
2025-12-03
bladder cancer
A new study shows that the BK virus, which up to 80% of the population get infected with during childhood, can drive the development of bladder cancer down the line. The post Common Childhood Virus Causes DNA Damage Driving Bladder Cancer appeared first on Inside Precision Medicine .
FDA expands pirtobrutinib use in chronic lymphocytic leukemia, small lymphocytic lymphoma
Healio
2025-12-03
leukemia
accelerated approval
small lymphocytic lymphoma
chronic lymphocytic leukemia
The FDA has approved pirtobrutinib for certain adults with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma.The new indication applies to adults who previously have been treated with a covalent Bruton tyrosine kinase (BTK) inhibitor, expanding on the 2023 accelerated approval for pirtobrutinib (Jaypirca, Eli Lilly and Co.) for patients who had received at least
Capricor soars on positive results for Duchenne cell therapy
Biopharma Dive
2025-12-03
cell therapy
Shares quadrupled now that the company appears on track to reverse an earlier FDA rejection of what could be the first treatment for heart-related complications of Duchenne.
8-K - CAPRICOR THERAPEUTICS, INC. (0001133869) (Filer)
SEC Filing (8-K)
2025-12-03
capricor therapeutics
Filed: 2025-12-03 AccNo: 0001104659-25-117947 Size: 211 KB Item 7.01: Regulation FD Disclosure Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
GenSight Biologics Announces Regulatory Approval for GS010/LUMEVOQ® REVISE Dose-Ranging Study in France
GenSight Biologics
2025-12-03
lumevoq
eXmoor Pharma and UCL Collaborate to Accelerate the Advancement of Cell and Gene Therapies
GenEng News
2025-12-03
gene therapies
Researchers at UCL say they will benefit from structured early engagement with eXmoor’s teams of experts, which should help shape more practical and commercially aware development strategies. The post eXmoor Pharma and UCL Collaborate to Accelerate the Advancement of Cell and Gene Therapies appeared first on GEN - Genetic Engineering and Biotechnology News .
ImpriMed to Present Ex Vivo Drug Sensitivity Data in Acute Myeloid Leukemia in Oral Presentation at American Society of Hematology Annual Meeting
PR Newswire
2025-12-03
leukemia
acute myeloid leukemia
Studies Demonstrate xCellSense® Platform's Ability to Predict Clinical Response and Enable Patient Stratification for Drug Development. PALO ALTO, Calif., Dec. 3, 2025 /PRNewswire/ -- ImpriMed, a precision oncology CRO specializing in ex vivo drug sensitivity testing for hematologic...
Pharvaris’ hereditary angioedema drug gets Phase 3 win
Endpoints News
2025-12-03
hereditary angioedema
Pharvaris’ on-demand treatment for hereditary angioedema attacks can diminish symptoms significantly faster than placebo, according to topline data from a Phase 3 trial released Wednesday. The drug, called deucrictibant, is an ...
Focused ultrasound safely opens blood-brain barrier, may improve survival in glioblastoma
Healio
2025-12-03
phase 1/2
glioblastoma
Use of MRI-guided focused ultrasound with standard chemotherapy is safe and may improve outcomes for people with glioblastoma, results of a phase 1/2 trial showed.Patients treated with this regimen survived nearly 40% longer than those who received chemotherapy alone. No treatment-related deaths occurred, and only one adverse event deemed related to focused ultrasound exceeded grade 2.
Black Diamond reports mid-stage lung cancer data, continues its hunt for a partner
Endpoints News
2025-12-03
lung cancer
Black Diamond Therapeutics reported preliminary Phase 2 data for its EGFR inhibitor in lung cancer, but said it will need to find a partner to be able to move into a registrational program. Instead, the ...
Harmony’s Dravet Drug Halves Monthly Seizures in Late-Stage Study
BioSpace
2025-12-03
dravet syndrome
The Phase III, open-label extension study suggests EPX-100 has a “positive” risk/benefit profile for the treatment of Dravet syndrome, analysts at H.C. Wainwright & Co. said Tuesday.
Capricor Heads Back to FDA With Pivotal Results for DMD Cardiomyopathy Therapy
BioSpace
2025-12-03
dmd
pivotal
deramiocel
cell therapy
cardiomyopathy
dmd cardiomyopathy
Six months after receiving a surprise rejection due to what the FDA called “lack of substantial evidence of effectiveness,” Capricor’s cell therapy deramiocel showed significant benefits in upper-limb function and slowed decline in cardiac function in a Phase III trial.
FDA clears trial of 'vectorised' ALS antibody from VectorY
PharmaPhorum
2025-12-03
amyotrophic lateral sclerosis
VectorY has been cleared by the FDA to start human trials of a drug against a target implicated in up to 97% of amyotrophic lateral sclerosis cases.
Potential Cancer Therapy Through New Cell Death Pathway, Mitoxyperilysis
Inside Precision Medicine
2025-12-03
melanoma
St. Jude scientists have identified a new form of cell death, mitoxyperilysis, triggered by combined metabolic disruption and innate immune activation—and shown it can regress melanoma tumors, revealing a promising new direction for cancer therapy. The post Potential Cancer Therapy Through New Cell Death Pathway, Mitoxyperilysis appeared first on Inside Precision Medicine .
Cataract surgery necessary in patients with dementia
Healio
2025-12-03
diabetic retinopathy
Age-related dementia, cataract, age-related macular degeneration, glaucoma, diabetic retinopathy and hearing loss are all more common in the elderly patient. The average age of onset of a cataract in the U.S. is about 60 years and for cataract/IOL surgery, 74 years. The average age of onset for age-related dementia is 70 years, for glaucoma 55 years, for AMD 60 years, and for diabetic
With Phase 3 Duchenne success, Capricor plots path forward after FDA rejection
Endpoints News
2025-12-03
cell therapy
duchenne muscular dystrophy
Capricor Therapeutics said that its cell therapy for Duchenne muscular dystrophy met both a primary and key secondary endpoint in a Phase 3 trial, boosting the company’s odds for approval as it returns to the ...
Postmenopausal Estrone and Obesity Drive Deadly ER+ Breast Cancer
Inside Precision Medicine
2025-12-03
breast cancer
An analysis of prior research has identified estrone, the dominant estrogen present in women after menopause, as the key driver of the inflammation, tumor progression, and metastasis of estrogen receptor–positive (ER+) breast cancer in postmenopausal women with obesity. The post Postmenopausal Estrone and Obesity Drive Deadly ER+ Breast Cancer appeared first on Inside Precision Medicine .
Sonrotoclax Gets Priority Review for R/R Mantle Cell Lymphoma
Hematology Advisor News
2025-12-03
priority review
mantle cell lymphoma
A next-generation BCL2 inhibitor, sonrotoclax, receives FDA Priority Review for previously treated relapsed or refractory mantle cell lymphoma.
HIV Remission with Allo-SCT Endures in Heterozygous CCR5Δ32 Patient
Inside Precision Medicine
2025-12-03
allogeneic
This is the first documented instance of a sustained HIV-negative state after allogeneic stem-cell transplantation (allo-SCT) in both a donor and recipient who lacked complete resistance to HIV. The post HIV Remission with Allo-SCT Endures in Heterozygous CCR5Δ32 Patient appeared first on Inside Precision Medicine .
8-K - CAPRICOR THERAPEUTICS, INC. (0001133869) (Filer)
SEC Filing (8-K)
2025-12-03
capricor therapeutics
Filed: 2025-12-03 AccNo: 0001104659-25-117981 Size: 2 MB Item 7.01: Regulation FD Disclosure Item 9.01: Financial Statements and Exhibits
Protego Closes $130M Series B to Fund Pivotal Trial of AL Amyloidosis Candidate
GenEng News
2025-12-03
pivotal
PROT-001 is an oral small molecule kinetic stabilizer of lambda light chains (λLCs). PROT-001 reflects Protego’s approach of developing small-molecule pharmacological chaperones that directly bind to and modulate target protein stability. These chaperones get their name by acting as cellular “guides,” ensuring proteins fold correctly. The post Protego Closes $130M Series B to Fund Pivotal Trial of AL Amyloidosis Candidate appeared first on GEN - Genetic Engineering and Biotechnology News .
Granata Bio Reports Strong Enrollment Momentum in Pivotal Phase III GRACE Study of Investigational Gonadotropin for Assisted Reproductive Technology
PR Newswire
2025-12-03
pivotal
BOSTON, Dec. 3, 2025 /PRNewswire/ -- Granata Bio Corporation, a biotechnology company advancing innovative reproductive health solutions, announced strong momentum in its pivotal Phase III clinical study, with multiple subjects already screened to evaluate the safety and efficacy of its...
FDA selects enGene’s bladder cancer therapy for development program By Investing.com
enGene Google
2025-12-02
bladder cancer
FDA selects enGene’s bladder cancer therapy for development program
Tirzepatide heart effects do not vary by heart failure status
Healio
2025-12-02
heart failure
NEW ORLEANS — Tirzepatide conferred similar benefit for many cardiovascular outcomes to dulaglutide in patients with diabetes regardless of whether they also had heart failure, according to new data from the SURPASS-CVOT trial. However, in the outcomes of all-cause death/hospitalization or urgent visit for HF and all-cause death alone, tirzepatide (Mounjaro, Eli Lilly) was superior to
Treatment of Multiple Myeloma at Specialized Clinic Tied to Improved Survival
Hematology Advisor News
2025-12-02
multiple myeloma
Median progression-free survival was 22 months, overall survival was 152 months among patients with newly diagnosed MM.
GenSight Biologics Announces Regulatory Approval for GS010/LUMEVOQ® REVISE Dose-Ranging Study in France
GenSight Biologics
2025-12-02
gene therapies
PARIS, December 02, 2025--Regulatory News: GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that the French medicines safety agency ANSM (Agence nationale de sécurité du médicament et des produits de santé) has authorized the dose-ranging study REVISE, which will investigate the efficacy and
enGene (Nasdaq: ENGN) gene therapy chosen for FDA CDRP, backing BLA plans in second half 2026
enGene Google
2025-12-02
bla
gene therapy
enGene’s detalimogene enters the FDA’s CDRP to support commercial-scale CMC readiness, after a 62% six‑month CR rate and favorable safety in 125 patients.
Sangamo Therapeutics Receives U.S. FDA Fast Track Designation for ST-503 for the Treatment of Small Fiber Neuropathy
Sangamo BioSciences
2025-12-02
fast track
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Optimized Conditioning Regimens Improve FIX Restoration in Hemophilia Models
Hematology Advisor News
2025-12-02
hemophilia b
Combining granulocyte colony-stimulating factor, plerixafor, and timed 5-fluorouracil produced durable FIX expression and phenotypic rescue in hemophilia B mice.
enGene detalimogene voraplasmid selected for FDA manufacturing pilot program
enGene Google
2025-12-02
detalimogene voraplasmid
enGene (ENGN) announced that the FDA has selected detalimogene voraplasmid to participate in the Chemistry, Manufacturing, and Controls Development and Readiness Pi...
FDA approves Itvisma in spinal muscular atrophy for patients aged 2 years and older
Healio
2025-12-02
adeno-associated virus
spinal muscular atrophy
The FDA has approved an adeno-associated virus 9 gene replacement therapy to treat individuals aged 2 years and older with spinal muscular atrophy and a confirmed mutation of the survival motor neuron 1 gene, according to the manufacturer.In a press release, Novartis said the FDA’s approval positions Itvisma (onasemnogene abeparvovec-brve) as the first and only gene replacement therapy
Tinlarebant reduces lesion growth rate in Stargardt disease
Healio
2025-12-02
fast track
stargardt disease
breakthrough therapy
Belite Bio announced positive topline results from the phase 3 DRAGON trial of Tinlarebant in patients with Stargardt disease. Tinlarebant (LBS-008) is an oral therapy designed to reduce the accumulation of cytoxic bisretinoids, toxins that cause retinal disease in Stargardt disease type 1. The drug has obtained breakthrough therapy, fast track and rare pediatric disease designations from the
Baxdrostat New Drug Application accepted under FDA Priority Review in the US for patients with hard-to-control hypertension 02 December 2025
AstraZeneca
2025-12-02
priority review
FDA selects enGene’s bladder cancer therapy for development program By Investing.com
enGene Google
2025-12-02
bladder cancer
FDA selects enGene’s bladder cancer therapy for development program
enGene Shares Rise After FDA Taps Bladder Cancer Therapy for CMC Pilot
enGene Google
2025-12-02
gene therapy
bladder cancer
detalimogene voraplasmid
enGene Holdings Inc. (NASDAQ:ENGN) traded 2% higher on Tuesday after the company revealed that the FDA has selected its bladder cancer therapy for a specialized program aimed at accelerating manufacturing readiness. The gene-medicine developer announced that its lead non-viral gene therapy candidate, detalimogene voraplasmid, has been accepted into the FDA’s Chemistry, Manufacturing, and Controls (CMC) […]
Nkarta to Participate in Evercore Healthcare Conference
nkarta
2025-12-02
cell therapies
SOUTH SAN FRANCISCO, Calif., Dec. 02, 2025 (GLOBE NEWSWIRE) -- Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat autoimmune diseases, today announced its participation in the Evercore 8 th Annual Healthcare
Lower-Dose Emicizumab May Offer Comparable Protection in Hemophilia A
Hematology Advisor News
2025-12-02
hemophilia a
All participants opted to extend their dosing interval, typically from 7 to 14 days, without increasing individual doses, and the median individualized dose-free interval was 20 days.
enGene stock rises after FDA selects bladder cancer therapy for CMC pilot program By Investing.com
enGene Google
2025-12-02
bladder cancer
enGene stock rises after FDA selects bladder cancer therapy for CMC pilot program
Metastatic Melanoma Survival Worse in Hispanic Patients Than Non-Hispanic Patients
Hematology Advisor News
2025-12-02
melanoma
In this study, researchers explored disparities in metastatic cutaneous melanoma survival following the introduction of immunotherapies.
Sun Auto Network Raises $50,000 for Susan G. Komen to Drive Progress in Breast Cancer Research
PR Newswire
2025-12-02
breast cancer
Sun Auto Tire & Service celebrated the expansion of its nationwide October giving campaign for Susan G. Komen, presenting a $50,000 donation to advance breast cancer research and patient programs nationwide. This effort was further supported through Sun Auto's partnership with RelaDyne....
Around the Helix: Cell and Gene Therapy Company Updates – November 5, 2025
CGTLive
2025-12-02
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
enGene’s Detalimogene Chosen for FDA Manufacturing Pilot Program
enGene Google
2025-12-02
gene therapy
detalimogene voraplasmid
enGene Holdings ( ($ENGN) ) has provided an announcement. enGene Holdings announced that its investigational gene therapy, detalimogene voraplasmid, has been select...
J&J gets EU approval for Imaavy in gMG
PharmaPhorum
2025-12-02
myasthenia gravis
J&J's Imaavy has become the first FcRn blocker to be approved in the EU for both adolescent and adult patients with generalised myasthenia gravis.
enGene’s Detalimogene Selected for FDA Manufacturing Pilot Program to Support Manufacturing Readiness
enGene Google
2025-12-02
gene therapy
detalimogene voraplasmid
BOSTON & MONTREAL, December 02, 2025--enGene Holdings Inc. (Nasdaq: ENGN or "enGene" or the "Company"), a clinical-stage, non-viral genetic medicines company, today announced that the U.S. Food and Drug Administration (FDA) has selected detalimogene voraplasmid (also known as detalimogene and previously EG-70) to participate in the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program. Detalimogene is a novel, investigational, non-viral gene therapy for pati
Study Manager – Translational
coave therapeutics
2025-12-02
ind
gene therapy
The Study manager Translational plays an important role in the development of Coave’s therapeutic programs by conducting IND-enabling preclinical studies that bridge Coave’s gene therapy lead candidates to clinical development. He/She is responsible for designing, planning and implementing IND-enabling preclinical studies including distribution and toxicology profile of our therapeutic products required to support clinical study authorization. The individual ensures that studies, outsourced to contract research organizations (CROs), are executed correctly, on time, within budget and with the quality standards to meet regulatory requirements.
House passes bill to reauthorize rare pediatric PRV program; Senate next
Endpoints News
2025-12-02
priority review
The US House of Representatives on Monday afternoon passed a bill that would reauthorize the FDA's rare pediatric priority review voucher (PRV) incentive program, which expired in late 2024. The program, created in 2012, gave ...
enGene stock rises after FDA selects bladder cancer therapy for CMC pilot program By Investing.com
enGene Google
2025-12-02
bladder cancer
enGene stock rises after FDA selects bladder cancer therapy for CMC pilot program
Colorectal Cancer Market Set to Grow Substantially Through 2034, DelveInsight Projects | Takeda, Genentech/Seagen, AstraZeneca and Daiichi Sankyo, Merck, Amgen, Daiichi Sankyo Company, Mirati Therapeu
Genentech
2025-12-02
colorectal cancer
The Key Colorectal Cancer Companies in the market include - Plus Therapeutics, Processa Pharmaceutic als, Shanghai Henlius Biotech, Apollomics, Novartis Pharmaceuticals, Sumitomo Pharma Oncology, Purple Biotech Ltd., Nektar Therapeutics, ALX Oncology...
Prior mental health diagnosis may predict neuropsychiatric outcomes in childhood lupus
Healio
2025-12-02
lupus
CHICAGO — A previous diagnosis of a mental health disorder may predict neuropsychiatric complications in children with systemic lupus erythematosus, according to data presented at ACR Convergence 2025.“Childhood-onset systemic lupus erythematosus is the second most common chronic rheumatologic disease of childhood,” Ekemini Ogbu, MD, MSc, director of neuroinflammatory disease
SUO and Ferring Pharmaceuticals Announce the SUO Non-Muscle Invasive Bladder Cancer (NMIBC) Fellows Research Grant
Ferring Pharmaceuticals
2025-12-02
bladder cancer
The Society of Urologic Oncology (SUO) and Ferring Pharmaceuticals today announced the SUO NMIBC (non-muscle invasive bladder cancer) Fellows Research Grant,...
Imvax shares Phase 2b data for glioblastoma treatment, plans to meet with FDA
Endpoints News
2025-12-02
glioblastoma
Imvax, a biotech led by former Spark Therapeutics executives, says its glioblastoma treatment was successful at keeping patients alive about six months longer than standard of care in a Phase 2b study. But the experimental ...
enGene stock rises after FDA selects bladder cancer therapy for CMC pilot program By Investing.com
enGene Google
2025-12-02
bladder cancer
enGene stock rises after FDA selects bladder cancer therapy for CMC pilot program
Janux sinks on ‘overreaction’ to prostate cancer data; RFK Jr. replaces ACIP chair
Biopharma Dive
2025-12-02
prostate cancer
Janux’s “masked” T cell engager had a lower response rate than reported last year. Elsewhere, Lilly dropped Zepbound’s price and ACIP named a new chairman days before a key vaccine meeting.
Cullinan Therapeutics Receives FDA Fast Track Designation for CLN-049, a Novel FLT3xCD3 T Cell Engager, in Relapsed/Refractory Acute Myeloid Leukemia
Cullinan Therapeutics
2025-12-02
leukemia
fast track
acute myeloid leukemia
Nanoscope Therapeutics’ MCO-010 Optogenetic Therapy Featured at FLORetina-ICOOR 2025 Congress
Nanoscope Therapeutics
2025-12-02
mco-010
DALLAS, Dec. 2, 2025 — NanoscopeTherapeutics Inc., a biotechnology company committed to developing and commercializing novel, disease-agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degeneration, today announced that Dr. Jordi Monés and Dr. Allen C. Ho will present Phase 2b/3 RESTORE and Long-Term-Follow-Up REMAIN study results at the FLORetina-ICOOR 2025 Congress, December […] The post Nanoscope Therapeutics’ MCO-010 Optogenetic Therapy Featured at FLORetina-ICOOR 2025 Congress appeared first on Nanoscope Therapeutics .
Follicular Lymphoma Market Predicted to See Upsurge Through 2034, Highlights DelveInsight | Roche, MEI Pharma, Novartis, Innovent Biologics, Regeneron, Genentech, Xynomic Pharma, Eli Lilly
Roche Group
2025-12-02
follicular lymphoma
The Key Follicular Lymphoma Companies in the market include - Roche, BeiGene, Kite Pharma, AstraZeneca, Incyte Corp, Xynomic Pharmaceuticals, MEI Pharma, Novartis, Innovent Biologics, Regeneron, Genentech, Eli Lilly and Company, ADC Therapeutics S.A,...
enGene stock rises after FDA selects bladder cancer therapy for CMC pilot program By Investing.com
enGene Google
2025-12-02
bladder cancer
enGene stock rises after FDA selects bladder cancer therapy for CMC pilot program
Obesity Can Speed Progression of Alzheimer’s Disease
Inside Precision Medicine
2025-12-02
alzheimer's disease
Research shows obesity can speed the progression of amyloid and tau buildup in the brain of people with Alzheimer's disease over time. The post Obesity Can Speed Progression of Alzheimer’s Disease appeared first on Inside Precision Medicine .
Imvax Heads to FDA With 6-Month Survival Gain in Glioblastoma, Despite Endpoint Miss
BioSpace
2025-12-02
autologous
glioblastoma
While Imvax’s autologous immunotherapy IGV-001 missed the primary endpoint of progression-free survival in a Phase IIb trial, the company will request a meeting with the FDA to discuss next steps for “synergistic” treatment.
Muscle Invasive Bladder Cancer Market to Reach New Heights in Growth by 2034, DelveInsight Predicts | CG Oncology, Pfizer, UroGen Pharma, Janssen R&D, LLC, Protara Therapeutics, Binhui Biopharma
CG Oncology
2025-12-02
bladder cancer
The Key Muscle Invasive Bladder Cancer Companies in the market include CG Oncology Pfizer UroGen Pharma Janssen Research Development LLC Protara Therapeutics Binhui Biopharmaceutical SURGE Therapeutics Pfizer AstraZeneca ImmunityBio Inc Guarionex J Decastro Janssen Research Development Tollys Aura Biosciences Vaxiion ...
Blood Cancer United, formerly The Leukemia & Lymphoma Society, Announces New Funding for Lifesaving Cancer Breakthroughs
PR Newswire
2025-12-02
leukemia
With the latest funding round, Blood Cancer United's cumulative research funding tops $2 billion, continuing to help increase survival rates and improve patients' lives Blood Cancer United's new research funding supports foundational science, AI, big data and global collaborations to...
Engene's detalimogene selected for FDA manufacturing pilot program to support manufacturing readiness
enGene Google
2025-12-02
detalimogene voraplasmid
enGene Holdings Inc: enGene's detalimogene selected for FDA manufacturing pilot program to support manufacturing readiness enGene Holdings - FDA selects detalimogene voraplasmid to participate...
123Invent Inventor Develops Compress Vibes for the Legs & Feet (SGM-724)
PR Newswire
2025-12-02
ind
PITTSBURGH, Dec. 2, 2025 /PRNewswire/ -- "I am paralyzed, and I experience nerve pain in my legs and feet. I wanted to create a new massage device to help reduce pain," said an inventor, from Alexandria, Ind., "so I invented COMPRESS VIBES. My design also offers hot/cold therapy, and it...
Novartis-Backed Protego Eyes Pivotal Amyloidosis Study With $130M Series B
BioSpace
2025-12-01
pivotal
Protego Biopharma is advancing a small-molecule drug that helps light chain proteins fold correctly, in turn addressing the underlying biological cause of AL amyloidosis.
Autolus Therapeutics Appoints Ryan Richardson to Board of Directors
Autolus Therapeutics
2025-12-01
cell therapies
LONDON and GAITHERSBURG, Md., Dec. 01, 2025 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, today announced the appointment of Ryan Richardson to the Company’s Board of Directors. Mr. Richardson brings more than 20 years of strategy and corporate development experience and in the healthcare and investment banking industries to Autolus’ Board. “Aut
YuviTal wins Roche-backed breast cancer innovation challenge
Roche Group
2025-12-01
breast cancer
The Israeli digital health startup will pilot an AI-driven platform at Sheba Medical Center to provide personalized guidance and identify concerns.
AML drug Komzifti added to national treatment guidelines
Rare Cancer News
2025-12-01
aml
leukemia
acute myeloid leukemia
Komzifti (ziftomenib), a once-daily oral therapy approved for certain people with acute myeloid leukemia (AML), is now included in the National Comprehensive Cancer Network (NCCN) clinical practice guidelines in oncology for AML. The NCCN guidelines provide evidence-based recommendations for the prevention, diagnosis, and treatment of cancer. They incorporate real-time updates to reflect rapid advancements in […] The post AML drug Komzifti added to national treatment guidelines appeared first on Rare Cancer News .
Roche shares hit milestone with 19% November surge on breast cancer and promising MS drug trials
Roche Group
2025-12-01
breast cancer
Sure! Please provide the article or input text you'd like a meta description for.
Actinium Pharmaceuticals Announces ATNM-400 Data Demonstrating Potent Efficacy in Triple-Negative Breast Cancer and Ability to Overcome Endocrine and HER2-Targeted Therapy Resistance Being Presented at the San Antonio Breast Cancer Symposium
Actinium Pharma
2025-12-01
her2
lung cancer
breast cancer
prostate cancer
Presentation to include new data in triple-negative disease, a breast cancer subtype with poor outcomes and limited viable treatment options ATNM-400 overcomes HER2 therapy resistance and represents a novel targeted radiotherapy with the potential to avoid off-target toxicities including ILD that constrains other therapeutic modalities such as antibody drug conjugates ATNM-400 has demonstrated the potential to overcome resistance to first-line tamoxifen endocrine therapy Data to be presented on December 11, 2025 at 5:00 PM CT NEW YORK , Dec. 1, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE American: ATNM), a leader in the development of differentiated targeted radiotherapies, today announced compelling preclinical data for ATNM-400, a first-in-class Actinium-225 (AC-225) antibody radioconjugate, in hormone receptor positive (HR+), HER2 positive (HER2+) and triple-negative breast cancer (TNBC) that will be presented at the San Antonio Breast Cancer Symposium (SABCS) being held December 10‑14, 2025 in San Antonio, Texas. The data demonstrates significant anti-tumor activity in breast cancer models resistant to standard-of-care therapies including endocrine therapy…
Belite plans filing for oral Stargardt drug after phase 3 win
PharmaPhorum
2025-12-01
stargardt disease
The first therapy for sight-robbing Stargardt disease could be on the way, as Belite Bio's tinlarebant hits the mark in a phase 3 trial.
As New Hereditary Angioedema Drugs Flood the Market, Will Uptake Match Innovation?
BioSpace
2025-12-01
hereditary angioedema
This year has seen the approval of several first-in-class therapies for HAE, but in a fragmented space, experts question whether they will be enough to net their developers a significant share of the entrenched market.
FDA Action Alert: BMS, GSK, Amgen and More
BioSpace
2025-12-01
breyanzi
The FDA’s docket in December includes decisions for two big biologic franchises: BMS’s Breyanzi and Amgen’s Uplizna.
PreBLA Meeting With FDA Sinks uniQure’s Hopes for Approval of Huntington Disease Gene Therapy AMT-130 Based Primarily on Phase 1/2 Data
CGTLive
2025-12-01
amt-130
phase 1/2
gene therapy
huntington disease
uniQure pointed out that this new feedback stands in contrast to its previous communications with the FDA in several Type B meetings.
Limited prostate cancer screening backed in UK
PharmaPhorum
2025-12-01
prostate cancer
A recommendation on prostate cancer screening has been delivered in the UK, upsetting patient groups whilst being endorsed by medical organisations.
Regeneron Makes $275M Gene Editing Play With Tessera Partnership Targeting AATD
BioSpace
2025-12-01
gene editing
alpha-1 antitrypsin deficiency
The centerpiece of the deal is the in vivo editor TSRA-196, which in preclinical studies has shown robust editing at SERPINA1, the locus linked to alpha-1 antitrypsin deficiency.
Krystal Biotech doses first patient in EMERALD-1 phase 1/2 trial for neurotrophic keratitis | Ophthalmology Times - Clinical Insights for Eye Specialists
Krystal Biotech
2025-12-01
phase 1/2
gene therapy
Krystal Biotech initiates EMERALD-1 trial for KB801, a redosable eye drop gene therapy.
European Medicines Agency Validates Type II Variation Application for PADCEV TM (enfortumab vedotin) with KEYTRUDA TM (pembrolizumab) for Certain Patients with Muscle-Invasive Bladder Cancer
astellas
2025-12-01
bladder cancer
TOKYO, November 28, 2025 – Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, “Astellas”) today announced that the European Medicines Agency (EMA) validated for review a Type...
Regeneron Puts Up $150M to Partner on Tessera Gene-Editing Med for Rare Liver & Lung Disorder
MedCity News
2025-12-01
alpha-1 antitrypsin deficiency
Regeneron Pharmaceuticals will share in development of Tessera Therapeutics’ TSRA-196, a Phase 1-ready gene-editing therapy for alpha-1 antitrypsin deficiency (AATD). Other companies developing genetic medicines for this rare disease include Beam Therapeutics, Wave Life Sciences, Korro Bio, and AIRNA. The post Regeneron Puts Up $150M to Partner on Tessera Gene-Editing Med for Rare Liver & Lung Disorder appeared first on MedCity News .
Startup Protego Bio Lands $130M for First-in-Class Drug’s Pivotal Test in Rare Plasma Disorder
MedCity News
2025-12-01
pivotal
Protego Biopharma’s lead program is a potential treatment for the rare disease light chain amyloidosis. The startup says the novel mechanism of its oral small molecule should have better outcomes than antibodies from AstraZeneca and Prothena that failed their respective pivotal studies in the disorder earlier this year. The post Startup Protego Bio Lands $130M for First-in-Class Drug’s Pivotal Test in Rare Plasma Disorder appeared first on MedCity News .
Romidepsin Identified as Candidate for Treating Relapsed Neuroblastoma
Inside Precision Medicine
2025-12-01
neuroblastoma
An approved drug for lymphoma called romidepsin can induce cell death in neuroblastoma through pathways that remain active even when standard chemotherapy drugs lose effectiveness. The post Romidepsin Identified as Candidate for Treating Relapsed Neuroblastoma appeared first on Inside Precision Medicine .
Belite Bio aims for first approval of Stargardt disease treatment with Phase 3 success
Endpoints News
2025-12-01
stargardt disease
Belite Bio reported Monday that its pill for Stargardt disease succeeded in a Phase 3 trial, and it's heading to the FDA to seek approval. There are currently no approved treatments ...
Caribou Biosciences to Host In-Person and Virtual KOL Event at ASH 2025
caribou biosciences
2025-12-01
cd19
car-t
allogeneic
cell therapy
KOL panel to discuss how vispa-cel, an anti-CD19 allogeneic CAR-T cell therapy, can broaden access for patients with second-line large B cell lymphoma including through use in the community setting BERKELEY, Calif., Dec. 01, 2025 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc.
Specialty pharmacy’s secret weapon: The unsung power of the enrollment form
Biopharma Dive
2025-12-01
orphan drug
One form can make or break orphan drug success and patient outcomes.
AstraZeneca’s Imfinzi receives FDA approval for gastric and gastro-oesophageal cancers
PMLive Regulatory
2025-12-01
gastric cancer
In 2022, nearly one million new patients were diagnosed with gastric cancer
Janux’s prostate cancer immunotherapy’s response rate drops, according to new early-stage data
Endpoints News
2025-12-01
prostate cancer
Janux Therapeutics’ lead T cell engager has shown response rates of 30% in a new data cut from an early-stage study in prostate cancer. The product, named JANX007, is in a Phase 1 trial called ...
6 Companies Hanging On in Cell Therapy
BioSpace
2025-12-01
cell therapy
As big pharmas including Takeda and Novo Nordisk flee the cell therapy space and smaller biotechs shutter their operations, these players are sticking around to take the modality as far as it can go.
AbbVie to Present Phase 3 ECLIPSE Data Demonstrating Atogepant (AQUIPTA®) Superiority Over Placebo in Achieving Pain Freedom for the Acute Treatment of Migraine at the 19th European Headache Congress
Abbvie
2025-12-01
pivotal
Pivotal Phase 3 ECLIPSE study demonstrated atogepant is superior to placebo for achieving pain freedom at two hours after treatment of the first migraine attack (24.3% for atogepant vs. 13.1% for...
Mogamulizumab Plus CHOP Improved PFS in Older Patients With TCL
Hematology Advisor News
2025-12-01
leukemia
Researchers determined adding mogamulizumab to CHOP improved PFS in older patients with adult T-cell leukemia/lymphoma.
Protego gets $130M for AL amyloidosis drug from leaders behind Vyndaqel
Endpoints News
2025-12-01
pivotal
A San Diego biotech hoping to crack into a rare protein misfolding disease has drummed up $130 million in investor support to get into a potential pivotal trial next year. Protego Biopharma, from the ...
EMA starts review of 'transformative' bladder cancer therapy
PharmaPhorum
2025-12-01
bladder cancer
The EMA has started a review of a regimen based on Pfizer/Astellas' Padcev and MSD's Keytruda that is described as a game-changer for bladder cancer.
Gene writing startup Tessera strikes $150M partnership with Regeneron
Endpoints News
2025-12-01
gene editing
Regeneron Pharmaceuticals is expanding its investment in gene editing through a new partnership with Tessera Therapeutics, a well-funded startup that is preparing to test its first experimental therapy in a clinical trial ...
Targeted Treg Boost Before Transplant Reduces GVHD in Stem Cell Models
Inside Precision Medicine
2025-12-01
leukemia
A pre-transplant immunotherapy designed to expand regulatory T cells reduces GVHD, protects tissues, and preserves anti-leukemia immunity in preclinical stem cell transplant models. The post Targeted Treg Boost Before Transplant Reduces GVHD in Stem Cell Models appeared first on Inside Precision Medicine .
Regeneron inks gene editing deal with startup Tessera
Biopharma Dive
2025-12-01
gene editing
alpha-1 antitrypsin deficiency
The big biotech, which has made genetic medicine a focus in recent years, is paying Tessera $150 million for rights to a potential one-and-done treatment for alpha-1 antitrypsin deficiency.
Naji Gehchan, MD, MBA, on Evaluating CAR-T KYV-101 for Generalized Myasthenia Gravis
CGTLive
2025-11-30
car-t
kyv-101
myasthenia gravis
Guess the Therapy Answer November 26, 2025
CGTLive
2025-11-29
gene therapy
Cell and Gene Therapy Live
Adicet Bio Reports Inducement Grant under Nasdaq Listing Rule 5635(c)(4)
Adicet Bio
2025-11-29
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. --(BUSINESS WIRE)--Nov. 28, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced it granted an inducement award on November 28, 2025
Innovation. ‘Obe-cel’ – A breakthrough immunotherapy that helps in treating aggressive blood cancer
Autolus Therapeutics
2025-11-28
car t
leukemia
cell therapy
Researchers have developed a breakthrough immunotherapy to treat an aggressive form of leukemia. The CAR T-cell therapy – known as ‘obe-cel’ involves taking a patient’s immune cells and reprogramming them in a lab to identify and target their cancer, before returning them to the body as ‘living medicine’. Obe-cel is a second-generation CAR T cell […]
enGene Holdings: A Deep Dive (NASDAQ:ENGN)
enGene Google
2025-11-28
gene therapy
bladder cancer
Shares of bladder cancer gene therapy concern enGene Holdings Inc.
Novartis’ Itvisma gets FDA approval for spinal muscular atrophy treatment
PMLive Regulatory
2025-11-28
sma
spinal muscular atrophy
Around 9,000 people in the US currently live with SMA
November 2025: This Month in Huntington’s Disease Research
uniQure
2025-11-28
gene therapy
From stem-cell “tooth fairy therapy” to gene therapy roadblocks and new genetic insights, November was big for HD research. Catch all of these stories in our monthly summary!
Orphan Drugs Market to Surge to USD 486.51 Billion by 2032 as Rare Disease Innovation, Gene Therapies & Precision Medicine Reshape Global Healthcare
PR Newswire
2025-11-27
gene therapies
AUSTIN, Texas and TOKYO, Nov. 27, 2025 /PRNewswire/ -- According to DataM Intelligence, the Orphan Drugs Market Size was USD 223.76 billion in 2023 and is forecasted to soar to USD 486.51 billion by 2032, expanding at a strong CAGR of 9.1% between 2024 and 2032. This remarkable growth...
Kura and Kyowa’s Komzifti moves ahead at pace in US market
Kyowa Kirin
2025-11-27
leukemia
acute myeloid leukemia
US biotech Kura Oncology (Nasdaq: KURA) and Japanese specialty pharma Kyowa Kirin (TYO: 4151) have secured early guideline recognition for Komzifti (ziftomenib), their new treatment for a subset of adults with relapsed or refractory acute myeloid leukemia.
uniQure (QURE): Evaluating Valuation as FDA Sets New Hurdles for Huntington’s Gene Therapy
uniQure
2025-11-27
amt-130
phase 1/2
gene therapy
Investors are closely watching uniQure (QURE) after the FDA signaled that Phase 1/2 results for its AMT-130 gene therapy are not sufficient to support a Biologics License Application. The company now faces new regulatory hurdles for its Huntington’s disease program. See our latest analysis for uniQure. Despite recent regulatory challenges for AMT-130, uniQure’s story has been marked by both volatility and notable gains. While a sharp 59.3% decline in the share price over the past month has...
Sandoz launches first and only multiple sclerosis biosimilar in US
PMLive Regulatory
2025-11-27
multiple sclerosis
The treatment is FDA-approved to treat MS and Crohn’s disease
Intratumoral Cancer Therapies Market to Reach USD 630.7 Million by 2033 as Next-Generation Localized Immunotherapies Transform Solid Tumor Treatment
PR Newswire
2025-11-27
solid tumor
AUSTIN, Texas and TOKYO, Nov. 27, 2025 /PRNewswire/ -- According to DataM Intelligence, the Intratumoral Cancer Therapies Market Size increased from USD 219.79 million in 2023 to USD 246.4 million in 2024, and is expected to climb to USD 630.7 million by 2033, growing at a strong CAGR of...
Roche supports Sidra’s Global DMD Exchange, spotlighting Qatar as a gene therapy hub
Roche Group
2025-11-27
dmd
gene therapy
Tribune News NetworkDohaRoche Pharmaceuticals, through its local representative Ebn Sina Medical, a fully owned subsidiary of Aamal Company has co-organized the Duchenne Muscular...
European Medicines Agency (EMA) grants Orphan Drug Designation to Kedrion's plasma-derived treatment for Congenital Aceruloplasminemia, a rare genetic disorder of iron metabolism
PR Newswire
2025-11-27
orphan drug
This designation marks a significant milestone for patients and reaffirms Kedrion's commitment to addressing unmet medical needs in rare and ultra-rare diseases CASTELVECCHIO PASCOLI, Italy, Nov. 27, 2025 /PRNewswire/ -- Kedrion announces that the European Medicines Agency (EMA) has...
Roche supports Sidra Medicine’s global DMD exchange, spotlighting Qatar as a gene therapy hub
Roche Group
2025-11-27
dmd
gene therapy
Doha, Qatar:Roche Pharmaceuticals, through its local representative Ebn Sina Medical, a fully owned subsidiary of Aamal Company Q.P.S.C will co organi...
Innocan Pharma Reports Financial Results for Q3 2025, with Revenues at US $21.6M
PR Newswire
2025-11-27
financial results
HERZLIYA, Israel and CALGARY, AB, Nov. 27, 2025 /PRNewswire/ -- Innocan Pharma Corporation (CSE: INNO) (FSE: IP4) (OTC: INNPF) (the "Company" or "Innocan"), a pharmaceutical technology company focusing on developing innovative drug delivery platform technologies, is pleased to announce...
FDA starts priority review of BeOne's Venclexta rival
PharmaPhorum
2025-11-27
priority review
mantle cell lymphoma
BeOne nears a possible FDA okay for sonrotoclax in mantle cell lymphoma, hoping to be the second BCL-2 inhibitor in the US after AbbVie's Venclexta.
BioMarin Pharmaceutical (BMRN): Assessing Valuation Following Robust Q3 Growth and Upgraded Full-Year Guidance
BioMarin Pharmaceutical
2025-11-27
quarterly
BioMarin Pharmaceutical (BMRN) grabbed attention after its Q3 update, where it reported solid revenue growth and raised its full-year guidance higher than any of its peers. The big drivers were its PALYNZIQ and VOXZOGO products, each delivering over 20% revenue growth. See our latest analysis for BioMarin Pharmaceutical. BioMarin’s latest quarterly momentum has drawn a spotlight, but the share price tells a more cautious story. At $55.52, the 1-year total shareholder return is still down 15%,...
BioMarin Pharmaceutical (BMRN): Evaluating Valuation After Raised Guidance on Revenue Strength
BioMarin Pharmaceutical
2025-11-27
quarterly
BioMarin Pharmaceutical (BMRN) just posted quarterly revenues rising 4% to $776 million, matching Wall Street views. Growth was fueled by strong performance in PALYNZIQ and VOXZOGO, and this prompted BioMarin to raise its full-year outlook more than its competitors. See our latest analysis for BioMarin Pharmaceutical. BioMarin’s raised outlook has drawn fresh investor attention, but shares are still down nearly 17% year-to-date and have delivered a -15% total shareholder return over the past...
Die Europäische Arzneimittelagentur (EMA) erteilt die Orphan Drug Designation für das aus Plasma gewonnene Medikament von Kedrion zur Behandlung der kongenitalen Aceruloplasminämie, einer seltenen genetischen Störung des Eisenstoffwechsels
PR Newswire
2025-11-27
orphan drug
Diese Einstufung stellt einen bedeutenden Meilenstein für die Patienten dar und bekräftigt Kedrions Engagement, den ungedeckten medizinischen Bedarf bei seltenen und extrem seltenen Krankheiten zu decken CASTELVECCHIO PASCOLI, Italien, 27. November 2025 /PRNewswire/ -- Kedrion gibt...
IASO Bio Announces BLA Approval of Fucaso (Equecabtagene Autoleucel) by the Hong Kong Department of Health for the Treatment of Relapsed or Refractory Multiple Myeloma
PR Newswire
2025-11-27
bla
cell therapies
multiple myeloma
SHANGHAI and NANJING, China and PLEASANTON, Calif., Nov. 27, 2025 /PRNewswire/ -- IASO Biotechnology ("IASO Bio"), a commercial-stage biopharmaceutical company dedicated to the discovery, development, manufacturing, and commercialization of novel cell therapies and biologics for...
nuEra Cannabis Chicago Southland Is Now Open -- Green Wednesday Deals Start This Week, Grand Opening Event Coming Next Week
PR Newswire
2025-11-26
open
CHICAGO SOUTHLAND, Ill., Nov. 26, 2025 /PRNewswire/ -- nuEra Cannabis is excited to announce that its newest Illinois dispensary, nuEra Chicago Southland, is officially open at 16950 S Halsted Street. The modern, customer-focused dispensary is now welcoming shoppers seven days a week with...
Computer-guided surgery delivers gene therapy to 2 children
AADC News/BioNews
2025-11-26
upstaza
kebilidi
gene therapy
Surgery guided by MRI and computer navigation safely and accurately delivered Upstaza (eladocagene exuparvovec, marketed as Kebilidi in the U.S.) in two children with AADC deficiency, a study reported. “Gene therapy for AADC deficiency proved both feasible and safe, with early clinical improvements observed in motor function and symptom control,” the researchers wrote. “This technique […] The post Computer-guided surgery delivers gene therapy to 2 children appeared first on AADC News .
Backbone Potential: Actuate’s Elraglusib Shows Promise vs. Pancreatic Cancer
GenEng News
2025-11-26
complete response
pancreatic cancer
Among 29 efficacy evaluable patients, elraglusib showed median overall survival (mOS) of 15.3 months with confirmations of complete response (CR) in two patients and partial response in nine others. When a separate single-arm trial is included, elraglusib has led to four CRs among 200+ patients. The post Backbone Potential: Actuate’s Elraglusib Shows Promise vs. Pancreatic Cancer appeared first on GEN - Genetic Engineering and Biotechnology News .
Otsuka’s anti-APRIL antibody gets FDA approval for kidney disease IgAN
Endpoints News
2025-11-26
accelerated approval
Otsuka secured FDA accelerated approval for its antibody drug in a disease that impacts the kidneys’ ability to filter waste. The Japanese drugmaker said late Tuesday the agency greenlit sibeprenlimab for ...
New center promotes advocacy for gynecological cancer patients
Rare Cancer News
2025-11-26
ovarian cancer
The Ovarian Cancer Research Alliance (OCRA) launched a project to push lawmakers to enact legislation that will improve the lives of people affected by ovarian cancer and other types of gynecological cancers. OCRA’s new Center for Public Policy is a dedicated arm of the organization focused on driving legislative changes at all levels of government […] The post New center promotes advocacy for gynecological cancer patients appeared first on Rare Cancer News .
CG Oncology (NASDAQ: CGON) names Rossi to board amid BLA initiation
CG Oncology
2025-11-26
bla
CG Oncology appoints Christina Rossi, a 25-year biotech veteran, to its Board as Simone Song resigns; the company recently initiated a BLA for cretostimogene.
Potential adverse reaction to gene therapy in a patient treated with Strimvelis for the treatment of ADA-SCID
Fondazione Telethon
2025-11-26
ada-scid
gene therapy
Il paziente è stato trattato con Strimvelis per la cura di ADA-SCID.
Kelonia's in vivo CAR-T data will feature at ASH
PharmaPhorum
2025-11-26
car-t
multiple myeloma
Kelonia only started human testing of its in vivo CAR-T therapy for multiple myeloma in August, but will report impressive results at ASH next month.
Cure SMA Welcomes Broad FDA Approval of Itvisma™ for Spinal Muscular Atrophy, Reinforces Commitment to Patient Access and Support
PR Newswire
2025-11-26
sma
spinal muscular atrophy
SCHAUMBURG, Ill., Nov. 26, 2025 /PRNewswire/ -- Cure SMA, the leading nonprofit organization dedicated to supporting those impacted by spinal muscular atrophy (SMA), welcomes the FDA approval of Itvisma™ (onasemnogene abeparvovec-brve) by Novartis for adults and children 2 years of age...
‘Strong economic justification’ for directly mailing HPV testing kits to patients
Healio
2025-11-26
cervical cancer
Health care centers could reduce costs if they mailed HPV self-sampling kits directly to patients, including those who are up to date with cervical cancer screening.The Self-Testing options in the Era for Primary HPV screening for cervical cancer (STEP) trial previously showed mailing HPV kits to women who were screening adherent or overdue for testing increased uptake.
KOMZIFTI™ (ziftomenib) Added to National Comprehensive Cancer Network® (NCCN) Guidelines for Acute Myeloid Leukemia (AML)
Kyowa Kirin Co.
2025-11-26
aml
leukemia
acute myeloid leukemia
uniQure (QURE) Is Down 5.9% After FDA Calls AMT-130 Data Insufficient for BLA Approval – Has the Bull Case Changed?
uniQure
2025-11-26
bla
amt-130
phase 1/2
gene therapy
accelerated approval
In recent days, William Blair reaffirmed a Market Perform rating on uniQure N.V. after the FDA stated that the Phase 1/2 data for its AMT-130 gene therapy is not sufficient for a Biologics License Application, introducing new regulatory challenges for the Huntington’s disease program. uniQure must now engage further with the FDA and awaits final minutes from a pre-BLA meeting while seeking potential accelerated approval for AMT-130. This development highlights how shifts in regulatory...
Advancements in Breast Cancer, Lifestyle Medicine and A Preview of SABCS with Amy Comander, MD
Healio
2025-11-26
breast cancer
Ianalumab shows ‘meaningful, disease-modifying results’ vs placebo in Sjögren’s
Healio
2025-11-26
ianalumab
CHICAGO — The novel B cell-targeting therapy ianalumab demonstrated improvement over placebo in key Sjögren’s disease parameters, according to data presented at ACR Convergence 2025.“For a long time, Sjögren’s therapy has been limited to symptomatic relief without altering the underlying disease process,” Thomas Grader-Beck, MD, an associate professor of clinical
ETI May Improve Bronchial Dilatations in Adolescents With Cystic Fibrosis
Hematology Advisor News
2025-11-26
cf
cystic fibrosis
Elexacaftor-tezacaftor-ivacaftor (ETI) may improve bronchial dilatations in adolescents with cystic fibrosis (CF).
Cystic fibrosis mortality falls, sickle cell disease mortality rises over recent years
Healio
2025-11-26
cystic fibrosis
sickle cell disease
From 2008 to 2023, there has been a significant decrease in cystic fibrosis mortality rates and a significant rise in sickle cell disease mortality rates in the U.S., according to findings published in JAMA Pediatrics.“For frontline clinicians, these results are a call to action,” Nansi S. Boghossian, PhD, associate professor in the Arnold School of Public Health at the University of
Oscar Qualifying Documentary Exposing Pediatric Healthcare Crisis Screens in NYC
PR Newswire
2025-11-26
open
Complicated exposes the misunderstood medical condition behind families' experiences with misdiagnoses, medical gaslighting, and child-protection overreach. LOS ANGELES, Nov. 26, 2025 /PRNewswire/ -- Two-time Oscar-shortlisted and Emmy nominated director Andrew Abrahams of Open Eye...
Frequent CGM use results in greater glycemic benefits for adults with type 2 diabetes
Healio
2025-11-26
open
Adults with type 2 diabetes who used a continuous glucose monitor and wore their sensor for more than 270 days in 1 year had greater HbA1c improvements than adults who did not use a CGM, researchers reported. In a retrospective cross-sectional analysis of Optum Market Clarity data published in JAMA Network Open, researchers observed that improvements in HbA1c at 1 year varied based on how often a
Q&A: Single AI system enables early detection of multiple causes of preventable blindness
Healio
2025-11-26
diabetic retinopathy
SAN DIEGO — Multiple diseases potentially leading to blindness can be detected early by a single AI platform analyzing OCT and fundus photography, according to a presenter at AIMed25. The multimodal, explainable AI platform capable of detecting diabetic retinopathy, glaucoma and retinopathy of prematurity was described in a poster presented by Tom Murickan, an ophthalmic technician at Tennessee
Up the bladder ladder, Engene tweak means phase II bell rung
enGene Google
2025-11-26
pivotal
gene therapy
bladder cancer
detalimogene voraplasmid
Engene Holdings Inc.’s protocol amendment to its phase II trial with detalimogene voraplasmid in bladder cancer worked out in a big way, and shares of the firm closed Nov. 11 at $8.82, up $2.81, or 47%. Engene rolled out additional preliminary data from the pivotal cohort of the ongoing Legend study testing the nonviral gene therapy in high-risk, Bacillus Calmette-Guérin-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, with or without concomitant papillary disease.
Time-Limited Venetoclax Regimens Match Continuous Ibrutinib in Untreated CLL
Hematology Advisor News
2025-11-26
cll
Undetectable minimal residual disease in peripheral blood reached 73.3% for VO and 47.2% for VI vs 0% for ibrutinib.
Older Patients With High-Risk MCL May Benefit From RBAC Plus Venetoclax
Hematology Advisor News
2025-11-26
mcl
Researchers determined adding fixed-duration venetoclax to RBAC in the first line may be effective in older patients with high-risk MCL.
NHS rolls out breakthrough CAR-T therapy for aggressive leukaemia
Autolus Therapeutics
2025-11-26
car-t
CAR-T therapy obe-cel is now available on the NHS, offering new hope and remission for adults with aggressive leukaemia.
CG Oncology Appoints Christina Rossi to Board of Directors
CG Oncology
2025-11-26
bla
CG Oncology appoints Christina Rossi to its Board of Directors, following Simone Song's resignation.Quiver AI SummaryCG Oncology, Inc. has announced the appointment of Christina Rossi to its Board of Directors, enhancing its leadership team as it prepares for the potential launch of its therapeutic candidate, cretostimogene, following recent BLA initiation. Rossi brings over 25 years of experience in the pharmaceutical and biotechnology sectors,
China's Zhongmou seeks to create a ‘one-size-fits-all’ rival to Luxturna
Endpoints News
2025-11-26
luxturna
gene therapy
An under-the-radar Wuhan-based biotech startup says that its gene therapy for an inherited form of vision loss has generated early but promising results in a handful of patients. The startup, Zhongmou Therapeutics, has attracted little ...
New CAR T Cells Target Glioblastoma Tumor Microenvironment Protein
Inside Precision Medicine
2025-11-26
car t
glioblastoma
The new lab-engineered therapy targets Tenascin-C (TNC), a protein secreted in the extracellular matrix by glioblastoma cells that is found within the glioblastoma tumor microenvironment. The post New CAR T Cells Target Glioblastoma Tumor Microenvironment Protein appeared first on Inside Precision Medicine .
Devonian Health Group Reports its Fourth Quarter and Annual 2025 Financial Results and Appointment of a New Board Member
PR Newswire
2025-11-26
financial results
Fourth quarter distribution revenues of $1.3 million Fourth quarter net loss of $0.4 million, $0.003 per share Annual distribution revenues of $23.6 million Annual net loss of $6 million, $0.041 per share Devonian is debt free with $7 million in cash as of July 31, 2025 Pierre Labbé...
8-K - CG Oncology, Inc. (0001991792) (Filer)
SEC Filing (8-K)
2025-11-26
cg oncology
Filed: 2025-11-26 AccNo: 0001193125-25-298160 Size: 162 KB Item 5.02: Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers: Compensatory Arrangements of Certain Officers
Major prostate cancer screening trial launched in UK
PMLive Healthcare
2025-11-25
prostate cancer
The £42m Transform trial will recruit men aged 50-74, with a lower age limit of 45 for some men
FDA approves Novartis’ Itvisma for older patients with spinal muscular atrophy
Endpoints News
2025-11-25
sma
gene therapy
spinal muscular atrophy
Novartis has secured FDA approval for a new version of its one-time gene therapy for spinal muscular atrophy (SMA) that it says could benefit a much broader swathe of patients. The Swiss drugmaker
New Standards Aim to Support Gene Therapy Manufacturers
GenEng News
2025-11-25
gene therapy
The U.S. Pharmacopeia has created new standards for viral vectors to support manufacturers in the rapidly developing fields of cell and gene therapy. They hope the standards will help support the quality and consistency of medicines. The post New Standards Aim to Support Gene Therapy Manufacturers appeared first on GEN - Genetic Engineering and Biotechnology News .
William Blair Maintains Market Perform Rating on uniQure (QURE) Despite AMT-130 Approval Uncertainty
uniQure
2025-11-25
amt-130
uniQure N.V. (NASDAQ:QURE) ranks among the most oversold biotech stocks to invest in. William Blair reaffirmed its Market Perform rating for uniQure N.V. (NASDAQ:QURE) on November 11 while the company anticipates comments from regulators regarding its AMT-130 therapy. William Blair suggests that lobbyists and the medical community might put pressure on regulators to negotiate a […]
Industry-Academia Collaboration Formed to Improve Cell and Gene Therapy Manufacturing
GenEng News
2025-11-25
gene therapy
The concept of using buoyancy for the capture of cells and other components of interest is already being used in life science applications, including in cell and gene therapy manufacturing. The post Industry-Academia Collaboration Formed to Improve Cell and Gene Therapy Manufacturing appeared first on GEN - Genetic Engineering and Biotechnology News .
Kura Oncology (KURA) Is Up 8.1% After FDA Approval of KOMZIFTI for NPM1-Mutated AML
Kyowa Kirin
2025-11-25
aml
leukemia
acute myeloid leukemia
On November 13, 2025, Kyowa Kirin and Kura Oncology announced that the FDA has granted full approval for KOMZIFTI (ziftomenib) as the first once-daily targeted therapy for adults with relapsed or refractory NPM1-mutated acute myeloid leukemia with no satisfactory alternatives. This long-awaited approval addresses a significant unmet medical need for a patient group with historically high relapse rates and limited treatment options. We’ll take a look at how FDA approval of KOMZIFTI as a new...
Kura Oncology and Kyowa Kirin's KOMZIFTI™ Approved and Included in NCCN Guidelines for Treatment of Relapsed/Refractory AML with NPM1 Mutation
Kyowa Kirin
2025-11-25
aml
leukemia
acute myeloid leukemia
Kura Oncology's KOMZIFTI approved for relapsed/refractory AML, included in NCCN guidelines as a recommended treatment.Quiver AI SummaryKura Oncology, Inc. and Kyowa Kirin Co., Ltd. announced that KOMZIFTI™ (ziftomenib), a once-daily oral menin inhibitor, has been approved by the FDA for treating adults with relapsed or refractory acute myeloid leukemia (AML) with a susceptible NPM1 mutation. The drug has been added to the National Comprehensive C
NICE backs NHS use of Autolus' leukaemia CAR-T
PharmaPhorum
2025-11-25
car-t
Hundreds of people with an aggressive leukaemia will be able to access a CAR-T therapy developed by UK biotech Autolus after a NICE recommendation.
Autolus Therapeutics announces NICE draft guidance recommending Aucatzyl
Autolus Therapeutics
2025-11-25
aucatzyl
Autolus Therapeutics (AUTL) announced that the National Institute for Health and Care Excellence has published draft guidance recommending Aucatzyl for use in the N...
Novartis wins approval to use SMA gene therapy in older patients
Biopharma Dive
2025-11-25
sma
zolgensma
gene therapy
Itvisma, an intrathecal version of Zolgensma, can be used in children, teens and adults to stabilize or improve motor function.
Novartis' SMA gene therapy cleared for wider use in US
PharmaPhorum
2025-11-25
sma
gene therapy
spinal muscular atrophy
A new FDA approval means Novartis now has a gene therapy option for almost everyone with spinal muscular atrophy (SMA), regardless of their age.
Autolus Therapeutics Showcases Promising Innovations and Stock Resilience
Autolus Therapeutics
2025-11-25
cell therapies
Autolus Therapeutics plc stocks have been trading up by 10.52 percent following promising clinical trial developments boosting investor confidence. Key Takeaways Investor excitement is building as Autolus presents its T cell therapies at the ASH Annual Meeting, poised to make significant strides against difficult-to-treat conditions. The ongoing CARLYSLE trial yields promising results with high remission […]
uniQure Investigation Notification: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2025-11-25
bla
amt-130
gene therapy
RADNOR, PA - November 25, 2025 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Mixed Results for Mitapivat in Sickle Cell Disease Trial
Hematology Advisor News
2025-11-25
sickle cell disease
Topline results from the RISE UP phase 3 trial demonstrated that mitapivat significantly improved hemoglobin response in patients with sickle cell disease.
Novartis Expands Spinal Muscular Atrophy Gene Therapy Market With FDA’s Itvisma Greenlight
BioSpace
2025-11-25
zolgensma
gene therapy
spinal muscular atrophy
The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access to patients two years and older in what one Stanford Medicine professor called a “game changing advance” for the field.
As CGT Struggles To Scale, FDA's New Designation Could Smooth Post-Approval Pivots
BioSpace
2025-11-25
gene therapies
Experts suggest the FDA’s Advanced Manufacturing Technologies designation could be a lifeline for improving production processes for approved cell and gene therapies.
New Approvals in Hematologic Malignancies, Skin Cancer, and Small Cell Lung Cancer
ASCO Post FDA
2025-11-25
lung cancer
Here are brief reports on four oncology approvals by the U.S. Food and Drug Administration (FDA) in October 2025.
NICE Recommends AUCATZYL® (obecabtagene autoleucel) as a Treatment Option for Adult Patients (≥26 years) with Relapsed or Refractory B-Cell Precursor Acute Lymphoblastic Leukemia (R/R B-ALL)¹
syncona
2025-11-25
b-all
aucatzyl
leukemia
acute lymphoblastic leukemia
Kura Oncology (KURA) Is Up 8.1% After FDA Clears First Targeted Therapy for NPM1-Mutated AML
Kyowa Kirin
2025-11-25
aml
pivotal
leukemia
acute myeloid leukemia
On November 13, 2025, Kura Oncology and Kyowa Kirin announced that the FDA had granted full approval for KOMZIFTI (ziftomenib), the first once-daily targeted therapy for adults with relapsed or refractory NPM1-mutated acute myeloid leukemia, following positive results from the pivotal KOMET-001 trial. This approval addresses a significant unmet need in a patient group where around 30% of AML cases involve NPM1 mutations and treatment options have historically been limited. We'll examine how...
England Becomes First In Europe To Reimburse Autolus’ CAR-T Aucatzyl
Autolus Therapeutics
2025-11-25
car-t
aucatzyl
leukemia
acute lymphoblastic leukemia
England is set to become the first market outside of the US where Autolus’ CAR-T therapy Aucatzyl is reimbursed for acute lymphoblastic leukemia, after the country’s health technology assessment institution, NICE, recommended its use for certain patients.
Kura Oncology (NASDAQ: KURA) KOMZIFTI Named NCCN Category 2A Option for R/R NPM1 AML
Kyowa Kirin
2025-11-25
aml
KOMZIFTI, a once-daily oral menin inhibitor, is now in NCCN AML Guidelines after FDA approval on Nov. 13, 2025, with 21.4% CR/CRh and 5‑month response.
VIDEO: Calabrese discusses advances in lupus nephritis, ‘narrative medicine’
Healio
2025-11-25
car t
lupus
In this Healio video exclusive, Leonard H. Calabrese, DO, chief medical editor of Healio Rheumatology, highlights how the lupus armamentarium has “exploded” recently in terms of therapeutics and research into new approaches.“Lupus has exploded in terms of therapeutics, biologics and advanced cellular therapies, including CAR T and bispecifics,” he said.
2025 Lupus Research Alliance Gala Raises Millions to Bring New Treatments and A Cure
PR Newswire
2025-11-25
lupus
GRAMMY® Award-winning Artist Rob Thomas Headlines Evening Honoring Partners iHeart Media and Genentech NEW YORK, Nov. 25, 2025 /PRNewswire/ -- The Lupus Research Alliance, the largest private funder of lupus research, hosted its annual Breaking Through Gala – raising over $2.8 million to...
Sarepta Announces Approval to Begin ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne
Sarepta Therapeutics
2025-11-25
sarepta
elevidys
gene therapy
Approximately 25 non-ambulatory participants will receive sirolimus as part of the regimen in Cohort 8 of the ENDEAVOR study, which is expected to begin before the end of the year The enhanced immunosuppressive regimen is designed to mitigate the risk of acute liver injury (ALI) and acute liver
Merck KGaA given FDA Fast Track designation for generalised myasthenia gravis drug
PMLive Research
2025-11-25
fast track
myasthenia gravis
Cladribine capsules could become the first ever oral treatment for gMG
KOMZIFTI™ (ziftomenib) Added to National Comprehensive Cancer Network® (NCCN) Guidelines for Acute Myeloid Leukemia (AML)
Kyowa Kirin
2025-11-25
aml
leukemia
acute myeloid leukemia
KOMZIFTI, the first and only once-daily FDA-approved menin inhibitor for R/R NPM1-mutated AML, is now commercially available in the United States...
William Blair Maintains Market Perform Rating on uniQure (QURE) Despite AMT-130 Approval Uncertainty
uniQure
2025-11-25
amt-130
uniQure N.V. (NASDAQ:QURE) ranks among the most oversold biotech stocks to invest in. William Blair reaffirmed its Market Perform rating for uniQure N.V. (NASDAQ:QURE) on November 11 while the company anticipates comments from regulators regarding its AMT-130 therapy. William Blair suggests that lobbyists and the medical community might put pressure on regulators to negotiate a […]
Neurophet and The Florey Strengthen Research Collaboration on 'Neurophet AQUA' to Advance Alzheimer's Disease Diagnosis
PR Newswire
2025-11-25
alzheimer's disease
Aiming to evaluate the utility of blood-based and other biomarkers for Alzheimer's disease diagnosis and management Joint research to enhance dementia diagnosis using the brain neurodegeneration imaging software 'Neurophet AQUA' SEOUL, South Korea, Nov. 25, 2025 /PRNewswire/ -- Neurophet...
NICE Recommends AUCATZYL® (obecabtagene autoleucel) as a Treatment Option for Adult Patients (≥26 years) with Relapsed or Refractory B-Cell Precursor Acute Lymphoblastic Leukemia (R/R B-ALL)¹
Autolus Therapeutics
2025-11-25
b-all
aucatzyl
leukemia
cell therapies
acute lymphoblastic leukemia
AUCATZYL® will be available through routine commissioning by the UK National Health Service (NHS)LONDON, Nov. 25, 2025 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, announces that the National Institute for Health and Care Excellence (NICE) has published draft guidance1 recommending AUCATZYL® (obecabtagene autoleucel, or “obe-cel”)2 for use in t
William Blair Maintains Market Perform Rating on uniQure (QURE) Despite AMT-130 Approval Uncertainty
uniQure
2025-11-25
amt-130
uniQure N.V. (NASDAQ:QURE) ranks among the most oversold biotech stocks to invest in. William Blair reaffirmed its Market Perform rating for uniQure N.V.
Why Is Autolus Therapeutics Stock Trading Higher On Tuesday?
Autolus Therapeutics
2025-11-25
b-all
aucatzyl
U.K.'s NICE issues draft guidance recommending Autolus' Aucatzyl for adult r/r B-ALL, setting up imminent NHS launch and broader patient access.
Autolus Says UK Health Body Recommended Aucatzyl as Treatment Option for Leukemia
Autolus Therapeutics
2025-11-25
aucatzyl
leukemia
Autolus Therapeutics said Tuesday that the National Institute for Health and Care Excellence has recommended leukemia drug Aucatzyl for use in the National Health Service in England and Wales to...
U.S. FDA Approves PADCEV® plus Keytruda® for Certain Patients with Bladder Cancer
astellas
2025-11-25
bladder cancer
PADCEV plus Keytruda is the first and only approved perioperative treatment regimen that can significantly improve survival over current standard of care (surgery alone) in cisplatin-ineligible...
FDA Approves Daratumumab and Hyaluronidase-fihj for High-Risk Smoldering Multiple Myeloma
ASCO Post FDA
2025-11-25
multiple myeloma
On November 6, 2025, the U.S. Food and Drug Administration approved daratumumab and hyaluronidase-fihj (Darzalex Faspro) for adults with high-risk smoldering multiple myeloma.
Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)
Novartis
2025-11-25
sma
spinal muscular atrophy
Autolus Therapeutics Announces NICE Draft Guidance for AUCATZYL® as Treatment for Adult Patients with Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia
Autolus Therapeutics
2025-11-25
b-all
car t
aucatzyl
leukemia
cell therapy
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
NICE recommends AUCATZYL for adult patients with relapsed B-ALL in England and Wales, enabling NHS access.Quiver AI SummaryAutolus Therapeutics has announced that the National Institute for Health and Care Excellence (NICE) has published draft guidance recommending its CAR T cell therapy, AUCATZYL® (obecabtagene autoleucel, or "obe-cel"), for adult patients in England and Wales with relapsed or refractory B-cell precursor acute lymphoblastic leuk
Late-Stage Data For J&J’s Tecvayli/Darzalex Combo Could "Shift Paradigm" in Myeloma
BioSpace
2025-11-25
multiple myeloma
Tecvayli plus Darzalex led to an 83% boost to progression-free survival versus the current standard therapy in relapsed or refractory multiple myeloma, results analysts at Guggenheim Securities called "remarkable."
Autolus Therapeutics plc Announces National Institute for Health and Care Excellence Recommends Aucatzyl®? (Obecabtagene Autoleucel) as Treatment Option for Adult Patients with Relapsed or Refractory B-Cell Precursor Acute Lymphoblastic Leukemia (R/R B-All)
Autolus Therapeutics
2025-11-25
b-all
aucatzyl
leukemia
acute lymphoblastic leukemia
Autolus Therapeutics plc announced that the National Institute for Health and Care Excellence has published draft guidance1 recommending AUCATZYL®? for use in the National Health Service in...
Quizartinib Improves Survival in Newly Diagnosed FLT3-ITD–Negative AML
Hematology Advisor News
2025-11-25
aml
Researchers determined quizartinib plus chemotherapy significantly improved event-free survival and overall survival in patients with newly diagnosed FLT-ITD–negative AML.
FDA clears Thermo Fisher EXTENT system for myeloma diagnosis
Rare Cancer News
2025-11-25
multiple myeloma
Thermo Fisher Scientific has received U.S. Food and Drug Administration (FDA) clearance for its EXENT System, an automated test that helps doctors diagnose multiple myeloma. The system detects and identifies abnormal antibodies called M-proteins, which are produced by cancerous plasma cells and play a crucial role in the disease. By providing fast, sensitive, and reliable […] The post FDA clears Thermo Fisher EXTENT system for myeloma diagnosis appeared first on Rare Cancer News .
Fate Therapeutics to Present at Piper Sandler 37th Annual Healthcare Conference
Fate Therapeutics
2025-11-25
ipsc
SAN DIEGO , Nov. 25, 2025 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (the “Company” or “Fate Therapeutics”) (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to
Krystal Biotech's B-VEC for Dystrophic Epidermolysis Bullosa Gets Expanded FDA Label | Dermatology Times - Dermatology News and Clinical Insights
Krystal Biotech
2025-11-25
gene therapy
dystrophic epidermolysis bullosa
Krystal Biotech enhances B-VEC's label, empowering DEB patients with at-home treatment options and improving quality of life through innovative gene therapy.
FDA OKs Sarepta study of new immune suppression approach with Elevidys; Gilead buys preclinical TREX1 program
Endpoints News
2025-11-25
sarepta
elevidys
gene therapy
duchenne muscular dystrophy
Plus, news about Nuvalent and Phrontline: 🟢 FDA clears Sarepta’s next step: Sarepta said it will study its Duchenne muscular dystrophy gene therapy Elevidys with a new ...
Around the Helix: Cell and Gene Therapy Company Updates – October 29, 2025
CGTLive
2025-11-25
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Data sets up phase 3 trials for Novo's amycretin in diabetes
PharmaPhorum
2025-11-25
pivotal
Novo Nordisk has decided to move its amycretin into pivotal trials in type 2 diabetes after a phase 2 readout.
Intellia Puts Phase 3 Trials for Transthyretin Amyloidosis Gene Editing Therapy Nex-Z on Hold Following Grade 4 Liver AE
CGTLive
2025-11-25
phase 3
gene editing
Autolus (Nasdaq: AUTL) to Launch AUCATZYL on NHS in England and Wales After NICE Backing
Autolus Therapeutics
2025-11-25
aucatzyl
NICE guidance recommends AUCATZYL for adults ≥26 with relapsed or refractory B-cell ALL, enabling NHS commissioning after MHRA approval in April 2025.
Lilly to present data from two positive Phase 3 studies of Jaypirca (pirtobrutinib) in chronic lymphocytic leukemia at the 2025 American Society of Hematology (ASH) Annual Meeting
PR Newswire
2025-11-24
cll
leukemia
chronic lymphocytic leukemia
Results from the BRUIN CLL-314 study comparing Jaypirca (pirtobrutinib) to Imbruvica (ibrutinib) – the first-ever head-to-head Phase 3 study versus a covalent BTK inhibitor to include treatment-naïve CLL/SLL patients – will be presented as an oral presentation Results from the Phase 3...
CAR-T Availability Necessary for Combatting Health Disparities in MM
Hematology Advisor News
2025-11-24
bcma
car-t
Patients with MM who are part of racial and ethnic minority populations have reduced access to BCMA-targeting CAR-T therapies.
Made Scientific and Cellergy Therapeutics Announce Manufacturing Partnership to Advance First-in-Human Mitochondrial Therapy
PR Newswire
2025-11-24
cell therapy
PRINCETON, N.J. and TEL AVIV, Israel, Nov. 24, 2025 /PRNewswire/ -- Made Scientific, Inc., a leading U.S.-based clinical- and commercial-stage cell therapy contract development and manufacturing organization (CDMO), and Cellergy Therapeutics Ltd., a biotechnology company pioneering...
Sarepta to pay $200M milestone to Arrowhead; Enlivex reports osteoarthritis data
Endpoints News
2025-11-24
sarepta
Plus, news about Clearside Biomedical, Pfizer, Astellas, Merck, MindImmune Therapeutics and Poxel: 💵 Sarepta to pay Arrowhead $200M as it continues type 1 myotonic dystrophy study: Sarepta
Trusted Physician Megan J. Engelen on Bringing Personalized Care to Salt Lake City
PR Newswire
2025-11-24
open
SALT LAKE CITY, Nov. 24, 2025 /PRNewswire/ -- In the heart of Utah's Wasatch Front, longtime local physician Megan J. Engelen, DO, FACP, is proud to open a new practice model in partnership with MDVIP that redefines primary care by putting patients first. Dr. Engelen opened her doors on...
Disappointment as Novo's oral GLP-1 fails in Alzheimer's
PharmaPhorum
2025-11-24
alzheimer's disease
GLP-1 agonists have been shown to be beneficial in a lengthening list of diseases, but it seems Alzheimer's disease may not be among them.
Ryoncil Revenue Growth Highlighted in AGM Trading Update
Mesoblast
2025-11-24
ryoncil
Did Kyowa Kirin's (TSE:4151) New FDA Approval and Trials Just Shift Its Investment Narrative?
Kyowa Kirin
2025-11-24
leukemia
acute myeloid leukemia
In the past week, Kyowa Kirin announced a series of product milestones, including FDA approval of KOMZIFTI (ziftomenib) for NPM1-mutated acute myeloid leukemia, new Japan launches of LUMICEF and Crysvita formulations, and the start of a Phase 3 achondroplasia study. These developments reflect Kyowa Kirin’s focus on expanding its rare disease and oncology portfolios while enhancing treatment convenience and patient outcomes. We'll explore how Kyowa Kirin's new ACHONDROPLASIA trial launch is...
Quarter Century Update: The Continued Unknowns of Cell and Gene Therapy
CGTLive
2025-11-24
gene therapy
VIDEO: Future of gene editing, epigenetic silencing in HBV
Healio
2025-11-24
gene editing
In this video, Mark Sulkowski, MD, discusses clinical trials in gene editing therapy and basic studies in epigenetic silencing as presented at The Liver Meeting.
Is Autolus Therapeutics PLC (AUTL) One of the Fastest-Growing Penny Stocks to Buy Now?
Autolus Therapeutics
2025-11-24
car-t
Autolus Therapeutics plc. (NASDAQ:AUTL) is one of the fastest-growing penny stocks to buy now. On November 18 at the Jefferies London Healthcare Conference 2025, CEO Christian Itin reiterated that Autolus Therapeutics PLC (NASDAQ:AUTL) is positioning itself to be a leader in CAR-T therapies. The push comes as the company generated $21.1 million in product revenue […]
Untreated Sleep Apnea Can Increase Parkinson’s Disease Risk
Inside Precision Medicine
2025-11-24
parkinson's disease
People with untreated obstructive sleep apnea are more likely to develop Parkinson's disease than those without the sleep condition, although CPAP treatment can help reduce the risk. The post Untreated Sleep Apnea Can Increase Parkinson’s Disease Risk appeared first on Inside Precision Medicine .
Scott LaNeve nommé président de Labstat International
PR Newswire
2025-11-24
ind
Un cadre expérimenté dans le domaine des sciences de la vie pour diriger la stratégie d'expansion mondiale de Labstat KITCHENER, Ontario, 24 novembre 2025 /PRNewswire/ -- Labstat Inc., une société de Certified Group et le plus grand laboratoire mondial indépendant de tests de nicotine, a...
Sarepta Provides Progress Update for SRP-1003, its Investigational siRNA treatment for Myotonic Dystrophy Type 1
Sarepta Therapeutics
2025-11-24
sarepta
phase 1/2
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Nov. 24, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared progress in the Phase 1/2 multiple ascending dose (MAD) clinical study of SRP-1003 (formerly ARO-DM1), an investigational small
Readout of Phase 3 Semaglutide Trials Marks Critical Moment in Alzheimer's Research and Suggests Potential for Combination Therapies
PR Newswire
2025-11-24
alzheimer's disease
Data provides new insights into targeting the diverse pathobiology of Alzheimer's disease, an approach spearheaded by the ADDF NEW YORK, Nov. 24, 2025 /PRNewswire/ -- Today, Novo Nordisk released topline results from their phase 3 Evoke and Evoke+ trials of semaglutide, an oral GLP-1...
Asundexian successful in secondary stroke prevention: Topline data
Healio
2025-11-24
ischemic stroke
Once-daily asundexian 50 mg, on top of antiplatelet therapy, reduced risk for recurrent ischemic stroke after a non-cardioembolic ischemic stroke or high-risk ischemic attack compared with placebo, Bayer reported.Asundexian, an investigational oral factor XIa inhibitor, met its primary efficacy and safety endpoints with no increase in the risk of International Society on Thrombosis and
#ASH25: Kelonia’s first in vivo CAR-T data; Lilly, J&J and Novartis spell out Phase 3 wins
Endpoints News
2025-11-24
car-t
Late-breaking abstracts from the annual American Society of Hematology meeting are early this year, coming right before Thanksgiving instead of the previously planned drop on Dec. 1. Big pharma companies are seeking to expand the ...
Is Autolus Therapeutics PLC (AUTL) One of the Fastest-Growing Penny Stocks to Buy Now?
Autolus Therapeutics
2025-11-24
car-t
Autolus Therapeutics plc. (NASDAQ:AUTL) is one of the fastest-growing penny stocks to buy now. On November 18 at the Jefferies London Healthcare Conference 2025, CEO Christian Itin reiterated that Autolus Therapeutics PLC (NASDAQ:AUTL) is positioning itself to be a leader in CAR-T therapies. The push comes as the company generated $21.1 million in product revenue […]
Data from Genprex’s Acclaim-1 Phase 1 Gene Therapy Clinical Trial Published in Clinical Lung Cancer
Genprex
2025-11-24
lung cancer
gene therapy
Phase 1 Study in Non-Small Cell Lung Cancer Combined Reqorsa® Gene Therapy with Tagrisso® AUSTIN, Texas — (Nov. 24, 2025) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced the publication of data from its Acclaim-1 Phase 1 clinical trial […]
Cystic fibrosis triple therapy linked to reductions in airway, systemic inflammation
Healio
2025-11-24
cystic fibrosis
Among adults and adolescents with cystic fibrosis starting triple therapy for the first time, inflammatory markers fell and stayed this way at 2.5 years of therapy, according to data published in Annals of the American Thoracic Society.“Understanding how inflammation changes in people on elexacaftor/tezacaftor/ivacaftor (ETI) therapy is important because neutrophilic inflammation is
InventHelp Inventor Develops New CRPS Cushion (SGM-577)
PR Newswire
2025-11-24
ind
PITTSBURGH, Nov. 24, 2025 /PRNewswire/ -- "I suffer with CRPS, and I wanted to create a way for individuals with this disorder to sit without significant pain," said an inventor, from Terre Haute, Ind., "so I invented the FINALLY FREE. My design could reduce pain while improving the...
Lupus nephritis takes center stage
Healio
2025-11-24
car t
lupus
This month, our Healio Exclusive entitled “Recent approvals, CAR T-cell advances signal ‘exciting time’ in lupus nephritis,” highlights the literal tsunami of therapeutic investigations in a complication of lupus that still remains the leading cause of death in our patients.I want to thank our panel — Shivani Garg, MD, PhD; Dafna Gladman, MD; Fahmeedah Kamal, MD; and
CG Oncology initiated with a Buy at Truist
CG Oncology
2025-11-24
complete response
Truist analyst Gregory Renza initiated coverage of CG Oncology (CGON) with a Buy rating and $62 price target The firm views Creto’s high complete response rates, fa...
Repeated Surveys Strengthen Preventive Care Engagement in CLL
Hematology Advisor News
2025-11-24
cll
Serial participation was associated with improved cumulative vaccine coverage.
Early Intervention Improves Outcomes in Pregnancy-Associated Colorectal Cancer
Hematology Advisor News
2025-11-24
colorectal cancer
In this systematic review, researchers examined the clinical features, diagnosis, treatment, and prognosis of pregnancy-associated colorectal cancer.
Cell and Gene Therapy Will Transform Lives—If We Let It
BioSpace
2025-11-24
gene therapy
Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only warranted but essential if these therapies are to reach their full potential.
APOE4 and CRISPR are reframing Alzheimer’s Risk
Biopharma Dive
2025-11-24
crispr
New research on APOE4 and CRISPR brings hope for slowing Alzheimer’s progression.
‘Exciting’ brepocitinib data may open door for first targeted dermatomyositis drug
Healio
2025-11-24
open
Treatment with once-daily brepocitinib improved skin disease, muscle and physical function measures for adults with dermatomyositis while minimizing steroid burden, with benefits as early as 4 weeks and sustained for 1 year, data show.Findings from the phase 3 VALOR study assessing brepocitinib (Priovant Therapeutics), an oral dual selective tyrosine kinase (TYK) 2 and JAK 1 inhibitor, were
FDA Approves Gene Therapy for Treatment of Spinal Muscular Atrophy
FDA Press
2025-11-24
sma
gene therapy
spinal muscular atrophy
The U.S. Food and Drug Administration today approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in the survival motor neuron 1 (SMN1) gene.
Pfizer’s PADCEV gets FDA approval for bladder cancer
PMLive Research
2025-11-24
bladder cancer
Bladder cancer affects more than 614,000 people globally each year
How Investors May Respond To CG Oncology (CGON) Leadership Transition Amid Rising Revenue and Expanding Losses
CG Oncology
2025-11-23
financial results
On November 14, 2025, CG Oncology reported its third quarter and nine-month financial results, highlighting a significant year-over-year increase in revenue to US$1.67 million and US$1.72 million respectively, but with a wider net loss for both periods. In addition, the company announced the appointment of Jim DeTore as interim principal financial and accounting officer effective November 17, signifying a transition in leadership amid financial changes. We'll examine how the arrival of a new...
VIDEO: MacTel trials shape how clinical trial endpoints will be used
Healio
2025-11-23
macular telangiectasia type 2
In this video from the American Academy of Ophthalmology meeting, Lejla Vajzovic, MD, FASRS, highlights a presentation on macular telangiectasia type 2.
Makary’s user fee comments; M&A activity stays hot; A new use for prime editing; and more
Endpoints News
2025-11-22
prime editing
Welcome back to another edition of Endpoints Weekly. Our Saturday newsletter will be off next week for Thanksgiving, so let us be the first to wish you all a happy and healthy holiday! I’m personally ...
New frontiers for gene therapy
Fondazione Telethon
2025-11-22
gene therapy
All'SR-Tiget si lavora per applicare a sempre più malattie la terapia genica che ha già dato risultati importanti, ottimizzando tempi e costi di sviluppo.
CG Oncology (CGON): Valuation Insights Following Strong Revenue Growth and Widening Losses in Latest Results
CG Oncology
2025-11-22
quarterly
financial results
CG Oncology (CGON) has just released its latest quarterly and nine-month financial results, showing revenue growth compared to a year ago, even as net losses widened. This update offers a fresh angle for investors tracking CGON’s momentum and financial health. See our latest analysis for CG Oncology. After rallying strongly through the last quarter, CG Oncology’s share price has climbed 57.7% over the past 90 days and sits at $42.44. This momentum has sparked wider interest, even as the...
Kyowa Kirin (TSE:4151): Assessing Valuation After FDA Green Light for KOMZIFTI in AML Treatment
Kyowa Kirin
2025-11-22
aml
leukemia
acute myeloid leukemia
Kyowa Kirin (TSE:4151) just scored a milestone with full FDA approval of KOMZIFTI for adults battling relapsed or refractory acute myeloid leukemia with a susceptible NPM1 mutation. This development provides a new option in an area where choices have been limited. See our latest analysis for Kyowa Kirin. Kyowa Kirin’s momentum has picked up following the KOMZIFTI approval. The share price has climbed more than 11% over the past month and delivered a 10.4% year-to-date gain. While its one-year...
Sangamo Therapeutics to Present Data From Its Next-Generation Technologies at 2022 Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
Sangamo BioSciences
2025-11-21
cell therapy
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Oncology Trends: The latest innovations in cancer care
Prime Therapeutics
2025-11-21
car-t
Recent webinar featuring Prime Therapeutics experts shares some of the latest breakthrough therapies — including ADCs, BiTes and CAR-T treatments — and regulatory changes and market shifts that are guiding the future of cancer treatment
Novartis Plans to Build Flagship Manufacturing Hub in North Carolina
GenEng News
2025-11-21
open
Anticipated to open in 2027-2028, the flagship manufacturing hub, encompassing more than 700,000 square feet between the existing campus and new facilities, is expected to create 700 new jobs at Novartis. The post Novartis Plans to Build Flagship Manufacturing Hub in North Carolina appeared first on GEN - Genetic Engineering and Biotechnology News .
VIDEO: Predicting relapse risk in multiple myeloma
Healio
2025-11-21
multiple myeloma
In this video, Omar Nadeem, MD, an assistant professor of medicine at Harvard Medical School, discusses the factors that impact if or when a patient with multiple myeloma will relapse.
Sangamo Therapeutics Announces FDA Acceptance of BLA Rolling Submission Request for ST-920 in Fabry Disease
Sangamo BioSciences
2025-11-21
bla
st-920
fabry disease
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
6 Biotechs With a Shot at the FDA’s Platform Technology Award
BioSpace
2025-11-21
sarepta
After revoking Sarepta’s designation in July and awarding one to Krystal last month, the FDA’s platform technology designation program appears to be back on track. These six biotechs could be on the regulator’s radar.
6 Biotechs With a Shot at the FDA’s Platform Technology Designation
BioSpace
2025-11-21
sarepta
After revoking Sarepta’s award in July and awarding one to Krystal last month, the FDA’s platform technology designation program appears to be back on track. These six biotechs could be on the regulator’s radar.
Why It’s Time to Bring HCC Coding In-House
MedCity News
2025-11-21
hcc
Recent advances in generative AI have made it a lot easier and safer to bring HCC coding in-house, lowering costs, and strengthening audit readiness. The post Why It’s Time to Bring HCC Coding In-House appeared first on MedCity News .
European Orphan Medicinal Product Designation Granted to Sangamo Therapeutics Investigational CAR-Treg Cell Therapy TX200 for Solid Organ Transplantation
Sangamo BioSciences
2025-11-21
cell therapy
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
How Investors Are Reacting To Krystal Biotech (KRYS) Surging Revenue and Global VYJUVEK Expansion
Krystal Biotech
2025-11-21
vyjuvek
dystrophic epidermolysis bullosa
Krystal Biotech recently reported strong third quarter 2025 results, attributing improved gross margins and significant revenue growth to its lead product VYJUVEK and streamlined manufacturing operations. Management also emphasized accelerating global expansion, launching VYJUVEK in markets such as Germany, France, and Japan, and forming distribution partnerships across multiple regions to improve patient access for dystrophic epidermolysis bullosa treatment. We will now explore how...
Ginkgo Datapoints Launches Virtual Cell Initiative for AI Drug Discovery
GenEng News
2025-11-21
open
The Virtual Cell Pharmacology Initiative (VCPI) is an open-source platform designed to build a standardized framework for virtual cell models. The community-driven effort will aim to test at least 100,000 compounds and generate over 12 billion data points. The post Ginkgo Datapoints Launches Virtual Cell Initiative for AI Drug Discovery appeared first on GEN - Genetic Engineering and Biotechnology News .
Adicet Bio to Participate in a Fireside Chat at the Guggenheim 2nd Annual Healthcare Innovation Conference
Adicet Bio
2025-11-21
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. --(BUSINESS WIRE)--Oct. 28, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced that Chen Schor , President and Chief Executive
Italian non-profit set for EU gene therapy first in Wiskott-Aldrich
Fondazione Telethon
2025-11-21
gene therapy
wiskott-aldrich
etuvetidigene autotemcel
Fondazione Telethon’s Waskyra (etuvetidigene autotemcel) has been recommended for approval by the CHMP as a treatment for the rare disease.
Iovance Biotherapeutics Reports Inducement Grants under NASDAQ Listing Rule 5635(c)(4)
Iovance Biotherapeutics
2025-11-21
til
SAN CARLOS, Calif. , Nov. 21, 2025 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA) ("Iovance" or the “Company”), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (“TIL”) therapies for patients with cancer,
Alnylam's ATTR cardiomyopathy drug backed for NHS use
PharmaPhorum
2025-11-21
cardiomyopathy
Patients with ATTR-CM in England and Wales will soon have a new treatment option, after NICE backed use of Alnylam's three-month injectable Amvuttra.
CAR T Cell Therapy for Coronary Artery Disease Shows Early Success
Inside Precision Medicine
2025-11-21
car t
cell therapy
The preclinical findings show that redirecting regulatory T cells to suppress inflammation associated with OxLDL may reduce plaque development in the arteries. The post CAR T Cell Therapy for Coronary Artery Disease Shows Early Success appeared first on Inside Precision Medicine .
Sangamo Therapeutics Reports Recent Business Highlights and Second Quarter 2022 Financial Results
Sangamo BioSciences
2025-11-21
financial results
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Sangamo Therapeutics Reports Recent Business Highlights and First Quarter 2022 Financial Results
Sangamo BioSciences
2025-11-21
financial results
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
MiNK Therapeutics Announces New Data Showing MiNK-215 Drives Potent Anti-Tumor Activity in Treatment-resistant Solid Tumors
Mink Therapeutics
2025-11-21
fap
colorectal cancer
MiNK-215, an IL-15 armoured FAP-targeting CAR-iNKT, targets and clears tumor-protective FAP+ fibroblasts to allow immune cells to infiltrate and kills cancer cells Activates multiple immune pathways to generate potent, lasting anti-tumor activity in lung and MSS colorectal cancer models NEW YORK ,
Taiho Oncology, Taiho Pharmaceutical and Cullinan Therapeutics Initiate Rolling Submission of New Drug Application to U.S. Food and Drug Administration for Zipalertinib for Treatment of Locally Advanced or Metastatic Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations
Cullinan Therapeutics
2025-11-21
lung cancer
Sangamo Therapeutics Announces Updated Preliminary Phase 1/2 Data in Fabry Disease Showing Continued Tolerability and Sustained Elevated α-Gal A Enzyme Activity in Nine Patients
Sangamo BioSciences
2025-11-21
phase 1/2
fabry disease
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
First Patient Dosed in SYNERGY-101, a Global, Randomized Phase 2 Clinical Trial of STK-012 in First-Line, PD-L1 Negative Nonsquamous Non-Small Cell Lung Cancer
Synthekine
2025-11-21
lung cancer
November 13, 2025 The post First Patient Dosed in SYNERGY-101, a Global, Randomized Phase 2 Clinical Trial of STK-012 in First-Line, PD-L1 Negative Nonsquamous Non-Small Cell Lung Cancer first appeared on Synthekine .
PFIZER AND SANGAMO THERAPEUTICS ANNOUNCE PHASE 3 TRIAL OF INVESTIGATIONAL GENE THERAPY FOR HEMOPHILIA A HAS RE-OPENED RECRUITMENT
Sangamo BioSciences
2025-11-21
gene therapy
hemophilia a
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Gilead (GILD) Unit Receives FDA Orphan Drug Designation
Gilead Sciences
2025-11-21
orphan drug
multiple myeloma
Key Takeaways: Kite Pharma, a subsidiary of Gilead Sciences (GILD), has received orphan drug status for its multiple myeloma treatment, potentially accelera
J&J, Contineum Fail ‘Risky’ Mid-Stage Multiple Sclerosis Trial
BioSpace
2025-11-21
multiple sclerosis
While expressing disappointment, William Blair analysts were unsurprised by the Phase II failure, having assigned the VISTA study a high level of risk given the “mixed” performance of a similar drug in a prior multiple sclerosis study.
Adicet Bio Announces Positive Preliminary Data from ADI-001 Phase 1 Study in Patients with Lupus Nephritis (LN) and Systemic Lupus Erythematosus (SLE)
Adicet Bio
2025-11-21
phase 1
Clinical data to date supports a potentially transformational approach to treating autoimmune diseases with an off-the-shelf, one-time therapy- -As of the August 31, 2025 data cut-off date, all seven evaluable LN and SLE patients experienced rapid and sustained reductions in the Systemic Lupus
CERAN wrap: Roche phase III BC win seals thesis, boosts Olema
Roche Group
2025-11-21
her2
breast cancer
Roche AG’s Genentech unit landed another clinical victory with giredestrant that provided a major stock boost for competitor Olema Pharmaceuticals Inc. Made public were positive phase III data from the Lidera study testing selective estrogen receptor degrader and complete estrogen receptor antagonist (CERAN) as an adjuvant endocrine treatment for people with ER-positive, HER2-negative, early stage breast cancer (BC).
Case: Growth on pancreas shows estrogen link to pancreatic cancer
Rare Cancer News
2025-11-21
pancreatic cancer
Changes in how cells respond to the hormone estrogen may underlie the development of certain forms of pancreatic cancer. That’s according to findings from testing done on a growth removed from the pancreas of a woman in Japan who had given birth several months before. While analyses showed the abnormal growth — identified as a […] The post Case: Growth on pancreas shows estrogen link to pancreatic cancer appeared first on Rare Cancer News .
Sarepta ends Elevidys trial with Hansa's pre-treatment; Some pipeline cuts at Moderna
Endpoints News
2025-11-21
sarepta
elevidys
Plus, news about Blue Lake, Bambusa, Inhibikase Therapeutics and Olema Oncology: 🛑 Sarepta terminates Elevidys trial with Hansa Biopharma’s imlifidase: The study was examining whether Hansa’s imlifidase, an IgG antibody-cleaving enzyme, could help reduce ...
Sangamo Therapeutics Announces Updated Preliminary Phase 1/2 Data in Fabry Disease Clinical Study Showing Continued Tolerability and Sustained Elevated α-gal A Enzyme Activity in Five Longest Treated Patients
Sangamo BioSciences
2025-11-21
phase 1/2
fabry disease
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
FDA Approves New Regimen for Cisplatin-Ineligible MIBC Patients
Hematology Advisor News
2025-11-21
open
The approval was supported by data from the open-label, phase 3 KEYNOTE-905/EV-303 study.
On-demand sebetralstat generally safe in pediatric patients with hereditary angioedema
Healio
2025-11-21
hereditary angioedema
ORLANDO — Among pediatric patients with hereditary angioedema, on-demand sebetralstat was generally safe and used within a median time of 30 minutes after attack onset, according to interim phase 3 trial results.These data were presented at the American College of Allergy, Asthma & Immunology Annual Scientific Meeting.
Shortened dual antithrombotic duration after PCI may be safe in patients with AF
Healio
2025-11-21
atrial fibrillation
NEW ORLEANS — One month of dual antithrombotic therapy followed by direct oral anticoagulation alone may be as safe vs. 1 year of dual therapy after percutaneous coronary intervention in patients with atrial fibrillation, a speaker reported.The results of the prospective, randomized controlled OPTIMA-AF trial evaluating optimal dual antiplatelet duration strategies in patients with AF who
How Investors Are Reacting To Krystal Biotech (KRYS) Surging Revenue and Global VYJUVEK Expansion
Krystal Biotech
2025-11-21
vyjuvek
U.S. stocks, Saudi stocks, stock trading and investment platforms
BioMarin Pharmaceutical (BMRN): Buy, Sell, or Hold Post Q3 Earnings?
BioMarin Pharmaceutical
2025-11-21
quarterly
Over the past six months, BioMarin Pharmaceutical’s stock price fell to $51.76. Shareholders have lost 11.5% of their capital, which is disappointing considering the S&P 500 has climbed by 13.7%. This was partly driven by its softer quarterly results and may have investors wondering how to approach…
Contineum's J&J-partnered drug fails Phase 2 multiple sclerosis trial
Endpoints News
2025-11-21
multiple sclerosis
Contineum Therapeutics reported a Phase 2 failure in multiple sclerosis on Thursday afternoon, but most analysts don’t seem to view the miss as catastrophic. Contineum said its drug PIPE-307 did not ...
QURE Investigation Reminder: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2025-11-21
bla
amt-130
gene therapy
RADNOR, PA - November 21, 2025 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Strength exercise may be safe for people with arrhythmogenic cardiomyopathy
Healio
2025-11-20
cardiomyopathy
arrhythmogenic cardiomyopathy
NEW ORLEANS — Strength-based exercise may be safe for people with high inherited risk for ventricular arrhythmia, a speaker reported.Guidelines previously recommended that people with arrhythmogenic cardiomyopathy avoid vigorous exercise, but strength training could be a safe alternative to endurance exercise for them, according to the presentation at the American Heart Association
Trikafta improves ventilation MRI outcomes in pediatric cystic fibrosis
Healio
2025-11-20
cf
cystic fibrosis
Pediatric patients with cystic fibrosis starting triple combination therapy had larger relative improvements in 129Xenon MRI vs. lung function measures, according to data published in Annals of the American Thoracic Society.“These findings highlight the potential for Xe MRI as a sensitive tool for future multi-center, interventional studies, especially in people with CF with preserved
Hiring Outlook: Job Openings Will Likely Drop as 2025 Winds Down But Could Rise in 2026
BioSpace
2025-11-20
open
Biopharma professionals will probably find decreasing employment opportunities this month and next even as layoffs continue, based on BioSpace data. However, hundreds of open roles are expected this year in Massachusetts, and a job market turnaround could start late next year.
Krystal Biotech (KRYS) Secures FDA Orphan Status for Hailey-Hailey Treatment
Krystal Biotech
2025-11-20
orphan drug
Key Takeaways: Krystal Biotech (KRYS) has been granted orphan drug designation by the FDA for its treatment targeting Hailey-Hailey disease. The company
Aspen raises $115M for a Parkinson’s stem cell treatment
Biopharma Dive
2025-11-20
cell therapy
The cell therapy maker said Thursday the raise will support an ongoing trial of its experimental treatment for the neurodegenerative condition, as well as plans for building its manufacturing capabilities.
Glioma trial of intranasal therapy NEO100 fills all patient slots
Rare Cancer News
2025-11-20
glioma
Neonc Technologies has completed enrollment for its Phase 2a clinical trial of NEO100, an intranasal treatment for high-grade glioma, the company announced. This follows the release of positive interim data suggesting the experimental treatment, which uses a compound found in plants, may help reduce tumors and extend survival in patients with this aggressive brain cancer. […] The post Glioma trial of intranasal therapy NEO100 fills all patient slots appeared first on Rare Cancer News .
Krystal Biotech (KRYS): Assessing Valuation Following Strong Q3 Results and Global Expansion Moves
Krystal Biotech
2025-11-20
vyjuvek
Krystal Biotech (KRYS) is drawing fresh attention after reporting strong third quarter results. The report included higher gross margins and impressive revenue from its VYJUVEK therapy. The company is also expanding into Europe, Asia, and the Middle East. See our latest analysis for Krystal Biotech. Momentum has clearly been building for Krystal Biotech. After a quieter start to the year, shares have surged more than 40% over the last 90 days, and the one-year total shareholder return now...
PeproMene Bio’s PMB-CT01 BAFFR-CAR T Data Selected for Two Oral Presentations at 67th ASH 2025 Annual Meeting
pepromene bio
2025-11-20
car t
To find the article on the web: Click here IRVINE, Calif., Nov. 20, 2025 /PRNewswire/ — PeproMene Bio, Inc., a clinical-stage biotechnology company developing novel therapies for relapsed and refractory (r/r) B-cell malignancies, announced that two abstracts from its ongoing Phase 1 … Continue reading → The post PeproMene Bio’s PMB-CT01 BAFFR-CAR T Data Selected for Two Oral Presentations at 67th ASH 2025 Annual Meeting appeared first on PeproMene Bio Inc .
Olema Pharmaceuticals (OLMA): Evaluating Valuation After Surge Linked to Roche’s Positive Breast Cancer Drug Results
Roche Group
2025-11-20
breast cancer
Olema Pharmaceuticals (OLMA) shares jumped after Roche reported positive Phase 3 data for giredestrant, a breast cancer treatment in the same class as Olema’s investigational drug palazestrant. This announcement quickly drew market attention. See our latest analysis for Olema Pharmaceuticals. Momentum around Olema Pharmaceuticals has exploded, with the share price up 141% over the past week and a striking 250% in the past three months. Confidence is high thanks to Roche’s positive trial...
Krystal Biotech (KRYS) Secures FDA Orphan Status for Hailey-Hailey Treatment
Krystal Biotech
2025-11-20
orphan drug
Key Takeaways: Krystal Biotech (KRYS) has been granted orphan drug designation by the FDA for its treatment targeting Hailey-Hailey disease. The company
VIDEO: Iqirvo may stabilize primary biliary cholangitis progression after long-term use
Healio
2025-11-20
open
In this video, Nancy S. Reau, MD, FAASLD, AGAF, discusses long-term data from the ELATIVE open-label extension trial of Iqirvo in patients with primary biliary cholangitis.Results presented at The Liver Meeting demonstrated 72% of patients achieved a biochemical response after 182 weeks of receiving Iqirvo (elafibranor, Ipsen). Further, alkaline phosphatase normalized in 19% of patients which
VIDEO: Late-stage wet AMD assets garner excitement at AAO
Healio
2025-11-20
gene therapy
wet age-related macular degeneration
ORLANDO — In this video, David A. Eichenbaum, MD, FASRS, discusses late-stage wet age-related macular degeneration assets presented at the American Academy of Ophthalmology meeting.Eichenbaum noted that tyrosine kinase inhibitors and gene therapy programs are moving toward being commercial products in the near future, adding to the treatment options available to patients with wet AMD.
M. Peter Marinkovich, MD, on Integrating Gene Therapy into Clinical Practice for Dermatologists
CGTLive
2025-11-20
gene therapy
gene therapies
The associate professor of dermatology at Stanford University discussed practical considerations for the introduction of gene therapies in dermatology.
Aspen gets $115M for Parkinson's cell therapy, potential IPO in sight next year
Endpoints News
2025-11-20
cell therapy
parkinson's disease
Aspen Neuroscience will put itself to the test in 2026. The San Diego biotech aims to enter Phase 3 with its lead cell therapy for Parkinson's disease next year, CEO Damien McDevitt told Endpoints News ...
Q&A: ‘Immediate action’ needed to address rising CKD prevalence
Healio
2025-11-20
msc
Recent data published in The Lancet revealed chronic kidney disease is the ninth leading cause of death globally, a sharp increase from previous 2017 data. Marcello Tonelli, MD, SM, MSc, FRCPC, CAHS, FRSC, professor at the University of Calgary Cumming School of Medicine, the 2025-2027 president of the International Society of Nephrology (ISN) and a member of the Global Burden of
City of Hope expands Orange County presence with opening of new cancer specialty hospital
Healio
2025-11-20
open
A vision more than 7 years in the making will become reality next month.City of Hope will open a new cancer specialty hospital Dec. 1 in Orange County, California.
Aspen’s $115M Series C Opens Doors to Late-Stage Development for Parkinson’s Program
BioSpace
2025-11-20
cell therapy
Aspen is now also considering the possibility of an initial public offering next year in an effort to bring its cell therapy to the market.
Reporter’s Notebook: The Dawn of Disease-Agnostic Gene Editing Therapies
Inside Precision Medicine
2025-11-20
gene editing
prime editing
Prime editing-mediated readthrough of premature termination codons (PERT) permanently converts a “dispensable” endogenous tRNA into an optimized suppressor tRNA (sup-tRNA) that can read through a premature stop codon. The post Reporter’s Notebook: The Dawn of Disease-Agnostic Gene Editing Therapies appeared first on Inside Precision Medicine .
Bayer Lung Cancer Drug Lands Speedy FDA Nod to Stay Competitive With a Boehringer Ingelheim Med
MedCity News
2025-11-20
her2
lung cancer
The accelerated FDA approval of Bayer’s Hyrnuo covers treatment of advanced cases of non-small cell lung cancer with HER2 mutations. It will compete against an HER2-targeting Boehringer Ingelheim pill approved in August. The post Bayer Lung Cancer Drug Lands Speedy FDA Nod to Stay Competitive With a Boehringer Ingelheim Med appeared first on MedCity News .
Northwest Biotherapeutics Announces That Construction Activities Are Under Way For the First Grade C Manufacturing Suite in the Sawston, UK Facility
PR Newswire
2025-11-20
solid tumor
BETHESDA, Md., Nov. 20, 2025 /PRNewswire/ -- Northwest Biotherapeutics (OTCQB:NWBO) (the "Company" or "NWBio"), a biotechnology company developing DCVax® personalized immune therapies for solid tumor cancers, announced that it has engaged a construction firm to carry out the construction...
Bayer takes on Boehringer with FDA nod for lung cancer drug
PharmaPhorum
2025-11-20
her2
lung cancer
Bayer joins Boehringer with FDA approval for a second-line, oral therapy for HER2-positive lung cancer, setting up a marketing battle in the US.
FDA plans boxed warning, revised indication for Elevidys in children with Duchenne
Healio
2025-11-20
elevidys
gene therapy
adeno-associated virus
duchenne muscular dystrophy
The FDA will submit a boxed warning and other labeling limits for Elevidys, an adeno-associated virus and vector-based gene therapy for ambulatory patients aged 4 years and older with Duchenne muscular dystrophy.In a press release, the FDA said it was made aware of reports of fatal acute liver failure in non-ambulatory patients with the rare pediatric condition treated with Elevidys
Natural Grocers by Vitamin Cottage, Inc. Declares Quarterly Dividend
PR Newswire
2025-11-20
quarterly
Increases Quarterly Cash Dividend by 25% to $0.15 per Common Share LAKEWOOD, Colo., Nov. 20, 2025 /PRNewswire/ -- Natural Grocers by Vitamin Cottage, Inc. (NYSE: NGVC) today announced that the Company's Board of Directors has declared a quarterly cash dividend of $0.15 per common share....
Bayer Wins Accelerated Lung Cancer Approval for Kinase Inhibitor
BioSpace
2025-11-20
her2
lung cancer
The nod brings Bayer face-to-face in the market with Boehringer Ingelheim and AstraZeneca, each of which has its own HER2 blocker for non-small cell lung cancer.
Mesoblast & BMT CTN to Initiate Adult aGvHD Trial of RYONCIL
Mesoblast
2025-11-20
ryoncil
Daten zu PMB-CT01 BAFFR-CAR T von PeproMene Bio für zwei mündliche Präsentationen auf der 67. Jahrestagung 2025 von ASH ausgewählt
PR Newswire
2025-11-20
car t
IRVINE, Kalifornien, 20. November 2025 /PRNewswire/ -- PeproMene Bio, Inc., ein Biotechnologieunternehmen im klinischen Stadium, das neuartige Therapien für rezidivierte und refraktäre (r/r) B-Zell-Malignome entwickelt, gab bekannt, dass zwei Abstracts aus seinen laufenden Phase-1-Studien...
Nadofaragene firadenovec under regulatory review in Japan
Ferring Pharmaceuticals
2025-11-20
phase 3
complete response
The NDA is supported by data from a phase 3 trial, which demonstrated a 75% complete response rate at 3 months.
Guess the Therapy Answer November 19, 2025
CGTLive
2025-11-20
gene therapy
Cell and Gene Therapy Live
J&J receives positive NICE recommendation for multiple myeloma treatment
PMLive Regulatory
2025-11-20
multiple myeloma
The treatment is approved for use within the NHS in England and Wales
Adicet Bio to Present at the H.C. Wainwright 27th Annual Global Investment Conference
Adicet Bio
2025-11-20
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Sep. 2, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced that Chen Schor , President and Chief
Los datos del estudio PMB-CT01 BAFFR-CAR T de PeproMene Bio seleccionados para dos ponencias en la ASH 2025
PR Newswire
2025-11-20
car t
Los datos del estudio PMB-CT01 BAFFR-CAR T de PeproMene Bio fueron seleccionados para dos ponencias en la 67ª Reunión Anual de la ASH 2025 IRVINE, Calif., 20 de noviembre de 2025 /PRNewswire/ -- PeproMene Bio, Inc., una compañía de biotecnología en fase clínica que desarrolla nuevas...
Agios shares slump on sickle cell disease data
PharmaPhorum
2025-11-19
sickle cell disease
Shares in Agios lost nearly half their value after it posted mixed clinical results from a phase 3 trial of its sickle cell disease therapy mitapivat.
Genprex Adds Clinical Trial Site for Acclaim-1 and Acclaim-3 Lung Cancer Clinical Trials
Genprex
2025-11-19
fast track
lung cancer
gene therapy
New Clinical Trial Site Added for Clinical Studies of Reqorsa® Gene Therapy to Treat Lung Cancer Acclaim-1 and Acclaim-3 Clinical Trials Supported by FDA Fast Track Designation AUSTIN, Texas — (Nov. 19, 2025) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today […]
Agios' sickle cell trial falls short as drug doesn't appear to help patient pain crises
Endpoints News
2025-11-19
pivotal
sickle cell disease
Agios Pharmaceuticals’ drug Pyrukynd hit only one of the two primary endpoints in its pivotal trial in sickle cell disease, the company said Wednesday, a significant setback for the company's plans ...
Adicet Bio Reports First Quarter 2025 Financial Results and Provides Business Updates
Adicet Bio
2025-11-19
financial results
Aperture Therapeutics Announces Development Candidate Targeting CD33 to Restore Microglial Homeostasis in FTD and ALS
PR Newswire
2025-11-19
cd33
SAN CARLOS, Calif., Nov. 19, 2025 /PRNewswire/ -- Aperture Therapeutics, a biotechnology company pioneering next-generation precision medicines for neurodegenerative diseases, today announced the nomination of APRTX-001 as its development candidate. APRTX-001 is a CD33-targeting antisense...
[Form 4] GILEAD SCIENCES, INC. Insider Trading Activity
Gilead Sciences
2025-11-19
open
Gilead Sciences, Inc. (GILD)11/17/2025, the CFO sold 2,500 shares of Gilead common stock at a price of $125.23 per share in an open-market sale coded as an "S"
QURE Investigation: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2025-11-19
bla
amt-130
gene therapy
RADNOR, PA - November 19, 2025 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
First Look at Roche/Genentech’s Gazyva Shows Strong Early Enthusiasm in Lupus Nephritis Launch, According to Spherix Global Insights
Genentech
2025-11-19
lupus
One-month post-launch, Gazyva’s perceived degree of advance exceeds benchmarks achieved by GSK’s Benlysta and Aurinia’s Lupkynis at comparable post-launch...
VIDEO: Elafibranor shows promise as potential treatment for patients with PSC
Healio
2025-11-19
open
In this video, Julius Wilder, MD, PhD, discusses early results from the ongoing phase 2 ELMWOOD open-label extension trial of elafibranor in patients with primary sclerosing cholangitis.“Some of the early data is exciting, because currently we don’t have any great treatments for primary sclerosis cholangitis,” Wilder, associate professor of medicine at Duke Health, told Healio.
New prime editing approach could create one-size-fits-many therapies
Endpoints News
2025-11-19
gene editing
prime editing
Researchers led by Broad Institute gene editing star David Liu have come up with a new use for prime editing that can tackle a bucket of diseases with the same root cause, which they say ...
FDA grants 510(k) clearance for TMS as adjunctive for major depressive disorder in young people
Healio
2025-11-19
clearance
The FDA has granted 510(k) clearance and a label expansion for a transcranial magnetic stimulation device to treat patients aged 15 to 21 years with major depressive disorder, according to a press release from the manufacturer. The FDA’s decision clears the way for the BrainsWay Deep TMS system to be utilized as an adjunctive therapy for adolescents and young adults, the company said in the
WHO grants nonproprietary name to ovarian cancer cell therapy
Rare Cancer News
2025-11-19
cell therapy
ovarian cancer
A committee of the World Health Organization (WHO) has approved an international nonproprietary name (INN) for Anixa Biosciences’ experimental cell therapy for ovarian cancer. The therapy, which is already in clinical testing, will now carry the INN liraltagene autoleucel, or lira-cel for short. An INN — sometimes referred to as a generic name — is […] The post WHO grants nonproprietary name to ovarian cancer cell therapy appeared first on Rare Cancer News .
First Look at Roche/Genentech’s Gazyva Shows Strong Early Enthusiasm in Lupus Nephritis Launch, According to Spherix Global Insights
Genentech
2025-11-19
lupus
One-month post-launch, Gazyva’s perceived degree of advance exceeds benchmarks achieved by GSK’s Benlysta and Aurinia’s Lupkynis at comparable post-launch time points.EXTON, PA, Nov. 19, 2025 (GLOBE NEWSWIRE) -- Spherix Global Insights has released the first wave of Launch Dynamix™: Gazyva in Lupus Nephritis, providing the earliest independent read on the commercial introduction of Gazyva® (obinutuzumab, Genentech/Roche). The monthly service benchmarks uptake, perceptions, and key performance me
Cell Line Ups Titer 7.5-Fold for Adenovirus Production
GenEng News
2025-11-19
adenovirus
Perfusion culture for the still-developing SF-BMAdR cell line for adenovirus production increased titer to levels comparable to optimized HEK293 and PER.C6 cell lines, without producing replication-competent adenoviruses. The post Cell Line Ups Titer 7.5-Fold for Adenovirus Production appeared first on GEN - Genetic Engineering and Biotechnology News .
Approvals extend use of Roche, AbbVie drugs in lymphoma
PharmaPhorum
2025-11-19
follicular lymphoma
Patients in the US and EU with follicular lymphoma have new treatment options, thanks to new approvals for CD20xCD3 bispecifics Lunsumio and Epkinly.
Genentech continues oral SERD momentum in breast cancer: Clinical Report
Genentech
2025-11-19
breast cancer
Amid a week of clinical setbacks, a notable exception was Genentech’s oral SERD, which had its second Phase III win this year — this tim...
Roche (OTCQX: RHHBY) Lunsumio SC Offers 1-Minute Injection for R/R Follicular Lymphoma
Roche Group
2025-11-19
follicular lymphoma
EU grants conditional authorisation for subcutaneous Lunsumio in R/R follicular lymphoma, with 1 minute injection vs 2–4 hour IV infusion backed by phase I/II GO29781.
Cellectis Publishes Nature Communications Article on a Non-Viral Gene Editing Process Enabling Efficient Gene Insertion in Hematopoietic Stem Cells
cellectis
2025-11-19
aav6
talen
pivotal
gene editing
gene therapy
gene therapies
New York, NY – November 19, 2025 – Cellectis (the “Company”) (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today publishes an article in Nature Communications establishing circular single-stranded DNA (CssDNA) as a highly efficient non-viral DNA donor template, for gene insertion in hematopoietic stem and progenitor cells (HSPCs). Gene editing of HSPCs offers the potential for long-term therapeutic benefit. While viral vectors such as AAV6 are commonly used for gene insertion, they raise safety and efficacy concerns. Over the past decade, non-viral DNA templates delivery has emerged as promising alternatives and have been used with nucleases to target short single-stranded linear DNA corrective template in HSPCs. Although non-viral approaches were initially limited to making only small corrections within defective genes, Cellectis harnessed its TALEN ® technology and CssDNA donor templates, to develop a robust gene insertion process. This process enables precise and efficient integration of large genetic sequences within therapeutically relevant…
Cellipont Bioservices and Ronawk Partner to Advance Scalable Expansion of High-Quality Mesenchymal Stem Cells and Biologics
PR Newswire
2025-11-19
cell therapy
mesenchymal stem cells
THE WOODLANDS, Texas and OVERLAND PARK, Kan., Nov. 19, 2025 /PRNewswire/ -- Cellipont Bioservices, a leading cell therapy Contract Development and Manufacturing Organization (CDMO), today announced a strategic collaboration with Ronawk, developer of the Bio-OS™ biological operating...
Bayer gets EU approval for menopause drug Lynkuet
PharmaPhorum
2025-11-19
breast cancer
Bayer now has approval on both sides of the Atlantic for Lynkuet, with the EU backing it for symptoms caused by both menopause and breast cancer.
Healthy food supplementation improved quality of life in patients with heart failure
Healio
2025-11-19
heart failure
NEW ORLEANS — An intervention of healthy food deliveries improved quality of life among patients with heart failure after recent hospitalization compared with usual care and dietary counseling alone, a speaker reported.The results of the FOOD-HF trial, presented at the American Heart Association Scientific Sessions, indicated larger trials to better understand the impact of dietary
FDA Approves Darzalex Faspro for High-Risk Smoldering Multiple Myeloma
Hematology Advisor News
2025-11-19
multiple myeloma
Compared with active monitoring, treatment reduced risk for disease progression by 51 percent.
FDA approves Bayer’s HER2-mutated lung cancer drug
Endpoints News
2025-11-19
her2
lung cancer
accelerated approval
The FDA on Wednesday granted accelerated approval to Bayer’s targeted lung cancer drug for certain HER2 mutations, setting up competition with a Boehringer Ingelheim drug that was recently approved for a ...
Granulomatous Acute Interstitial Nephritis Can Cause Renal Dysfunction in CLL
Hematology Advisor News
2025-11-19
cll
The patient initiated treatment with zanubrutinib and high-dose corticosteroids, resulting in partial improvement in renal function.
European Commission approves Roche’s Lunsumio subcutaneous for relapsed or refractory follicular lymphoma
Roche Group
2025-11-19
follicular lymphoma
Lunsumio provides high rates of deep and long-lasting responses in third-line and later follicular lymphoma, a disease that typically becomes harder to treat each time a patient relapses1,2Lunsumio subcutaneous offers a new treatment option that can significantly reduce administration time to approximately one minuteAvailability of Lunsumio SC allows patients to receive treatment aligned to clinical requirements and lifestyle preferences Basel, 19 November 2025 - Roche (SIX: RO, ROG; OTCQX: RHHB
VIDEO: New understanding of abnormal proteins in Parkinson’s
Healio
2025-11-19
clearance
In this Healio video, David G. Standaert, MD, PhD, discusses a plenary session on protein aggregate clearance in aging brains presented at the 2025 International Congress of Parkinson’s Disease and Movement Disorders.
CRISPR Restores Chemotherapy Sensitivity in Lung Tumors with NRF2 Mutation
Inside Precision Medicine
2025-11-19
crispr
The NRF2 gene functions as a master regulator of genes that enable cancer cells to withstand chemotherapy, radiotherapy, and immunotherapy. The level of NRF2 in tumor cells is often a clear determinant of the effectiveness of standard of care treatments. The post CRISPR Restores Chemotherapy Sensitivity in Lung Tumors with NRF2 Mutation appeared first on Inside Precision Medicine .
Precision BioSciences Announces Presentation of Clinical Data from the ELIMINATE-B Trial at HEP-DART 2025
precision biosciences
2025-11-19
gene editing
DURHAM, N.C. --(BUSINESS WIRE)--Nov. 19, 2025-- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that it will deliver an oral
Single Prime Editing Platform Shows Potential vs. Multiple Diseases
GenEng News
2025-11-19
prime editing
Rather than edit individual genes, the prime editing technique focuses on a class of mutations that halt protein production early, raising the possibility of targeting multiple unrelated rare genetic diseases with a single treatment. The post Single Prime Editing Platform Shows Potential vs. Multiple Diseases appeared first on GEN - Genetic Engineering and Biotechnology News .
Hemostemix Closes CDN $280,594 Private Placement
Hemostemix
2025-11-19
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta – November 18, 2025 – Hemostemix Inc. (TSX-V: HEM; OTCQB: HMTXF; FSE: 2VF0) (" Hemostemix " or the " Company ") , the leading autologous (patient’s own) stem cell therapy company offering VesCell™ (ACP-01) to individuals suffering from peripheral arterial disease, chronic limb threatening ischemia, angina, ischemic cardiomyopathy, non-ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, in Florida under Florida’s SB 1768, announces the TSX Venture Exchange (" TSXV ") conditionally approved the Company’s previously disclosed non-brokered private placement (the “ Offering ”) in the amount of CDN $280,594. The Offering consisted of the issuance of 2,244,752 Common Shares at a price of CDN $0.125 per Common Share. Subject to final TSXV approval, the Company will issue 2,244,752 Common Shares.
Olema Pharmaceuticals: Shares Double On Roche's MBC Data - I Preach Caution (NASDAQ:OLMA)
Roche Group
2025-11-19
breast cancer
Olema Pharmaceuticals (OLMA) surges after Roche's breast cancer data, but caution is urged as key clinical results are awaited.
Amy Rudolph, PhD, Joins Thryv Therapeutics' Board of Directors to Support Portfolio Progression into Heart Failure
PR Newswire
2025-11-19
heart failure
MONTREAL, Nov. 19, 2025 /PRNewswire/ - Thryv Therapeutics Inc., a clinical-stage biotechnology company advancing a series of novel serum glucocorticoid inducible kinase 1 (SGK1) inhibitors for inherited cardiac arrhythmias, cardiometabolic diseases, and cardiomyopathies, today announced...
VIDEO: Livdelzi maintains improvements in cholestatic markers, liver stiffness in PBC
Healio
2025-11-19
open
In this video, Nancy S. Reau, MD, FAASLD, AGAF, discusses 36-month results from the ongoing phase 3 ASSURE open-label extension trial of Livdelzi in patients with primary biliary cholangitis.“Earlier from the study, we realized the agent worked quickly, and that quick resolution or improvement in the composite endpoint of increased or improved alkaline phosphatase of at least 15% from
Olema Pharmaceuticals (OLMA) Rethinks Competitive Edge After Roche’s Breast Cancer Trial Success
Roche Group
2025-11-19
breast cancer
Roche recently announced positive Phase 3 trial results for its breast cancer therapy, giredestrant, which operates through a mechanism that directly parallels Olema Pharmaceuticals’ lead candidate, palazestrant. This development has been widely interpreted as clinical validation for Olema’s ongoing drug program and a potential inflection point for its competitive positioning in breast cancer treatment. We will assess how this external clinical milestone, signaling broader confidence in the...
Protara trumpets small rare disease dataset in children for cell therapy
Endpoints News
2025-11-19
open
cell therapy
bladder cancer
Protara Therapeutics on Wednesday revealed new data showing its experimental bladder cancer cell therapy could also have potential in a rare pediatric disease. In a small, open-label Phase 2 dataset, Protara’s TARA-002
The American Board of Medical Specialties Research and Education Foundation 2026-2028 Independent Research Grant Application Process Is Now Open
PR Newswire
2025-11-19
open
CHICAGO, Nov. 19, 2025 /PRNewswire/ -- The American Board of Medical Specialties Research and Education Foundation (ABMS REF) has announced the opening of the 2026–2028 Research Grant Application process for independent investigators. The grant program, funded by the ABMS REF, seeks...
Roche survey: Many millennial women skipping cervical cancer screenings
Roche Group
2025-11-19
cervical cancer
Indianapolis-based Roche Diagnostics said while the survey was conducted in Europe, its implications are global and reflect challenges in the United States.
FYR Bio and NuvOx Therapeutics Awarded NCI Direct-to-Phase II SBIR Grant to Advance Liquid-Biopsy Collaboration in Phase IIb Glioblastoma Trial
PR Newswire
2025-11-19
glioblastoma
Funding builds on FYR's neuro-oncology expertise and applies EV-based biomarkers to de-risk NuvOx's clinical development MISSOULA, Mont., Nov. 19, 2025 /PRNewswire/ -- FYR Bio ("FYR"), a leader in liquid-biopsy precision medicine for neuro-oncology, today announced a collaboration with...
Gilead reports Trodelvy breast cancer trial missed primary survival endpoint
PMLive Research
2025-11-19
breast cancer
The drug treatment is being evaluated in several ongoing phase 3 trials
Genentech’s Giredestrant Becomes the First Oral SERD to Show Superior Invasive Disease-Free Survival in Early Breast Cancer
Genentech
2025-11-18
her2
breast cancer
SOUTH SAN FRANCISCO, Calif., November 18, 2025--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today positive Phase III results from the lidERA Breast Cancer study evaluating investigational giredestrant as an adjuvant endocrine treatment for people with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, early-stage breast cancer. The study met its primary endpoint at a pre-planned interim analysis, showing a statistically s
European Commission Approves BRINSUPRI® (brensocatib) as the First and Only Treatment To Date Approved for Non-Cystic Fibrosis Bronchiectasis in the European Union
PR Newswire
2025-11-18
cystic fibrosis
— Non-Cystic Fibrosis Bronchiectasis (NCFB) Is a Serious, Progressive Lung Disease That Can Lead to Permanent Lung Damagei — — BRINSUPRI Is a First-in-Disease, First-in-Class DPP1 Inhibitor Targeting Neutrophilic Inflammation — — BRINSUPRI Was Reviewed Under EMA's Accelerated Assessment...
Why Is Olema Pharmaceuticals Stock Soaring Today? - Olema Pharmaceuticals (NASDAQ:OLMA), Roche Holding (OTC:RHHBY)
Roche Group
2025-11-18
breast cancer
Roche posts positive phase 3 results for giredestrant in early breast cancer, showing improved disease-free survival and a favorable safety profile.
ENGN: Non-viral gene therapy for NMIBC achieves 62% six-month CR rate, targeting 2027 approval
enGene Google
2025-11-18
cr rate
gene therapy
detalimogene voraplasmid
Detalimogene voraplasmid, a non-viral gene therapy for NMIBC, shows strong efficacy and tolerability, with a six-month CR rate of 62% and low adverse events. Positioned for community urologists, it offers simple logistics and is on track for regulatory filing next year.Based on enGene Holdings Inc…
Immune and Genetic Changes Signal CLL Progression to Richter Transformation
Hematology Advisor News
2025-11-18
cll
Declines in CD4 central memory T cells and T-cell receptor clonal diversity at transformation may signify worse clinical outcomes.
VIDEO: Improving access to multiple myeloma care
Healio
2025-11-18
multiple myeloma
In this video, Omar Nadeem, MD, an assistant professor of medicine at Harvard Medical School, discusses improving access to relapsed/refractory multiple myeloma care options.
Genentech announces lidERA Breast Cancer study meets primary endpoint
Genentech
2025-11-18
breast cancer
Genentech, a member of Roche (RHHBY), announced Phase III results from the lidERA Breast Cancer study evaluating investigational giredestrant as an adjuvant endocri...
Arrowhead Secures Maiden Approval, First FDA Nod for Rare Genetic Metabolic Disorder
BioSpace
2025-11-18
sarepta
gene therapy
The FDA approval of the siRNA drug Redemplo caps off a tumultuous 12 months for Arrowhead, whose partnership with Sarepta caused its own stock to drop during the gene therapy maker’s safety troubles this summer.
Amicus Therapeutics Announces Second Quarter 2025 Financial Results and Corporate Updates
amicus therapeutics
2025-11-18
financial results
Arcellx to Host Investor Relations Event During the 67th ASH Annual Meeting and Exposition
arcellx, inc
2025-11-18
pivotal
cell therapy
cell therapies
multiple myeloma
Saturday, December 6, 2025 at 8:00 p.m. ET Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, today announced that management will host an Investor Relations event on Saturday, December 6, 2025 at 8:00 p.m. ET. The event will include a panel of clinician experts and a discussion of the latest data from the Company’s Phase 2 pivotal iMMagine-1 study in patients with relapsed or refractory multiple myeloma. A live webcast of this event will be accessible from Arcellx's website at www.arcellx.com in the Investors section. A replay of the webcast will be archived and available for 30 days following the event. About Arcellx, Inc. Arcellx, Inc. is a clinical-stage biotechnology company reimagining cell therapy by engineering innovative immunotherapies for patients with cancer and other incurable diseases. Arcellx believes that cell therapies are one of the
Adicet Bio Announces Oral Presentation Highlighting Preclinical ADI-270 Data at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting
Adicet Bio
2025-11-18
cell therapy
European Commission Approves BRINSUPRI™ (brensocatib) as the First and Only Treatment Approved for Non-Cystic Fibrosis Bronchiectasis in the European Union
PR Newswire
2025-11-18
cystic fibrosis
— Non-Cystic Fibrosis Bronchiectasis (NCFB) Is a Serious, Progressive Lung Disease That Can Lead to Permanent Lung Damage — — BRINSUPRI Is a First-in-Disease, First-in-Class DPP1 Inhibitor Targeting Neutrophilic Inflammation — — BRINSUPRI Was Reviewed Under EMA's Accelerated Assessment...
Do Rising Losses at uniQure (QURE) Reflect Strategic Investment or Escalating Cost Pressure?
uniQure
2025-11-18
quarterly
uniQure N.V. reported its third quarter 2025 financial results on November 10, with revenue rising to US$3.7 million from US$2.29 million a year earlier, accompanied by a net loss of US$80.53 million. An interesting insight from the announcement is that, despite higher quarterly revenue, the company’s losses deepened compared to the previous year, pointing to ongoing cost pressures or investment needs. We’ll now explore how uniQure’s widening quarterly losses and revenue changes could impact...
Carvedilol linked to fewer exacerbations vs. metoprolol in heart failure, asthma
Healio
2025-11-18
heart failure
ORLANDO — Fewer adults with heart failure and asthma treated with carvedilol vs. metoprolol had an asthma exacerbation, according to a poster presented at the American College of Allergy, Asthma & Immunology Annual Scientific Meeting.“This study is not enough to say we should recommend carvedilol in patients with asthma, but it’s enough to maybe plant a seed to consider
Hemophilia B Research Report 2025-2035: Market Led by UniQure, Pfizer, Novo Nordisk, HEMA Biologics, and Alnylam Pharmaceuticals as Next-Generation Therapies and R&D Investment Drive Innovation
uniQure
2025-11-18
beqvez
hemgenix
gene therapy
hemophilia b
The Hemophilia B market is growing rapidly due to advancements in gene therapy and diagnostics. This rare genetic disorder, caused by F9 gene mutations, primarily affects males. Revolutionary treatments like Hemgenix and Beqvez offer potential functional cures by reducing bleeding rates. Early detection using next-generation sequencing enhances patient management and personalized care. Despite challenges such as high costs and limited long-term safety data, leading companies like UniQure and Pfi
8-K - BRISTOL MYERS SQUIBB CO (0000014272) (Filer)
SEC Filing (8-K)
2025-11-18
bristol myers squibb
Filed: 2025-11-18 AccNo: 0001140361-25-042607 Size: 937 KB Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
AbbVie Announces U.S. FDA Approval of EPKINLY® (epcoritamab-bysp) in Combination with Rituximab and Lenalidomide for Relapsed or Refractory Follicular Lymphoma
Abbvie
2025-11-18
follicular lymphoma
EPKINLY plus rituximab and lenalidomide (EPKINLY + R2) is now the first and only bispecific antibody combination therapy available for patients with relapsed or refractory follicular lymphoma...
Roche boosts Olema after breast cancer trial win (RHHBY)
Roche Group
2025-11-18
breast cancer
Roche (RHHBY) boosts Olema Pharma (OLMA) stock as its breast cancer drug, giredestrant, similar to Olma's lead drug, succeeds in a Phase 3 trial. Read more here.
Our gene therapy milestone honors all those fighting AADC deficiency
AADC News/BioNews
2025-11-18
gene therapy
Within the community of people with aromatic L-amino acid decarboxylase (AADC) deficiency, we refer to the day of gene therapy as “Reborn Day” because it truly marks a new beginning. Our family celebrated our daughter Rylae-Ann’s seventh Reborn Day on Nov. 13. For my wife, Judy, and me, it was not only a celebration of […] The post Our gene therapy milestone honors all those fighting AADC deficiency appeared first on AADC News .
FDA approves epcoritamab for two follicular lymphoma indications
Healio
2025-11-18
follicular lymphoma
accelerated approval
The FDA approved epcoritamab-bysp in combination with lenalidomide and rituximab for patients with relapsed or refractory follicular lymphoma and as monotherapy for these patients after two or more lines of systemic therapy.Healio previously reported that epcoritamab-bysp (Epkinly; Genmab, AbbVie) — a CD3xCD20 bispecific antibody — received accelerated approval in 2024 for patients
Carvykti label updated to include survival benefit data in myeloma
Rare Cancer News
2025-11-18
carvykti
cell therapy
Regulators in the U.S. and Europe have approved updates to the prescribing information for Carvykti (ciltacabtagene autoleucel), a cell therapy used to treat hard-to-manage myeloma. The updates include survival data from the ongoing CARTITUDE-4 (NCT04181827) Phase 3 clinical trial. This study compares Carvykti with standard myeloma treatments in people who have received one to three prior […] The post Carvykti label updated to include survival benefit data in myeloma appeared first on Rare Cancer News .
RNAi biotech Arrowhead wins first FDA approval
Biopharma Dive
2025-11-18
clearance
The clearance of Redemplo for a rare genetic disorder marks Arrowhead’s transition, after two decades, into a commercial-stage company and sets up a turf war with Ionis Pharmaceuticals.
Kazia Therapeutics Achieves Initial iCR (Immune-Complete Response) in Metastatic TNBC and Delivers Q4 Business Update with Breakthroughs Across Breast Cancer, Immuno-Oncology, and GBM Regulatory Strategy
PR Newswire
2025-11-18
gbm
breast cancer
complete response
SYDNEY, Nov. 18, 2025 /PRNewswire/ -- Kazia Therapeutics Limited (Nasdaq: KZIA), an oncology-focused drug development company, today announced that a patient with stage IV triple-negative breast cancer (TNBC) treated under an FDA-authorized single-patient expanded access protocol...
Genentech’s Multiple Sclerosis Drug Shows Promising Phase III Results
Genentech
2025-11-18
multiple sclerosis
On Tuesday, Roche Holding AG ADR surged 7.78% after positive Aviceda results, boosting investor confidence. Key takeaways Genentech’s Phase III trial of fenebrutinib for multiple sclerosis revealed a reduction in relapses compared to teriflunomide, showing promising outcomes. Roche Group reports a 7% increase in 2025 sales, driven by strong pharmaceutical demand and positive clinical trial […]
Top 3 Genomics Stocks to Consider for Your Portfolio
Krystal Biotech
2025-11-18
gene editing
Genomics gains momentum as EDIT, SANA, and PACB emerge as standout picks amid rapid advances in sequencing and gene editing.
Genprex Announces U.S. Patent for Reqorsa® Gene Therapy in Combination with PD-L1 Antibodies to Treat Cancers
Genprex
2025-11-18
gene therapy
Strengthens Intellectual Property Portfolio and Provides Protection for Therapeutic Combination in Acclaim-3 Clinical Trial AUSTIN, Texas — (Nov. 18, 2025) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that the United States Patent and Trademark Office (USTPO) has granted Genprex […]
Fixed-Duration Therapy May Revise Definitions of Progression in CLL
Hematology Advisor News
2025-11-18
cll
Importantly, the 12-month threshold was consistent across treatment modalities, confirmed in both chemoimmunotherapy and targeted therapy subgroups.
Amicus Therapeutics to Announce Second Quarter 2025 Financial Results on July 31, 2025
amicus therapeutics
2025-11-18
financial results
Roche obtains promising trial data with breast cancer tablet
Roche Group
2025-11-18
breast cancer
In the study, drug giredestrant has helped women with a form of early breast cancer, allowing them to live longer without the disease progressing.
Genentech (OTCQX: RHHBY) Oral SERD Giredestrant First to Improve IDFS in Breast Cancer
Genentech
2025-11-18
her2
breast cancer
Phase III lidERA study in ER-positive, HER2-negative early breast cancer met its primary endpoint, showing meaningful IDFS benefit vs standard endocrine therapy.
Gilead single tablet regimen of Bictegravir, Lenacapavir for HIV-1 meets primary endpoint in Phase 3 trial
Gilead Sciences
2025-11-18
open
Foster City: Gilead Sciences has announced positive topline results from the Phase 3 ARTISTRY-1 trial. The open-label trial evaluated the treatment responses of adults with HIV who are...
BioCytics Successfully Completes a Key Regulatory Milestone with Recent FDA INTERACT Meeting
PR Newswire
2025-11-18
cell therapies
HUNTERSVILLE, N.C., Nov. 18, 2025 /PRNewswire/ -- BioCytics is pleased to announce they successfully reached a regulatory milestone for de-risking clinical development plans with a productive FDA INTERACT meeting during which they discussed manufacturing immune cell therapies at the point...
Amicus Therapeutics Announces Third Quarter 2025 Financial Results and Corporate Updates
amicus therapeutics
2025-11-18
financial results
VIDEO: Myeloma treatment has ‘evolved significantly over the past decade’
Healio
2025-11-18
multiple myeloma
In this video, Omar Nadeem, MD, an assistant professor of medicine at Harvard Medical School, discusses advances in relapsed/refractory multiple myeloma care.
Roche (OTCQX: RHHBY) drug first oral SERD to improve iDFS in ER+ early breast cancer
Roche Group
2025-11-18
her2
breast cancer
Phase III lidERA study showed statistically significant, meaningful invasive disease-free survival benefit for giredestrant in ER+, HER2- early breast cancer.
OLMA Stock Doubles After Roche Cancer Drug Trial Yields Positive Results
Roche Group
2025-11-18
breast cancer
Roche was evaluating Giredestrant as an adjuvant endocrine treatment for people with certain types of early-stage breast cancer.
Roche oral drug shown to keep breast cancer at bay, boosting shares
Roche Group
2025-11-18
breast cancer
FRANKFURT (Reuters) -Roche's experimental oral drug giredestrant has been shown to cut the risk of recurrence of a common form of breast cancer after surgery, boosting the company's shares and underpinning its traditional credentials in oncology. The Swiss drugmaker said on Tuesday that a scheduled interim analysis of a late-stage trial showed the experimental pill resulted in a clinically meaningful improvement in keeping patients disease-free after surgery compared with standard endocrine
Adicet Bio Receives FDA Fast Track Designation for ADI-001 for the Treatment of Systemic Sclerosis (SSc)
Adicet Bio
2025-11-18
fast track
Roche's $15 Billion Breast Cancer Pill Just Stunned the Market--But AstraZeneca Is Lurking
Roche Group
2025-11-18
breast cancer
A surprise late-stage win just lit up Roche stock, but the real fight for dominance may only be beginning.
Adicet Bio Receives FDA Fast Track Designation for ADI-001 for the Treatment of Refractory Systemic Lupus Erythematosus (SLE) with Extrarenal Involvement
Adicet Bio
2025-11-18
fast track
Genentech’s Giredestrant Becomes the First Oral SERD to Show Superior Invasive Disease-Free Survival in Early Breast Cancer
Roche Group
2025-11-18
breast cancer
VIDEO: Choosing the best therapy after multiple myeloma relapse
Healio
2025-11-18
multiple myeloma
In this video, Omar Nadeem, MD, an assistant professor of medicine at Harvard Medical School, discusses strategies for choosing the best treatment option for a patient after multiple myeloma recurrence.
Q&A: ACLM launches free online resources to address gap in nutrition education
Healio
2025-11-18
open
DALLAS — The American College of Lifestyle Medicine recently unveiled two open-source resources to educate health care professionals and patients about healthy eating and cooking techniques.These resources — the Culinary Medicine Program and updated second edition of the Culinary Medicine Curriculum — are designed to be practical and accessible “to help people discover
Roche shares soar on breast cancer drug breakthrough
Roche Group
2025-11-18
breast cancer
Roche's giredestrant pill successfully reduced breast cancer recurrence risk in trials, boosting shares 6% and potentially generating $5 billion annually.
Hemophilia Research Analysis Report 2025-2035: Market Advances as Leading Players Accelerate Gene Therapy Innovation and Strategic Partnerships, Led by CSL Behring, Takeda, and Pfizer
CSL Behring
2025-11-18
gene therapy
The global hemophilia market is experiencing robust growth, fueled by advances in gene therapy and recombinant clotting factors. Increased awareness and healthcare investment worldwide are expanding access to innovative treatments such as extended half-life therapies and novel biologics, enhancing patient outcomes. The U.S. market drives this expansion, supported by a strong healthcare infrastructure and evolving regulatory landscape. However, high therapy costs and regulatory hurdles remain cha
PADCEV™ (enfortumab vedotin-ejfv) Plus KEYTRUDA® (pembrolizumab) sBLA Granted FDA Priority Review for Treatment of Certain Patients with Muscle-Invasive Bladder Cancer
astellas
2025-11-18
pivotal
bladder cancer
priority review
Results from the pivotal EV-303 trial demonstrated that, when used before and after surgery, the combination reduced the risk of recurrence, progression or death by 60% and the risk of death by...
uniQure N.V. Investigation: Kessler Topaz Meltzer & Check, LLP Encourages uniQure N.V. (NASDAQ: QURE) Investors With Significant Losses to Contact the Firm
uniQure
2025-11-18
bla
amt-130
gene therapy
RADNOR, PA - November 18, 2025 (NEWMEDIAWIRE) - The law firm of Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) is currently investigating potential violations of the federal securities laws on behalf of investors of uniQure N.V. (NASDAQ: QURE) (“uniQure”). On November 3, 2025, uniQure issued a press release revealing that the FDA notified the company that data for its AMT-130, an investigational gene therapy for Huntington’s disease, did not provide sufficient evidence to support uniQure’s Biologics License Application (“BLA”) submission. Specifically, uniQure disclosed that the company believes the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate to provide the primary evidence in support of a BLA submission, and that the timing of the BLA submission for AMT-130 is now unclear as a result. On this news, the price of uniQure’s stock fell over 50%, from a close of $67.69 on October 31, 2025, to close at $34.29 on November 3, 2025. If you are a uniQure investor and would like to learn more about our investigation, please CLICK HERE to fill out our online form or contact Kessler Topaz Meltzer & Check, LLP: Jonathan Naji, Esq. (484)…
Amicus Therapeutics to Announce Third Quarter 2025 Financial Results on November 4, 2025
amicus therapeutics
2025-11-18
financial results
Merck’s blockbuster-to-be cardiovascular drug scores in another heart study
Biopharma Dive
2025-11-18
heart failure
Winrevair, which Merck has identified as a “growth driver,” significantly improved blood flow through the lungs to the heart in a study of adults with a common form of heart failure.
VIDEO: Good disease management impacts quality of life in myeloma
Healio
2025-11-18
multiple myeloma
In this video, Omar Nadeem, MD, an assistant professor of medicine at Harvard Medical School, discusses the impact of multiple myeloma treatment on quality of life and strategies to manage this impact.
J&J’s $3B Halda Acquisition Brings a Way to Overcome Drug Resistance in Prostate Cancer & More
MedCity News
2025-11-17
phase 1
prostate cancer
Johnson & Johnson is acquiring Halda Therapeutics for its lead program, a drug that recently posted encouraging Phase 1 data in advanced prostate cancer. This drug comes from a proprietary Halda platform technology that yields oral small molecules offering a novel mechanism for killing cancer cells. The post J&J’s $3B Halda Acquisition Brings a Way to Overcome Drug Resistance in Prostate Cancer & More appeared first on MedCity News .
Securing quality in every dose: 5 essential capabilities for CGT developers to look for in a fill-finish CDMO
Biopharma Dive
2025-11-17
gene therapies
With their promise to transform disease trajectories, cell and gene therapies (CGTs) are bringing new hope to patients with an ever-expanding array of high-burden and chronic conditions as well as rare genetic disorders.
Komzifti Approved for Adults With R/R NPM1-Mutated AML
Hematology Advisor News
2025-11-17
aml
The FDA has approved Komzifti for the treatment of R/R AML in adults with a susceptible NPM1 mutation who have no satisfactory alternative treatment options.
Blood test shows ‘fantastic’ predictive value for preeclampsia in sickle cell disease
Healio
2025-11-17
sickle cell disease
Women with sickle cell disease have a significantly higher likelihood of developing preeclampsia than the general population, but a blood test could identify which patients have the greatest risk.A retrospective analysis showed low placental growth factor (PlGF) determined early-onset preeclampsia with 100% sensitivity and specificity between 20 and 24 weeks gestation, and had near 100%
Peanut patch safe, effective in toddlers 3 years after placebo use
Healio
2025-11-17
msc
ORLANDO — Toddlers with a peanut allergy built tolerance with 3 years of epicutaneous immunotherapy after a year of placebo use, according to a presentation at the American College of Allergy, Asthma & Immunology Annual Scientific Meeting.There were no new safety signals with this treatment group either, Matthew Greenhawt, MD, MBA, MSc, FACAAI, FAAAAI, chief medical officer of the
Artios Pushes Cancer Pipeline Forward With $115M Series D
BioSpace
2025-11-17
pipeline
second-line
colorectal cancer
pancreatic cancer
Artios Pharma is working on a pipeline of oncology assets, led by alnodesertib, currently being tested for second-line pancreatic cancer and third-line colorectal cancer.
Scholar Rock plans to launch spinal muscular atrophy drug next year
Endpoints News
2025-11-17
spinal muscular atrophy
Scholar Rock said it intends to launch its spinal muscular atrophy drug apitegromab next year, even after the FDA rejected the drug over issues it found at a manufacturing site run by Novo Nordisk. ...
Sarepta rises after FDA adds warning label to Elevidys
PharmaPhorum
2025-11-17
sarepta
elevidys
gene therapy
Uncertainty ends Sarepta's Duchenne gene therapy Elevidys ends after an FDA label update, with the company's shares on the up despite restrictions.
Jeffrey Bluestone hands Sonoma CEO post to Stephen Dilly
Endpoints News
2025-11-17
regulatory t cell
Jeffrey Bluestone is stepping down from the CEO role at Sonoma Biotherapeutics, a regulatory T cell company he co-founded six years ago. He'll be replaced by Stephen Dilly, who previously led RNAi therapy manufacturer Codexis ...
Immatics Announces Third Quarter 2025 Financial Results and Business Update
Immatics Biotechnologies
2025-11-17
phase 3
melanoma
cell therapy
Anzu-cel (anzutresgene autoleucel, IMA203) PRAME Cell Therapy: Global, randomized, controlled Phase 3 trial, SUPRAME, in previously treated advanced melanoma ongoing; interim and final analyses will occur in 2026 Anzu-cel (IMA203) PRAME Cell Therapy: One-time infusion continues to show strong
Cincinnati Children's accelerating breakthroughs in care for cancer, blood diseases and genetic disorders with new center
PR Newswire
2025-11-17
cell therapy
Applied Gene and Cell Therapy Center's cutting-edge facility boosts capacity to manufacture innovative therapies for clinical trials CINCINNATI, Nov. 17, 2025 /PRNewswire/ -- Cincinnati Children's today celebrated the opening of a new state-of-art facility for its Applied Gene and Cell...
VRD With BUMEL or MEL200 Yields Long PFS in Newly-Diagnosed Multiple Myeloma Following ASCT
Hematology Advisor News
2025-11-17
multiple myeloma
Induction and consolidation with VRD and conditioning with either BUMEL or MEL200 was associated with long PFS among patients with NDMM following ASCT.
Anixa Biosciences Announces World Health Organization (WHO) Approval of International Non-Proprietary Name for its CAR-T Therapy, Marking a Key Milestone Toward Global Recognition and Future Commercialization
anixa biosciences
2025-11-17
car-t
phase 1
cell therapies
first-in-human
ovarian cancer
chimeric antigen receptor
SAN JOSE, Calif. , Nov. 17, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the International Nonproprietary Names (INN) Expert Committee of the World Health Organization (WHO) approved "liraltagene autoleucel" for the non-proprietary name of the Company's novel FSHR-targeted CAR-T therapy for recurrent ovarian cancer. The INN nomenclature scheme for CAR-T cell therapies follows a two-word structure describing the gene and cell component. "The assignment of the non-proprietary name represents an important step in the development and potential future commercialization of our CAR-T therapy. The INN naming process meticulously evaluates proposed drug names for adherence to nomenclature guidelines and potential conflicts, followed by expert consensus and public review. With this INN approval, we have the ability to establish a universally recognized and conflict-free non-proprietary drug name for our CAR-T therapy," said Dr. Amit Kumar, Chairman and CEO of Anixa. "Looking ahead, we remain focused on the successful execution of our ongoing Phase 1…
CG Oncology stock rises as RBC Capital raises price target on BLA filing progress By Investing.com
CG Oncology
2025-11-17
bla
CG Oncology stock rises as RBC Capital raises price target on BLA filing progress
Zymeworks and Jazz tout HER2 bispecific success in first Phase 3 readout
Endpoints News
2025-11-17
her2
Zymeworks and Jazz Pharmaceuticals said that the addition of their HER2 bispecific Ziihera helped stave off disease progression in certain patients with gastroesophageal adenocarcinoma — a type of cancer that occurs in the stomach, esophagus and ...
9 Best Gene-Editing Stocks to Buy According to Hedge Funds
Krystal Biotech
2025-11-17
gene editing
Genetic manipulation – or gene editing – is a concept that describes altering the genetic material (DNA) of an organism.
Study: Autoimmune impacts of CAR T-cell therapy require monitoring
Healio
2025-11-17
car t
icans
lupus
cell therapy
chimeric antigen receptor
cytokine release syndrome
CHICAGO — De novo autoimmune diseases, including myositis, vasculitis and lupus, may occur after chimeric antigen receptor T-cell therapy for cancer, according to data presented at ACR Convergence 2025. “Although immune-medicated toxicities such as cytokine release syndrome and ICANs are well described, the autoimmune safety profile remains poorly defined,” said Malvika Gupta, MD.
J&J adds to pharma’s M&A spree with $3B buyout of startup Halda
Biopharma Dive
2025-11-17
pipeline
prostate cancer
The deal gives J&J access to a pipeline of drugs designed to “hold and kill” tumors, led by a prostate cancer medicine that’s shown promise in early-stage testing.
Pressure On, Purpose Questioned as Second Round of FDA Priority Review Vouchers Revealed
BioSpace
2025-11-17
priority review
Priority voucher awardees and regulators could feel pressure to “meet the moment” as FDA watchers question the intent and feasibility of the Commissioner’s National Priority Voucher program.
Updated NCCN guidelines on NSCLC put spotlight on biomarker testing
Healio
2025-11-17
first-line
lung cancer
Updates to National Comprehensive Cancer Network guidelines on non-small cell lung cancer emphasize the importance of biomarker testing in determining correct treatment course.NCCN now recommends osimertinib (Tagrisso, AstraZeneca) plus chemotherapy and lazertinib (Lazcluze, Janssen) plus amivantamab-vmjw (Rybrevant, Janssen) as first-line treatment for EGFR-mutant metastatic NSCLC. They join
Solid Biosciences Licenses Use of Next-Generation Capsid AAV-SLB101 to Andelyn Biosciences
GenEng News
2025-11-17
aav
gene therapy
Solid grants Andelyn a non-exclusive worldwide license to provide its gene therapy clients with access to utilize AAV-SLB101 in combination with Andelyn’s suspension and adherent modular platform process—AAV Curator. The post Solid Biosciences Licenses Use of Next-Generation Capsid AAV-SLB101 to Andelyn Biosciences appeared first on GEN - Genetic Engineering and Biotechnology News .
FDA Imposes Boxed Warning, Narrows Patient Pool for Sarepta’s Duchenne Gene Therapy Elevidys
BioSpace
2025-11-17
sarepta
elevidys
gene therapy
Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed with the gene therapy.
ONE HEMP Urges Immediate Action on Misguided Federal Hemp Restrictions
PR Newswire
2025-11-17
open
WASHINGTON, Nov. 17, 2025 /PRNewswire/ - Congress has introduced measures that require swift, united action to safeguard the hemp wellness sector. Language buried in the compromise package to open up the government – in the Agriculture, Rural Development, and FDA Appropriations Act - bans...
Protara Therapeutics to Present New Interim Data from Phase 2 ADVANCED-2 Trial of TARA-002 in BCG-Naïve NMIBC Patients at the 26th Annual Meeting of the Society of Urologic Oncology
protara therapeutics
2025-11-17
open
phase 2
NEW YORK , Nov. 17, 2025 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced that interim data from Cohort A of the ongoing Phase 2 open-label ADVANCED-2 trial of
VIDEO: Differences in CAR-T therapy examined at LLM
Healio
2025-11-17
cd19
car-t
cell therapy
chimeric antigen receptor
In this video, Catherine S. Diefenbach, MD, discusses the differences between three FDA-approved CD19-directed chimeric antigen receptor T-cell therapy products for the treatment of lymphoma.
Adaptimmune Announces Changes to Board and Executive Leadership Team
Adaptimmune Therapeutics
2025-11-17
tecelra
cell therapy
Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - November 17, 2025) - Adaptimmune Therapeutics plc (OTC Pink: ADAPY) ("Adaptimmune" or the "Company") today announced changes to the Company's Board of Directors ("Board") and its Executive Leadership team, which are effective from November 14, 2025. Christopher (Chris) Hill, currently Chief Financial Officer ("CFO") of the Company, has been appointed as Chief Executive Officer ("CEO") and a Director of the Company, succeeding Adrian Rawcliffe who has left in connection with the Company's restructuring. Mr. Hill is continuing to serve as CFO. William (Bill) Bertrand, formerly Chief Operating Officer, has also left the Company in connection with the restructuring. Lawrence Alleva, a current Board member, has been appointed as Chairman of the Company succeeding David Mott who has served as a director since 2014 and as Chairman since 2017. Garry Menzel will also continue to serve on the Board. In addition to Mr. Mott, the following Board members have resigned: Andrew Allen, Ali Behbahani, John Furey, Priti Hegde and Kristen Hege. Following the changes, the Board comprises Lawrence Alleva (Chairman), Garry Menzel…
Cryptic AML Gene Fusions Identified Through Enhanced NGS Panel
Inside Precision Medicine
2025-11-17
aml
leukemia
The added element is a probe-ligation–based targeted gene fusion assay that is designed to identify 112 gene fusions that are known to be associated with leukemia. The post Cryptic AML Gene Fusions Identified Through Enhanced NGS Panel appeared first on Inside Precision Medicine .
AMCP Nexus 2025: The specialty surge — How to stay atop the latest in trend and spend
Prime Therapeutics
2025-11-17
pipeline
Prime Therapeutics clinical experts shared insights on the specialty drug pipeline at recent industry event
VIDEO: Advancements in T-cell, Hodgkin lymphomas, DLBCL highlighted at LLM 2025
Healio
2025-11-17
dlbcl
leukemia
In this video, Catherine S. Diefenbach, MD, discusses highlights from the 2025 Lymphoma, Leukemia & Myeloma Congress.
FDA approves ziftomenib to treat some relapsed leukemia patients
Rare Cancer News
2025-11-17
aml
leukemia
acute myeloid leukemia
The U.S. Food and Drug Administration (FDA) has approved ziftomenib to treat adults with relapsed or refractory acute myeloid leukemia (AML) with a susceptible NPM1 gene mutation who have no satisfactory alternative treatment options. The once-daily oral therapy, developed by Kura Oncology and Kyowa Kirin, will be sold under the brand name Komzifti. “Together with […] The post FDA approves ziftomenib to treat some relapsed leukemia patients appeared first on Rare Cancer News .
CG Oncology, Inc.: Promising Future with Creto’s Accelerated Approval Path and Market Differentiation
CG Oncology
2025-11-17
accelerated approval
TD Cowen analyst Tyler Van Buren has maintained their bullish stance on CGON stock, giving a Buy rating on November 14. Tyler Van Buren has given his Buy rating due...
Wall Street Has a Positive Outlook on uniQure N.V. (QURE) Despite Challenges From The FDA
uniQure
2025-11-17
amt-130
gene therapy
uniQure N.V. (NASDAQ:QURE) is one of the Hottest Small Cap Stocks to Buy Now. Wall Street has a positive outlook on uniQure N.V. (NASDAQ:QURE) despite the regulatory challenges from the FDA for its experimental gene therapy called AMT-130. On November 12, Yanan Zhu from Wells Fargo also reiterated a Buy rating on uniQure N.V. (NASDAQ:QURE), […]
FDA limits Elevidys use; Nxera to lay off staff
Biopharma Dive
2025-11-17
elevidys
Elevidys now has a “boxed” warning and is no longer available for Duchenne patients who can’t walk. Elsewhere, Nuvalent and Zymeworks unveiled data for targeted cancer drugs.
BMS, J&J give up on milvexian for acute coronary syndrome
PharmaPhorum
2025-11-17
atrial fibrillation
BMS and J&J call time on oral anticoagulant milvexian in acute coronary syndrome, but are still running trials in atrial fibrillation and stroke.
Wall Street Has a Positive Outlook on uniQure N.V. (QURE) Despite Challenges From The FDA
uniQure
2025-11-17
amt-130
gene therapy
uniQure N.V. (NASDAQ:QURE) is one of the Hottest Small Cap Stocks to Buy Now. Wall Street has a positive outlook on uniQure N.V. (NASDAQ:QURE) despite the regulatory challenges from the FDA for its experimental gene therapy called AMT-130. On November 12, Yanan Zhu from Wells Fargo also reiterated a Buy rating on uniQure N.V. (NASDAQ:QURE), […]
Sandoz launches first biosimilar Tysabri in US
PharmaPhorum
2025-11-17
multiple sclerosis
Biogen is facing biosimilar competition to its multiple sclerosis therapy Tysabri in the US for the first time, as Sandoz enters the market.
Nuvalent shares Phase 1/2 data for ALK inhibitor that could battle with Pfizer’s Lorbrena
Endpoints News
2025-11-17
phase 1
phase 1/2
lung cancer
Nuvalent reported positive mid-stage data for its ALK inhibitor in patients with a genetically driven form of lung cancer, reinforcing the company’s plans for an FDA filing. The biotech is testing its drug, called neladalkib ...
AHA: Long-Term Melatonin Use for Insomnia Tied to Higher Risk for Heart Failure
Hematology Advisor News
2025-11-17
heart failure
Similarly, doubled risk for all-cause mortality seen during five-year study period.
CG Oncology Plans To Complete BLA Submission For Cretostimogene In Bladder Cancer In 2026
CG Oncology
2025-11-17
bla
bladder cancer
(RTTNews) - CG Oncology Inc. (CGON), a clinical-stage biopharmaceutical company, is making steady progress in the development of its lead investigational oncolytic immunotherapy, Cretostimogene, as monotherapy for patients with high-risk BCG-unresponsive non-muscle invasive bladd
Accelerated Cretostimogene Advances Could Be a Game Changer for CG Oncology (CGON)
CG Oncology
2025-11-16
phase 3
pivotal
pipeline
bladder cancer
CG Oncology announced it has initiated a rolling Biologics License Application submission to the U.S. FDA for cretostimogene, its therapy for high-risk non-muscle invasive bladder cancer, and completed enrollment ahead of schedule for its pivotal Phase 3 trial in intermediate-risk disease. These key steps reflect strong clinical momentum and signal growing stakeholder and physician interest in the company’s urologic oncology pipeline. With these milestones, we’ll explore how accelerated...
Krystal Biotech: VYJUVEK Gene Therapy Well Positioned For Growth (NASDAQ:KRYS)
Krystal Biotech
2025-11-16
vyjuvek
pipeline
gene therapy
Krystal Biotech drives Vyjuvek growth, premium margins, global expansion, a $1B goal, and pipeline data ahead with a $219.50 target. See why KRYS stock is a buy.
Merck to Acquire Cidara for $9.2B, Bolstering Antiviral Pipeline
GenEng News
2025-11-16
pipeline
Cidara’s lead pipeline candidate, CD388, is a long-acting antiviral designed to achieve universal prevention of seasonal and pandemic influenza. CD388 consists of a small molecule neuraminidase inhibitor stably conjugated to a proprietary Fc fragment of a human antibody designed to prevent influenza A and B. The post Merck to Acquire Cidara for $9.2B, Bolstering Antiviral Pipeline appeared first on GEN - Genetic Engineering and Biotechnology News .
CARsgen’s CAR-T Satri-Cel Significantly Reduces CA19-9 Levels in Patients With Pancreatic Ductal Adenocarcinoma
CGTLive
2025-11-16
car-t
It was noted that the CA19-9 reductions in the 5 patients observed after infusion of satri-cel ranged from 51.3% to 96.1%.
MiNK Therapeutics Reports Q3 2025 Results and Accelerates iNKT Platform Toward Pivotal Development Across Oncology, Pulmonary Disease, and Transplantation
Mink Therapeutics
2025-11-15
pivotal
New clinical data show durable remissions and long-tail survivors including >2-year complete remissions in chemotherapy- and checkpoint-refractory cancers GVHD trial launching through non-dilutive funding from NIH - and philanthropic grants Launching Phase 2+ trial in severe pulmonary disease in US
Die Fondazione Telethon gibt bekannt, dass der CHMP eine positive Stellungnahme zu Waskyra™ abgegeben hat, einer Gentherapie zur Behandlung des Wiskott-Aldrich-Syndroms (WAS)
Fondazione Telethon
2025-11-15
wiskott-aldrich
/PRNewswire/ -- Fondazione Telethon gibt die positive Stellungnahme des Ausschusses für Humanarzneimittel (CHMP) der Europäischen Arzneimittelagentur (EMA)...
What CG Oncology (CGON)'s FDA BLA Submission and Phase 3 Progress Mean for Shareholders
CG Oncology
2025-11-15
bla
phase 3
quarterly
bladder cancer
CG Oncology announced the initiation of a rolling Biologics License Application submission to the U.S. FDA for cretostimogene, its therapy targeting high-risk BCG-unresponsive non-muscle invasive bladder cancer, along with early completion of enrollment in the Phase 3 PIVOT-006 trial. These regulatory milestones follow the company’s report of a very large year-over-year increase in quarterly revenue, highlighting momentum in both clinical and commercial efforts. We’ll take a look at how the...
AskBio’s Heart Failure Gene Therapy AB-1002 Shows Ability to Improve NYHA Class in Phase 1 Trial
CGTLive
2025-11-15
phase 1
gene therapy
A Fresh Look at Autolus Therapeutics (AUTL) Valuation Following Earnings, Obe-cel Expansion, and Anticipated ASH Data
Autolus Therapeutics
2025-11-15
quarterly
Autolus Therapeutics (AUTL) just released its latest quarterly earnings and is drawing increased investor attention thanks to several key developments. The company continues to expand commercial access for Obe-cel, reports strong manufacturing results, and anticipates new data at the upcoming ASH conference. See our latest analysis for Autolus Therapeutics. Autolus Therapeutics has seen a burst of activity lately, from broadening commercial access and updating its leadership team to advancing...
EMA’s CHMP recommends five new products
Fondazione Telethon
2025-11-15
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
CHMP recommended approval of the first gene therapy for Wiskott-Aldrich syndrome, a rare genetic disease affecting blood and immune cells, at its November ...
Update on Phase 3 Librexia ACS Trial
Bristol Myers Squibb
2025-11-14
phase 3
Penn and CHOP will test gene therapy for rare diseases with a new FDA trial protocol
uniQure
2025-11-14
crispr
gene therapy
Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy.
Bristol Myers, J&J’s next-gen blood thinner fails key trial in another setback for factor XI
Endpoints News
2025-11-14
phase 3
Pharma’s big quest to make safer blood thinners isn't going well. On Friday, Bristol Myers Squibb and Johnson & Johnson announced that a Phase 3 trial of their experimental drug milvexian ...
Coave Therapeutics unveils lead gene therapy program for retinal vascular diseases, powered by first-in-class vector for suprachoroidal delivery
coave therapeutics
2025-11-14
gene therapy
How the FDA Is Accelerating Cell and Gene Therapy’s Next Chapter
BioSpace
2025-11-14
gene therapy
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.
Can Autolus Therapeutics' (AUTL) Expanding Obe-cel Reach Bolster Its Position in Cell Therapy?
Autolus Therapeutics
2025-11-14
cell therapy
U.S. stocks, Saudi stocks, stock trading and investment platforms
Autolus Therapeutics: Trading At Cash Is Not Justified With This Pipeline (NASDAQ:AUTL)
Autolus Therapeutics
2025-11-14
pipeline
cell therapy
Autolus Therapeutics plc is continuing its work to become a pioneering cell therapy company in the space of AD. Learn more about AUTL stock here.
enGene Holdings Soars: Is a Buy Warranted?
enGene
2025-11-14
gene therapy
bladder cancer
enGene Holdings Inc. stocks have been trading up by 37.94 percent, driven by optimistic market sentiment and positive developments. Shine in the Spotlight: enGene’s Best Place to Work Recognition Recognized as a Best Place to Work by BioSpace, enGene is marking its territory in the non-viral gene therapy landscape, especially focusing on bladder cancer treatment…
VIDEO: Gildeuretinol in Stargardt disease improves low luminance visual acuity
Healio
2025-11-14
stargardt disease
In this video from the American Academy of Ophthalmology meeting, Philip J. Ferrone, MD, discusses the TEASE-2 trial results in treating Stargardt disease with gildeuretinol.
Cognision Announces Partnership with Kynexis to Utilize its COGNISION® System in a Phase 2 Trial of KYN-5356 in Subjects with Schizophrenia
PR Newswire
2025-11-14
phase 2
LOUISVILLE, Ky., Nov. 14, 2025 /PRNewswire/ -- Cognision, provider of advanced neurobiomarker technology, announces its partnership with Kynexis to utilize the COGNISION® System in Kynexis' Phase 2 clinical trial of KYN-5356 in subjects with schizophrenia. KYN‑5356 is designed to inhibit...
Gilead’s investigational single-tablet regimen of bictegravir and lenacapavir for HIV-1 treatment meets primary endpoint in Phase 3 ARTISTRY-1 trial
Gilead Sciences
2025-11-14
phase 3
The novel HIV therapy has the potential to expand options for virologically suppressed adults treated with complex ART regimens.
CG Oncology Initiates Rolling BLA Submission for Cretostimogene Monotherapy in High-Risk BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
CG Oncology
2025-11-14
bla
bladder cancer
complete response
CG Oncology announces BLA submission for cretostimogene in high-risk bladder cancer, showing promising clinical results and strong financial position.Quiver AI SummaryCG Oncology, Inc. has initiated a rolling Biologics License Application (BLA) submission to the U.S. FDA for cretostimogene monotherapy targeting high-risk, BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). The company reported a 41.8% complete response rate at 24 months
Benitec Biopharma Releases First Quarter 2026 Financial Results and Provides Operational Update
benitec biopharma
2025-11-14
financial results
MSD said to be close to $9bn+ deal for flu biotech Cidara
PharmaPhorum
2025-11-14
phase 3
MSD is reported to be on the brink of announcing a takeover deal for Cidara, hoping to bolt on a universal flu prevention candidate in phase 3.
8-K - CG Oncology, Inc. (0001991792) (Filer)
SEC Filing (8-K)
2025-11-14
cg oncology
Filed: 2025-11-14 AccNo: 0001193125-25-281672 Size: 405 KB Item 2.02: Results of Operations and Financial Condition Item 9.01: Financial Statements and Exhibits
Fondazione Telethon announces CHMP positive opinion for Waskyra™, a gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS)
Fondazione Telethon
2025-11-14
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
CG Oncology (NASDAQ: CGON) reports 41.8% 24-month CR in BOND-003; initiates rolling BLA
CG Oncology
2025-11-14
bla
Cash $680.3M funds ops into H1 2028. Phase 3 PIVOT-006 enrollment completed. Q3 net loss $43.8M. Rolling BLA for cretostimogene underway; completion in 2026.
Social deprivation increases diabetic retinopathy incidence
Healio
2025-11-14
diabetic retinopathy
Housing instability, food insecurity and other factors related to social deprivation come with significantly greater risks for diabetic retinopathy, even among patients adherent to treatment protocols, according to a study published in Eye.“Socially deprived individuals — including those with food insecurity, housing instability and low income — are more likely to present with
[8-K] CG Oncology, Inc. Reports Material Event
CG Oncology
2025-11-14
financial results
CG Oncology (CGON) furnished an update on its financial results under Item 2.02. The company announced it issued a press release covering results for the quarte
LAST CALL: Join Nobel Laureate & Global Leaders to Explore the Next Era of Cell & Gene Therapy in London
PR Newswire
2025-11-14
gene therapy
PISCATAWAY, N.J., Nov. 14, 2025 /PRNewswire/ -- On November 20, London will become the global spotlight for cell and gene therapy (CGT) as a Nobel Laureate, scientific leaders, biotech innovators, and top investors gather to chart the future of one of the fastest-growing fields in...
BrainStorm Cell Therapeutics Announces Third Quarter 2025 Financial Results and Provides Corporate Update
BrainStorm Cell Therapeutics
2025-11-14
financial results
Is Gilead’s Successful Phase 3 HIV Regimen Trial Shifting the Investment Case for GILD?
Gilead Sciences
2025-11-14
phase 3
Gilead Sciences recently announced that its Phase 3 ARTISTRY-1 trial met the primary endpoint for a novel single-tablet HIV regimen combining bictegravir and lenacapavir, confirming non-inferiority versus multi-tablet therapy and reporting a favorable safety profile. This development highlights Gilead’s continued progress in simplifying treatment for people living with HIV and advancing next-generation regimens that address patient needs for convenience and tolerability. We’ll examine how...
CG Oncology reports Q3 EPS (57c), consensus (55c)
CG Oncology
2025-11-14
bla
Reports Q3 revenue $1.666M, consensus $72.56k. “I am proud of the meaningful progress we have made over the last few months, most notably, the initiation of our BLA...
FDA Approves New Safety Warning and Revised Indication that Limits Use for Elevidys Following Reports of Fatal Liver Injury
FDA Press
2025-11-14
elevidys
The U.S. Food and Drug Administration today announced it is taking action to approve new labeling submitted by the company that includes the addition of a Boxed Warning, the agency’s most prominent safety warning, to Elevidys (delandistrogene moxeparvovec-rokl), and that the indication section of th
FDA updates Sarepta’s Duchenne gene therapy label, requests postmarketing study
Endpoints News
2025-11-14
sarepta
elevidys
gene therapy
duchenne muscular dystrophy
The FDA on Friday revised the label for Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys to add a boxed safety warning and restrict the therapy’s use to ambulatory patients, reflecting changes that the agency and ...
Fondazione Telethon announces CHMP positive opinion for Waskyra™, a gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS)
Fondazione Telethon
2025-11-14
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
/PRNewswire/ -- Fondazione Telethon announces the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European...
Kura Oncology and Kyowa Kirin Announce FDA Approval of KOMZIFTI™ (ziftomenib), the First and Only Once-Daily Targeted Therapy for Adults with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia
Kyowa Kirin Co.
2025-11-14
acute myeloid leukemia
VIDEO: MCO-010 targets severe Stargardt disease in STARLIGHT study
Healio
2025-11-14
mco-010
In this video from the American Academy of Ophthalmology meeting, Kenneth C. Fan, MD, MBA, highlights outcomes of the STARLIGHT study in Stargardt disease.
enGene Holdings Inc. (ENGN) Discusses Preliminary Data From LEGEND Pivotal Cohort in High-Risk BCG-Unresponsive NMIBC - Slideshow (NASDAQ:ENGN) 2025-11-14
enGene Google
2025-11-14
pivotal
2025-11-14. The following slide deck was published by enGene Holdings Inc.
La Fondazione Telethon anuncia la opinión positiva del CHMP sobre Waskyra™
PR Newswire
2025-11-14
wiskott-aldrich
La Fondazione Telethon anuncia la opinión positiva del CHMP sobre Waskyra™, una terapia génica para el tratamiento del síndrome de Wiskott-Aldrich (WAS) ROMA, 14 de noviembre de 2025 /PRNewswire/ -- La Fondazione Telethon anuncia la opinión positiva emitida por el Comité de Medicamentos...
Alpha-1 Trailblazers
CSL Behring
2025-11-14
alpha-1 antitrypsin deficiency
During Alpha-1 Awareness Month, get inspired by the forward-moving, always-reaching Alpha-1 Antitrypsin Deficiency community.
Examining BioMarin Stock After Pipeline Advances and Recent Price Rebound in 2025
BioMarin Pharmaceutical
2025-11-14
pipeline
Ever wondered if BioMarin Pharmaceutical is really worth its current price? Let's break down what the market is seeing and whether there might be untapped value here. After a rollercoaster year, BioMarin’s stock is up 4.6% this past week and 3.3% over the past month, though it's still down more than 17% for the last year. This reflects both renewed optimism and lingering caution. Much of this recent movement follows news about advances in their pipeline therapies and expanded regulatory...
Update: MSD's $9.2bn deal for flu biotech Cidara confirmed
PharmaPhorum
2025-11-14
phase 3
MSD has signed a $9.2 billion takeover deal for Cidara that will bolt on a universal flu prevention candidate in phase 3.
BMS Jettisons Coronary Trial for J&J-Partnered Anticoagulant
BioSpace
2025-11-14
atrial fibrillation
Bristol Myers Squibb and Johnson & Johnson will continue to test the asset in stroke and atrial fibrillation.
AbbVie Partners with Award-Winning Filmmaker on Film About the Dynamic and Resilient Lives of Those Living with Blood Cancer
Abbvie
2025-11-14
cll
Film captures inspiring journeys of three individuals living with chronic lymphocytic leukemia (CLL) CLL is one of the most common leukemias among adults affecting more than 200,000 people in the...
Celebrating a new, faster path to gene-editing medicines on demand
uniQure
2025-11-14
crispr
The FDA’s Marty Makary and Vinay Prasad have laid out a path for CRISPR treatments on demand. An expert weighs in on what it would mean for families.
Merck Makes Big Antiviral Move With $9B Deal to Land Cidara’s Late-Stage Drug for Flu Prevention
MedCity News
2025-11-14
phase 3
Merck’s Cidara Therapeutics acquisition brings a lead program with potential to become a first-in-class drug for preventing influenza infection. A Phase 3 study is already underway, timed to coincide with the start of the flu season in the Northern Hemisphere. The post Merck Makes Big Antiviral Move With $9B Deal to Land Cidara’s Late-Stage Drug for Flu Prevention appeared first on MedCity News .
AbbVie Partners with Award-Winning Filmmaker on Film About the Dynamic and Resilient Lives of Those Living with Blood Cancer
PR Newswire
2025-11-14
cll
leukemia
chronic lymphocytic leukemia
Film captures inspiring journeys of three individuals living with chronic lymphocytic leukemia (CLL) CLL is one of the most common leukemias among adults affecting more than 200,000 people in the United States1,2 Film premiered yesterday in Los Angeles, hosted by actor and advocate Jesse...
CG Oncology Reports Third Quarter 2025 Financial Results and Provides Business Updates
CG Oncology
2025-11-14
bla
phase 3
bladder cancer
complete response
Initiated rolling Biologics License Application (BLA) submission to U.S. FDA for cretostimogene monotherapy in high-risk (HR) BCG-unresponsive non-muscle invasive bladder cancer (NMIBC)Demonstrated continued best-in-disease durability and tolerability in BOND-003 Cohort C with robust 24-month complete response (CR) rate of 41.8% observed for cretostimogene monotherapy in patients with HR NMIBC unresponsive toBacillus Calmette Guerin (BCG)Completed enrollment of PIVOT-006, one of the largest randomized Phase 3 studies in intermediate risk (IR) NMIBC encompassing broadest range of patients per AUA/SUO GuidelinesWell-positioned to deliver on key milestones with $680.3 million cash and cash equivalents and marketable securities sufficient to fund operations into the first half of 2028 IRVINE, Calif., Nov. 14, 2025 (GLOBE NEWSWIRE) -- CG Oncology, Inc. (NASDAQ: CGON), a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients with bladder cancer, today reported financial results for the third quarter ended September 30, 2025, and provided business updates.
How Investors May Respond To Krystal Biotech (KRYS) Surging Net Income and EPS Growth in Q3 2025
Krystal Biotech
2025-11-14
quarterly
Krystal Biotech reported third quarter 2025 results showing net income of US$79.37 million, up from US$27.18 million a year ago, and basic earnings per share of US$2.74 versus US$0.95 in the prior-year quarter. This marked improvement in both quarterly and year-to-date profitability, with net income and per-share earnings rising significantly, reflects growing operational efficiencies and stronger market adoption of its key products. We'll explore how this substantial growth in net income...
Update on Phase 3 Librexia ACS Trial
bristol myers squibb
2025-11-14
phase 3
Fondazione Telethon achieves milestone with Waskyra approval for Wiskott-Aldrich Syndrome
Fondazione Telethon
2025-11-14
gene therapy
wiskott-aldrich
ex vivo gene therapy
wiskott-aldrich syndrome
Fondazione Telethon announces the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending marketing authorization in the European Union for Waskyra™, an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS), a rar...
Can Autolus Therapeutics' (AUTL) Expanding Obe-cel Reach Bolster Its Position in Cell Therapy?
Autolus Therapeutics
2025-11-14
cell therapy
Autolus Therapeutics recently reported its third quarter results, showing a reduction in net loss to US$79.12 million versus US$82.09 million a year ago, and announced operational advances including broad US market access for Obe-cel with 60 authorized treatment centers and a manufacturing success rate above 90%. The company is also progressing with clinical programs in pediatric ALL, lupus nephritis, and progressive multiple sclerosis, and announced leadership changes as it prepares for key...
Kura and Kyowa Kirin secure FDA go-ahead for targeted leukaemia therapy
Kyowa Kirin
2025-11-14
aml
Komzifti now becomes the first once-daily targeted therapy for r/r AML, though it will have to fight Syndax’s Revuforj for market share.
enGene Holdings (ENGN) Valuation in Focus Following Promising Phase 2 Bladder Cancer Trial Results
enGene Google
2025-11-14
phase 2
pivotal
bladder cancer
complete response
enGene Holdings (ENGN) is drawing attention after announcing positive preliminary results from the pivotal cohort of its Phase 2 LEGEND trial. The data showed a high complete response rate and a favorable safety profile in challenging bladder cancer cases. See our latest analysis for enGene Holdings. enGene Holdings’ impressive momentum really kicked in after its recent LEGEND trial data sent the stock soaring, highlighted by a 40% seven-day share price return and a staggering 129% gain over...
Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information
Sarepta Therapeutics
2025-11-14
sarepta
elevidys
gene therapy
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Nov. 14, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced an update to the prescribing information for ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for
VIDEO: Real-world data show extended injection intervals with aflibercept 8 mg
Healio
2025-11-14
wet age-related macular degeneration
In this video from the American Academy of Ophthalmology meeting, Diana V. Do, MD, discusses the real-world use of aflibercept 8 mg in wet age-related macular degeneration or diabetic macular edema.
Fondazione Telethon announces CHMP positive opinion for Waskyra™, a gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS)
PR Newswire
2025-11-14
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
ROME, Nov. 14, 2025 /PRNewswire/ -- Fondazione Telethon announces the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), recommending marketing authorisation in the European Union for Waskyra™, an ex vivo gene...
FDA approves first-ever interchangeable biosimilar to pertuzumab
Healio
2025-11-14
her2
The FDA has approved the first-ever interchangeable biosimilar to pertuzumab for patients with certain HER2-positive breast cancers.Poherdy (pertuzumab-dpzb, Shanghai Henlius Biologics Co.), a HER2 receptor antagonist and biosimilar to pertuzumab (Perjeta, Genentech), is indicated for use in combination with trastuzumab (Herceptin, Genentech) and docetaxel for adults with HER2-positive metastatic
Fondazione Telethon achieves milestone with Waskyra approval for Wiskott-Aldrich Syndrome
Fondazione Telethon
2025-11-14
gene therapy
wiskott-aldrich
ex vivo gene therapy
wiskott-aldrich syndrome
Fondazione Telethon announces the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending marketing authorization in the European Union for Waskyra, an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening primary immunodeficiency.
Cartesian to pause work in form of lupus, despite trial success
Endpoints News
2025-11-14
cell therapy
Cartesian Therapeutics said it will focus development of its lead cell therapy candidate on patients with myasthenia gravis and myositis, and will pause work in another immune condition known as systemic lupus erythematosus. That's despite ...
FDA approves Kura and Kyowa Kirin's drug for form of acute myeloid leukemia
Endpoints News
2025-11-13
acute myeloid leukemia
The FDA gave the go-ahead to Kura Oncology and Kyowa Kirin’s treatment for certain acute myeloid leukemia patients, just weeks after a rival drug was approved for a similar population. Thursday's
Kura/Kyowa Kirin’s Komzifti Comes With QTc Safety Advantage
Kyowa Kirin
2025-11-13
leukemia
acute myeloid leukemia
The US FDA approved the menin inhibitor for NPM1-mutant relapsed/refractory acute myeloid leukemia.
Elicio Therapeutics Reports Third Quarter 2025 Financial Results and Provides Corporate Updates
Elicio Therapeutics
2025-11-13
financial results
November 13, 2025 16:01 ETIn the ongoing Phase 2 AMPLIFY-7P study evaluating ELI-002 7P in patients with pancreatic ductal adenocarcinoma (“PDAC”), ...
4D-150 maintains visual acuity in wet AMD at 2 years
Healio
2025-11-13
wet age-related macular degeneration
A single intravitreal injection of 4D-150 helped patients with wet age-related macular degeneration maintain visual acuity for up to 2 years, according to a press release from 4DMT.
Century Therapeutics Reports Third Quarter 2025 Financial Results and Provides Business Update
Century Therapeutics
2025-11-13
financial results
FDA Approves Kura Oncology Leukemia Drug, Teeing Up Competition With Syndax Pharma
MedCity News
2025-11-13
acute myeloid leukemia
Kura Oncology’s Komzifti is approved for treating advanced cases of acute myeloid leukemia with an NPM1 mutation. A Syndax Pharmaceuticals drug is already approved in this indication, but Kura says its drug has advantages in safety and patient convenience. The post FDA Approves Kura Oncology Leukemia Drug, Teeing Up Competition With Syndax Pharma appeared first on MedCity News .
Day One to buy struggling ADC maker Mersana for $129M
Endpoints News
2025-11-13
pivotal
ovarian cancer
Day One Biopharmaceuticals is bailing out Mersana Therapeutics two years after the antibody-drug conjugate maker’s lead ovarian cancer drug failed a pivotal trial. Day One plans to pay $129 million upfront to buy Mersana, the ...
Traumatic spinal cord injury linked to elevated morbidity, mortality
Healio
2025-11-13
open
Traumatic spinal cord injuries were linked to elevated risks for long-term morbidity and mortality, regardless of health prior to injury, underscoring the need for improved longitudinal care, data published in JAMA Network Open show.“This study fills a major gap by quantifying the long-term, multisystem disease burden after previously healthy traumatic spinal cord injury across two large
Kura Oncology and Kyowa Kirin Announce FDA Approval of KOMZIFTI for Relapsed or Refractory Acute Myeloid Leukemia with NPM1 Mutation
Kyowa Kirin
2025-11-13
aml
leukemia
acute myeloid leukemia
FDA fully approves KOMZIFTI for relapsed or refractory NPM1-mutated AML, offering a new treatment option with favorable safety.Quiver AI SummaryKura Oncology and Kyowa Kirin announced that the FDA has granted full approval for KOMZIFTI™ (ziftomenib) for adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible NPM1 mutation. This marks KOMZIFTI as the first once-daily oral menin inhibitor approved for this indicat
EnGene squares up to J&J
enGene Google
2025-11-13
bladder cancer
New detalimogene bladder cancer data send the minnow’s stock up 47%.
Instil Bio Reports Third Quarter 2025 Financial Results and Provides Corporate Update
Instil Bio
2025-11-13
til
phase 1
First patient dosed in Phase 1 clinical trial evaluating AXN-2510/IMM2510 (“’2510”) monotherapy in adult patients with advanced solid tumors DALLAS, Nov. 13, 2025 (GLOBE NEWSWIRE) -- Instil Bio, Inc. (“Instil”) (Nasdaq: TIL), a clinical-stage biopharmaceutical company focused on developing a
Arcellx to Present New Data for Its iMMagine-1 Study and Continues Scientific Momentum with Multiple Presentations at the 67th ASH Annual Meeting and Exposition
arcellx, inc
2025-11-13
bla
phase 2
pivotal
cell therapy
A more recent data cut will be shared during an oral presentation for the iMMagine-1 study - - To date, no delayed neurotoxicities, including no Parkinsonism, no cranial nerve palsies, no Guillain-Barré syndrome, and no immune effector cell-associated enterocolitis have been observed with anito-cel - - FDA pre-BLA meeting for anito-cel has occurred; reiterating planned 2026 commercial launch - - Company to host a live webcast event with an expert panel of clinicians during ASH 2025 - Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, today announced it will share two presentations at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition taking place December 6-9, 2025, in Orlando, Florida, including updated clinical data from iMMagine-1, its Phase 2 pivotal study (publication #256) of anitocabtagene autoleucel (anito-cel) in
Vor Bio Reports Third Quarter 2025 Financial Results and Provides Corporate Update
Vor Biopharma
2025-11-13
financial results
Multiple Late-Stage Data Readouts Reinforce Telitacicept’s Broad Potential Across Autoimmune Diseases Expansion of Executive Leadership and Board Strengthens Global Development Capabilities Expected gross proceeds of $115 million raised in the November 2025 underwritten public offering, including
Zealand pauses development of GLP-1/GLP-2 obesity candidate
Endpoints News
2025-11-13
phase 2
Zealand Pharma said Thursday it has paused one of its assets for obesity. The GLP-1/GLP-2 dual agonist dapiglutide had completed an investigator-led Phase 2 trial and was slated to begin another under Zealand’s direction. ...
Conservative dialysis may improve recovery for patients with acute kidney injury
Healio
2025-11-13
msc
HOUSTON — More patients with AKI recovered kidney function after a conservative hemodialysis strategy compared with patients on hemodialysis three times a week, according to data presented at ASN Kidney Week. Hemodialysis three times a week remains a widespread practice in nephrology, but Chi-yuan Hsu, MD, MSc, chief of the division of nephrology at University of California-San Francisco Health,
Legend Biotech Celebrates Official Opening of New State-of-the-Art Cell Therapy Research and Development Facility in Philadelphia
Legend Biotech
2025-11-13
cell therapy
Makary, Prasad outline pathway for bespoke gene therapy
uniQure
2025-11-13
gene therapy
And other biotech news, brought to you by The Readout
enGene Holdings Inc. (ENGN) Stock: Declines After Announcing Major $130M Share & Warrant Offering
enGene Google
2025-11-13
pipeline
enGene Holdings prices a major equity and warrant offering to raise $130M, impacting its stock while supporting clinical pipeline growth and long-term capital strategy.
Opus Genetics Announces Dosing of First Participant in OPGx-BEST1 Phase 1/2 Gene Therapy Clinical Trial for Best Disease
opus genetics
2025-11-13
phase 1
phase 1/2
gene therapy
Kura Oncology and Kyowa Kirin Announce FDA Approval of KOMZIFTI™ (ziftomenib), the First and Only Once-Daily Targeted Therapy for Adults with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia
Kyowa Kirin
2025-11-13
aml
leukemia
acute myeloid leukemia
– NPM1 mutations, one of the most common genetic drivers of AML, are now actionable for patients – – Acute unmet need in R/R NPM1-mutated AML defined by...
MeiraGTx Reports Third Quarter 2025 Financial and Operational Results
MeiraGTx
2025-11-13
aav
leber congenital amaurosis
Entered into broad strategic collaboration with Eli Lilly and Company (“Lilly”) in the area of ophthalmology, granting Lilly worldwide exclusive rights to the Company’s AAV-AIPL1 program for treatment of one of the most severe inherited retinopathies, Leber congenital amaurosis 4 (LCA4) Released
MS drug fenebrutinib shows ‘unprecedented results’ in trials –...
Genentech
2025-11-13
phase 3
multiple sclerosis
Two Phase 3 trials testing the investigational oral therapy fenebrutinib in people with multiple sclerosis (MS) have hit their main goals.
Gilead (GILD) Reveals Promising Phase 3 Trial Results for HIV Treatment
Gilead Sciences
2025-11-13
phase 3
Key Takeaways: Gilead Sciences (GILD) reports promising results from its Phase 3 ARTISTRY-1 clinical trial for a new single-tablet HIV therapy. The compa
VIDEO: Livdelzi appears safe for patients with PBC, advanced liver disease
Healio
2025-11-13
phase 3
second-line
PHOENIX — In this video, Barry Crittenden, MD, discusses data that show Livdelzi appears safe for second-line treatment of patients with primary biliary cholangitis with cirrhosis and clinical signs of portal hypertension.Crittenden, executive director of clinical development at Gilead Sciences, and colleagues pooled safety data from the phase 3, placebo-controlled ENHANCE and RESPONSE
Common Epstein–Barr Virus Reprograms B Cells to Drive Lupus
GenEng News
2025-11-13
lupus
Using a single-cell RNA-sequencing platform, a new study demonstrates that common Epstein–Barr virus, found in 95% of the population, reprograms autoreactive B cells to drive lupus autoimmunity. The post Common Epstein–Barr Virus Reprograms B Cells to Drive Lupus appeared first on GEN - Genetic Engineering and Biotechnology News .
Gilead hails phase 3 victory for single-tablet combo of bictegravir and lenacapavir, preps filings
Gilead Sciences
2025-11-13
phase 3
Gilead Sciences has developed an industry-leading HIV portfolio in recent years with its megablockbuster daily treatment Biktarvy and its new long-acting pre-exposure prophylaxis (PrEP) medicine Ye | For people who are currently on multi-tablet HIV regimens, many of whom are also on medicines for other conditions, the findings are "significant," an expert said.
Gilead Reports Positive Phase 3 Results For Bictegravir/ Lenacapavir Single-tablet Regimen For HIV
Gilead Sciences
2025-11-13
phase 3
(RTTNews) - Gilead Sciences, Inc. (GILD) on Thursday announced positive topline results from its Phase 3 ARTISTRY-1 trial evaluating the company's single-tablet regimen of bictegravir and lenacapavir (BIC/LEN) for the treatment of HIV.
Takeda says dengue vaccine provides yearslong protection
Healio
2025-11-13
phase 3
Takeda’s dengue vaccine demonstrated strong protection against infection and hospitalization for up to 7 years, findings shared by the company showed. The company reported data from the phase 3 TIDES trial, which assessed the vaccine — marketed as Qdenga — among more than 20,000 healthy children and adolescents in eight dengue-endemic countries in Latin America and Asia.
Gilead's One-Pill HIV Treatment Shows Promise In Late-Stage Trial
Gilead Sciences
2025-11-13
phase 3
Gilead's Phase 3 ARTISTRY-1 trial shows its single-tablet HIV regimen of bictegravir and lenacapavir matches the efficacy and safety of current therapy.
Caribou Biosciences to Participate in the Jefferies Global Healthcare Conference
caribou biosciences
2025-11-13
crispr
BERKELEY, Calif., May 28, 2025 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today announced Rachel Haurwitz, PhD, Caribou’s president and chief executive officer, will participate in a fireside chat at the
New Survey Reveals Non-Muscle Invasive Bladder Cancer Patients Seek More Care Conversations
Immunity Bio
2025-11-13
bladder cancer
The post New Survey Reveals Non-Muscle Invasive Bladder Cancer Patients Seek More Care Conversations appeared first on ImmunityBio .
Kyowa's bet on Kura pays off as partners' oral med for AML subset wins FDA approval
Kyowa Kirin
2025-11-13
aml
leukemia
acute myeloid leukemia
Kyowa Kirin’s big bet on Kura Oncology has paid off in short order, delivering an FDA approval for a medicine to treat a subset of patients with acute myeloid leukemia. | Kyowa Kirin’s big bet on Kura Oncology has paid off in short order, delivering an FDA approval for a medicine to treat a subset of patients with acute myeloid leukemia.
FDA approves Kura and Kyowa Kirin's drug for form of acute myeloid leukemia
Kyowa Kirin
2025-11-13
leukemia
acute myeloid leukemia
The FDA gave the go-ahead to Kura Oncology and Kyowa Kirin’s treatment for certain acute myeloid leukemia patients, just weeks after a rival drug was approved for a similar population.
Support from Harrington Discovery Institute at University Hospitals Helped Advance Heart Attack Drug into Clinical Trials
PR Newswire
2025-11-13
phase 3
pivotal
Positive results of zalunfiban announced at AHA Scientific Sessions CLEVELAND, Nov. 13, 2025 /PRNewswire/ -- Promising results from a pivotal Phase 3 clinical trial of an investigational heart-attack drug were announced this week as part of the late-breaking sessions at the American Heart...
Esaote and Schiller Americas announce strategic partnership to expand the sales channel in the cardiology imaging space
PR Newswire
2025-11-13
ind
FISHERS, Ind., Nov. 13, 2025 /PRNewswire/ -- Esaote, one of the world's leading medical imaging companies, is proud to announce a new strategic partnership with Schiller Americas, a global leader in cardiopulmonary diagnostics. This collaboration strengthens both companies' presence in...
Gilead hits late-stage trial win for new HIV therapy (GILD)
Gilead Sciences
2025-11-13
phase 3
Gilead (GILD) stock gains as the company's new single-tablet HIV regimen is proven as effective as multi-tablet therapies in a Phase 3 trial. Read more here.
Korro to cut staff, shift strategy as RNA editing drug misses mark in early testing
Biopharma Dive
2025-11-13
alpha-1 antitrypsin deficiency
Korro’s lead program for alpha-1 antitrypsin deficiency wasn’t as potent as anticipated, leading the company to change course and lay off a third of its workforce.
VIDEO: Resmetirom may be beneficial for patients with MASH cirrhosis
Healio
2025-11-13
phase 3
PHOENIX — In this video, Paul Kwo, MD, discusses preliminary results from the phase 3 MAESTRO-NAFLD-1 trial assessing resmetirom in patients with compensated metabolic dysfunction-associated steatohepatitis cirrhosis.“There’s a larger effort ongoing, but this initial glimpse suggests that there may be benefit to resmetirom in patients with cirrhosis, in addition to its known
New Survey Reveals Non-Muscle Invasive Bladder Cancer Patients Seek More Care Conversations
Immunity Bio
2025-11-13
bladder cancer
The post New Survey Reveals Non-Muscle Invasive Bladder Cancer Patients Seek More Care Conversations appeared first on ImmunityBio .
Korro Suffers Cacophony of Bad News as Lead Candidate Fails, Novo Pauses RNA Deal
BioSpace
2025-11-13
alpha-1 antitrypsin deficiency
Korro Bio is moving back to square one as a preclinical biotech after the failure of KRRO-110 in alpha-1 antitrypsin deficiency. The company's stock is down 80% on all the news.
How Investors Are Reacting To Autolus Therapeutics (AUTL) Trimming Losses and Appointing a New Finance Chief
Autolus Therapeutics
2025-11-13
quarterly
Autolus Therapeutics recently reported third quarter 2025 earnings, showing a net loss of US$79.12 million, a slight improvement from the same period last year, and also appointed Patrick McIlvenny as Senior Vice President, Finance and Chief Accounting Officer. The combination of a modest year-over-year reduction in quarterly loss and the addition of an experienced finance executive suggests a renewed focus on operational discipline and financial management. We'll examine how the appointment...
Tinostamustine named orphan drug for treating malignant gliomas
Rare Cancer News
2025-11-13
orphan drug
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to tinostamustine, a treatment candidate from Purdue Pharma that aims to extend survival for people with malignant gliomas, the developer announced. Gliomas are a group of tumors that form from glial cells — those that support and protect the nerve cells in the […] The post Tinostamustine named orphan drug for treating malignant gliomas appeared first on Rare Cancer News .
AbbVie Ends 11-Year Relationship With Calico, Lays Off 100+
BioSpace
2025-11-13
amyotrophic lateral sclerosis
In January, AbbVie and Calico’s fosigotifator failed to show significant signs of efficacy in the HEALEY ALS platform trial for amyotrophic lateral sclerosis.
Genetic Agency on Display at GATC 2025
GenEng News
2025-11-13
gene editing
gene therapy
The Genetic Agency Technology Conference brought together a broad coalition of researchers and executives in cell and gene therapy, gene editing, drug discovery and AI as well as patients and advocates. The post Genetic Agency on Display at GATC 2025 appeared first on GEN - Genetic Engineering and Biotechnology News .
Kura (NASDAQ: KURA) receives FDA approval; KOMZIFTI shows 21.4% CR+CRh in NPM1‑mutated AML
Kyowa Kirin
2025-11-13
aml
Full FDA approval for KOMZIFTI in R/R NPM1‑mutated AML. Trial in 112 patients showed 21.4% CR+CRh and 5.0‑month median duration; no QTc Boxed Warning.
NICE backs Roche's non-Hodgkin lymphoma drug Columvi
PharmaPhorum
2025-11-13
second-line
NICE backs NHS use of Roche's Columvi as a second-line therapy for a form of non-Hodgkin lymphoma affecting thousands of people in the UK each year.
Rapid Nexus Becomes the First Company to Receive FDA Clearance for its Advanced Wound-Healing Device Hemastyl
PR Newswire
2025-11-13
clearance
FDA 510(k) clearance for its Hemastyl gel device marks the first technology that has treated the tissue surrounding chronic wounds and prevented numerous amputations. BREA, Calif., Nov. 13, 2025 /PRNewswire/ -- Rapid Nexus Nanotech Wound Solutions, Inc., a California-based med-tech...
FDA Unwraps ‘Plausible Mechanism Pathway’ for Personalized Therapies
BioSpace
2025-11-13
gene therapy
The plausible mechanism pathway "could accelerate gene therapy/editing development," analysts at William Blair said Thursday, while adding that additional clarity is needed.
Kura Oncology, Kyowa Kirin announce FDA approval of KOMZIFTI for AML
Kyowa Kirin
2025-11-13
aml
Kura Oncology (KURA) and Kyowa Kirin announced the U.S. Food and Drug Administration has granted full approval of KOMZIFTI for adult patients with ...
enGene Holdings (ENGN) Is Up 36.9% After Strong Phase 2 Bladder Cancer Gene Therapy Results
enGene Google
2025-11-13
phase 2
gene therapy
bladder cancer
complete response
detalimogene voraplasmid
enGene Holdings Inc. recently reported additional positive preliminary data from its Phase 2 LEGEND trial, highlighting a 62% complete response rate at six months for detalimogene voraplasmid in patients with high-risk, BCG-unresponsive non-muscle invasive bladder cancer. The updated trial enrolled 125 patients, surpassing original targets, and enGene plans to file for regulatory approval in the second half of 2026 for this new gene therapy approach. We'll explore how these encouraging...
Gilead’s Investigational Single-Tablet Regimen of Bictegravir and Lenacapavir for HIV-1 Treatment Meets Primary Endpoint in Phase 3 ARTISTRY-1 Trial
Gilead Sciences
2025-11-13
phase 3
FOSTER CITY, Calif., November 13, 2025--Gilead Sciences, Inc. (NASDAQ: GILD) today announced positive topline results from the Phase 3 ARTISTRY-1 trial. The open-label trial evaluated the treatment responses of adults with HIV who are virologically suppressed switching from a multi-tablet regimen to an investigational single-tablet regimen of bictegravir 75 mg/lenacapavir 50 mg (BIC/LEN). BIC/LEN efficacy was found to be statistically non-inferior to multi-tablet regimens. Gilead plans to file t
Predictors Found for Durable Response to BCMA CAR T Therapy in Multiple Myeloma
Inside Precision Medicine
2025-11-13
bcma
car t
durable response
multiple myeloma
Patients who remained cancer-free for more than five years had a focused, early expansion of CAR T cells and maintained a broad, diverse pool of endogenous CD4 helper T cells. The post Predictors Found for Durable Response to BCMA CAR T Therapy in Multiple Myeloma appeared first on Inside Precision Medicine .
Novel Novartis Malaria Drug Shows Promise to Become New Asset Against Resistant Strains
MedCity News
2025-11-13
phase 3
Phase 3 results for the Novartis malaria drug “GanLum” show it was comparable to the standard of care and led to cure rates that top the threshold recommended by the World Health Organization. If approved, this new combination drug would become the first novel malaria therapy in more than 25 years. The post Novel Novartis Malaria Drug Shows Promise to Become New Asset Against Resistant Strains appeared first on MedCity News .
BiomEdit Announces USDA Finding of No Significant Impact Letter for Non-Antibiotic Poultry Biologic, Clearing Path to Field Safety Trials
PR Newswire
2025-11-13
ind
Regulatory milestone allows product to proceed to final phase of USDA conditional licensure process for necrotic enteritis prevention in broiler chickens GREENFIELD, Ind., Nov. 13, 2025 /PRNewswire/ -- BiomEdit, Inc., a pioneering animal health biotechnology company, today announced it...
Gilead Sciences (GILD) Achieves Key Milestone in HIV Treatment Trial
Gilead Sciences
2025-11-13
phase 3
Key Takeaways: Gilead Sciences' (GILD) Phase 3 ARTISTRY-1 trial achieved its primary endpoint, showcasing the efficacy of a new single-tablet HIV regimen.
Abeona’s skin disease cell therapy launch stymied by false-positive sterility test
Endpoints News
2025-11-13
cell therapy
Abeona Therapeutics said the commercial rollout of its rare skin disease cell therapy has hit a roadbump, delaying the treatment of its first patient by several months. Although the company noted on Wednesday morning that ...
Could a Shift in enGene (ENGN) Trial Endpoints Reveal New Priorities in Its Clinical Strategy?
enGene Google
2025-11-13
phase 2
pivotal
bladder cancer
complete response
detalimogene voraplasmid
enGene Holdings recently reported additional preliminary efficacy data from its pivotal Phase 2 LEGEND trial for detalimogene voraplasmid in patients with high-risk, BCG-unresponsive non-muscle invasive bladder cancer, along with a protocol amendment changing the primary clinical endpoint after discussions with the FDA. This update reveals that earlier patients in the study experienced a lower 12-month complete response rate compared to FDA-approved therapies, leading to significant...
Extracellular Vesicles Show Promise for Gene Therapy Alternative
Inside Precision Medicine
2025-11-13
gene therapy
Extracellular vesicles could offer a way of editing genes that does not involve viral vectors, with promising preclinical findings for genetic hearing loss. The post Extracellular Vesicles Show Promise for Gene Therapy Alternative appeared first on Inside Precision Medicine .
Eureka’s ARTEMIS® CAR T Cells Show Breakthrough Potential in Solid Tumors in NCI-Led Study Published in Cell Reports Medicine
Eureka Therapeutics
2025-11-13
gpc2
car t
neuroblastoma
cell therapies
Emeryville, CA – November 13, 2025 – Eureka Therapeutics, Inc., a clinical-stage biotechnology company developing novel T cell therapies for cancer, today announced the publication of a peer-reviewed study titled “Antibody–Gamma/Delta T Cell Receptors Targeting GPC2 Regress Neuroblastoma with Low Antigen Density” in Cell Reports Medicine. The study, led by researchers at the National Cancer The post Eureka’s ARTEMIS® CAR T Cells Show Breakthrough Potential in Solid Tumors in NCI-Led Study Published in Cell Reports Medicine appeared first on Eureka .
A Landmark Year for LDT Oversight, Advocacy, and What Comes Next
Inside Precision Medicine
2025-11-13
pivotal
The LDT breakout session outlined how the field arrived at this pivotal moment and what AMP believes must happen next to safeguard patient access, innovation, and the clinical laboratory ecosystem. The post A Landmark Year for LDT Oversight, Advocacy, and What Comes Next appeared first on Inside Precision Medicine .
Novartis says novel drug could counter malaria resistance
PharmaPhorum
2025-11-13
phase 3
Novartis has reported what looks like another big step forward in the treatment of malaria, as a drug with a novel mechanism hits the mark in phase 3.
Fate Therapeutics Reports Third Quarter 2025 Financial Results and Business Updates
Fate Therapeutics
2025-11-13
cd19
car t
Authorization Received from UK and EU Authorities to Activate ex-US Clinical Trial Sites Supporting the Ongoing Patient Enrollment of FT819, an Off-the-Shelf CD19-targeted CAR T-cell Product Candidate that Utilizes Less-intensive or No Conditioning, Enabling Broad Patient Accessibility First
FDA approves ziftomenib for adults with relapsed, refractory AML
Healio
2025-11-13
aml
acute myeloid leukemia
The FDA approved ziftomenib for adult patients with relapsed or refractory acute myeloid leukemia with a susceptible nucleophosmin 1 mutation. This indication for ziftomenib (Komzifti, Kura Oncology), an oral menin inhibitor, applies to adults who do not have satisfactory alternative treatment options.
Gilead price attacks pivotal resistance - Forecast today - 13-11-2025
Gilead Sciences
2025-11-13
pivotal
Gilead price attacks pivotal resistance - Forecast today - 13-11-2025
Neurogene Reports Third Quarter 2025 Financial Results and Highlights Recent Updates
neurogene
2025-11-13
phase 1
gene therapy
rett syndrome
Reported positive interim data from NGN-401 Phase 1 /2 gene therapy trial for Rett syndrome demonstrating multidomain, durable gains, with 35 total developmental milestones/skills acquired across eight participants First participant dosed in Embolden™ registrational trial of NGN-401 for Rett
enGene Holdings Inc Announces Updates on Phase 2 LEGEND Trial
enGene Google
2025-11-12
phase 2
detalimogene voraplasmid
On November 11, 2025, enGene Holdings Inc announced new preliminary data from its Phase 2 LEGEND trial for detalimogene voraplasmid. Key Highlights: Original SEC Filing: enGene Holdings Inc. [ ENGN ] - 8-K - Nov. 12, 2025This is an AI-powered summary. It may contain inaccuracies. Consider verifyin…
Kyverna Therapeutics Provides Business Update and Reports Third Quarter 2025 Financial Results
Kyverna Therapeutics
2025-11-12
bla
myasthenia gravis
stiff person syndrome
Topline data from registrational trial in stiff person syndrome (SPS) now expected in early 2026; narrowed from previous guidance of first half 2026; BLA submission anticipated in 1H 2026 Positive interim Phase 2 data in generalized myasthenia gravis (gMG); on-track to enroll first patient for
VIDEO: No impact on quality of life with addition of tafasitamab in follicular lymphoma
Healio
2025-11-12
phase 3
follicular lymphoma
In this video, Paolo Strati, MD, of The University of Texas MD Anderson Cancer Center, discusses quality of life outcomes from the phase 3 inMIND trial, which he presented at Society of Hematologic Oncology’s Annual Meeting.Results of the inMIND trial showed that tafasitamab-cxix (Monjuvi, Incyte) plus lenalidomide and rituximab improved PFS compared with placebo plus lenalidomide and
Immatics Achieves Clinical Proof-of-Concept of its Next-Generation TCR Bispecific (TCER®) Pipeline with Data on IMA402 (PRAME) and IMA401 (MAGEA4/8) and Announces Next Development Steps
Immatics Biotechnologies
2025-11-12
pipeline
Company to host conference call and webcast today, November 12 , at 8:30 am EST / 2:30 pm CET IMA402 and IMA401 TCR Bispecifics showed favorable tolerability at RP2D as well as deep and durable responses in heavily pre-treated, last-line patients with a range of solid tumors IMA402 PRAME Bispecific
Autolus Therapeutics Reports Third Quarter 2025 Financial Results and Business Updates
Autolus Therapeutics
2025-11-12
aucatzyl
Company reports Q3 2025 AUCATZYL® net product revenue of $21.1 million and deferred revenue of $7.6 million; 60 authorized treatment centers achieved ahead of targetClinical execution and data...
Johnson & Jonson secures FDA approval for multiple myeloma treatment
PMLive Regulatory
2025-11-12
phase 3
multiple myeloma
Support for first and only high-risk smouldering multiple myeloma treatment came from positive phase 3 trial results
A Deep Dive into uniQure (QURE) Valuation After FDA Uncertainty Shakes Huntington’s Disease Program
uniQure
2025-11-12
amt-130
uniQure (NasdaqGS:QURE) shares experienced a sharp reaction after the FDA unexpectedly indicated that Phase I/II data for AMT-130 in Huntington’s disease may not support the company’s Biologics License Application. This change introduces new regulatory uncertainty for investors. See our latest analysis for uniQure. This regulatory curveball comes after a volatile year for uniQure. Enthusiasm from promising clinical trial results quickly turned to caution as investors reassessed the timeline...
Autolus outlines margin improvement and market share growth focus while expanding AUCATZYL to 60 U.S. centers
Autolus Therapeutics
2025-11-12
b-all
aucatzyl
Earnings Call Insights: Autolus Therapeutics plc (AUTL) Q3 2025 Management View CEO Christian Itin reported the launch of AUCATZYL in the U.S. has reached market leadership in relapsed/refractory B-ALL, with broad access and reliable delivery, emphasizing, "We managed, in the first 9 months, to achieve market leadership, broad market access and cov...
FDA approves 1st treatment for high-risk smoldering myeloma
Rare Cancer News
2025-11-12
multiple myeloma
The U.S. Food and Drug Administration (FDA) has approved Darzalex Faspro (daratumumab and hyaluronidase) for adults with high-risk smoldering multiple myeloma, making it the first and only approved treatment for this slow-growing type of multiple myeloma. People with smoldering multiple myeloma typically don’t show visible symptoms, but the condition carries a high risk of progressing […] The post FDA approves 1st treatment for high-risk smoldering myeloma appeared first on Rare Cancer News .
Updated BOND-003 data support durability of cretostimogene grenadenorepvec in NMIBC
CG Oncology
2025-11-12
bla
cretostimogene grenadenorepvec
CG Oncology plans to initiate a BLA submission with the FDA in Q4 of 2025.
BLOG: Preparing transplant, cellular therapy recipients for the respiratory viral season
Healio
2025-11-12
cell therapy
chimeric antigen receptor
The 2025-2026 respiratory viral season is approaching.With it comes the familiar challenge of protecting our most vulnerable patients — those who have undergone hematopoietic cell transplantation or received chimeric antigen receptor T-cell therapy.
8-K - Atara Biotherapeutics, Inc. (0001604464) (Filer)
SEC Filing (8-K)
2025-11-12
atara biotherapeutics
Filed: 2025-11-12 AccNo: 0001193125-25-276209 Size: 228 KB Item 2.02: Results of Operations and Financial Condition Item 9.01: Financial Statements and Exhibits
Autolus reports Q3 revenue of $21.1 million from AUCATZYL sales By Investing.com
Autolus Therapeutics
2025-11-12
aucatzyl
Autolus reports Q3 revenue of $21.1 million from AUCATZYL sales
ProKidney’s Autologous Cell Therapy for CKD and Diabetes Advances to Phase III
Inside Precision Medicine
2025-11-12
autologous
cell therapy
CEO Bruce Culleton, MD, talks about the findings of the rilparencel Phase II REGEN-007 trial during Kidney Week 2025 of the American Society of Nephrology (ASN). The post ProKidney’s Autologous Cell Therapy for CKD and Diabetes Advances to Phase III appeared first on Inside Precision Medicine .
PAI Named as 'Action for Women's Health Awardee' to Fight for Reproductive Health and Rights
PR Newswire
2025-11-12
pivotal
PAI receives the largest grant amount possible from Action for Women's Health, a $250 million global open call funded by Pivotal to improve women's health around the world WASHINGTON, Nov. 12, 2025 /PRNewswire/ -- PAI, a U.S.-based NGO with 60 years of leadership in global sexual and...
FDA chiefs offer roadmap to expand custom gene editing treatments like Baby KJ's
uniQure
2025-11-12
gene editing
Marty Makary and Vinay Prasad offered a roadmap for expanding access to custom gene-editing treatments like the one used to treat Baby KJ's ultra-rare disease.
Gene therapy outcomes in AADC deficiency best tracked by doctors
AADC News/BioNews
2025-11-12
gene therapy
Analysis of certain chemicals, known as neurotransmitters, in the cerebrospinal fluid (CSF) that bathes the brain and spinal cord does not predict clinical outcomes following gene therapy in children and adolescents with aromatic L-amino acid decarboxylase (AADC) deficiency. That’s according to a recent study that analyzed CSF and clinical data in 30 children and adolescents […] The post Gene therapy outcomes in AADC deficiency best tracked by doctors appeared first on AADC News .
Humacyte Announces Third Quarter 2025 Financial Results and Provides Business Update
Humacyte
2025-11-12
bla
pipeline
first-in-human
Total revenues of $753,000 for third quarter, and $1,571,000 for first nine months of 2025, from sales and collaborative research agreement - - Major advances in pipeline as Humacyte moves closer to planned BLA filing in dialysis and first-in-human studies in cardiac bypass graft surgery – –
Lyell Immunopharma Reports Business Highlights and Financial Results for the Third Quarter 2025
lyell immunopharma
2025-11-12
car t
overall response rate
Acquired exclusive global rights to LYL273, a novel GCC-targeted CAR T-cell product candidate that has demonstrated a 67% overall response rate, an 83% disease control rate and a manageable safety profile at the highest dose level studied to date in patients with refractory metastatic colorectal
Alkermes breaks new ground in narcolepsy with orexin drug
PharmaPhorum
2025-11-12
phase 2
Alkermes' alixorexton is the first orexin 2 receptor agonist to show efficacy in phase 2 trials involving patients with both main narcolepsy subtypes.
FDA Unveils New Path for Approving Rare Disease Drugs for Just One Person
uniQure
2025-11-12
gene editing
US regulators laid out new guidelines for approving custom-made treatments for individual patients, a move that could bring the promise of gene editing to more people with devastating diseases.
Gilead Sciences (GILD): Assessing Valuation After Trodelvy Breast Cancer and Livdelzi PBC Data Updates
Gilead Sciences
2025-11-12
phase 3
Gilead Sciences (GILD) is drawing interest following two major clinical updates: mixed results in the Phase 3 ASCENT-07 study of Trodelvy in metastatic breast cancer, and encouraging long-term data for Livdelzi in primary biliary cholangitis. See our latest analysis for Gilead Sciences. After a strong run this year, Gilead’s share price has gained 33.4% year-to-date, and the company’s latest news is keeping momentum alive. Recent product updates and raised earnings guidance are clearly...
Atara Biotherapeutics Announces Third Quarter Financial Results and Operational Progress
Atara Biotherapeutics
2025-11-12
financial results
Health Insurance Enrollment: Winning OEP With a Tech-Driven Call Center
MedCity News
2025-11-12
open
Adding more agents or buying more leads are just pieces of the puzzle, but they don’t address the multifaceted challenge of open enrollment. Teams must be aligned around meaningful metrics rather than vanity measures like total dials. The post Health Insurance Enrollment: Winning OEP With a Tech-Driven Call Center appeared first on MedCity News .
Pfizer Wins Metsera, Trump Strikes GLP-1 Pricing Deal, FDA Awards More Priority Vouchers
BioSpace
2025-11-12
priority review
Pfizer seals the deal with Metsera for $10 billion after Novo Nordisk bowed out; President Donald Trump welcomes executives from Novo and Eli Lilly to the White House to announce that the companies’ GLP-1 medicines would be sold at a reduced cost; and the FDA grants the second round of priority review vouchers—primarily to already marketed drugs.
Immix Biopharma Appoints Chief Commercial Officer to Drive NXC-201 Launch
Immix Biopharma
2025-11-12
nxc-201
– Former Chimerix (CMRX, acquired by Jazz for $935 million) Senior Commercial Leader Michael Grabow led Go-To-Market Strategy and Launch for rare disease therapeutic MODEYSO® – – Rare disease commercial veteran Mr. Grabow to lead Immix’s U.S. launch and commercial buildout for NXC-201 in relapsed/refractory AL Amyloidosis, a serious, rare, blood disease with no currently […] The post Immix Biopharma Appoints Chief Commercial Officer to Drive NXC-201 Launch first appeared on IMMIX BIOPHARMA INC .
Autolus Therapeutics reports Q3 EPS (30c), consensus (23c)
Autolus Therapeutics
2025-11-12
b-all
Reports Q3 revenue $21.144M, consensus $21.31M. “Through three quarters of launch, we are encouraged by our progress to increase the overall market in r/r B-ALL, re...
AstraZeneca reports positive results from phase 3 blood pressure trial
PMLive Research
2025-11-12
phase 3
The study demonstrated reduction in systolic blood pressure compared to placebo in patient with hypertension
Complete Remission Improves Survival in MDS Transforming to AML
Hematology Advisor News
2025-11-12
aml
ECOG performance status, IPSS risk scores, bone marrow blast percentage, TP53 mutations, and cytogenetic abnormalities independently predicted prognosis.
Use of Alternative Donor HCT Limited in Patients With Severe Aplastic Anemia
Hematology Advisor News
2025-11-12
allogeneic
In patients with SAA, upfront allogeneic HCT using alternative donors has become an option for some, but this method has had limited utilization.
Roche Holding (SWX:ROG) Is Up 9.4% After Positive Phase III Results in MS and Lupus—Has the Bull Case Changed?
Roche Group
2025-11-12
lupus
multiple sclerosis
Roche has reported highly positive Phase III results for its investigational drugs fenebrutinib in multiple sclerosis and Gazyva/Gazyvaro in systemic lupus erythematosus, showing significant clinical benefits over standard therapies and meeting all primary and key secondary endpoints. This breadth of late-stage success highlights Roche's capabilities in advancing novel treatments for large, high-unmet-need autoimmune diseases, reinforcing its position as a leader in neurology and immunology...
Novartis says Phase 3 data prove new antimalarial could address resistance
Endpoints News
2025-11-12
phase 3
Novartis’ experimental malaria treatment passed a Phase 3 trial, bringing it one step closer to becoming the newest tool in an aging arsenal of antimalarials. The drug, called GanLum, was non-inferior to Novartis’ standard-of-care treatment ...
Kalaris Reports Third Quarter 2025 Financial Results and Provides Business Updates | Wed, 11/12/2025
Kalaris Therapeutics
2025-11-12
phase 3
Phase 1a Single Ascending Dose trial remains on track for initial clinical data to be reported by year end 2025 Phase 1b/2 Multiple Ascending Dose trial currently enrolling, intended to inform dose selection for potential Phase 3 development program; initial clinical data expected in 2H 2026
Stem Cell Transplant and Targeted Therapy Extend MDS Survival After HMA Failure
Hematology Advisor News
2025-11-12
allogeneic
Allogeneic stem cell transplantation and targeted therapy conferred the longest median survival after HMA failure.
Prime Therapeutics introduces new publication, Cell & Gene Pipeline Outlook
Prime Therapeutics
2025-11-12
gene therapies
Maryam Tabatabai, PharmD, associate vice president of clinical information, shares more about how cell and gene therapies are transforming the treatment landscape, and how payers and providers can keep up with the latest insights
Metagenomi Chops 25% of Workforce, CEO To Advance Hemophilia A Program
BioSpace
2025-11-12
hemophilia a
The strategic initiative will extend Metagenomi’s cash runway into the fourth quarter of 2027. That same year, the company expects to generate initial Phase I data for its lead asset MGX-001 in hemophilia A.
Novartis, Vertex, More Optimizing Shots on Multiple Goals in Lucrative I&I Space
BioSpace
2025-11-12
pipeline
With immunology and inflammation blockbusters like AbbVie’s Skyrizi and Rinvoq reeling in nearly $7 billion combined in the third quarter, the pipeline-in-a-product strategy has never been more attractive.
Beam Therapeutics to Participate in 2025 Jefferies London Healthcare Conference
beam therapeutics
2025-11-12
base editing
CAMBRIDGE, Mass., Nov. 12, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that management will participate in a fireside chat during the 2025 Jefferies London Healthcare Conference
VIDEO: Potential treatment options emerge for patients with marginal zone lymphoma
Healio
2025-11-12
phase 2
marginal zone lymphoma
In this video, Paolo Strati, MD, discusses two clinically impactful phase 2 studies that evaluated the safety and efficacy of lisocabtagene maraleucel and mosunetuzumab-axgb for patients with marginal zone lymphoma.The results were presented at Society of Hematologic Oncology’s Annual Meeting.
Abeona Therapeutics® Reports Third Quarter 2025 Financial Results and Corporate Updates
Abeona Therapeutics
2025-11-12
financial results
Melanoma Risk Increased After Radioactive Iodine Treatment for Primary Thyroid Cancer
Hematology Advisor News
2025-11-12
melanoma
Researchers found that 45.5% of the 174,916 patients with primary thyroid cancer diagnosed from 2000 to 2019 had first-course treatment of some form of radiation.
Immatics shares early bispecific data as it seeks to make its name beyond cell therapy
Endpoints News
2025-11-12
phase 1
cell therapy
Immatics is planning to advance two T cell receptor-based bispecific antibodies based on results reported Wednesday across Phase 1 trials. The company’s bispecific work previously faced questions when Bristol Myers Squibb ...
Caribou Biosciences Reports Third Quarter 2025 Financial Results and Provides Business Update
caribou biosciences
2025-11-12
car-t
phase 1
allogeneic
autologous
cell therapy
Vispa-cel (CB-010) ANTLER phase 1 data demonstrate efficacy and durability on par with autologous CAR-T cell therapy and safety allows for outpatient use, highlighting its potential as a best-in-class allogeneic CAR-T cell therapy for LBCL CB-011 CaMMouflage phase 1 data demonstrate deep, durable
Autolus outlines margin improvement and market share growth focus while expanding AUCATZYL to 60 U.S. centers
Autolus Therapeutics
2025-11-12
b-all
aucatzyl
Earnings Call Insights: Autolus Therapeutics plc (AUTL) Q3 2025 Management View CEO Christian Itin reported the launch of AUCATZYL in the U.S. has reached market leadership in relapsed/refractory B-ALL, with broad access and reliable delivery, emphasizing, "We managed, in the first 9 months, to achieve market leadership, broad market access and cov...
Venture Global (VG): Assessing Valuation Following Ongoing Share Price Decline
uniQure
2025-11-12
financial results
Venture Global (VG) shares moved lower this week, continuing a trend that has seen the stock fall 45% over the past 3 months. Investors remain focused on the company’s recent financial results and evolving growth outlook. See our latest analysis for Venture Global. Venture Global's recent slide comes after a tough stretch this year, with the share price return now down 68.6% year-to-date and momentum clearly fading. A string of weaker financial updates and cautious investor sentiment seem to...
Opus Genetics Announces Financial Results for Third Quarter 2025 and Provides Corporate Update
opus genetics
2025-11-12
financial results
10-Q - Atara Biotherapeutics, Inc. (0001604464) (Filer)
SEC Filing (10-Q)
2025-11-12
atara biotherapeutics
Filed: 2025-11-12 AccNo: 0001193125-25-276219 Size: 12 MB
Darzalex Faspro Approved for High-Risk Smoldering Multiple Myeloma
Hematology Advisor News
2025-11-12
multiple myeloma
Darzalex Faspro reduced the risk of disease progression to active multiple myeloma or death by 51% compared with active monitoring.
Autolus Therapeutics Reports Q3 2025 Financial Results and Highlights Progress in AUCATZYL® Launch and Clinical Development
Autolus Therapeutics
2025-11-12
car t
aucatzyl
Autolus Therapeutics reports $21.1 million in Q3 2025 revenue and advances its AUCATZYL therapy in multiple clinical trials.Quiver AI SummaryAutolus Therapeutics plc reported net product revenue of $21.1 million for the third quarter of 2025 and a deferred revenue of $7.6 million, with 60 authorized treatment centers established ahead of schedule. The company is focused on expanding the market for its CAR T therapy, AUCATZYL®, particularly in rel
VIDEO: Bispecific antibody, antibody-drug conjugate combination promising in DLBCL
Healio
2025-11-12
dlbcl
Blood Test Helps Identify Ovarian Cancer Patients for PARP Therapy
Inside Precision Medicine
2025-11-12
ccr4
ovarian cancer
The new diagnostic, called the CUP-CC assay, analyzes a set of immune biomarkers in blood, including upregulation of the chemokine receptor CCR4, and circulating levels of chemokines CCL17 and CCL22 and cytokines IL-6 and IL-8. The post Blood Test Helps Identify Ovarian Cancer Patients for PARP Therapy appeared first on Inside Precision Medicine .
Alkermes reports messy Phase 2 narcolepsy data, stock drops
Endpoints News
2025-11-12
phase 2
Alkermes’ quest to develop a new narcolepsy drug became murkier on Wednesday following another Phase 2 readout. The company claimed success, noting the 18 mg high dose achieved a statistically significant ...
Autolus Therapeutics : 251112 Q3 2025 Earnings Final v2
Autolus Therapeutics
2025-11-12
financial results
Q3 2025 Financial Results and Business Updates November 12, 2025 For Investor communication only. Not for use in product promotion. Not for further...
[8-K] enGene Holdings Inc. Reports Material Event
enGene Google
2025-11-12
phase 2
detalimogene voraplasmid
enGene Holdings Inc. reported updates from its ongoing Phase 2 LEGEND trial of detalimogene voraplasmid in high-risk, BCG‑unresponsive NMIBC. After discussions
enGene Holdings: Still A Buy After A Massive Surge (ENGN)
enGene Google
2025-11-12
detalimogene voraplasmid
enGene Holdings Inc. remains a Buy, driven by promising LEGEND trial data for detalimogene voraplasmid. Learn more about ENGN stock here.
Neurogene Reports Positive Interim Data in Pediatric Cohort from NGN-401 Gene Therapy Trial for Rett Syndrome
neurogene
2025-11-12
gene therapy
rett syndrome
Multidomain, durable gains with continued skill acquisition over time All 8 participants showed functional gains across spectrum of disease severity 35 total developmental milestones/skills acquired across 8 participants NGN-401 at the registrational dose continues to be generally well-tolerated,
J&J, Legend withdraw ASH abstract assessing Carvykti against Gilead, Arcellx’s rival CAR-T
Gilead Sciences
2025-11-12
car-t
carvykti
Johnson & Johnson and Legend Biotech are said to have withdrawn an abstract originally planned for next month’s Amer | “At this point, the abstract was withdrawn in alignment with the authors, and we are looking forward to future opportunities to share the data,” Legend’s president of Carvykti, Alan Bash, said Wednesday.
Around the Helix: Cell and Gene Therapy Company Updates – October 22, 2025
CGTLive
2025-11-12
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
enGene price target raised to $10 from $4 at UBS
enGene Google
2025-11-12
phase 2
UBS raised the firm’s price target on enGene (ENGN) to $10 from $4 and keeps a Neutral rating on the shares. EnGene reported updated Phase 2 LEGEND trial data for
UPDATED: Prasad, Makary unveil new FDA path for accelerating custom gene editing therapies
uniQure
2025-11-12
gene editing
In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory pathway that could trigger a seismic shift in how bespoke gene editing therap | In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory pathway that could trigger a seismic shift in how bespoke gene editing therapies are developed and approved.
Anixa Biosciences Awarded Key U.S. Patent Expanding Breast Cancer Vaccine IP Protection into 2040s
anixa biosciences
2025-11-12
car-t
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
Patent Extends Protection for Novel Vaccine Targeting a Cancer Projected to Strike Nearly 300,000 U.S. Women in 2026 SAN JOSE, Calif. , Nov. 12, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the United States Patent and Trademark Office (USPTO) will issue U.S. Patent Number 12,472,205 on November 18, 2025, covering key aspects of the Company's breast cancer vaccine technology. The patent protects novel methods of inducing an immune response to α-lactalbumin protein—a protein typically found only in normal breast tissue during lactation but also expressed in certain breast cancers, making it an attractive target for immunoprevention strategies. The co-inventors of the patent are the late Dr. Vincent Tuohy, and Dr. Justin Johnson of Cleveland Clinic. "Our breast cancer vaccine program is a cornerstone of our strategy to develop next-generation preventive immunotherapies," stated Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences. "This new patent meaningfully extends the duration of our intellectual property protection and reinforces our…
Biohaven Seeks $150 Million From Wall Street Following FDA Rejection
BioSpace
2025-11-12
pipeline
After the FDA rejected its spinocerebellar ataxia treatment, Biohaven missed out on a $150 million payment from Oberland Capital. Now the company is reshuffling its pipeline to stay alive.
VIDEO: Combination therapy could ‘challenge the paradigm’ of relapsed/refractory DLBCL care
Healio
2025-11-12
dlbcl
In this video, Paolo Strati, MD, discusses the potential of bispecific antibody and antibody-drug conjugate combination therapy for relapsed or refractory diffuse large B-cell lymphoma, based on initial results from the LOTIS-7 trial.The trial was presented at Society of Hematologic Oncology’s Annual Meeting.
Adicet Bio Announces First Systemic Sclerosis (SSc) Patient Dosed in Ongoing Phase 1 Clinical Trial of ADI-001 in Autoimmune Diseases
Adicet Bio
2025-11-11
phase 1
EnGene Shares Surge 53% On Strong Bladder Cancer Gene Therapy Data
enGene
2025-11-11
phase 2
gene therapy
bladder cancer
complete response
(RTTNews) - enGene Holdings Inc. (ENGN) jumped 53.08% to $9.20, up $3.19, after its press release showing that its gene-therapy candidate detalimogene achieved a 62% complete response rate at six months in the Phase 2 LEGEND trial for high-risk, BCG-unresponsive non-muscle-invasi
AAVantgarde’s Stargardt Gene Therapy AAVB-039 Garners FDA Orphan Drug Designation, Cleared for Trial in UK
CGTLive
2025-11-11
ind
orphan drug
gene therapy
The IND application enabling the launch of the CELESTE trial in the US was previously cleared by the FDA in July 2025.
Why EnGene Is Rising In Pre-market?
enGene
2025-11-11
phase 2
bladder cancer
detalimogene voraplasmid
(RTTNews) - enGene (ENGN) reported additional preliminary data from the cohort of its Phase 2 LEGEND trial of detalimogene voraplasmid in high-risk, Bacillus Calmette-Gurin-unresponsive non-muscle invasive bladder cancer patients with carcinoma in situ with or without concomitant
enGene reports detalimogene shows complete response rate of 62% at 6 months
enGene
2025-11-11
phase 2
pivotal
complete response
detalimogene voraplasmid
enGene (ENGN) Holdings reported additional preliminary data from the pivotal cohort of its ongoing, Phase 2 LEGEND trial of detalimogene voraplasmid, also known as ...
Why enGene Holdings Stock Is Trading Higher Today
enGene
2025-11-11
phase 2
bladder cancer
enGene's Phase 2 LEGEND trial shows 63% response in BCG-unresponsive bladder cancer, supporting plans for a 2026 FDA filing.
VIDEO: Tyrosine kinase inhibitors, gene therapy being investigated for wet AMD
Healio
2025-11-11
gene therapy
wet age-related macular degeneration
ORLANDO — In this video from the American Academy of Ophthalmology meeting, Esther M. Bowie, MD, discusses what is “new and exciting” in wet age-related macular degeneration.Tyrosine kinase inhibitors being investigated for wet AMD include EYP-1901 (vorolanib intravitreal insert, EyePoint Pharmaceuticals) and OTX-TKI, also known as Axpaxli (axitinib intravitreal implant, Ocular
enGene’s NMIBC gene therapy touts 63% complete response in pivotal trial
enGene
2025-11-11
bla
pivotal
gene therapy
complete response
detalimogene voraplasmid
enGene anticipates filing a biologics licence application (BLA) for detalimogene voraplasmid to the FDA in H2 2026.
Neurocrine reports Phase 2 fail for depression drug it licensed from Takeda
Endpoints News
2025-11-11
phase 2
Neurocrine Biosciences said that a candidate for major depressive disorder that it got from Takeda did not succeed in a mid-stage trial. The oral drug, codenamed NBI-1070770, did not meet the primary endpoint. This used ...
Book value per share of enGene Holdings Inc. Warrant - on enGene Holdings – NASDAQ:ENGNW
enGene
2025-11-11
quarterly
Book value per share, quarterly and annual stats of enGene Holdings Inc. Warrant - on enGene Holdings.
uniQure (QURE) Is Down 14.1% After FDA Alters AMT-130 Approval Pathway - Has The Bull Case Changed?
uniQure
2025-11-11
amt-130
gene therapy
In early November 2025, uniQure announced that the U.S. Food and Drug Administration no longer supports using Phase I/II data with external controls as the primary evidence for a Biologics License Application for AMT-130, its investigational gene therapy for Huntington's disease. This sudden regulatory shift created uncertainty regarding the product’s approval pathway, despite recent promising clinical data and prior breakthrough designations for AMT-130. We'll explore how increased...
Unlocking gene therapy’s potential for neurotransmitter disorders
AADC News/BioNews
2025-11-11
gene therapy
Last week, Judy, my wife, and I had the privilege of attending the International Neurotransmitter Disorders Conference (INDConf), held Nov. 5-7 at Goodenough College in London. Nestled in the heart of Bloomsbury, the historic venue provided an inspiring setting filled with warmth, collaboration, and academic energy. The event brought together scientists, clinicians, and families united […] The post Unlocking gene therapy’s potential for neurotransmitter disorders appeared first on AADC News .
Detalimogene Demonstrates Improved Complete Response Rate of 62% at 6 Months
enGene
2025-11-11
complete response
enGene Holdings Inc. (Nasdaq: ENGN or “enGene” or the “Company”), a clinical-stage, non-viral genetic medicines company, today reported additional preliminar...
Should FDA Priority Review for Expanded PALYNZIQ Use Prompt Action From BioMarin (BMRN) Investors?
BioMarin Pharmaceutical
2025-11-11
pipeline
priority review
BioMarin Pharmaceutical announced that the FDA has accepted for Priority Review its supplemental Biologics License Application to expand PALYNZIQ® treatment for adolescents with phenylketonuria (PKU), setting a target action date of February 28, 2026. This regulatory milestone could increase PALYNZIQ’s eligible patient base and signals ongoing progress in BioMarin’s rare disease pipeline. We’ll look at how expanded adolescent access to PALYNZIQ may influence BioMarin’s future revenue growth...
EnGene posts more pivotal data for bladder cancer gene therapy, plans 2026 filing
Endpoints News
2025-11-11
bla
pivotal
gene therapy
bladder cancer
EnGene has posted another tranche of encouraging bladder cancer treatment data from a pivotal trial, teeing up a BLA filing in the back half of next year. The company said that the gene therapy, called ...
Genentech announces positive clinical results for BTK inhibitor MS treatment
PMLive Research
2025-11-11
multiple sclerosis
Multiple sclerosis affects more than 2.9 million people globally
Buy Rating for enGene Holdings Amid Anticipated Positive Data from Phase 2 LEGEND Study
enGene Google
2025-11-11
phase 2
Judah Frommer, an analyst from Morgan Stanley, maintained the Buy rating on enGene Holdings. The associated price target remains the same with $18.00. Judah Frommer...
Trial sets up filing for enGene bladder cancer gene therapy
enGene
2025-11-11
phase 2
gene therapy
bladder cancer
complete response
enGene is hoping to file its gene for a form of bladder cancer before the end of 2026, based on phase 2 data revealing a 62% complete response rate
EnGene Holdings Inc. (ENGN) Stock: Soars Over 50% as Positive Phase 2 Data Boosts Investor Confidence
enGene Google
2025-11-11
phase 2
bladder cancer
ENGN jumps over 50% after strong Phase 2 data shows promising response rates and safety in bladder cancer treatment trials.
Alpha-1 Antitrypsin Deficiency Disease Market Research Report 2025, Competitive Analysis of Shire, GSK, CSL Behring, AstraZeneca, Grifols, Teva Pharma, Boehringer Ingelheim, Kamada, Pfizer, Baxter
CSL Behring
2025-11-11
gene therapies
alpha-1 antitrypsin deficiency
The Alpha-1 Antitrypsin Deficiency Disease market offers opportunities in developing targeted therapies due to an increasing diagnosed patient population, advances in genetic testing, and innovative treatments like enzyme replacement and gene therapies. Growing awareness and technological advancements further drive this niche market. Alpha-1 Antitrypsin Deficiency Disease Market Alpha-1 Antitrypsin Deficiency Disease Market Dublin, Nov. 11, 2025 (GLOBE NEWSWIRE) -- The "Alpha-1 Antitrypsin Defic
Net income of enGene Holdings Inc. – DUS:S0N
enGene
2025-11-11
quarterly
Net income, quarterly and annual stats of enGene Holdings Inc..
Genentech announces positive clinical results for BTK inhibitor MS treatment
Genentech
2025-11-11
multiple sclerosis
Multiple sclerosis affects more than 2.9 million people globally
Adicet Bio Announces First Patient Dosed in the Phase 1 Clinical Trial of ADI-270 in Metastatic/Advanced Clear Cell Renal Cell Carcinoma
Adicet Bio
2025-11-11
phase 1
Neurocrine Bio Drug Licensed From Takeda Flunks Mid-Stage Test in Depression
MedCity News
2025-11-11
phase 2
The Phase 2 failure in major depressive disorder marks the second time in a little more than a year that Neurocrine Biosciences has reported a drug licensed from Takeda Pharmaceutical fell short in the clinic. Last year, a different drug from this partnership failed a mid-stage test in schizophrenia. The post Neurocrine Bio Drug Licensed From Takeda Flunks Mid-Stage Test in Depression appeared first on MedCity News .
Voyager, Transition Bio's small molecule drug pact; CRISPR Therapeutics ends work on a CAR-T therapy
Endpoints News
2025-11-11
car-t
crispr
Plus, news about InflaRx, Arcturus, Vor Biopharma and Precision BioSciences: 🤝 Al Sandrock’s Voyager makes a deal with another company he oversees: Voyager announced a deal with ...
HMA-Venetoclax Shows Clinical Benefit in Post-Transplant MDS/AML Relapse
Hematology Advisor News
2025-11-11
aml
Venetoclax dosing was individualized based on cytopenias or concomitant antifungal therapy. The median number of treatment cycles was 4, with a median of 21 days of venetoclax per cycle.
enGene (Nasdaq: ENGN) reports 62% CR at 6 months in NMIBC; LEGEND enrollment completed
enGene
2025-11-11
bla
phase 2
Phase 2 LEGEND analysis shows 63% any-time CR and 56% at 3 months, with TRAEs in 42% and dose interruptions 1.6%. Enrollment 125; BLA planned H2 2026.
GenomOncology Partners with Glioblastoma Foundation to Accelerate Precision Glioblastoma Brain Cancer Treatment
PR Newswire
2025-11-11
glioblastoma
CLEVELAND and DURHAM, N.C., Nov. 11, 2025 /PRNewswire/ -- GenomOncology today announced a strategic partnership with the Glioblastoma Foundation to transform genomic testing for the nation's most aggressive brain cancer, glioblastoma. The collaboration integrates GenomOncology's advanced...
Generative AI often spreads false, stigmatizing obesity content
Healio
2025-11-11
phase 2
ATLANTA — Both conversational and image-based generative AI often spreads inaccurate and stigmatizing information about people with obesity, worsening the stigma around weight gain, according to phase 2 data presented at ObesityWeek. “Not only is generative AI being increasingly used, but [it is] increasingly being used in health care,” Stuart W. Flint, PhD, associate professor of psychology at
enGene’s Detalimogene Shows Promising Results in Bladder Cancer Trial
enGene
2025-11-11
phase 2
bladder cancer
enGene Holdings ( ($ENGN) ) has issued an announcement. enGene Holdings Inc. has reported promising preliminary data from its Phase 2 LEGEND trial for detalimogene ...
Here’s Why enGene Holdings Stock Is Soaring Today
enGene Google
2025-11-11
phase 2
bladder cancer
enGene Holdings ($ENGN) stock is soaring on Tuesday after releasing promising preliminarydata from its Phase 2 LEGEND trial for its bladder cancer gene therap...
Childhood hereditary angioedema worries, interrupts lives of patients, caregivers
Healio
2025-11-11
hereditary angioedema
ORLANDO — The impact of hereditary angioedema on pediatric patients and their caregivers goes beyond the disease itself and can take a toll on mental health, according to study findings.These data were presented at the American College of Allergy, Asthma & Immunology Annual Scientific Meeting.
VIDEO: Future of MASH treatment likely to include combination therapy
Healio
2025-11-11
pipeline
PHOENIX — In this video, Mazen Noureddin, MD, MHSc, professor of medicine at Houston Methodist, highlights current treatments and others in the pipeline for metabolic dysfunction-associated steatohepatitis.Two medications are currently approved for MASH with fibrosis: Rezdiffra (resmetirom, Madrigal Pharmaceuticals), an oral thyroid hormone receptor-beta selective agonist, and Wegovy
uniQure’s Bumpy Ride: Stock Price Prospects
uniQure
2025-11-11
amt-130
gene therapy
uniQure N.V.’s stocks have been trading up by 19.12% following encouraging FDA designation, instilling investor optimism. Key Developments About QURE Q3 earnings fell short with an EPS of ($1.38), not meeting expectations, paired with unimpressive $3.7M revenue against a $4.46M consensus. AMT-130, a promising gene therapy for Huntington’s, showcased hopeful results in slowing disease progression […]
enGene soars on positive detalimogene data
enGene Google
2025-11-11
pivotal
detalimogene voraplasmid
Canadian genetic medicines company enGene Holdings saw its shares rocket as much as 65% to $9.93 in pre-market trading, after it reported additional preliminary data from the pivotal cohort of its ongoing, Phase II LEGEND trial of detalimogene voraplasmid (also known as detalimogene and previously EG-70).
Analgesia for sickle cell vaso-occlusive crises often delayed due to improper ED triage
Healio
2025-11-10
sickle cell disease
Most patients with sickle cell disease who go to the ED for vaso-occlusive crises do not get triaged according to guideline recommendations, resulting in significantly longer wait times for pain medication.Patients who receive an Emergency Severity Index (ESI) score of 3, instead of 2, can experience a 2-hour delay in analgesia, according to a retrospective analysis.
Roche's experimental MS drug hits main goal in one of two key trials
Roche Group
2025-11-10
multiple sclerosis
By Ludwig Burger Roche's SIX:RO experimental multiple sclerosis drug achieved its main goal in one of two key late-stage trials testing it against the relapsing form of the disease, the Swiss drugmaker said on Monday, sending its shares up nearly 2%. The drug, fenebrutinib, was shown to signific…
8-K - Intellia Therapeutics, Inc. (0001652130) (Filer)
SEC Filing (8-K)
2025-11-10
intellia therapeutics
inc.
Filed: 2025-11-10 AccNo: 0001193125-25-274596 Size: 238 KB Item 7.01: Regulation FD Disclosure Item 8.01: Other Events Item 9.01: Financial Statements and Exhibits
uniQure (QURE) Reveals Promising Data Amid Regulatory Challenges
uniQure
2025-11-10
amt-130
Key Takeaways: uniQure (QURE) reported third-quarter 2025 revenue of $3.7 million, missing expectations. Promising three-year data for AMT-130 shows sign
Anixa Biosciences to Participate in Water Tower Research Fireside Chat on Tuesday, November 11, 2025
anixa biosciences
2025-11-10
car-t
phase 1
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
SAN JOSE, Calif. , Nov. 10, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that its Chairman and CEO, Dr. Amit Kumar, will participate in the upcoming Water Tower Research Fireside Chat Series taking place on Tuesday, November 11, 2025, at 11:00am PT. The Fireside Chat wil be hosted by Robert Sassoon, Managing Director – Healthcare at Water Tower Research, and will cover several topics, including: Overview of the Company and its clinical programs Breast cancer vaccine Phase 1 clinical trial status This event is open access for all investors to participate. Interested parties can register for the event through Water Tower Research at: https://us06web.zoom.us/webinar/register/9617621959146/WN_zLl3VvR4TRyikSX7QdSoMQ About Anixa Biosciences, Inc. Anixa is a clinical-stage biotechnology company focused on the treatment and prevention of cancer. Anixa's therapeutic portfolio consists of an ovarian cancer immunotherapy program being developed in collaboration with Moffitt Cancer Center, which uses a novel type of CAR-T, known as chimeric endocrine receptor-T…
#AHA25: CRISPR Therapeutics’ gene editing therapy halves cholesterol and triglycerides in early trial
Endpoints News
2025-11-10
crispr
gene editing
One of CRISPR Therapeutics’ gene editing treatments cut levels of blood fats and cholesterol in an early-stage trial, according to data presented Saturday, raising the possibility of a once-and-done treatment for conditions that increase heart ...
Lilly picks up an eye gene therapy in deal with MeiraGTx
Biopharma Dive
2025-11-10
gene therapy
The alliance extends a deal streak in genetic medicine for Lilly and hands the company rights to an experimental treatment for an ultra-rare form of childhood blindness.
Vir Biotechnology Stock Set For Premarket Surge? Retail Trader Hopes Swell As ‘Phenomenal’ HDV Data Sets Stage For Gilead Showdown
Gilead Sciences
2025-11-10
phase 2
Phase 2 trial showed 66% of patients achieved undetectable hepatitis delta virus RNA with Vir’s tobevibart-elebsiran combo, with no serious safety issues reported.
Precision BioSciences Presents Late-Breaking Phase 1 PBGENE-HBV Data at AASLD The Liver Meeting® Showing Safety, Tolerability and Cumulative, Dose-Dependent Antiviral Activity in First Three Cohorts
precision biosciences
2025-11-10
phase 1
gene editing
PBGENE-HBV, the first gene editing therapy designed to treat chronic Hepatitis B by directly targeting HBV cccDNA and integrated HBV DNA, showcased as the final oral presentation in the late-breaking AASLD session at 5:45pm EST on Monday, November 10, 2025 PBGENE-HBV was well-tolerated across
Kyverna Therapeutics to Present at the Jefferies London Healthcare Conference
Kyverna Therapeutics
2025-11-10
cell therapies
EMERYVILLE, Calif., Nov. 10, 2025 (GLOBE NEWSWIRE) -- Kyverna Therapeutics, Inc. (Kyverna, Nasdaq: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, today announced that Warner Biddle, Chief Executive Officer of Kyverna,
Protara Therapeutics Announces Third Quarter 2025 Financial Results and Provides Business Update
protara therapeutics
2025-11-10
phase 2
Results from an interim analysis in the Phase 2 STARBORN-1 trial of TARA-002 in pediatric LMs patients remain on track for 4Q 2025 Expect to present interim analysis from approximately 25 six-month evaluable BCG-Unresponsive NMIBC patients in the ongoing ADVANCED-2 trial in 1Q 2026 Dosing of first
Humacyte Announces Presentation of Positive Two-Year Results from Phase 3 Dialysis Access Trial at the American Society of Nephrology’s Kidney Week 2025
Humacyte
2025-11-10
phase 3
– The ATEV™ was observed to have superior duration of use over 24 months compared to autogenous fistula in high-need subgroups with historically poor outcomes with AV fistula procedures – – The significantly longer duration of ATEV use in these high-need patients could greatly reduce reliance on
Genentech’s Fenebrutinib Shows Unprecedented Positive Phase III Results as the Potential First and Only BTK Inhibitor in Both Relapsing and Primary Progressive Multiple Sclerosis
Genentech
2025-11-10
multiple sclerosis
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the first Phase III
UniQure outlines urgent FDA engagement for AMT-130 as BLA timing turns uncertain
uniQure
2025-11-10
bla
amt-130
pivotal
gene therapy
Earnings Call Insights: uniQure N.V. (QURE) Q3 2025 Management View CEO Matthew Kapusta reported, "in the third quarter, we announced positive top line data from our pivotal Phase I/II study of AMT-130 in Huntington's disease, the first gene therapy to demonstrate statistically significant slowing of disease progression in Huntington's disease". He...
Catalent lays off more gene therapy workers in Baltimore
Endpoints News
2025-11-10
gene therapy
Catalent will let go of more gene therapy staff in Maryland over continued issues in demand from a "large commercial customer," a company spokesperson told Endpoints News. A total of 77 workers will be laid ...
Allogene Therapeutics Announces Participation in Upcoming Investor Conferences
Allogene
2025-11-10
car t
allogeneic
SOUTH SAN FRANCISCO, Calif. , Nov. 10, 2025 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™) products for cancer and autoimmune disease, today announced that it will participate in
Genentech’s Fenebrutinib Shows Unprecedented Positive Phase III Results as the Potential First and Only BTK Inhibitor in Both Relapsing and Primary Progressive Multiple Sclerosis
Genentech
2025-11-10
pivotal
SOUTH SAN FRANCISCO, Calif., November 10, 2025--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the first Phase III (FENhance 2) of two pivotal, similarly-designed Phase III studies (FENhance 1 and 2) in patients with relapsing multiple sclerosis (RMS) met its primary endpoint. Fenebrutinib, an investigational Bruton’s tyrosine kinase (BTK) inhibitor, significantly reduced the annualized relapse rate (ARR) compared to teriflunomide over a period of at le
Cabaletta Bio Reports Third Quarter 2025 Financial Results and Provides Business Update
cabaletta bio
2025-11-10
financial results
INOVIO Reports Third Quarter 2025 Financial Results and Recent Business Highlights
Inovio Pharmaceuticals
2025-11-10
financial results
Multidrug-Resistant Ulcerative Colitis: Response to CD19 CAR T-Cell Therapy in a Single Case
ASCO Post Journals
2025-11-10
cd19
car t
cell therapy
In a letter to the editor published in The New England Journal of Medicine, Markus F. Neurath, MD, of Friedrich-Alexander-University Erlangen–Nuremberg, Erlangen, Germany, and colleagues described the...
Precision BioSciences Announces $75 Million Offering of Common Stock, Pre-Funded Warrants and Warrants
precision biosciences
2025-11-10
gene editing
DURHAM, N.C. --(BUSINESS WIRE)--Nov. 10, 2025-- Precision BioSciences, Inc. (Nasdaq: DTIL) (“Precision”), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that it has agreed
Genentech reports first relapsing multiple sclerosis trial met primary endpoint
Genentech
2025-11-10
pivotal
Genentech, a member of the Roche (RHHBY), announced that the first Phase III of two pivotal, similarly-designed Phase III studies in patients with relapsing multipl...
AnaptysBio reports Phase 2 fail in ulcerative colitis; Lyell's CAR-T licensing deal
Endpoints News
2025-11-10
car-t
phase 2
Plus, news about Tenaya, AtaiBeckley, Galecto, Damora, A2 Biotherapeutics, AstraZeneca and Evommune: 📉 AnaptysBio's lead drug fails ulcerative colitis study: The company said the T cell depleter ...
Nkarta Reports Third Quarter 2025 Financial Results and Corporate Highlights
nkarta
2025-11-10
nkx019
dose-escalation
b-cell depletion
Enrollment now underway in second dose-escalation cohort Deep B-cell depletion observed in all patients treated to date who received NKX019 with lymphodepletion using fludarabine and cyclophosphamide versus partial B-cell depletion in patients receiving only cyclophosphamide Enrollment streamlined
Intellia Therapeutics Presents Positive Longer-Term Phase 1 Data of Nexiguran Ziclumeran (nex-z) in Patients with Transthyretin (ATTR) Amyloidosis with Cardiomyopathy
Intellia Therapeutics Inc.
2025-11-10
phase 1
One-time treatment of nex-z led to consistently rapid, deep and durable reduction in serum TTR through three years of follow-up Consistent trend in disease stability or improvement in multiple measures of cardiomyopathy, regardless of NYHA Class, at 24 months compared to baseline Longer-term safety
Roche posts late-stage trial wins for multiple sclerosis drug
Roche Group
2025-11-10
multiple sclerosis
Roche (RHHBY) stock gains and TG Therapeutics (TGTX) stock falls as the Swiss firm's new drug for multiple sclerosis succeeds in two late-stage trials. Read more here.
Roche (RHHBY) Shares Rise on Positive Phase 3 Trial Results for Fenebrutinib
Roche Group
2025-11-10
phase 3
Key Takeaways: Roche's experimental drug fenebrutinib shows promising results in Phase 3 trials, boosting investor confidence. The company maintains a st
Cogent soars on back of Phase 3 success in stromal tumors
Endpoints News
2025-11-10
phase 3
Cogent Biosciences’ share price more than doubled on Monday after its drug candidate halved the risk of death or disease progression in a late-stage trial of a type of rare tumor that grows in the ...
10-Q - CAPRICOR THERAPEUTICS, INC. (0001133869) (Filer)
SEC Filing (10-Q)
2025-11-10
capricor therapeutics
Filed: 2025-11-10 AccNo: 0001104659-25-109298 Size: 7 MB
uniQure (QURE) Faces Uncertainty in BLA Submission Despite Positive AMT-130 Data
uniQure
2025-11-10
bla
amt-130
Key Takeaways: uniQure's AMT-130 shows a 75% reduction in Huntington's disease progression in Phase I/II study. FDA expresses concerns over data adequacy
Passage Bio Reports Third Quarter 2025 Financial Results and Provides Recent Business Highlights
passage bio
2025-11-10
financial results
Roche's experimental MS drug hits main goal in one of two key trials
Roche Group
2025-11-10
multiple sclerosis
Roche's experimental multiple sclerosis drug achieved its main goal in one of two key late-stage trials testing it against the relapsing form of the disease, the Swiss drugmaker said on Monday, sending its shares up nearly 2%.
CAR T-cell therapy candidate yields lasting response in tough lymphoma
Rare Cancer News
2025-11-10
car t
phase 1
allogeneic
cell therapy
One-time treatment with vispa-cel (CB-010), Caribou Biosciences’ off-the-shelf CAR T-cell therapy candidate, resulted in durable responses in people with hard-to-treat large B-cell lymphoma (LBCL). That’s according to data from the ongoing Phase 1 ANTLER clinical trial (NCT04637763), which is testing the therapy in adults with treatment-resistant or relapsed LBCL. Vispa-cel, an allogeneic CAR T-cell therapy […] The post CAR T-cell therapy candidate yields lasting response in tough lymphoma appeared first on Rare Cancer News .
Mediterranean diet shows potential as a first-line treatment for IBS
Healio
2025-11-10
first-line
The Mediterranean diet showed noninferiority and superiority in treating IBS vs. traditional dietary advice.The mechanism behind the findings remains unclear.
uniQure Announces Third Quarter 2025 Financial Results and Provides Corporate Update
uniQure
2025-11-10
bla
amt-130
pivotal
Announced pivotal topline data from Phase I/II study of AMT-130 in Huntington’s disease met its primary and key secondary endpoints, demonstrating statistically significant slowing of disease progression at 36 months and supportive trends across key clinical and biomarker endpoints ~ ~ Preliminary feedback from FDA at a recent pre-Biologics License Application (BLA) meeting for AMT-130 indicated a key shift from prior regulatory communications; uniQure plans to urgently interact with the FDA t
Prime Medicine Reports Second Quarter 2025 Financial Results and Provides Business Updates
prime medicine
2025-11-10
phase 1
phase 1/2
prime editing
Announced positive data from two patients in Phase 1/2 clinical trial in Chronic Granulomatous Disease (CGD), providing clinical proof-of-concept for Prime Editing; plan to have regulatory interactions based on current dataset -- -- Completed follow-on offering, raising $144.2 million in gross
Roche reports two Phase 3 wins for its BTK inhibitor in multiple sclerosis
Endpoints News
2025-11-10
phase 3
Roche’s BTK inhibitor has cleared two registrational studies in different types of multiple sclerosis, a surprise turn for a drug class that’s seen several trial failures and safety setbacks. The Swiss drugmaker said Monday that ...
Gilead Provides Update on Phase 3 ASCENT-07 Study
Gilead Sciences
2025-11-10
phase 3
Reporter’s Notebook: Is Intellia’s Patient Death the Nail in the Coffin for Cas9?
Inside Precision Medicine
2025-11-10
cas9
crispr
In light of the recent tragedy, Jonathan D. Grinstein, PhD, Inside Precision Medicine 's North American Editor, investigates whether the issue is specific to Intellia's program or if it affects all Cas9-based CRISPR therapies. The post Reporter’s Notebook: Is Intellia’s Patient Death the Nail in the Coffin for Cas9? appeared first on Inside Precision Medicine .
enGene to Host Conference Call to Provide Update on Pivotal Cohort of LEGEND Trial
enGene
2025-11-10
pivotal
enGene to Host Conference Call to Provide Update on Pivotal Cohort of LEGEND Trial
CRISPR Therapeutics Provides Business Update and Reports Third Quarter 2025 Financial Results
CRISPR
2025-11-10
crispr
casgevy
phase 1
Positive Phase 1 data for CTX310 ® presented in a late-breaking presentation at the American Heart Association (AHA) Scientific Sessions and simultaneously published in The New England Journal of Medicine - -CASGEVY ® momentum accelerating; nearly 300 patients have been referred to Authorized
Roche Just Unleashed a Drug That Could Disrupt the Entire MS Market
Roche Group
2025-11-10
multiple sclerosis
Roche Holding (RHHBF) could be entering a new growth chapter after unveiling strong late-stage trial data for its multiple sclerosis drug fenebrutinib. The oral BTK inhibitor sharply reduced relapse rates in patients with relapsing MS and slowed disability progression in those with the progressive…
#AHA25: Novo Nordisk posts mixed mid-stage data on ATTR-CM drug
Endpoints News
2025-11-10
phase 2
Three drugs are already approved for the progressive heart disease known as transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). Novo Nordisk appears keen to enter the market anyway, despite mixed Phase 2 trial results. ...
Capricor Therapeutics Reports Third Quarter 2025 Financial Results and Provides Corporate Update
Capricor Therapeutics
2025-11-10
financial results
UniQure Misses Estimates But Finds Support And Fresh Funding
uniQure
2025-11-10
gene therapy
Despite bigger losses and lower revenue, uniQure raised cash and kept investors optimistic as it eyes FDA talks for its gene therapy.
uniQure’s AMT-130: Promising Results Amid Regulatory Challenges Support Buy Rating
uniQure
2025-11-10
amt-130
TD Cowen analyst Joseph Thome has maintained their bullish stance on QURE stock, giving a Buy rating today. Joseph Thome’s rating is based on several factors that h...
Variation in PSA Levels in Annual Testing Among Individuals Without a Prostate Cancer Diagnosis
ASCO Post Journals
2025-11-10
prostate cancer
In a study reported in JAMA Oncology,1 Nicholas A. Pickersgill, MD, of the Department of Surgery (Urology Service) at Memorial Sloan Kettering Cancer Center, New York, and colleagues found that indivi...
Why Biohaven (BHVN) Is Down 52.0% After FDA Setback Forces R&D Overhaul
uniQure
2025-11-10
complete response
Earlier this month, Biohaven Ltd. announced it received a Complete Response Letter from the FDA declining approval of its drug candidate VYGLXIA (troriluzole) for spinocerebellar ataxia, prompting the company to restructure operations and R&D priorities. This regulatory setback marks a shift in Biohaven's resource allocation, with a heightened emphasis on advancing its most promising late-stage clinical programs while targeting a substantial reduction in direct R&D spending. We'll examine...
uniQure Announces Third Quarter 2025 Financial Results and Provides Corporate Update
uniQure
2025-11-10
amt-130
pivotal
Announced pivotal topline data from Phase I/II study of AMT-130 in Huntington’s disease met its primary and key secondary endpoints, demonstrating...
Lyell Immunopharma Acquires Exclusive Global Rights to a Next-Generation CAR T-Cell Product Candidate in Clinical Development for Metastatic Colorectal Cancer
lyell immunopharma
2025-11-10
car t
phase 1
colorectal cancer
overall response rate
LYL273 has demonstrated a 67% overall response rate, an 83% disease control rate, and a manageable safety profile at the highest dose level studied to date in patients with refractory metastatic colorectal cancer enrolled in an ongoing U.S. Phase 1 clinical trial LYL273 is a GCC-targeted CAR T-cell
Roche's oral MS hope fenebrutinib hits the mark in phase 3
PharmaPhorum
2025-11-10
phase 3
Roche's oral BTK inhibitor fenebrutinib is on track for filings next year for multiple sclerosis after clearing two phase 3 trials.
Microvesicles May Serve as Markers of Complication Risk in SCD
Hematology Advisor News
2025-11-10
scd
Patients with higher levels of RMVs, AMVs, and EMVs tended to have more severe disease, whereas those with higher fetal hemoglobin had milder disease.
[Ad hoc announcement pursuant to Art. 53 LR] Roche’s fenebrutinib shows unprecedented positive Phase III results as the potential first and only BTK inhibitor in both relapsing and primary progressive multiple sclerosis
Roche Group
2025-11-10
pivotal
The first (FENhance 2) of two pivotal RMS studies met its primary endpoint, showing investigational fenebrutinib significantly reduced relapses compared to...
Phase III Win for Roche with B Cell & Microglia Targeting MS Treatment
Roche Group
2025-11-10
multiple sclerosis
Fenebrutinib, Roche's Bruton’s tyrosine kinase inhibitor, has succeeded in two Phase III trials for treatment of multiple sclerosis.
Celldex Reports Third Quarter 2025 Financial Results and Provides Corporate UpdateDownload PDF
Celldex Therapeutics
2025-11-10
financial results
Strong execution and continued progress across pipeline Positive Phase 2 barzolvolimab data in Chronic Spontaneous Urticaria (CSU) demonstrating rapid, profound improvement in UCT7 scores with sustained disease control post treatment and strong efficacy regardless of baseline IgE levels Positive Download PDF
In string of deals, Lilly nabs ultra rare eye disease gene therapy and RNAi partner
Endpoints News
2025-11-10
gene therapy
Eli Lilly is expanding its genetic medicine repertoire through two new deals with biotech companies. It penned a deal on Monday with MeiraGTx for a gene therapy to treat a very ...
uniQure (QURE) Is Down 19.0% After FDA Questions Data for AMT-130 Gene Therapy Approval Pathway – Has The Bull Case Changed?
uniQure
2025-11-10
amt-130
gene therapy
gene therapies
On November 3, 2025, uniQure announced that the FDA now questions whether Phase I/II data for AMT-130 are sufficient as primary evidence for a Biologics License Application, introducing uncertainty about the therapy's U.S. regulatory pathway and timeline. This shift in the FDA’s stance marks a significant change from previous communications and could potentially delay the availability of one of the first gene therapies for Huntington’s disease. We’ll now consider how this FDA feedback, which...
Roivant Reports Financial Results for the Second Quarter Ended September 30, 2025, and Provides Business Update
roivant
2025-11-10
financial results
BASEL, Switzerland and LONDON and NEW YORK, Nov. 10, 2025 (GLOBE NEWSWIRE) -- Roivant (Nasdaq: ROIV) today reported its financial results for the second quarter ended September 30, 2025, and provided a business update. Brepocitinib 30 mg demonstrated clinically meaningful and statistically
Roche's MS drug candidate meets primary goal in late-stage trial
Roche Group
2025-11-10
multiple sclerosis
By Ludwig Burger Swiss drugmaker Roche SIX:RO said on Monday that a late-stage trial testing its multiple sclerosis drug candidate fenebrutinib against the relapsing form of the disease achieved its primary goal.Fenebrutinib was shown to significantly reduce the annualised relapse rate when comp…
enGene to Discuss New Data from LEGEND Trial for Bladder Cancer Therapy
enGene
2025-11-10
pivotal
bladder cancer
An update from enGene Holdings ( ($ENGN) ) is now available. enGene Holdings Inc. announced a conference call to discuss new preliminary data from the pivotal cohor...
Eli Lilly Strikes Another Genetic Meds Deal, Gaining Global Rights to MeiraGTx Retinal Gene Therapy
MedCity News
2025-11-10
aav
gene therapy
leber congenital amaurosis
Eli Lilly is paying $75 million up front for global rights to AAV-AIPL1, a gene therapy on track for U.S. and European regulatory submissions in the rare, inherited eye disorder Leber congenital amaurosis 4, or LCA4. The deal brings to Lilly another late-stage gene therapy for the eyes following the pharma giant’s October acquisition of Adverum Biotechnologies and its lead program for wet AMD. The post Eli Lilly Strikes Another Genetic Meds Deal, Gaining Global Rights to MeiraGTx Retinal Gene Therapy appeared first on MedCity News .
#AHA25: Merck reports pivotal PCSK9 pill data in race to beat AstraZeneca to market
Endpoints News
2025-11-09
pivotal
Merck’s lipid-lowering pill is on track to become the first oral PCSK9 inhibitor to reach the market, after positive data from two pivotal trials were presented at the American Heart Association's annual meeting over the ...
#AASLD25: Vir presents new hepatitis D data and ramps up bullish Phase 3 plans
Endpoints News
2025-11-09
phase 3
pivotal
New data from Vir Biotechnology’s chronic hepatitis D treatment reinforce the combo regimen’s promise of keeping viral levels low, as the company's pivotal trials advance ahead of schedule. Vir said Sunday evening that a monthly ...
Metformin reduced recurrence of atrial fibrillation after catheterization
Healio
2025-11-09
first-line
NEW ORLEANS — Adults with atrial fibrillation with overweight or obesity were less likely to experience recurrent atrial arrhythmia when taking metformin, a generic diabetes drug, before a catheter ablation, data from a small study show.Metformin, a first-line therapy for type 2 diabetes well known for its metabolic benefits, has been associated with a decrease in AF in observational
Olezarsen reduces triglycerides, cutting pancreatitis risk by 85%
Healio
2025-11-08
phase 3
pivotal
NEW ORLEANS — Monthly olezarsen reduced triglycerides by 65% for people with severe hypertriglyceridemia, resulting in a greater than 85% reduction in acute pancreatitis, data show.Findings from the pivotal CORETIMI 72a and CORE2TIMI 72b phase 3 trials were presented at the American Heart Association Scientific Sessions and simultaneously published in The New England Journal of Medicine.
Krystal Biotech doses first patient in phase 3 trial IOLITE | Ophthalmology Times - Clinical Insights for Eye Specialists
Krystal Biotech
2025-11-08
phase 3
dystrophic epidermolysis bullosa
The trial is evaluating KB803 for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa
Vertex Presents Updated Phase 1/2 Data From RUBY-3 Study That Continue to Demonstrate Best-in-Class Potential for Povetacicept in Adults with IgA Nephropathy and Primary Membranous Nephropathy at American Society of Nephrology Kidney Week
Vertex
2025-11-08
phase 1
phase 1/2
48-week data show a 64% decrease from baseline in proteinuria in IgA nephropathy, 82% decrease from baseline in proteinuria in primary membranous nephropathy, and stabilization of estimated glomerular filtration rate across both diseases - - Vertex on track to initiate rolling submission of
Patients Treated With AskBio’s Limb-Girdle Muscular Dystrophy Gene Therapy AB-1003 Free of Serious Adverse Events at Up to 52 Weeks
CGTLive
2025-11-08
gene therapy
Headaches, falls, and nausea constituted the common treatment-emergent AEs, and were deemed mild-to-moderate in terms of severity.
Gene-editing therapy for elevated lipid levels shows promise
Healio
2025-11-08
phase 1
NEW ORLEANS — In a phase 1 trial, a gene-editing therapy that targets ANGPTL3 was associated with few serious adverse events and at higher doses reduced levels of the hepatokine ANGPTL3, researchers reported. For the trial, presented at the American Heart Association Scientific Sessions and simultaneously published in The New England Journal of Medicine, the researchers administered a
Intellia Therapeutics Presents Positive Pooled Phase 1/2 Data of Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema
Intellia Therapeutics Inc.
2025-11-08
phase 1
phase 1/2
hereditary angioedema
Deep, stable and durable reductions in kallikrein observed Among 32 patients who received a 50 mg dose of lonvo-z as of data cutoff: 31 (97%) were attack-free and long-term prophylaxis (LTP)-free 24 (75%) were attack-free and LTP-free for at least seven months (up to 32 months) Among the 11
MiNK Therapeutics Reports Durable Responses and Immune Reactivation with Allo-iNKT Cell Therapy agenT-797 in PD-1–Refractory Solid Tumors at SITC 2025
Mink Therapeutics
2025-11-08
cell therapy
Durable survival and deep, lasting remissions in checkpoint-refractory, heavily pretreated cancers with median OS of ~23 months with agenT-797 plus anti-PD-1 Evidence of immune activation and tumor-immune remodeling underscore agenT-797’s potential to restore responsiveness in PD-1–resistant
CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310® Demonstrating Deep and Durable ANGPTL3 Editing, Triglyceride and Lipid Lowering
CRISPR
2025-11-08
crispr
phase 1
Data presented in a late-breaking presentation at the American Heart Association (AHA) Scientific Sessions 2025- -Phase 1 clinical data for CTX310® demonstrate robust, dose-dependent reductions in circulating ANGPTL3 with a mean reduction from baseline of -73% (maximum -89%), a mean reduction in
Tenaya Therapeutics Presents Promising Interim Clinical Data from MYPEAK™-1 Phase 1b/2a Clinical Trial of TN-201 Gene Therapy for the Treatment of MYBPC3-Associated Hypertrophic Cardiomyopathy
Tenaya Therapeutics
2025-11-08
gene therapy
cardiomyopathy
MyPEAK-1 Data Presented During Late-Breaking Session at AHA Scientific Sessions 2025 with Simultaneous Publication in Cardiovascular Research TN-201 Has Been Generally Well Tolerated at Both Doses Longer-term Follow Up of Cohort 1 Patients Showed Consistent, Deeper, and Durable Improvement in
ACAAI: Sanofi and Regeneron’s Dupixent pivotal study met all primary and secondary endpoints, reducing signs and symptoms of allergic fungal rhinosinusitis; sBLA accepted for FDA priority review
sanofi
2025-11-07
pivotal
priority review
Cullinan Therapeutics Provides Corporate Update and Reports Third Quarter 2025 Financial Results
Cullinan Therapeutics
2025-11-07
financial results
Abeona Therapeutics® Announces Date of Third Quarter Financial Results and Upcoming Investor Conference Participation
Abeona Therapeutics
2025-11-07
financial results
Gilead’s breast cancer drug fails to meet main goal of late-stage study
Gilead Sciences
2025-11-07
first-line
(Reuters) -Gilead Sciences said on Friday its breast cancer drug Trodelvy did not significantly lower the risk of disease progression in patients when used as a first-line treatment, failing to meet the main goal of a late-stage study. Gilead said an e...
PacBio Supports Berry Genomics in Achieving First Regulatory Approval for Clinical Long-Read Sequencing in China
GenEng News
2025-11-07
clearance
PacBio said that China’s approval marks the world’s first regulatory clearance of a clinical-grade long-read sequencer that signals a new era for precision medicine and high-accuracy genomic testing in China. The post PacBio Supports Berry Genomics in Achieving First Regulatory Approval for Clinical Long-Read Sequencing in China appeared first on GEN - Genetic Engineering and Biotechnology News .
GSK presents data from its advancing liver pipeline at AASLD 2025
GSK
2025-11-07
pipeline
21 abstracts highlight advances in the treatment of liver conditions, building from GSK’s expertise in inflammation and fibrosis
Intellia shares fall after hospitalised CRISPR patient dies
PharmaPhorum
2025-11-07
crispr
Shares in gene-editing specialist Intellia Therapeutics came under renewed pressure today after a patient in one of its clinical trials died.
Resolution Therapeutics announces new data showcasing the potential of Regenerative Macrophage Therapy in treating cirrhosis at AASLD 2025
syncona
2025-11-07
macrophage therapy
FDA speeds development of oral pancreatic cancer treatment
Rare Cancer News
2025-11-07
orphan drug
pancreatic cancer
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to daraxonrasib, an oral therapy being developed by Revolution Medicines to treat pancreatic cancer. The FDA gives this designation to experimental medicines designed to treat conditions affecting fewer than 200,000 people in the U.S. The goal is to provide additional economic incentives to […] The post FDA speeds development of oral pancreatic cancer treatment appeared first on Rare Cancer News .
Engineered Bone Marrow Model Advances CRISPR-CAR T Research in AML
GenEng News
2025-11-07
aml
car t
crispr
cell therapy
acute myeloid leukemia
Scientists developed a bioengineered bone marrow model incorporating human cells in a synthetic peptide hydrogel, which helped them to evaluate potential use of a combined CRISPR-CAR T cell therapy in the treatment of acute myeloid leukemia. The post Engineered Bone Marrow Model Advances CRISPR-CAR T Research in AML appeared first on GEN - Genetic Engineering and Biotechnology News .
Uniqure NV expected to post a loss of 90 cents a share
uniQure
2025-11-07
quarterly
Previous quarterly performance (using preferred earnings measure in US dollars). This summary was machine generated November 7 at 15:28 GMT. All figures in US dollars unless otherwise stated. (For questions concerning the data in this report, contact Estimates.Support@lseg.com. For any other ques…
Gilead's breast cancer drug fails to meet main goal of late-stage study
Gilead Sciences
2025-11-07
first-line
Gilead Sciences NASDAQ:GILD said on Friday its breast cancer drug Trodelvy did not significantly lower the risk of disease progression in patients when used as a first-line treatment, failing to meet the main goal of a late-stage study.Gilead said an early trend for extending overall survival, a ke…
Gilead Provides Update on Phase 3 ASCENT-07 Study
Gilead Sciences
2025-11-07
phase 3
Gilead Sciences, Inc. (Nasdaq: GILD) today announced the Phase 3 ASCENT-07 study investigating Trodelvy® (sacituzumab govitecan-hziy) versus chemotherapy as ...
Gilead Trodelvy trial fails in breast cancer (GILD:NASDAQ)
Gilead Sciences
2025-11-07
first-line
Gilead Sciences (GILD) stock is in focus as the company fails in a late-stage trial which tested its Trodelvy cancer drug as a first-line therapy for breast cancer. Read more here.
MedCity FemFwd: Inside Wellcome Leap and Pivotal’s $100M Commitment To Women’s Health
MedCity News
2025-11-07
pivotal
In this episode, we’re joined by Dr. Renee Wittemyer, vice president of program strategy at Pivotal Ventures, and Dr. Regina Dugan, CEO of Wellcome Leap. They discuss the organizations’ recent $100 million commitment to accelerate women’s health research. The post MedCity FemFwd: Inside Wellcome Leap and Pivotal’s $100M Commitment To Women’s Health appeared first on MedCity News .
Patients Treated With Spur Therapeutics’ Gaucher Disease Gene Therapy FLT201 Maintain Clinical Benefit for Up to 2 Years
CGTLive
2025-11-07
gene therapy
Gilead Sciences (GILD) Phase 3 Trial for Trodelvy Falls Short
Gilead Sciences
2025-11-07
phase 3
Key Takeaways: Gilead Sciences' Phase 3 trial for Trodelvy did not meet its primary endpoint in a specific breast cancer subtype. The company maintains a
J&J Wins Expansion for Top-Selling Cancer Drug in First for Smoldering Multiple Myeloma
BioSpace
2025-11-07
multiple myeloma
Darzalex Faspro’s approval for smoldering multiple myeloma could allow for earlier intervention and reduce the risk of progression to active disease.
Cellectis Reports Third Quarter 2025 Financial Results and Provides Business Update
cellectis
2025-11-07
aav
bla
orr
til
b-all
car-t
cr rate
phase 1
phase 2
pivotal
melanoma
pipeline
allogeneic
first line
second line
base editing
cell therapy
gene editing
gene therapy
gene therapies
complete response
overall response rate
b-cell acute lymphoblastic leukemia
Presented data underscore the potential of lasme-cel (UCART22) and eti-cel (UCART20x22) to improve outcomes in r/r B-ALL and r/r NHL: Lasme-cel in r/r B-ALL (BALLI-01) ORR of 68% with lasme-cel Process 2 (n=22), 83% at RP2D (n=12) and 100% in the target Phase 2 population (n=9) Median OS of 14.8 months in patients who achieved MRD-negative CR/CRi First interim analysis for the BALLI-01 trial expected in Q4 2026 Eti-cel in r/r NHL (NATHALI-01) ORR of 86% and 57% CR rate (n=7) Development update to be presented at the ASH 2025 annual meeting Full Phase 1 dataset expected to be shared in 2026 Servier arbitration: arbitral decision expected to be rendered on or before December 15, 2025 Cash, cash equivalents and fixed-term deposits of $225 million as of September 30, 2025 [1] provides runway into H2 2027 New York, NY – November 7, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, today provided financial results for the third quarter 2025 ending September 30, 2025 and business updates. “We are proud of the promising data from our…
FDA Approves Donislecel (Lantidra), an Allogeneic Cellular Therapy, for Type 1 Diabetes
CellTrans Inc.
2025-11-07
lantidra
allogeneic
Announced on June 28, 2023, the FDA's approval of donislecel (Lantidra) marks the first approval of an allogeneic pancreatic islet cellular therapy for the treatment of type 1 diabetes.
A2 Biotherapeutics Presents Initial Safety and Efficacy Data from Ongoing Phase 1/2 EVEREST-2 Study, Including First Report of a Complete Response to CAR T-Cell Therapy in a Patient with NSCLC
A2 Bio
2025-11-07
car t
phase 1
phase 1/2
cell therapy
complete response
The post A2 Biotherapeutics Presents Initial Safety and Efficacy Data from Ongoing Phase 1/2 EVEREST-2 Study, Including First Report of a Complete Response to CAR T-Cell Therapy in a Patient with NSCLC appeared first on A2 Biotherapeutics .
Biomarin plans to sell rights to hemophilia A therapy Roctavian
BioMarin Pharmaceutical
2025-11-07
roctavian
gene therapy
hemophilia a
Biomarin Pharmaceutical is looking to sell off rights to Roctavian, its approved gene therapy for hemophilia A.
Nurix Therapeutics Presents New Translational Data from First-in-Human Clinical Trial of Oral CBL-B Inhibitor NX-1607 Demonstrating Immune Activation and Tumor Microenvironment Remodeling
nurix therapeutics
2025-11-07
first-in-human
Data are being presented at the 2025 Society for Immunotherapy of Cancer (SITC) Annual Meeting SAN FRANCISCO, Nov. 07, 2025 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company developing targeted protein modulation therapies to treat cancer and
Prime Medicine Reports Third Quarter 2025 Financial Results and Provides Business Updates
prime medicine
2025-11-07
cta
ind
alpha-1 antitrypsin deficiency
New preclinical data for PM577 in Wilson’s Disease (WD) to be presented at AASLD; on-track to file IND and/or CTA in H1’26, with initial clinical data expected in 2027 -- -- Nominated PM647 as development candidate for Alpha-1 Antitrypsin Deficiency (AATD); on-track to file IND and/or CTA in
Gilead's Trodelvy stumbles in first-line breast cancer trial, adding to shaky clinical track record
Gilead Sciences
2025-11-07
first-line
Becoming a blockbuster in its third full year on the market put Gilead’s cancer treatment Trodelvy on a course to potentially justify the company’s $21 billion
Sarepta Therapeutics’ Limb-Girdle Muscular Dystrophy Gene Therapy SRP-9003 Produces “Robust Expression” of β-SG in Phase 3 Trial
CGTLive
2025-11-07
phase 3
sarepta
srp-9003
gene therapy
The primary end point of the study was met.
uniQure Staggers at FDA, Recursion’s Microglia Map, Leadership Transitions
GenEng News
2025-11-07
gene therapy
In this episode of GEN 's Touching Base , we discuss news about uniQure’s gene therapy candidate for Huntington’s disease and some updates from Recursion and Roche. We also discuss drug discovery improvements using AI. The post uniQure Staggers at FDA, Recursion’s Microglia Map, Leadership Transitions appeared first on GEN - Genetic Engineering and Biotechnology News .
Patient Dies After Receiving Intellia's CRISPR Gene Therapy Candidate
BioSpace
2025-11-07
crispr
gene therapy
The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred. Intellia is working with experts to create a risk management program for nex-z.
Gilead's breast cancer drug fails to meet main goal of late-stage study
Gilead Sciences
2025-11-07
first-line
Gilead Sciences said on Friday its drug Trodelvy did not significantly improve progression-free survival in patients with the most common type of breast cancer when used as a first-line treatment, failing to meet the main goal of a late-stage study.
Novel LNP Delivers Influenza mRNA Vaccine at 100-Fold Lower Dose
GenEng News
2025-11-07
lnp
For today, World Immunization Day, we highlight the development of a new LNP that delivers mRNA vaccines 100 times more efficiently, boosting immune response, reducing toxicity, and potentially cutting vaccine production costs significantly. The post Novel LNP Delivers Influenza mRNA Vaccine at 100-Fold Lower Dose appeared first on GEN - Genetic Engineering and Biotechnology News .
VIDEO: Aflibercept 8 mg shows benefit in treatment-naive, prior-treated wet AMD patients
Healio
2025-11-07
wet age-related macular degeneration
ORLANDO — In this video from the American Academy of Ophthalmology meeting, Esther M. Bowie, MD, discusses real-world data from the IRIS Registry on aflibercept 8 mg in wet age-related macular degeneration.Bowie, professor in the department of ophthalmology at Penn State Health, said that patients who were treatment-naive or previously treated with intravitreal injections did well. Patients
MCO-010 shows long-term safety, quality of life improvements in retinitis pigmentosa
Healio
2025-11-07
mco-010
retinitis pigmentosa
MCO-010, an optogenetic therapy, demonstrated long-term safety and improved quality of life in patients with retinitis pigmentosa after a single intravitreal injection, according to a press release from Nanoscope Therapeutics. “Patients with RP suffer from a progressive decline in vision following the natural course of disease progression, with no treatment available to halt or even slow the
Investigational phosphate-binding agent lowers pill size, number
Healio
2025-11-06
pivotal
HOUSTON — Patients with hyperphosphatemia taking an investigational phosphate-binding agent achieved clinical targets with lower pill burden, according to pivotal trial data presented at ASN Kidney Week. The medication, oxylanthanum carbonate (OCL; Unicycive Therapeutics), is “a next-generation lanthanum-based phosphate-binding agent utilizing proprietary nanoparticle technology being developed
Celldex Presents Results from Barzolvolimab Phase 2 Study in Cold Urticaria (ColdU) and Symptomatic Dermographism (SD) Demonstrating Sustained Efficacy and Favorable Safety Profile over 20 Week Placebo Controlled Treatment Period; Up to 66% of Patients with ColdU and 49% with SD Obtain Complete Response at Week 20Download PDF
Celldex Therapeutics
2025-11-06
complete response
First large, randomized, placebo-controlled study to demonstrate clinical benefit in patients with Cold Urticaria (ColdU) and Symptomatic Dermographism (SD) All primary and secondary endpoints met with high statistical significance at 12 weeks and sustained through end of treatment period (20 Download PDF
Rocket Pharmaceuticals Reports Third Quarter 2025 Financial Results and Highlights Recent Progress
Rocket Pharmaceuticals
2025-11-06
pdufa
phase 2
pivotal
kresladi
Pivotal Phase 2 trial of RP-A501 for Danon disease to resume in 1H 2026 KRESLADI™ for severe LAD-I on track for March 28, 2026 PDUFA date Leadership updates with the appointments of Dr. Syed Rizvi , Chief Medical Officer, Christopher Stevens , Chief Operating Officer, and Sarbani Chaudhuri , Chief
Opus Genetics Announces Successful FDA Meeting Supporting Advancement of OPGx-LCA5 Toward Pivotal Trial for LCA5-Related Inherited Retinal Disease
opus genetics
2025-11-06
pivotal
Adjuvant Ribociclib Offers Ongoing DFS Benefit in HR+/HER2- Early Breast Cancer
Hematology Advisor News
2025-11-06
her2
In HR+/HER- early breast cancer, the DFS benefits of adjuvant ribociclib plus NSAI treatment compared to NSAI alone persisted even a year after treatment completion.
Braveheart Bio Bags $185M for Drug That Could Rival a Bristol Myers Squibb Cardio Med
MedCity News
2025-11-06
phase 3
Braveheart Bio is preparing to advance to Phase 3 testing in obstructive hypertrophic cardiomyopathy, or oHCM. The startup says its small molecule could have safety advantages over Camzyos, a Bristol Myers Squibb capsule projected to become a blockbuster seller in this indication. The post Braveheart Bio Bags $185M for Drug That Could Rival a Bristol Myers Squibb Cardio Med appeared first on MedCity News .
Oral safusidenib begins trial as maintenance for high-grade glioma
Rare Cancer News
2025-11-06
glioma
Enrollment has begun in the second part of a clinical trial testing safusidenib, an experimental oral treatment, as a maintenance therapy for high-grade IDH1-mutant astrocytoma, an aggressive form of glioma. The study (NCT05303519) — sponsored by safusidenib’s developer, Nuvation Bio — aims to enroll 300 adults at sites in the U.S., Australia, and China. Participants must […] The post Oral safusidenib begins trial as maintenance for high-grade glioma appeared first on Rare Cancer News .
Novel amylin receptor agonist induces ‘clinically meaningful’ weight loss
Healio
2025-11-06
phase 2
ATLANTA — An investigational once-weekly amylin receptor agonist conferred substantial weight loss across six different dosing regimens for adults with overweight or obesity, researchers reported in a phase 2 trial.Eloralintide (Eli Lilly) is a selective, long-acting amylin receptor agonist designed to reduce body weight for adults with obesity. In a phase 2 trial presented at ObesityWeek
VIVET THERAPEUTICS ATTENDING JEFFERIES 2025 – LONDON HEALTHCARE CONFERENCE
Vivet Therapeutics
2025-11-06
aav
Vivet Therapeutics is pleased to announce that our CEO, Jean-Philippe Combal, will be attending the Jefferies Global Healthcare Conference in London from November 18th to 20th, 2025. As we join this leading global event, we are excited to present our latest advances, including the VTX-PID NAb depleting strategy. VTX-PID is designed to temporarily reduce anti-AAV […] The post VIVET THERAPEUTICS ATTENDING JEFFERIES 2025 – LONDON HEALTHCARE CONFERENCE appeared first on Vivet Therapeutics .
Wegovy, Casgevy Among Latest FDA Priority Review Voucher Recipients
BioSpace
2025-11-06
casgevy
priority review
The FDA awards a second round of Commissioner’s National Priority vouchers to six larger biopharma companies. And this time, with the exception of Eli Lilly’s orforglipron, the vouchers are for drugs that are already on the market.
Lisata Therapeutics Reports Third Quarter 2025 Financial Results and Provides Business Update
Lisata Therapeutics
2025-11-06
financial results
Data rich period continues to support certepetide’s broad applicability and effectiveness Catalent enters into global license agreement for the use of certepetide as part of their SMARTag® Antibody-Drug Conjugate Technology Platform Strategic Alliance formed with GATC Health to exploit their
Recurrent MDS Can Manifest as Orbital Lesion Before Other Signs of Malignancy
Hematology Advisor News
2025-11-06
aml
Initial biopsies showed only benign fibrous tissue and no signs of malignancy, but further testing after 3 months revealed clinical signs that confirmed MDS recurrence with AML transformation.
AZ's Q3 sales reach record $15bn, but it holds guidance
PharmaPhorum
2025-11-06
quarterly
AstraZeneca takes another step forwards towards its target of growing revenues to $80bn by 2030, as quarterly sales top $15bn for the first time.
ProQR Announces Third Quarter 2025 Operating and Financial Results
proqr therapeutics
2025-11-06
cta
phase 1
Received CTA authorization for Phase 1 trial of lead program AX-0810, targeting NTCP for cholestatic diseases Initiating Phase 1 study of AX-0810 in healthy volunteers, with initial safety, tolerability, and PK data from Cohort 1 expected by year-end, and target engagement data from all cohorts in H1 2026 € 106.9 million cash and cash equivalents as of end Q3 – providing runway into mid-2027 LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov. 06, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the third quarter ended September 30, 2025, and provided a business update.
Optimized Sequencing Advances Care for HR+/HER2+ Breast Cancer
Hematology Advisor News
2025-11-06
her2
Researchers present an overview of treatment and management strategies for patients with HR+/HER2+ breast cancer, including common adverse effects.
Lineage Cell Therapeutics Reports Third Quarter 2025 Financial Results and Provides Business Update
lineage cell therapeutics
2025-11-06
phase 1
cell therapy
Positive RG6501 (OpRegen ® ) Phase 1/2a Clinical Study 36 Month Results Featured at Clinical Trials at the Summit 2025 Entered Collaboration with William Demant Invest to Develop ReSonance™ (ANP1) for Sensorineural Hearing Loss Initiated Manufacturing Scale Project in Type 1 Diabetes Cell Therapy
Humacyte to Present Third Quarter Financial Results and Provide Business Update on November 12, 2025
Humacyte
2025-11-06
financial results
DURHAM, N.C. , Nov. 06, 2025 (GLOBE NEWSWIRE) -- Humacyte, Inc. (Nasdaq: HUMA), a clinical-stage biotechnology platform company developing universally implantable, bioengineered human tissue at commercial scale, will release its financial results for the quarter ended September 30, 2025 , on
Dianthus Therapeutics Highlights Recent Business Achievements and Reports Q3 Financial ResultsPDF VersionRead More
Dianthus Therapeutics
2025-11-06
myasthenia gravis
Claseprubart achieved statistically significant and clinically meaningful improvements in Myasthenia Gravis Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG), and other efficacy measures at Week 13 in Phase 2 MaGic trial in gMG New claseprubart data from the MaGic open-label PDF Version Read More
Low Placental Growth Factor Predicts Early-Onset Preeclampsia in Patients With SCD
Hematology Advisor News
2025-11-06
scd
PlGF cutoff
MiNK Therapeutics to Report Third Quarter 2025 Financial Results and Highlight Clinical and Corporate Milestones Advancing iNKT Platform Toward Pivotal Development
Mink Therapeutics
2025-11-06
pivotal
allogeneic
cell therapies
financial results
NEW YORK , Nov. 05, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics , Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering allogeneic invariant natural killer T (iNKT) cell therapies to treat cancer and immune disorders, today announced that the Company will release its third
Why Krystal Biotech (KRYS) Is Up 8.0% After Surging VYJUVEK Revenue and Global Expansion
Krystal Biotech
2025-11-06
vyjuvek
gene therapy
Krystal Biotech, Inc. recently reported third-quarter 2025 earnings, revealing net income of US$79.37 million and total revenue of US$97.8 million, reflecting substantial gains compared to the same period last year and continued global commercial expansion of its lead gene therapy, VYJUVEK. Key milestones included successful launches of VYJUVEK in Germany, France, and Japan, high US reimbursement approvals, and regulatory steps that broadened the eligible patient population, highlighting...
uniQure to Announce Third Quarter 2025 Financial Results
uniQure
2025-11-06
gene therapy
uniQure to host earnings call on Monday, November 10, 2025 at 8:30 a.m. ET ~LEXINGTON, Mass. and AMSTERDAM, Nov. 06, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report third quarter 2025 financial results before market open on Monday, November 10, 2025. Management will then host a conference call at 8:30 a.m. ET. The event will be webcast under the Events & Presentations sect
Synthekine Presents Positive Initial Results from Phase 1a/1b Clinical Trial of STK-012 Plus Pembrolizumab and Chemotherapy in First-Line, PD-L1 Negative Nonsquamous Non-Small Cell Lung Cancer
Synthekine
2025-11-06
lung cancer
November 7, 2025 The post Synthekine Presents Positive Initial Results from Phase 1a/1b Clinical Trial of STK-012 Plus Pembrolizumab and Chemotherapy in First-Line, PD-L1 Negative Nonsquamous Non-Small Cell Lung Cancer first appeared on Synthekine .
VIDEO: Extension studies shed light on wet AMD treatments
Healio
2025-11-06
wet age-related macular degeneration
ORLANDO — In this video from the American Academy of Ophthalmology meeting, Esther M. Bowie, MD, discusses results from the AVONELLE-X trial, the PULSAR trial and the Portal study in wet age-related macular degeneration.In AVONELLE-X, patients with wet AMD who received Vabysmo (faricimab-svoa, Genentech) were extended from 2 years to 4 years, with approximately 80% of patients extending
Has uniQure’s Volatile 63% Drop Created a Compelling Opportunity After Pipeline News?
uniQure
2025-11-06
gene therapy
Wondering if uniQure’s rollercoaster stock price means it’s a bargain or a trap? Let’s dig into what’s really driving its value. uniQure shares have seen wild swings lately, dropping 63.1% in just the past week, but are still up an eye-catching 264.0% over the last year. This highlights both volatility and dramatic growth potential. Much of this action has been fueled by recent headlines about regulatory milestones for its gene therapy pipeline and an announced partnership set to expand its...
US FDA approves injectable version of Roche's multiple sclerosis therapy
Roche Group
2025-11-06
multiple sclerosis
The U.S. Food and Drug Administration on Friday approved Roche's under-the-skin injection to treat patients with multiple sclerosis.
Lexeo Therapeutics Reports Third Quarter 2025 Financial Results and Operational Highlights
Lexeo Therapeutics
2025-11-06
financial results
Adicet Bio Reports Third Quarter 2025 Financial Results and Provides Business Updates
Adicet Bio
2025-11-06
phase 1
Announced positive preliminary safety and efficacy data from Phase 1 clinical trial of ADI-001 in patients with lupus nephritis (LN) and systemic lupus erythematosus (SLE); data highlighted rapid and sustained reductions in Systemic Lupus Erythematosus Disease Activity Index (SLEDAI-2K) score and
Iovance Biotherapeutics Highlights Business Achievements, Pipeline Milestones, and Third Quarter 2025 Results
Iovance Biotherapeutics
2025-11-06
pipeline
quarterly
Quarterly Revenue Growth of 13% to ~$68 Million Gross Margin Increased to 43% on Improved Execution and Operational Efficiency Best-in-Class Clinical Profile for Lifileucel in Previously Treated Advanced Non-Small Cell Lung Cancer (NSCLC) with Median Duration of Response Not Reached after 25+
Intellia Therapeutics Announces Third Quarter 2025 Financial Results and Recent Updates
Intellia Therapeutics Inc.
2025-11-06
phase 1
clinical hold
Awaiting FDA clinical hold letter on MAGNITUDE and MAGNITUDE-2 clinical trials of nex-z Presenting longer-term Phase 1 clinical data of nex-z for ATTR-CM on November 10 at AHA 2025; previously published longer-term Phase 1 clinical data for nex-z for ATTRv-PN in New England Journal of Medicine
FDA Faces Scrutiny as Biohaven, UniQure, and Sarepta Therapeutics Encounter Drug Development Delays
uniQure
2025-11-06
sarepta
uniqure
sarepta therapeutics
Recent challenges faced by several pharmaceutical compa […]
Allogene Therapeutics Reports Third Quarter 2025 Financial Results and Business Update
Allogene
2025-11-06
phase 2
pivotal
first line
Pivotal Phase 2 ALPHA3 Trial with Cemacabtagene Ansegedleucel (Cema-Cel) in First Line (1L) Consolidation in Large B-Cell Lymphoma (LBCL) ALPHA3 Positions Company at the Forefront of MRD-guided, Earlier-Line Oncology Treatment Additional Sites in Australia and South Korea Expected to Open in early
Why Krystal Biotech (KRYS) Is Up 8.0% After Surging VYJUVEK Revenue and Global Expansion
Krystal Biotech
2025-11-06
vyjuvek
U.S. stocks, Saudi stocks, stock trading and investment platforms
Pfizer and Novo Battle Over Metsera, Tidmarsh Fights FDA Exit, UniQure and Sarepta Face Setbacks
uniQure
2025-11-05
sarepta
gene therapy
duchenne muscular dystrophy
Pfizer and Novo Nordisk continue to fight for ownership of obesity startup Metsera; CDER Director George Tidmarsh leaves his position amid an ongoing probe into his “personal conduct”; FDA reverses course on approval requirements for uniQure’s Huntington’s gene therapy; Sarepta’s exon-skipping Duchenne muscular dystrophy drugs fail confirmatory study.
FDA Issues Complete Response Letter for Biohaven's VYGLXIA (troriluzole) New Drug Application for Spinocerebellar Ataxia
uniQure
2025-11-05
complete response
The troriluzole clinical development program encompassed the first industry clinical trials to generate data showing therapeutic potential in patients with...
CG Oncology (CGON) Projected to Post Quarterly Earnings on Tuesday
CG Oncology
2025-11-05
quarterly
CG Oncology (NASDAQ:CGON) will be releasing its Q3 2025 earnings before the market opens on Tuesday, November 11. (View Earnings Report at https://www.marketbeat.com/earnings/reports/2025-11-11-cg-oncology-inc-common-stock-stock/)
VIDEO: Scoring tools may help identify nonresponders in primary biliary cholangitis
Healio
2025-11-05
first-line
Ahmad Anouti, MD, a pediatric resident at UT Southwestern Medical Center, spoke about the current treatment landscape for primary biliary cholangitis.In addition, he noted there has been an increased use of scoring to help identify patients who will not respond to ursodeoxycholic acid, the first-line therapy for PBC. With these scoring tools, physicians may determine early on if patients with PBC
Arcellx Provides Third Quarter 2025 Financial Results
arcellx, inc
2025-11-05
cell therapy
Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, today reported financial results for the third quarter ended September 30, 2025. Third Quarter 2025 Financial Highlights Cash, cash equivalents, and marketable securities: As of September 30, 2025, Arcellx had cash, cash equivalents, and marketable securities of $576.0 million. Arcellx anticipates that its cash, cash equivalents, and marketable securities will fund its operations into 2028. Collaboration revenue: Collaboration revenue was $4.9 million and $26.0 million for the quarters ended September 30, 2025 and 2024, respectively, a decrease of $21.1 million. This decrease was primarily driven by completion of dosing and manufacturing of anito-cel in the iMMagine-1 trial in the fourth quarter of 2024. RD expenses: Research and development expenses were $35.1 million and $39.2 million for the
Anixa Biosciences Announces Execution of Data Transfer Agreement with Cleveland Clinic
anixa biosciences
2025-11-05
ind
car-t
phase 1
phase 2
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
Completion of key step in IND transfer for upcoming Phase 2 breast cancer vaccine clinical trial SAN JOSE, Calif. , Nov. 5, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that it has executed a data transfer agreement ("DTA") with Cleveland Clinic, as a key step in the process of transferring the breast cancer vaccine clinical trial data and sponsorship to Anixa for future clinical development. With enrollment completed and encouraging immune response data observed in the Phase 1 trial, Anixa plans to advance the vaccine into a Phase 2 clinical trial and will assume full sponsorship of the IND. The IND, currently sponsored by Cleveland Clinic, is in the process of being transferred to Anixa. The DTA will allow for the transfer of all relevant data and information, collected and generated from the Phase 1 clinical trial, to Anixa. Anixa's breast cancer vaccine, developed in collaboration with Cleveland Clinic, targets α-lactalbumin—a lactation-associated protein that is typically expressed only in breast tissue during lactation, but which re-emerges in…
LENZ Therapeutics Reports Third Quarter 2025 Financial Results and Recent Corporate Highlights
LENZ Therapeutics
2025-11-05
financial results
UNIQURE ALERT: Bragar Eagel & Squire, P.C. is Investigating uniQure N.V. on Behalf of uniQure Stockholders and Encourages Investors to Contact the Firm
uniQure
2025-11-05
bla
amt-130
uniQure disclosed the FDA no longer agrees its AMT-130 trial data may support a BLA submission, making timing unclear. Shares fell over 57% on the news....
Multidisciplinary comprehensive obesity care model boosts weight loss, medication persistence
Healio
2025-11-05
persistence
ATLANTA — Participants in a multidisciplinary comprehensive obesity care model had clinically meaningful weight loss and high obesity medication persistence at 1 year, according to results of a real-world analysis presented at ObesityWeek.“Multidisciplinary obesity treatment models that integrate the pillars of obesity care — behavioral health, nutrition intervention, physical
Guess the Therapy Answer November 5, 2025
CGTLive
2025-11-05
gene therapy
Cell and Gene Therapy Live
Psychedelic merger to create AtaiBeckley completes
PharmaPhorum
2025-11-05
pipeline
A $390m merger of two pioneers of psychedelic medicines has completed to create AtaiBeckley, with a pipeline of candidates for mental illnesses.
Optimizing CAR T Manufacturing With Automation and Global Networks
GenEng News
2025-11-05
car t
As demand for precision medicine grows, CAR T and CAR Treg therapies are transforming cancer and autoimmune treatment. Automation, scalable manufacturing, and global partnerships are driving progress, making personalized medicine more accessible and commercially viable worldwide. The post Optimizing CAR T Manufacturing With Automation and Global Networks appeared first on GEN - Genetic Engineering and Biotechnology News .
Weak Spot of Metastatic Melanoma Cells Identified
Inside Precision Medicine
2025-11-05
melanoma
A protein involved in ferroptosis has been revealed as a unique weakness of metastatic melanoma cells that have spread to the lymph nodes. The post Weak Spot of Metastatic Melanoma Cells Identified appeared first on Inside Precision Medicine .
Around the Helix: Cell and Gene Therapy Company Updates – October 15, 2025
CGTLive
2025-11-05
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
04 Nov 2025 Abstract for CARsgen’s Allogeneic BCMA CAR-T Product CT0596 Available on ASH Website Nov 04, 2025
Carsgen Therapeutics
2025-11-04
bcma
car-t
allogeneic
FDA halts uniQure’s plans for Huntington’s disease gene therapy
uniQure
2025-11-04
gene therapy
The company’s stock has plummeted after news of regulatory uncertainty despite positive results.
Women’s health and obesity: Understanding the role of menopause and hormones
Healio
2025-11-04
pivotal
The menopausal transition is a pivotal time in women’s health, bringing about profound hormonal, metabolic and physiologic changes.For many women, this stage of life raises new concerns about weight regulation, cardiometabolic health and overall well-being. Understanding how menopause influences these factors is essential for both patients and providers.
Vertex’s New Products Dim as Analysts Eye Kidney Portfolio
BioSpace
2025-11-04
casgevy
gene therapy
The potential approval of Vertex’s IgAN therapy povetacicept in 2026 comes amid launch headwinds for the company’s non-opioid pain medicine Journavx and gene therapy Casgevy.
Krystal Biotech (NASDAQ:KRYS) Announces Quarterly Earnings Results
Krystal Biotech
2025-11-04
quarterly
Krystal Biotech (NASDAQ:KRYS - Get Free Report) announced its earnings results on Monday. The company reported $2.66 earnings per share (EPS) for the quarter, topping analysts' consensus estimates of $1.04 by $1.62. Krystal Biotech had a return on equity of 15.21% and a net margin of 40.85%.
MCO-010 demonstrates visual acuity gains in Stargardt disease at 48 weeks
Healio
2025-11-04
mco-010
ORLANDO — At 48 weeks, MCO-010, an optogenetic therapy, demonstrated gains in visual acuity in Stargardt disease, according to a study presented at the American Academy of Ophthalmology meeting. Kenneth C. Fan, MD, MBA, of Retina Consultants of Texas, told Healio that the treatment of Stargardt disease is a well-known unmet need in ophthalmology.
Hemostemix Closes Private Placement of $461,230
Hemostemix
2025-11-04
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta, November 4, 2025 – Hemostemix Inc. (" Hemostemix " or the " Company ") (TSXV: HEM) (OTC: HMTXF) (FSE: 2VF0), the leading autologous (patient's own) stem cell therapy company offering VesCell™ (ACP-01) to no-option individuals suffering from pain related to angina, peripheral arterial disease, chronic limb threatening ischemia, ischemic cardiomyopathy, non-ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, is pleased to announce it has closed its first tranche of its previously announced non-brokered private placement of units (the “ Offering ”), as originally disclosed on October 8, 2025. The Company raised aggregate gross proceeds of $461,230 through the issuance of 4,193,000 units (“ Units ”) at a price of $0.11 per Unit.
Tr1X's Multiple Sclerosis Cell Therapy TRX319 Cleared for US Trial
CGTLive
2025-11-04
cell therapy
Kura Oncology and Kyowa Kirin Announce Presentations of Ziftomenib Ven/Aza Combination Data in Frontline and Relapsed/Refractory NPM1-m or KMT2A-r Acute Myeloid Leukemia at 2025 ASH Annual Meeting
Kyowa Kirin Co.
2025-11-04
acute myeloid leukemia
Cryoport Reports Third Quarter 2025 Financial Results
Cryoport Inc
2025-11-04
financial results
Caribou’s CB-011 may be effective, safe for hard-to-treat myeloma
Rare Cancer News
2025-11-04
car t
phase 1
cell therapy
multiple myeloma
One-time use of CB-011, Caribou Biosciences’ off-the-shelf CAR T-cell therapy candidate, showed a manageable safety profile and resulted in lasting responses in adults with treatment-resistant or relapsed multiple myeloma. That’s according to the first data from the ongoing Phase 1 CaMMouflage clinical trial (NCT05722418), which is underway at 16 sites across the U.S. The data […] The post Caribou’s CB-011 may be effective, safe for hard-to-treat myeloma appeared first on Rare Cancer News .
Krystal Biotech price target raised to $220 from $216 at Chardan
Krystal Biotech
2025-11-04
vyjuvek
Chardan raised the firm’s price target on Krystal Biotech (KRYS) to $220 from $216 and keeps a Buy rating on the shares. The company’s Q3 net Vyjuvek revenue topped...
enGene (Nasdaq: ENGN) Named to BioSpace 2026 Best Places to Work; among 50 U.S. employers
enGene
2025-11-04
phase 2
BioSpace names enGene to 2026 Best Places to Work among 50 U.S. employers. enGene advances detalimogene in Phase 2 LEGEND for BCG‑unresponsive NMIBC with CIS.
Nanoscope Therapeutics Reports Five-Year Safety Results from Phase 1/2a Follow-Up Study of MCO-010 Optogenetic Therapy in Retinitis Pigmentosa
Nanoscope Therapeutics
2025-11-04
mco-010
phase 1
retinitis pigmentosa
DALLAS, Nov. 4, 2025 — Nanoscope Therapeutics Inc. announced today positive long-term safety results from its EXTEND study, a five-year follow-up of participants who received a single intravitreal injection of MCO-010 in an earlier Phase 1/2a trial. The EXTEND study followed ten participants with advanced retinitis pigmentosa (RP) who had previously received MCO-010, an optogenetic therapy designed to restore […] The post Nanoscope Therapeutics Reports Five-Year Safety Results from Phase 1/2a Follow-Up Study of MCO-010 Optogenetic Therapy in Retinitis Pigmentosa appeared first on Nanoscope Therapeutics .
FDA Priority Review for PALYNZIQ Adolescent Expansion Could Be a Game Changer for BioMarin (BMRN)
BioMarin Pharmaceutical
2025-11-04
priority review
BioMarin Pharmaceutical recently announced that the FDA has accepted its supplemental Biologics License Application for Priority Review to expand PALYNZIQ® treatment to adolescents aged 12–17 with phenylketonuria (PKU), setting a target action date for February 28, 2026. This regulatory milestone not only demonstrates ongoing progress in BioMarin's therapy pipeline but also signals potential growth in its rare disease treatment market. We'll look at how PALYNZIQ's regulatory advancement...
New orphan drug, CSL’s garadacimab, cleared for hereditary angioedema
CSL Behring
2025-11-04
orphan drug
complete response
hereditary angioedema
Following a complete response letter issued last October over CMC issues, CSL Behring LLC gained U.S. FDA approval June 16 of its humanized anti-factor XIIa monoclonal antibody, garadacimab (CSL-312), to prevent hereditary angioedema attacks.
Support available for veterans’ unique respiratory challenges
Healio
2025-11-04
msc
CHICAGO — Veterans of the U.S. military face unique respiratory risks, but physicians can guide them through care with the aid of the Department of Veterans Affairs, according to a presentation at the CHEST Annual Meeting.“The key here, when caring for a veteran, is that they have different exposures than what you may be used to seeing in your office,” Laura J. Spece, MD, MSc,
Pretreatment Immune Composition Influences CAR-T Efficacy in DLBCL
Hematology Advisor News
2025-11-04
car-t
dlbcl
All patients with detectable circulating B cells prior to treatment achieved complete remission.
Actinium Pharmaceuticals, Inc. to Highlight ATNM-400 Data in Hormone-Resistant and HER2-Resistant Breast Cancer at the 2025 San Antonio Breast Cancer Symposium, Expanding Pan-Tumor Profile Across Three Solid Tumor Indications
Actinium Pharma
2025-11-04
aml
cd33
her2
psma
phase 2
pivotal
phase 2/3
quarterly
first-line
second-line
solid tumor
gene therapies
acute myeloid leukemia
New preclinical data to show anti-tumor activity of ATNM-400 in hormone-resistant and HER2-resistant breast cancer, addressing the need for new treatment options beyond tamoxifen and trastuzumab - ATNM-400 demonstrates efficacy across prostate cancer, non-small cell lung cancer (NSCLC), and breast cancer - three of the largest oncology indications - with potential as monotherapy, in combination, or as an alternative therapy NEW YORK , Nov. 4, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE American: ATNM) ("Actinium" or the "Company"), a leader in the development of differentiated targeted radiotherapies, announced today that new preclinical data for its lead antibody radioconjugate program, ATNM‑400, will be presented at the 2025 San Antonio Breast Cancer Symposium (SABCS) taking place December 10‑14, 2025 in San Antonio, Texas. Title : Anti‑tumor activity of ATNM‑400, a first‑in‑class Actinium‑225 antibody radioconjugate, in tamoxifen and trastuzumab resistant breast cancer models Abstract Number : 2069 Presentation Number : PS4‑04‑26 Date/Time : Thursday, December 11, 2025, 5:00 PM–6:30 PM CT Session : Poster Session 4 The ATNM-400 data presentation at SABCS follows…
Genprex Announces European Patent Office’s Intent to Grant a Patent for the Combination of Reqorsa® Gene Therapy and PD-1 Antibodies to Treat Cancer
Genprex
2025-11-04
gene therapy
Adds to the Previously Granted Patents Covering the Use of REQORSA in Combination with Immune Checkpoint Inhibitors Strengthens Patent Estate and Provides Additional Protection in Large Market AUSTIN, Texas — (Nov. 4, 2025) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, […]
Cullinan Therapeutics to Showcase New Data Demonstrating Compelling Clinical Activity for CLN-049, a Novel FLT3xCD3 T Cell Engager, in AML Patients in an Oral Presentation at the 67th ASH Annual Meeting
Cullinan Therapeutics
2025-11-04
aml
Sarepta shares slide as DMD treatment woes pile up
PharmaPhorum
2025-11-04
dmd
sarepta
The ESSENCE trial of Sarepta's Vyondys 53 and Amondys 45 failed to hit its objective, placing their FDA accelerated approvals at risk.
Lisata Therapeutics to Report Second Quarter 2025 Financial Results and Provide a Business Update on Thursday, August 7, 2025
Lisata Therapeutics
2025-11-04
financial results
BASKING RIDGE, N.J., July 31, 2025 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the
Beam Therapeutics Reports Third Quarter 2025 Financial Results and Recent Business Updates
beam therapeutics
2025-11-04
phase 1
phase 1/2
dose escalation
sickle cell disease
Expanded Dose Exploration in Part A and Dose Escalation in Part B of BEAM-302 Phase 1/2 Study in Alpha-1 Antitrypsin Deficiency Ongoing; Updated Data and Clinical Development Update Expected in Early 2026 Updated Data from BEACON Phase 1/2 Trial of BEAM-101 in Sickle Cell Disease Accepted for
Fate Therapeutics Reports New Employee Inducement Awards Under Nasdaq Listing Rule 5635(c)(4)
Fate Therapeutics
2025-11-04
pipeline
SAN DIEGO , Nov. 04, 2025 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases,
New treatments worsened income-based survival disparities for lung cancer, melanoma
Healio
2025-11-04
melanoma
Treatment innovations in advanced lung cancer and melanoma over the past 2 decades have significantly improved OS, but they also have worsened survival disparities between high- and low-income communities.Survival disparities based on income increased more than 2 percentage points in lung cancer and 6 percentage points in melanoma in the years after anti-PD-L1 immunotherapies garnered FDA
Despite Phase 3 Failure, Sarepta Still Sees Path for Muscle Disease Drugs to Secure Full FDA Approval
MedCity News
2025-11-04
phase 3
sarepta
duchenne muscular dystrophy
Sarepta Therapeutics attributed the Phase 3 failure to the Covid-19 pandemic, during which many patients missed multiple doses of its Duchenne muscular dystrophy therapies. The company plans to discuss with the FDA traditional regulatory approvals based on the totality of data that includes real world evidence from the years these drugs have been commercially available under accelerated approvals. The post Despite Phase 3 Failure, Sarepta Still Sees Path for Muscle Disease Drugs to Secure Full FDA Approval appeared first on MedCity News .
UniQure: Controversy Abounds As Stock Price Halves On FDA News (NASDAQ:QURE)
uniQure
2025-11-04
amt-130
accelerated approval
UniQure shares soared on positive AMT-130 Huntington's disease data, but plunged after the FDA reversed its stance on accelerated approval. Click to read.
Sarepta’s DMD Exon-Skippers Fail Confirmatory Study, Stock Craters
BioSpace
2025-11-04
dmd
sarepta
Sarepta nevertheless plans to push for full FDA approval of Vyondys 53 and Amondys 45 based on what it said are “encouraging trends” in efficacy.
BrainSTEM Atlas Maps the Developing Brain, Revealing Clues to Parkinson’s
GenEng News
2025-11-04
cell therapies
A detailed single-cell atlas of the developing human brain highlights how midbrain neurons form and evolve, offering a new benchmark for Parkinson’s disease models and potential cell therapies. The post BrainSTEM Atlas Maps the Developing Brain, Revealing Clues to Parkinson’s appeared first on GEN - Genetic Engineering and Biotechnology News .
New Novartis data at ASN Kidney Week and AHA Scientific Sessions demonstrate momentum of broad CRM portfolio and pipeline
Novartis
2025-11-04
pipeline
Is Now the Moment to Reassess uniQure After Its 516% Surge and Sharp Drop?
uniQure
2025-11-04
gene therapy
Wondering if uniQure is trading at a bargain or riding a speculative wave? You are not alone. Getting to the bottom of a stock's value is one of the most important things investors can do. The stock’s recent rollercoaster is hard to ignore, with a 90.0% gain year-to-date, 516.7% return over the last 12 months, but also a sharp 49.5% drop in just the past week. uniQure has been in the spotlight thanks to its advancements in gene therapy and shifts within the biotech sector overall, both of...
Once-Weekly Marstacimab Reduces Bleeding Rates and Is Well Tolerated in Severe Hemophilia A and B
Hematology Advisor News
2025-11-04
hemophilia a
hemophilia b
Marstacimab significantly reduced bleeding rates and was well tolerated in males with severe hemophilia A or moderately severe to severe hemophilia B without inhibitors.
Taysha Gene Therapies Reports Third Quarter 2025 Financial Results and Provides Corporate Update
Taysha Gene Therapies
2025-11-04
bla
pivotal
phase 1/2
gene therapies
financial results
breakthrough therapy
TSHA-102 granted Breakthrough Therapy designation by FDA Finalized FDA alignment on REVEAL pivotal trial protocol and SAP, including 6-month interim analysis that may expedite BLA submission, which was enabled by the rigorous developmental milestone evaluation in Part A REVEAL Phase 1/2 trials
Tenaya Therapeutics Announces New Clinical Data for TN-201 Gene Therapy will be Featured in Late-Breaking Presentation at the American Heart Association Scientific Sessions 2025
Tenaya Therapeutics
2025-11-04
gene therapy
cardiomyopathy
Oral Presentation on Saturday to Highlight Interim Safety and Efficacy Results from the MyPEAK ™ -1 Phase 1b/2a Clinical Trial in Adults with MYBPC3-Associated Hypertrophic Cardiomyopathy Second Late-Breaking Presentation on Sunday to Showcase Results of Cellular Reprogramming Gene Therapy
Revuforj Gains Approval for R/R NPM1 Mutated AML
Hematology Advisor News
2025-11-04
aml
Among 65 evaluable participants with NPM1 mutated AML, the rate of complete remission (CR) plus CR with partial hematological recovery was 23.1%.
Ultragenyx Reports Third Quarter 2025 Financial Results and Corporate Update—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-11-04
financial results
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Positive Outlook for Krystal Biotech: Growth Potential Driven by Vyjuvek, Clinical Pipeline, and Global Expansion
Krystal Biotech
2025-11-04
vyjuvek
William Blair analyst Sami Corwin has maintained their bullish stance on KRYS stock, giving a Buy rating yesterday. Sami Corwin has given his Buy rating due to a co...
HIV, Livdelzi Fuel GILD's Q3 Top Line Amid Cell Therapy Sales Decline
Gilead Sciences
2025-11-04
cell therapy
Gilead Sciences, Inc.’s GILD posted 3% year-over-year growth in total revenues for the third quarter of 2025.Total revenues comprise product sales and royalty, contract and other revenues.While higher royalty, contract and other revenues propelled the growth in revenues, GILD’s HIV franchise and li…
A Closer Look at uniQure (QURE) Valuation Following FDA Shift on Huntington’s Gene Therapy Data
uniQure
2025-11-04
amt-130
gene therapy
uniQure (QURE) shares have taken a hit after the FDA withdrew support for using Phase I/II data from its AMT-130 Huntington's disease gene therapy in a key regulatory submission. This unexpected shift has jolted investor sentiment and increased scrutiny. See our latest analysis for uniQure. uniQure’s share price has swung dramatically alongside this regulatory surprise, plunging 56.5% in the past week and 44.6% over the last month. Despite this volatility, momentum for the stock had been...
Positive phase III data for Roche’s Gazyva/Gazyvaro show significant reduction in disease activity for systemic lupus erythematosus
Roche Group
2025-11-03
cd20
Phase III ALLEGORY study met primary and all key secondary endpoints with Gazyva/Gazyvaro, an anti-CD20 monoclonal antibody designed for enhanced B cell...
From bluebird bio to Genetix | Press Release
Genetix Biotherapeutics
2025-11-03
bluebird bio
genetix biotherapeutics
bluebird bio Rebrands to Genetix Biotherapeutics, Returning to Its Foundational Roots
Krystal Biotech targets global expansion for VYJUVEK with launches in Germany, France, and Japan amid pipeline acceleration
Krystal Biotech
2025-11-03
vyjuvek
Earnings Call Insights: Krystal Biotech (KRYS) Q3 2025 Management View CEO Krish Krishnan opened the call highlighting the continued momentum in the VYJUVEK launch. He stated, "In Q3, VYJUVEK launch continued to build momentum and the updated U.S. label clearly strengthens long-term outlook in the U.S. We're now launched in Germany, France and Japa...
Kyverna Therapeutics Secures up to $150 Million in Non-Dilutive Financing from Oxford Finance
Kyverna Therapeutics
2025-11-03
stiff person syndrome
Initial funding of $25 million from the first of four tranches Facility strengthens Kyverna’s financial flexibility, further supporting advancement of its late-stage indications in generalized myasthenia gravis (gMG) and stiff person syndrome (SPS), while also accelerating pre-launch activities
Gilead and Kite Showcase Continued Progress in Transforming Blood Cancer Care With New Cell Therapy Data at ASH 2025
Gilead Sciences
2025-11-03
cell therapy
Gilead Sciences, Inc. (Nasdaq: GILD) and Kite, a Gilead Company, will present 21 abstracts, including 5 oral presentations, during the 67th American Society ...
Roche's Gazyva breaks new ground in lupus
PharmaPhorum
2025-11-03
lupus
Already approved for kidney damage in lupus, Roche's Gazyva could be heading for broader use in the potentially life-threatening autoimmune disorder.
Immix Biopharma’s NXC-201 NEXICART-2 Clinical Trial Results Selected for Oral Presentation at ASH 2025
Immix Biopharma
2025-11-03
nxc-201
phase 1/2
– Oral presentation Sunday, December 7, at ASH 2025 – LOS ANGELES, Nov. 03, 2025 (GLOBE NEWSWIRE) — Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), the global leader in relapsed/refractory AL Amyloidosis, today announced that Phase 1/2 interim results from its U.S. NXC-201 NEXICART-2 trial in relapsed/refractory AL Amyloidosis has been selected […] The post Immix Biopharma’s NXC-201 NEXICART-2 Clinical Trial Results Selected for Oral Presentation at ASH 2025 first appeared on IMMIX BIOPHARMA INC .
ALS and FTD Neuronal Overactivity Linked to TDP-43 Dysfunction
Inside Precision Medicine
2025-11-03
amyotrophic lateral sclerosis
A new study has described how the TDP-43 protein moves from its normal location in the cell nucleus to the cytoplasm, resulting in excessive neuronal firing in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The post ALS and FTD Neuronal Overactivity Linked to TDP-43 Dysfunction appeared first on Inside Precision Medicine .
UniQure shares plummet as FDA says inadequate clinical data for Huntington’s therapy
uniQure
2025-11-03
gene therapy
(Reuters) -UniQure, said on Monday the U.S. health regulator has deemed clinical data for its gene therapy for Huntington's disease as inadequate, sending the Dutch company's U.S.-listed shares plummeting over 68% in premarket trading. The company's sh...
Caribou Biosciences to Participate in Upcoming Investor Conferences
caribou biosciences
2025-11-03
crispr
BERKELEY, Calif., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today announced management will participate in the following investor conferences: 2025 Truist Securities BioPharma Symposium , New
Roche's Gazyva, on an autoimmune disease hot streak, looks to further its new foothold in lupus with phase 3 win
Roche Group
2025-11-03
phase 3
Roche’s Gazyva is at it again. | After Roche's Gazyva won an FDA approval in lupus-related kidney disease, a phase 3 study now shows the med's worth in systemic lupus erythematosus, the most common type of lupus.
uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease
uniQure
2025-11-03
bla
amt-130
gene therapy
LEXINGTON, Mass. and AMSTERDAM, Nov. 03, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it received feedback from the U.S. Food and Drug Administration (FDA) during a recent pre-Biologics License Application (BLA) meeting regarding AMT-130, an investigational gene therapy for Huntington’s disease (HD). Though final meeting minutes have not yet been received, based
Autolus (NASDAQ: AUTL) to present five ASH abstracts, 95% ORR in pediatric B-ALL
Autolus Therapeutics
2025-11-03
orr
b-all
persistence
Five ASH abstracts feature obe‑cel data: CARLYSLE SLE initial safety and biomarkers, CATULUS pediatric R/R B‑ALL 95% ORR, and Month 3 persistence tied to EFS/OS.
uniQure (NASDAQ: QURE) updates on AMT-130; FDA feedback shifts, BLA timing unclear
uniQure
2025-11-03
bla
rmat
amt-130
Pre-BLA feedback says Phase I/II external-control data may not suffice as primary evidence. AMT-130 has Breakthrough (Apr 2025) and RMAT (May 2024); minutes due in 30 days.
Qlosi demonstrates consistent pupil size reduction in presbyopia
Healio
2025-11-03
phase 3
ORLANDO — Qlosi demonstrated consistent pupil size reduction and increasing rates of near visual acuity in patients with presbyopia over 8 days, according to a study. Mitchell A. Jackson, MD, of Jacksoneye in Lake Villa, Illinois, presented data from the phase 3 NEAR trials at the American Academy of Ophthalmology meeting.
Allogene Therapeutics Announces ALPHA3 Trial-in-Progress Poster Presentation at ASH Annual Meeting
Allogene
2025-11-03
car t
allogeneic
SOUTH SAN FRANCISCO, Calif. , Nov. 03, 2025 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™) products for cancer and autoimmune disease, today announced that a Trial-in-Progress (TIP)
Cero seeking more safety, dosing data on T-cell therapy for AML
Rare Cancer News
2025-11-03
aml
phase 1
cell therapy
acute myeloid leukemia
A third dose of CER-1236, Cero Therapeutics’ T-cell therapy candidate for acute myeloid leukemia (AML), was administered to an AML patient taking part in an early clinical trial, with the goal of generating further safety and dosing information. The additional dosing — within the Phase 1/1b trial’s first group of participants — was given to […] The post Cero seeking more safety, dosing data on T-cell therapy for AML appeared first on Rare Cancer News .
Autolus Therapeutics to Present Clinical Data Updates at the American Society of Hematology (ASH) Annual Meeting 2025
Autolus Therapeutics
2025-11-03
cell therapies
LONDON and GAITHERSBURG, Md., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, announces the online publication of five abstracts submitted to the American Society of Hematology (ASH) Annual Meeting, to be held December 6-9, 2025, in Orlando, Florida.
Genetix at ASH 2025 | Press Release
Genetix Biotherapeutics
2025-11-03
gene therapy
Genetix Biotherapeutics to Present Recent Patient Experience Data from U.S. Commercial Gene Therapy Implementation at the 67th American Society of Hematology (ASH) Annual Meeting
‘Enriched’ benefit: ctDNA could guide immunotherapy use in advanced urothelial cancer
Healio
2025-11-03
phase 3
Circulating tumor DNA could be used to identify which patients with muscle-invasive urothelial cancer would benefit from adjuvant immunotherapy and those who would not, according to data presented at ESMO Congress.Five-year follow-up data from the randomized, phase 3 CheckMate 274 trial, copublished in Annals of Oncology, showed patients who received nivolumab (Opdivo, Bristol Myers Squibb) had
UniQure dives after FDA’s ‘very surprising’ reversal on Huntington’s gene therapy
uniQure
2025-11-03
gene therapy
The FDA’s abrupt shift on UniQure’s treatment reflects an agency that, under current leadership, is as unpredictable as its been in years, some analysts said.
uniQure Staggers as FDA Questions Data for Huntington’s Gene Therapy Candidate
GenEng News
2025-11-03
bla
amt-130
gene therapy
In a statement, uniQure said that based on a recent pre-Biologics License Application (BLA) meeting with agency officials, the company “believes that the FDA currently no longer agrees” that data from the Phase I/II studies of AMT-130 may be sufficient as primary evidence in support of a BLA submission. The post uniQure Staggers as FDA Questions Data for Huntington’s Gene Therapy Candidate appeared first on GEN - Genetic Engineering and Biotechnology News .
03 Nov 2025 CARsgen Announces Positive Clinical Data for Allogeneic CAR-T Products CT0596 and CT1190B Nov 03, 2025
Carsgen Therapeutics
2025-11-03
car-t
allogeneic
Roche to present new data from its broad and innovative haematology portfolio at ASH 2025
Roche Group
2025-11-03
multiple myeloma
Findings further demonstrate the effectiveness of Roche’s approved medicines in advancing treatment standards for people with blood disorders Data from innovative pipeline signals progress toward improved outcomes in haemophilia A, lymphoma, and multiple myeloma Basel, 3 November 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that it will showcase 46 abstracts, including 12 oral presentations, from its industry-leading haematology portfolio at the 67th American Society of Hematology (
Vertex Reports Third Quarter 2025 Financial Results
Vertex
2025-11-03
pipeline
— Total revenue of $3.08 billion , an 11% increase compared to Q3 2024 — — Refined full year financial guidance: total revenue guidance now $11.9 to $12.0 billion and total combined non-GAAP R&D, AIPR&D, and SG&A expense guidance now $5.0 to $5.1 billion — — R&D pipeline continues to make
uniQure's ballyhooed gene therapy for Huntington's hits FDA roadblock
uniQure
2025-11-03
gene therapy
The FDA is said to be making a U-turn on uniQure’s one-time gene therapy candidate for Huntington’s disease despite the treatment being hailed as a game-changer. | FDA is said to be making a U-turn on uniQure’s one-time gene therapy candidate for Huntington’s disease despite the treatment being hailed as a game-changer.
Actinium Pharmaceuticals, Inc. to Participate in the Stephens Biotechnology Virtual Fireside Chat Conference
Actinium Pharma
2025-11-03
aml
cd33
psma
phase 2
pivotal
phase 2/3
quarterly
solid tumor
gene therapies
acute myeloid leukemia
NEW YORK , Nov. 3, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE American: ATNM) ("Actinium" or the "Company"), a leader in the development of differentiated targeted radiotherapies, announced today that Sandesh Seth, Chairman and CEO will be participating in the Stephens Biotechnology Virtual Fireside Chat. During the conference, members of Actinium's management team will also be available for one-on-one meetings with investors. Date: November 4, 2025 Time: 10:30-11:20 am ET Format: Virtual fireside chat To join the conference meetings or to obtain more information, please contact your Stephens representative. About Actinium Pharmaceuticals, Inc. Actinium is a pioneer in the development of targeted radiotherapies intended to meaningfully improve patient outcomes. ATNM-400, Actinium's lead product candidate, is a novel, first-in-class, and multi-indication Actinium-225 (Ac-225) in development for prostate cancer and non-small cell lung cancer (NSCLC). The antigen specifically targeted by ATNM-400 is highly expressed in metastatic castration-resistant prostate cancer (mCRPC), contributes directly to disease progression, poorer survival outcomes, and continues to be…
Antibody-gamma/delta T cell receptors targeting GPC2 regress neuroblastoma with low antigen density
Eureka Therapeutics
2025-11-03
gpc2
neuroblastoma
Cell Reports Medicine. 21 October 2025: Alex Quan, Mingyu Huo, Dan Li, Laura E. Hutchins, Constanza Rodriguez, Jangsuk Oh, Hsi-En Tsao, Madeline Spetz, Elijah Edmondson, Dana Ashworth, Rui Zheng, Jing Zhou, Jinyun Chen, Jingbao Liu, Guangyan Xiong, Hongbing Zhang, Cheng Liu, Rosa Nguyen, Nan Li, Mitchell Ho The post Antibody-gamma/delta T cell receptors targeting GPC2 regress neuroblastoma with low antigen density appeared first on Eureka .
Krystal's Q3: Platform Designation Adds Pipeline Leverage As Vyjuvek Scales (KRYS)
Krystal Biotech
2025-11-03
vyjuvek
Discover why Krystal Biotech’s Q3 results, Vyjuvek’s growth, & new FDA recognition make KRYS a Buy.
Appropriate ESI Severity Level Associated With Shorter TTFA for Vaso-Occlusive Crisis in SCD
Hematology Advisor News
2025-11-03
scd
Researchers determined assignment of an appropriate ESI score was associated with a shorter TTFA for patients with SCD presenting to the emergency department for VOC.
FDA Does 'About-Face' on UniQure’s Huntington’s Gene Therapy
uniQure
2025-11-03
amt-130
gene therapy
A month after sparking optimism for patients with Huntington’s disease with highly positive data for AMT-130, uniQure revealed Monday that the FDA may be changing its tune on the evidence required for an approval application, a change of direction Stifel called “very challenging” for uniQure.
Novartis reports positive phase 3 results for Cosentyx in polymyalgia rheumatica
PMLive Research
2025-11-03
phase 3
The REPLENISH trial met all primary and secondary endpoints, demonstrating sustained remission vs placebo
Krystal Biotech, Inc. SEC 10-Q Report
Krystal Biotech
2025-11-03
gene therapy
Krystal Biotech, Inc., a leading gene therapy company, has released its latest Form 10-Q report, showcasing robust financial and operational performance. The company, known for its innovative treatments for rare diseases, has demonstrated significant growth in both revenue and profitability, driven…
10-Q - Krystal Biotech, Inc. (0001711279) (Filer)
SEC Filing (10-Q)
2025-11-03
krystal biotech
Filed: 2025-11-03 AccNo: 0001711279-25-000087 Size: 6 MB
Lyell Announces Two Oral Presentations from the Phase 1/2 Clinical Trial of Ronde-Cel for the Treatment of Aggressive Large B-Cell Lymphoma at the 67th ASH Annual Meeting and Exposition
lyell immunopharma
2025-11-03
car t
phase 1
pipeline
phase 1/2
cell therapies
b-cell lymphoma
large b-cell lymphoma
SOUTH SAN FRANCISCO, Calif., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Lyell Immunopharma, Inc. (Nasdaq: LYEL), a late-stage clinical company advancing a pipeline of next-generation CAR T-cell therapies for patients with cancer, today announced that two abstracts highlighting new clinical and translational
UniQure Reeling As FDA Reverses Stance On Accelerated Approval For AMT-130
uniQure
2025-11-03
bla
amt-130
gene therapy
accelerated approval
UniQure’s plan for a 2026 BLA filing of gene therapy AMT-130 for Huntington’s is uncertain, as the FDA apparently reversed course after indicating it would consider accelerated approval.
Kura Oncology and Kyowa Kirin Announce Presentations of Ziftomenib Ven/Aza Combination Data in Frontline and Relapsed/Refractory NPM1-m or KMT2A-r Acute Myeloid Leukemia at 2025 ASH Annual Meeting
Kyowa Kirin
2025-11-03
aml
leukemia
acute myeloid leukemia
– Data to be featured in two oral presentations on December 8, 2025 – – Broad development program assesses ziftomenib across diverse AML segments and...
UniQure shares plummet as FDA says inadequate clinical data for Huntington’s therapy
uniQure
2025-11-03
gene therapy
(Reuters) -UniQure, said on Monday the U.S. health regulator has deemed clinical data for its gene therapy for Huntington's disease as inadequate, sending the Dutch company's U.S.-listed shares plummeting over 68% in premarket trading. The company's sh...
FDA Eyes Accelerated Reviews for Gene Editing Therapies
BioSpace
2025-11-03
gene editing
Vinay Prasad, chief of the FDA’s Center for Biologics Evaluation and Research, is planning to publish a paper this month to outline his office’s thinking of accelerating gene editing reviews.
Krystal Biotech’s Profits Rose As Vyjuvek Reached New Markets
Krystal Biotech
2025-11-03
vyjuvek
cystic fibrosis
Expanding its flagship product internationally and gaining US approval sent Krystal Biotech’s earnings sharply higher, while investors await new cystic fibrosis trial data.
uniQure poleaxed as FDA blocks Huntington's therapy
PharmaPhorum
2025-11-03
gene therapy
uniQure has revealed that the FDA has said it is not prepared to review its Huntington's gene therapy in what seems to be a change in policy.
BioMarin price target lowered to $88 from $95 at Bernstein
BioMarin Pharmaceutical
2025-11-03
quarterly
Bernstein lowered the firm’s price target on BioMarin (BMRN) to $88 from $95 and keeps a Market Perform rating on the shares following quarterly re...
Orna Therapeutics Announces Multiple Presentations Supporting its in vivo CAR Programs for Autoimmune Diseases at the 67th American Society of Hematology Annual Meeting
orna therapeutics
2025-11-03
car
WATERTOWN, Mass., Nov. 3, 2025 – Orna Therapeutics, a biotechnology company dedicated to engineering immune cells in vivo to treat autoimmune and oncology diseases, today announced upcoming presentations at the 67th American Society of Hematology (ASH) Annual Meeting being held December 6-9, 2025, in Orlando, Florida. The oral and poster presentations will highlight data supporting… The post Orna Therapeutics Announces Multiple Presentations Supporting its in vivo CAR Programs for Autoimmune Diseases at the 67th American Society of Hematology Annual Meeting appeared first on Orna Therapeutics .
Krystal Biotech reports Q3 EPS $2.66, consensus $1.12
Krystal Biotech
2025-11-03
vyjuvek
Reports Q3 revenue $97.8M, consensus $93.42M. “It is immensely gratifying to see a growing number of DEB patients worldwide benefit from access to VYJUVEK, and we l...
Adding durvalumab to BCG extends DFS for high-risk bladder cancer; toxicities a concern
Healio
2025-11-03
phase 3
bladder cancer
The addition of durvalumab to bacillus Calmette-Guérin therapy significantly extended DFS for patients with high-risk non-muscle-invasive bladder cancer, according to results of a randomized phase 3 trial.However, data from POTOMAC, presented at ESMO Congress and copublished in The Lancet, also showed a substantial increase in toxicities.
Krystal Biotech targets global expansion for VYJUVEK with launches in Germany, France, and Japan amid pipeline acceleration
Krystal Biotech
2025-11-03
vyjuvek
Earnings Call Insights: Krystal Biotech (KRYS) Q3 2025 Management View CEO Krish Krishnan opened the call highlighting the continued momentum in the VYJUVEK launch. He stated, "In Q3, VYJUVEK launch continued to build momentum and the updated U.S. label clearly strengthens long-term outlook in the U.S. We're now launched in Germany, France and Japa...
Sarepta Therapeutics Announces Third Quarter 2025 Financial Results and Recent Corporate Developments, Including Completion of Its Confirmatory Study, ESSENCE
Sarepta Therapeutics
2025-11-03
sarepta
Sarepta announces completion of the confirmatory trial commitment for its ultra-rare disease PMO therapies AMONDYS 45 and VYONDYS 53: While the ESSENCE study did not achieve statistical significance on its primary endpoint, results indicate positive and encouraging trends favoring therapy at 96
uniQure (QURE) Maintains Buy Rating as Chardan Capital Lowers Price Target
uniQure
2025-11-03
ind
Key Takeaways Chardan Capital maintains a Buy rating for uniQure (QURE) but lowers the price target from $76.00 to $53.00. The adjusted price target ind
Krystal Biotech Announces Third Quarter 2025 Financial and Operating Results
Krystal Biotech
2025-11-03
vyjuvek
$97.8 million in 3Q VYJUVEK revenue and $623.2 million since U.S. launch
FDA says clinical data for UniQure's Huntington's disease therapy not adequate
uniQure
2025-11-03
gene therapy
UniQure , said on Monday its experimental gene therapy for Huntington's disease does not have adequate clinical data to support its marketing application, according to the U.S. Food and Drug Administration.
INOVIO Completes Rolling BLA Submission Seeking Accelerated Approval for INO-3107 as a Treatment for RRP in Adults
Inovio Pharmaceuticals
2025-11-03
bla
accelerated approval
Optimistic Buy Rating for uniQure Amid Promising AMT-130 Data and Regulatory Prospects
uniQure
2025-11-03
amt-130
Patrick Trucchio, an analyst from H.C. Wainwright, reiterated the Buy rating on uniQure. The associated price target remains the same with $110.00. Patrick Trucchio...
Positive Phase III Data for Genentech’s Gazyva Show Significant Reduction in Disease Activity for Systemic Lupus Erythematosus
Genentech
2025-11-03
lupus
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today statistically significant
uniQure Stock Tumbled 55% Today: What’s The FDA Angle?
uniQure
2025-11-03
gene therapy
・It is now unclear when the company would apply for approval of the gene therapy, it said. ・uniQure, however, added that it intends to communicate with the FDA to determine a path forward toward approval of the therapy. ・uniQure CEO termed the meeting outcome “unexpected.” uniQure N.V. (QURE) no longer believes that the U.S. Food and Drug Administr...
Viral Vector Manufacturing: Case Studies and Considerations from a CDMO Perspective
BioSpace
2025-11-03
viral vector
Optimization Through Culture, Quality Control and Regulatory Standards
How FDA’s Revised Stance on AMT-130 Data Has Changed uniQure’s (QURE) Investment Story
uniQure
2025-11-03
amt-130
gene therapy
In recent news, uniQure announced that the FDA has reversed its previous guidance and now considers Phase I/II data inadequate to support a Biologics License Application submission for AMT-130, its gene therapy candidate for Huntington’s disease. This creates uncertainty in the regulatory pathway for a treatment that was positioned to be among the first disease-modifying options for this rare condition. This regulatory development introduces significant ambiguity regarding the timing and...
Beam Therapeutics to Present Updated Data from BEACON Phase 1/2 Trial of BEAM-101 in Sickle Cell Disease at American Society of Hematology (ASH) Annual Meeting
beam therapeutics
2025-11-03
phase 1
phase 1/2
base editing
sickle cell disease
CAMBRIDGE, Mass., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present updated safety and efficacy data from the BEACON Phase 1/2 clinical trial of
Positive Phase III Data for Genentech’s Gazyva Show Significant Reduction in Disease Activity for Systemic Lupus Erythematosus
Genentech
2025-11-03
lupus
Biohaven’s tror has ‘key differences’ from uniCure case, says Raymond James
uniQure
2025-11-03
gene therapy
Biohaven (BHVN) shares fell about 25% pre-market amid concern that the FDA’s reversal on uniCure’s (QURE) Huntington’s gene therapy signals trouble for Biohaven’s t...
Opus Genetics Featured on Good Morning America Spotlighting Breakthrough Gene Therapy Restoring Sight in Patients with Inherited Blindness
opus genetics
2025-11-03
gene therapy
uniQure (QURE) Faces FDA Reversal on AMT-130 Phase 1/2 Data
uniQure
2025-11-03
amt-130
phase 1
phase 1/2
gene therapy
Key Takeaways: The FDA's reconsideration of uniQure's (QURE) AMT-130 approval introduces uncertainty in the gene therapy sector. uniQure NV operates in t
Roche to present new data from its broad and innovative haematology portfolio at ASH 2025
Roche Group
2025-11-03
multiple myeloma
Findings further demonstrate the effectiveness of Roche’s approved medicines in advancing treatment standards for people with blood disordersData from innovative pipeline signals progress toward improved outcomes in haemophilia A, lymphoma, and multiple myeloma Basel, 3 November 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that it will showcase 46 abstracts, including 12 oral presentations, from its industry-leading haematology portfolio at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held 6-9 December 2025 in Orlando, Florida, US.
uniQure stock falls after FDA feedback on gene therapy application
uniQure
2025-11-03
bla
amt-130
gene therapy
uniQure (QURE) shares drop after FDA questions AMT-130 data for Huntington’s disease, delaying BLA submission. Read more here.
Caribou Biosciences Announces Positive Data from ANTLER Phase 1 Trial Demonstrating Efficacy and Durability of Vispa-cel (CB-010), an Allogeneic CAR-T Cell Therapy, on Par with Autologous CAR-T Cell Therapies
caribou biosciences
2025-11-03
orr
cd19
car-t
cr rate
phase 1
allogeneic
autologous
cell therapy
cell therapies
Data demonstrate efficacy and durability of vispa-cel, an allogeneic anti-CD19 CAR-T cell therapy, are on par with autologous CAR-T cell therapies in the confirmatory cohort (N=22) and with longer-term follow-up on patients who received optimized vispa-cel (N=35) 82% ORR, 64% CR rate, 51% PFS at 12
Cellectis to Present a Development Update for eti-cel at ASH 2025
cellectis
2025-11-03
orr
cd20
cd22
car-t
cr rate
phase 1
phase 2
pivotal
allogeneic
persistence
gene therapies
complete response
overall response rate
Preliminary data recently shared for eti-cel (UCART20x22) show an 86% ORR and a 57% CR rate (n=7), underscoring its potential to improve outcomes in r/r NHL Preclinical data demonstrated that combining eti-cel with low-dose IL-2 may deepen and extend anti-tumor activity in patients with r/r NHL Eti-cel full Phase 1 dataset, including low-dose IL-2 combination cohorts, expected to be presented in 2026 Correlation between alemtuzumab exposure and response with lasme-cel (UCART22) allows optimization of efficacy without an increase in toxicities New York, NY – November 3 , 202 5 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the acceptance of two abstracts for poster presentation at the American Society of Hematology (ASH) 2025 annual meeting taking place from December 6 to 9, 2025, in Orlando, FL. First poster – Development update on eti-cel The first poster provides a development update on eti-cel product candidate (UCART20x22), an allogeneic dual CAR-T targeting CD20 and CD22 being developed in Phase 1 of the…
Precision BioSciences Reports Third Quarter 2025 Financial Results and Provides Business Update
precision biosciences
2025-11-03
phase 1
Late-breaking oral presentation at the Liver Meeting ® 2025 to feature new data from multiple cohorts of the Phase 1 ELIMINATE-B trial of PBGENE-HBV in chronic hepatitis B - Commenced dosing in Cohort 3 of the ELIMINATE-B trial with additional data readouts planned in early 2026 - Investigational
Caribou Biosciences Announces Positive Data from CaMMouflage Phase 1 Trial of CB-011 in Multiple Myeloma
caribou biosciences
2025-11-03
car-t
phase 1
allogeneic
cell therapy
multiple myeloma
First clinical data disclosure for CB-011 highlights its potential as a best-in-class allogeneic CAR-T cell therapy for relapsed or refractory multiple myeloma 450 million cell dose is the recommended dose for expansion (RDE); dose expansion to initiate by year end and data expected in 2026 92%
Positive Phase III Data for Genentech’s Gazyva Show Significant Reduction in Disease Activity for Systemic Lupus Erythematosus
Genentech
2025-11-03
lupus
SOUTH SAN FRANCISCO, Calif., November 03, 2025--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today statistically significant and clinically meaningful results from the Phase III ALLEGORY study of Gazyva® (obinutuzumab) in adults with systemic lupus erythematosus (SLE) on standard therapy. The study met its primary endpoint showing a higher percentage of people achieved a minimum four-point improvement in SLE Responder Index 4 (SRI-4) at one year (52 weeks) with
FDA Grants DPTX3186 Orphan Drug Designation for Gastric Cancer
Hematology Advisor News
2025-11-03
orphan drug
A phase 1a/2a trial of DPTX3186 will begin at 8 US cancer centers in December 2025, with plans to expand to sites in South Korea and Taiwan.
UniQure and FDA No Longer in Alignment on Approval Pathway for AMT-130
uniQure
2025-11-03
amt-130
Women Have Higher Odds of Chronic Rhinosinusitis Without Nasal Polyps
Hematology Advisor News
2025-11-03
crs
Females have a higher odds of chronic rhinosinusitis (CRS) without nasal polyps than men.
Capricor Therapeutics Publishes Peer-Reviewed Study in Biomedicines Describing the Mechanism of Action and Potency Assay for its Investigational Cell Therapy, Deramiocel
Capricor Therapeutics
2025-11-03
deramiocel
cell therapy
Krystal Biotech (KRYS) Q3 2025 Earnings Transcript
Krystal Biotech
2025-11-03
vyjuvek
Total Net VYJUVEK Revenue Since Launch -- Over $623 million through the end of the reported quarter. Patient Compliance -- Remained in the low eighties percentage, consistent with prior trends for weekly VYJUVEK therapy. Ex-US Launches -- VYJUVEK launched in Germany, France, and Japan, with pricing negotiations completed in Japan in October and early French hospital and community access achieved.
Iovance Biotherapeutics Reports Potential Best-in-Class Clinical Data for Lifileucel TIL Cell Therapy in Advanced Non-Small Cell Lung Cancer (NSCLC)
Iovance Biotherapeutics
2025-11-03
til
cell therapy
26% Objective Response Rate Median Duration of Response Not Reached after 25 Months Follow Up Lifileucel Launch in Previously Treated Advanced NSCLC Expected in Second Half of 2027 SAN CARLOS, Calif. , Nov. 03, 2025 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc.
Benitec Biopharma Provides Positive Interim Clinical Study Results for BB-301 Phase 1b/2a Clinical Trial and Receives FDA Fast Track Designation for BB-301
benitec biopharma
2025-11-03
fast track
UniQure shares plummet as FDA says inadequate clinical data for Huntington's therapy
uniQure
2025-11-03
amt-130
gene therapy
(Reuters) -UniQure, said on Monday the U.S. health regulator has deemed clinical data for its gene therapy for Huntington's disease as inadequate, sending the Dutch company's U.S.-listed shares plummeting over 68% in premarket trading. The company's shares had surged nearly 200% in September after reporting trial data that showed its gene therapy, AMT-130, slowed progression of the brain disorder
StockWatch: Intellia Shares Sink as Clinical Hold Follows Safety Setback
GenEng News
2025-11-02
clinical hold
The patient, a man in his early 80s with a high body mass index, was dosed with nex-z on September 30. Intellia learned of his condition approximately 24 days post-dosing, the company disclosed during a conference call with analysts held to discuss the dosing pauses. The post StockWatch: Intellia Shares Sink as Clinical Hold Follows Safety Setback appeared first on GEN - Genetic Engineering and Biotechnology News .
Renier Brentjens, MD, PhD | Authors
CGTLive
2025-11-02
gene therapy
Renier Brentjens, MD, PhD | Authors | Cell and Gene Therapy Live
FDA Picks Abeona’s XLRS Gene Therapy ABO-503 for RDEA Pilot Program
CGTLive
2025-11-02
gene therapy
The RDEA program is meant to support use of novel end points for measuring efficacy in clinical studies for therapies aimed at treating rare diseases.
Caribou Biosciences to Host Webcast to Report New Data Updates from Two Allogeneic CAR-T Cell Therapy Programs in Lymphoma and Multiple Myeloma
caribou biosciences
2025-11-02
car-t
crispr
allogeneic
cell therapy
multiple myeloma
BERKELEY, Calif., Nov. 02, 2025 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today announced that it will hold a webcast beginning at 8:00 am ET on Monday, November 3, 2025, to report new data from the ANTLER
Regeneron’s Gene Therapy DB-OTO Improves Speech Perception in Children With Otoferlin-Related Hearing Loss
CGTLive
2025-11-01
gene therapy
Pending talks with the FDA, Regeneron intends to submit a regulatory application for DB-OTO before the end of 2025.
Adicet Bio Reports Inducement Grants under Nasdaq Listing Rule 5635(c)(4)
Adicet Bio
2025-11-01
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. --(BUSINESS WIRE)--Oct. 31, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced it granted inducement awards on October 31, 2025 .
What Catalysts Could Shift the Narrative for uniQure After Breakthrough AMT-130 Data?
uniQure
2025-11-01
amt-130
pivotal
gene therapy
uniQure's stock narrative has been recently revised, with the Fair Value Estimate remaining constant at $75.09 per share while the discount rate ticked up marginally from 6.70% to 6.76%. This comes amid intensified analyst attention, prompted by encouraging three-year clinical data for the company's AMT-130 gene therapy in Huntington's disease. As investors consider both opportunities and emerging risks, stay tuned for guidance on how to track these pivotal developments moving...
Gene Therapy for Bladder Cancer Now Fully Available
Ferring Pharmaceuticals
2025-11-01
adstiladrin
gene therapy
bladder cancer
Adstiladrin delivers a gene that encodes for the interferon alpha-2b protein, and it provides an option for those at high risk of recurrence and metastatic bladder cancer.
Cell & Gene Pipeline Outlook: November 2025
Prime Therapeutics
2025-11-01
pipeline
BLA for Rocket’s LAD-I Gene Therapy Under Review by FDA Again
CGTLive
2025-11-01
bla
gene therapy
Liver Complications Lead FDA to Stop Tests of Intellia Gene-Editing Therapy for a Rare Disease
MedCity News
2025-10-31
crispr
phase 3
clinical hold
The FDA clinical hold covers two Phase 3 studies for nexiguran ziclumeran, or nex-z, an experimental gene-editing therapy for transthyretin amyloidosis. Nex-z uses the CRISPR gene-editing technology to inactivate the gene that codes for the protein driving this rare disease. The post Liver Complications Lead FDA to Stop Tests of Intellia Gene-Editing Therapy for a Rare Disease appeared first on MedCity News .
Cellectis to Report Third Quarter Financial Results on November 7, 2025
cellectis
2025-10-31
gene therapies
New York, NY - October 31, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the third quarter 2025 ending September 30, 2025 on Friday, November 7, 2025 after the close of the US market. The press release will be available in the Investors section of Cellectis’ website: https://www.cellectis.com/en/investors/press-releases/ Cellectis will not host a conference call to discuss these results. Our investors relations team remains available for questions at investors@cellectis.com
Gilead Sciences Inc (GILD) Q3 2025 Earnings Call Highlights: Strong HIV Sales Drive Growth ...
Gilead Sciences
2025-10-31
cell therapy
Gilead Sciences Inc (GILD) reports robust performance in HIV therapies, offsetting challenges in Veklury and cell therapy sales.
Myrtelle’s Canavan Disease Gene Therapy Effects Significant Decrease in CSF NAA and Increases in Brain Myelin Volume
CGTLive
2025-10-31
gene therapy
Adagene Announces First Patient Dosed in Randomized Dose Optimization Cohort of the Phase 2 Study of Muzastotug (ADG126) in Combination with KEYTRUDA® (pembrolizumab) in Microsatellite Stable Colorectal Cancer
AdaGene
2025-10-31
phase 2
phase 3
Phase 2 clinical trial underway with first patient dosed in October to support a clear path to Phase 3 based on previous alignment with FDA Patients randomized to either 10 or 20 mg/kg of muzastotug, in combination with KEYTRUDA with up to 30 patients per arm Company anticipates trial completion
Cell therapy sales dip 11% for Gilead, as HIV franchise drives quarter
Gilead Sciences
2025-10-31
tecartus
yescarta
cell therapy
Gilead Sciences reports 11% drop in Q3 cell therapy sales, with Yescarta and Tecartus seeing lower demand, while HIV business grows 4% to $5.3B in quarterly sales
In Vivo Gene Editing for Rare Diseases Begins with Umbrella Clinical Trial
Inside Precision Medicine
2025-10-31
crispr
gene editing
To translate their success into scalable clinical trials, the FDA has teamed up with the team that developed the first customized, on-demand CRISPR-based treatment. The post In Vivo Gene Editing for Rare Diseases Begins with Umbrella Clinical Trial appeared first on Inside Precision Medicine .
RSV mRNA vaccine safe, effective for transplant patients
Healio
2025-10-31
phase 3
ATLANTA — Moderna’s respiratory syncytial virus vaccine is safe and effective for adults who have received a solid organ transplant, a group that is at an increased risk for severe infection, according to phase 3 data presented at IDWeek. The FDA approved the vaccine, mRNA-1345 (mResvia), in 2024 for adults aged 60 years or older, and expanded the approval this year to include adults aged 18 to
Gilead’s long-acting PrEP Yeztugo takes off while cell therapy declines continue to haunt sales
Gilead Sciences
2025-10-31
cell therapy
For the last several quarters, Gilead Sciences’ earnings calls have been colored by anticipation for the launch of the California drugmaker's long-acting HIV pre-exposure prophylaxis (PrEP) option. | Gilead’s long-acting pre-exposure prophylaxis med Yeztugo has garnered $54 million in sales since its June launch in the U.S., the company reported, bolstering its HIV franchise as cell therapy sales continue to decline.
Precision BioSciences to Report Third Quarter 2025 Financial Results on November 3, 2025
precision biosciences
2025-10-31
gene editing
DURHAM, N.C. --(BUSINESS WIRE)--Oct. 31, 2025-- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that it will publish financial
Individualized Infection Strategies Improve Outcomes in CLL, ALL
Hematology Advisor News
2025-10-31
cll
Mortality was strongly associated with older age, comorbidities, and infection type, underscoring the need for early and diagnosis-specific antimicrobial intervention.
Systematic review substantiates ‘compelling’ potential of CAR T-cell approach in lupus
Healio
2025-10-31
cd19
car t
cell therapy
chimeric antigen receptor
CHICAGO — Chimeric antigen receptor T-cell therapy has exhibited “compelling” efficacy and safety in lupus, allowing 84% of patients to cease other immunosuppressive drugs through follow-up, according to a presenter at ACR Convergence 2025.“CD19-directed CAR T-cell therapy has transformed the treatment of relapsed/refractory B-cell malignancies,” Alberto
Novartis presents new positive data for rheumatic autoimmune disease treatment
PMLive Research
2025-10-31
phase 3
Patient outcome benefits were observed in two ongoing phase 3 trials
MiNK Therapeutics to Present Late-Breaking Data on AgenT-797 in Solid Tumors at the 40th Annual Society for Immunotherapy of Cancer (SITC) Meeting
Mink Therapeutics
2025-10-31
allogeneic
cell therapies
NEW YORK , Oct. 30, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics , Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering allogeneic invariant natural killer T (iNKT) cell therapies to treat cancer and immune disorders, today announced that late-breaking data demonstrating durable
AbbVie Reports Third-Quarter 2025 Financial Results
Abbvie Investors
2025-10-31
financial results
Reports Third-Quarter Diluted EPS of $0.10 on a GAAP Basis, a Decrease of 88.6 Percent; Adjusted Diluted EPS of $1.86 , a Decrease of 38.0 Percent; These Results Include an Unfavorable Impact of $1.50 Per Share Related to Acquired IPR&D and Milestones Expense Delivers Third-Quarter Net Revenues
Hemostemix Boots on the Ground in Florida - October Update
Hemostemix
2025-10-31
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta – October 30, 2025 – Hemostemix Inc. (TSXV: HEM | OTCQB: HMTXF | FSE: 2VF0), the leading autologous (patient's own) stem cell therapy company offering VesCell™ (ACP-01) to no-option individuals suffering from pain related to angina, peripheral arterial disease, chronic limb threatening ischemia, ischemic cardiomyopathy, non-ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, today announced major progress in Florida as it builds upon the success of its weekly webinar series, which since August has drawn strong interest from vascular surgeons statewide. These efforts mark the next phase of the Company’s “Boots on the Ground” initiative, accelerating patient access to ACP-01, Hemostemix’s autologous angiogenic-cell-precursor therapy for chronic limb-threatening ischemia (CLTI), pain relief, and wound healing, under Florida Senate Bill 1768 (SB 1768). Florida Opens the Door to Compassionate Innovation
Capricor Therapeutics to Present Third Quarter 2025 Financial Results and Recent Corporate Update on November 10
Capricor Therapeutics
2025-10-31
financial results
Krystal Biotech Q3 2025 Earnings Preview
Krystal Biotech
2025-10-31
open
Krystal Biotech (KRYS) is scheduled to announce Q3 earnings results on Monday, November 3rd, before market open.The consensus EPS Estimate is $1.12 (+23.1% Y/Y) and the consensus Revenue Estimate is $93.42M (+11.4% Y/Y).Over the last 2 years, KRYS has beaten EPS estimates 25% of the time and has beaten revenue estimates 50% of the time.Over the las...
Hemostemix Announces its Webinar for Florida Vascular Surgeons: Autologous Stem Cell Therapy for CLTI
Hemostemix
2025-10-31
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta – October 28, 2025 – Hemostemix Inc. (TSXV: HEM | OTCQB: HMTXF | FSE: 2VF0), the leading autologous (patient's own) stem cell therapy company offering VesCell™ (ACP-01) to no-option individuals suffering from pain related to angina, peripheral arterial disease, chronic limb threatening ischemia, ischemic cardiomyopathy, non-ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, is pleased to announce a one-hour educational webinar for Florida vascular surgeons and healthcare professionals. The session will introduce ACP-01, Hemostemix’s innovative autologous stem cell therapy designed for patients who suffer in pain with chronic limb-threatening ischemia (CLTI) who have no revascularization options.
Dianthus Therapeutics Highlights New Claseprubart Data Presented During 2025 AANEM Annual MeetingPDF VersionRead More
Dianthus Therapeutics
2025-10-30
myasthenia gravis
Positive data from Phase 2 MaGic trial presented for claseprubart in generalized Myasthenia Gravis, including new open-label extension data further supporting potential for 300mg/2mL Q4W dosing Phase 3 gMG trial planned to include QMG ≥ 10 screening criteria and two claseprubart treatment arms, PDF Version Read More
Lineage Cell Therapeutics to Report Third Quarter 2025 Financial Results and Provide Business Update on November 6, 2025
lineage cell therapeutics
2025-10-30
allogeneic
cell therapies
CARLSBAD, Calif. --(BUSINESS WIRE)--Oct. 30, 2025-- Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic, or “off the shelf”, cell therapies for serious medical conditions, today announced that it will report its third quarter
Cellipont Bioservices and Ernexa Therapeutics Enter Cell Therapy Manufacturing Partnership to Advance ERNA-101 Toward Clinical Trials in Ovarian Cancer
Ernexa Therapeutics
2025-10-30
cell therapy
ovarian cancer
10-Q - BRISTOL MYERS SQUIBB CO (0000014272) (Filer)
SEC Filing (10-Q)
2025-10-30
bristol myers squibb
Filed: 2025-10-30 AccNo: 0000014272-25-000149 Size: 13 MB
Humacyte Announces Publication of New Data Comparing Symvess™ to Autologous Vein in Extremity Arterial Trauma
Humacyte
2025-10-30
open
autologous
Results published in AAST’s Trauma Surgery & Acute Care Open Journal - - Study compared outcomes of patients treated with Symvess with patients in PROOVIT registry who were treated with vein - - In a comparison to prior results in the PROOVIT registry, outcomes for Symvess and autologous vein
Stem Cell Therapy Lowers Heart Failure Risk After Heart Attack
Inside Precision Medicine
2025-10-30
cell therapy
Phase III results show that patients who received a stem cell therapy within a week of a heart attack had a significantly lower risk of developing heart failure within the next three years. The post Stem Cell Therapy Lowers Heart Failure Risk After Heart Attack appeared first on Inside Precision Medicine .
AMCP Nexus 2025: A deep dive into prostate cancer treatment
Prime Therapeutics
2025-10-30
prostate cancer
Simone Ndujiuba, clinical oncology expert, shares strategies for managed care pharmacists to improve treatment
Roche gets US antitrust clearance for 89bio deal; intends to close Thursday | MLex | Specialist news and analysis on legal risk and regulation
Roche Group
2025-10-30
clearance
Roche said Thursday that it will close its $3.5 billion acquisition of liver drug developer 89bio. The announcement indicates that the companies have received clearance from the US antitrust authorities, which had until Wednesday to request additional information about the deal.
Intrathalamic delivery of adeno-associated viral vector expressing progranulin as gene therapy for GRN-related frontotemporal dementia
aviadobio
2025-10-30
gene therapy
viral vector
The post Intrathalamic delivery of adeno-associated viral vector expressing progranulin as gene therapy for GRN-related frontotemporal dementia appeared first on AviadoBio .
Abeona Therapeutics® Announces Permanent J-Code for ZEVASKYN® (prademagene zamikeracel)
Abeona Therapeutics
2025-10-30
zevaskyn
Financial Results of Astellas for Q2 YTD/FY2025
astellas
2025-10-30
financial results
View Financial Results of Astellas for Q2 YTD/FY2025.
Nkarta to Participate in November Investor Conferences
nkarta
2025-10-30
cell therapies
SOUTH SAN FRANCISCO, Calif., Oct. 30, 2025 (GLOBE NEWSWIRE) -- Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies, today announced its participation in the following investor conferences next month: Stifel 2025 Healthcare
Deucravacitinib improves renal dysfunction in systemic lupus erythematosus
Healio
2025-10-30
msc
CHICAGO — Deucravacitinib reduces circulating proteins associated with kidney dysfunction in patients with systemic lupus erythematosus, according to data presented at ACR Convergence 2025.“Specific plasma biomarkers may be useful for early detection and monitoring of response in lupus nephritis,” Amit Saxena, MD, MSc, of the New York University Grossman School of Medicine, told
Announcement of Consolidated Financial Results Fiscal 2025 Third Quarter
Kyowa Kirin Co.
2025-10-30
financial results
Ultragenyx Announces First Patient Dosed in Aurora Study Evaluating GTX-102 in Additional Angelman Syndrome Genotypes and Age Groups—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-10-30
angelman syndrome
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
SEC Filing (8-K)
2025-10-30
ultragenyx
Filed: 2025-10-30 AccNo: 0001193125-25-257538 Size: 170 KB Item 8.01: Other Events
Roche Sees China Drag Peaking In 2025 As Diagnostics Bets On Innovation Pipeline
Roche Group
2025-10-30
pipeline
Roche CEO Matthew Sause said that the company’s ambition for 2025 remains low single-digit growth in diagnostics, rising to mid-single-digit growth in 2026 as the effects of Chinese reforms begin to ease.
Kyverna’s CAR T Therapy Sets ’New Efficacy Standard’ in Myasthenia Gravis, De-Risking Phase III Trials
BioSpace
2025-10-30
car t
kyv-101
With a 100% response rate in a Phase II study, KYV-101 sets a new efficacy bar in generalized myasthenia gravis, according to analysts at William Blair.
Life-Threatening Liver Toxicity Triggers FDA Freeze on Two Intellia Gene Therapy Studies
BioSpace
2025-10-30
gene therapy
clinical hold
The clinical hold comes days after Intellia voluntarily paused enrollment and dosing in the same two studies.
GenSight Biologics announces regulatory authorizations for Individual Patient Expanded Access treatment with GS010/LUMEVOQ® in the US
GenSight Biologics
2025-10-30
lumevoq
First-line nivolumab-ipilimumab superior to nivolumab monotherapy for colorectal cancer
Healio
2025-10-30
phase 3
first-line
Patients with microsatellite instability-high/mismatch repair-deficient metastatic colorectal cancer derived “clinically meaningful” survival benefits from first-line nivolumab plus ipilimumab compared with nivolumab monotherapy.Updated data from the randomized phase 3 CheckMate 8HW trial, presented at ESMO Congress, showed individuals who received the doublet therapy had a 31%
Managed Healthcare Executive: Payers Are Bracing for the Specialty Trend of the Future
Prime Therapeutics
2025-10-30
gene therapy
Managed Healthcare Executive: Prime Therapeutics executives discuss specialty drug trends at AMCP Nexus. Key areas include autoimmune therapies, multiple sclerosis treatments, and emerging cell and gene therapy challenges for payers
PICCs Maintain Low Thrombotic Risk in Chronic Hematologic Disease, CLL
Hematology Advisor News
2025-10-30
cll
Thrombotic risk with PICCs matched that of totally implantable venous access devices, showing comparable long-term safety.
Ibrutinib May Raise Sudden Death Risk in CLL With Cardiac Comorbidities
Hematology Advisor News
2025-10-30
cll
Cardiac testing identified asymptomatic, clinically significant arrhythmias in 19% of patients on ibrutinib.
Allogene Therapeutics to Report Third Quarter 2025 Financial Results and Provide Business Update
Allogene
2025-10-30
car t
allogeneic
Conference Call and Webcast Scheduled for November 6, 2025 at 2:00 p.m. PT / 5:00 p.m. ET SOUTH SAN FRANCISCO, Calif. , Oct. 30, 2025 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™)
Brensocatib positively impacts Asian patients with bronchiectasis
Healio
2025-10-30
cystic fibrosis
CHICAGO — Asian patients with non-cystic fibrosis bronchiectasis had a lower annualized exacerbation rate and less lung function decline with 52-week brensocatib vs. placebo, according to data presented at the CHEST Annual Meeting.“Results of this study build on prior subgroup analysis findings, confirming that subgroups of patients with varying demographics and clinical
Lisata Therapeutics to Report Third Quarter 2025 Financial Results and Provide a Business Update on Thursday, November 6, 2025
Lisata Therapeutics
2025-10-30
financial results
BASKING RIDGE, N.J., Oct. 30, 2025 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the
Gilead ends ph. 1 trial after settling on twice-yearly treatment partner for lenacapavir
Gilead Sciences
2025-10-30
phase 1
Gilead has chosen a partner for lenacapavir in the race to develop new low-frequency treatments for HIV. | Gilead has chosen a partner for lenacapavir in the race to develop low-frequency treatments for HIV. After comparing two phase 1 integrase strand transfer inhibitors, the Bay Area pharma has elected to pair GS-3242 with lenacapavir moving forward and not GS-1219, Gilead announced during its third quarter earnings call on Oct. 30.
After a futile battle, Biomarin will sell the bleeding disorder drug Roctavian
BioMarin Pharmaceutical
2025-10-30
roctavian
gene therapy
hemophilia a
The US company is exploring options to divest Roctavian from its portfolio after two turbulent years since the hemophilia A gene therapy was approved.
Autolus Therapeutics to Report Third Quarter 2025 Financial Results and Host Conference Call on November 12, 2025
Autolus Therapeutics
2025-10-30
financial results
LONDON & GAITHERSBURG, Md., Oct. 30, 2025 -- Autolus Therapeutics plc , an early commercial stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T...
GenSight Biologics Announces Regulatory Authorizations for Individual Patient Expanded Access Treatment with GS010/LUMEVOQ® in the US
GenSight Biologics
2025-10-30
gene therapy
gene therapies
PARIS, October 30, 2025--Regulatory News: GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that the Company’s gene therapy GS010/LUMEVOQ® has been granted regulatory authorizations for an Individual Patient Expanded Access in the United States. The authorizations mark the first set of regula
VIDEO: Experts move toward viewing follicular lymphoma as ‘potentially a curable disease’
Healio
2025-10-29
follicular lymphoma
In this video, Leo I. Gordon, MD, FACP, discusses the growing view that follicular lymphoma could be viewed as “curable.”
Avobis Bio's Crohn Disease Cell Therapy AVB-114 Garners FDA RMAT Designation
CGTLive
2025-10-29
rmat
cell therapy
FDA updates Winrevair indication in PAH, highlighting impact in high-risk patients
Healio
2025-10-29
phase 3
The FDA approved a label update for Winrevair to include that it lowers the risk for clinical worsening events in adults with advanced pulmonary arterial hypertension, according to a press release from Merck.Winrevair (sotatercept, Merck) initially received FDA approval in March 2024 for treating adults with PAH. This decision was based, in part, on data from the phase 3 STELLAR trial.
Medicus Pharma recruits first patient in cancer patch trial
PMLive Research
2025-10-29
phase 2
The first patient has been dosed as part of a phase 2 trial in the UAE
First UK MS patient treated in Phase I CAR-T therapy trial
Autolus Therapeutics
2025-10-29
car-t
A 37-year-old woman has become the first patient in the UK to be treated with CAR-T therapy for Multiple Sclerosis.
VIDEO: Considerations, challenges associated with extranodal marginal zone lymphoma
Healio
2025-10-29
marginal zone lymphoma
In this video, Leo I. Gordon, MD, FACP, discusses highlights from his presentation on extranodal marginal zone lymphomas at the Lymphoma Leukemia & Myeloma Congress.
Kyverna Therapeutics Announces Positive Interim Phase 2 Data from KYSA-6 Study of KYV-101 in Generalized Myasthenia Gravis at AANEM 2025
Kyverna Therapeutics
2025-10-29
kyv-101
phase 2
phase 3
Compelling results set new clinical standard in generalized myasthenia gravis (gMG), increasing confidence in the Company’s registrational KYSA-6 Phase 3 MG trial 100% of patients achieved clinically meaningful responses in MG-ADL and QMG -- the co-primary endpoints of the Phase 3 trial -- with
IN8bio Expands INB-100 Phase 1 Clinical Trial with Addition of The Ohio State University as New Site
In8Bio
2025-10-29
phase 1
cell therapies
gamma-delta t cell
NEW YORK, Oct. 29, 2025 (GLOBE NEWSWIRE) -- IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies for cancer and autoimmune diseases, today announced that The Ohio State University has been added as a new clinical site in the
Roche (SWX:ROG) Valuation in Focus Following Positive Phase III Gazyva Results in Kidney Disease
Roche Group
2025-10-29
pivotal
Roche Holding (SWX:ROG) has released new Phase III INShore study results showing Gazyva delivers better sustained complete remission at one year for children and young adults with idiopathic nephrotic syndrome. The therapy also demonstrated benefits on key secondary outcomes, such as relapse-free survival. See our latest analysis for Roche Holding. Roche shares have recently slipped, with a 7.0% decline in the last week despite pivotal clinical wins like the Gazyva INShore results and several...
Neurogene Reaches Accord With FDA on Plan for Registrational Trial for Rett Syndrome Gene Therapy NGN-401
CGTLive
2025-10-29
gene therapy
Neurogene also relayed new preclinical findings on the use of intracerebroventricular and intrathecal lumbar delivery of NGN-401.
Axogen, Inc. Reports Third Quarter 2025 Financial Results
axogen
2025-10-29
financial results
Kyowa Kirin (TSE:4151) Valuation in Focus Following Orphan Drug Designation for OTL-200 Gene Therapy
Kyowa Kirin
2025-10-29
open
orphan drug
gene therapy
metachromatic leukodystrophy
Kyowa Kirin (TSE:4151) recently announced that Japan's Ministry of Health, Labor and Welfare has granted Orphan Regenerative Medicine Product Designation to OTL-200, an investigational gene therapy for early-onset metachromatic leukodystrophy. This regulatory milestone could open doors to future opportunities in rare disease treatments. See our latest analysis for Kyowa Kirin. Regulatory momentum around OTL-200 arrives as Kyowa Kirin’s 1-year total shareholder return stands at -5.6%,...
Telitacicept Demonstrates Sustained Efficacy and Favorable Safety Profile in 48-Week China Phase 3 Open-Label Extension Generalized Myasthenia Gravis Data
Vor Biopharma
2025-10-29
myasthenia gravis
Telitacicept delivered consistent quality-of-life improvement across both treatment and placebo crossover arms 100% of patients on telitacicept for 48 weeks achieved ≥2-point Myasthenia Gravis Activities of Daily Living (MG-ADL) improvement, with a mean reduction of -7.5 points Sustained efficacy
MIMEDX Announces Record Revenue for Third Quarter 2025
mimedx
2025-10-29
quarterly
Second Consecutive Quarter of Highest Quarterly Net Sales in MIMEDX History Third Quarter Net Sales of $114 million Grew 35% Year-Over-Year Third Quarter GAAP Net Income and Diluted Earnings Per Share were $17 Million and $0.11 , Respectively Third Quarter Adjusted EBITDA was $35 Million, or 31%
Managed Healthcare Executive: Real-World Study Shows $330,000 Cost Difference Between CAR T and BiTE Therapies
Prime Therapeutics
2025-10-29
car t
neurotoxicity
follicular lymphoma
Managed Healthcare Executive: Bispecific T-cell engagers demonstrate lower total costs and reduced cytokine release and neurotoxicity rates compared with CAR T therapy for patients with follicular lymphoma
BioMarin (NASDAQ: BMRN) PALYNZIQ sBLA gets FDA Priority Review to expand to ages 12–17
BioMarin Pharmaceutical
2025-10-29
pdufa
phase 3
priority review
FDA accepted the sBLA with Priority Review; PDUFA target Feb. 28, 2026. Submission cites Phase 3 PEGASUS showing significant blood Phe lowering vs diet in ages 12–17.
Novartis’ Big Buy, Q3 Earnings, Regeneron’s Dropped Cell Therapy, More
BioSpace
2025-10-29
cell therapy
As third-quarter earnings continue to roll out, Novartis makes headlines with the second biggest acquisition of the year; Novartis’ CEO also downplayed the impact of Big Pharma pricing deals with the Trump administration; Regeneron continued the trend of dropping cell therapy assets; BioSpace takes a look at how the FDA is functioning mid-shutdown.
FDA Fast Track designation given to Enterome’s follicular lymphoma treatment
PMLive Regulatory
2025-10-29
fast track
follicular lymphoma
Follicular lymphoma, a type of non-Hodgkin’s lymphoma, has few initial symptoms
AMCP Nexus 2025: Prime’s GLP-1 persistence research recognized with platinum award
Prime Therapeutics
2025-10-29
persistence
Prime Therapeutics researchers share more about award-winning real-world research
Will FDA Platform Technology Designation for HSV-1 Vector Change Krystal Biotech's (KRYS) Narrative?
Krystal Biotech
2025-10-29
gene therapy
viral vector
Krystal Biotech recently announced that its HSV-1 viral vector, used in KB801 for the treatment of Neurotrophic Keratitis, received FDA platform technology designation. This regulatory milestone highlights the growing recognition of Krystal's gene therapy platform and could strengthen its position in advancing additional therapies across several indications. We'll examine how the FDA platform technology designation could strengthen Krystal Biotech's investment narrative and support future...
Guess the Therapy Answer October 29, 2025
CGTLive
2025-10-29
gene therapy
Cell and Gene Therapy Live
Novartis ianalumab first drug to reduce disease activity and patient burden in Sjögren’s disease Phase III trials
Novartis
2025-10-29
ianalumab
BridgeBio two for two as rare disease drug aces ph3 trial
PharmaPhorum
2025-10-29
pivotal
BridgeBio's week just went from great to superb, as it reports a second positive topline result in a pivotal trial of a late-stage pipeline drug.
Trastuzumab Deruxtecan Superior to T-DM1 in High-Risk HER2+ Early Breast Cancer
Hematology Advisor News
2025-10-29
her2
The DESTINY-Breast05 trial included patients with HER2-positive primary breast cancer who were at high risk of recurrence and had residual invasive disease in breast or axillary lymph nodes following neoadjuvant therapy.
AbbVie Announces Positive Topline Results from Phase 3 Pivotal Studies Evaluating Upadacitinib (RINVOQ®) in Adults and Adolescents with Vitiligo
Abbvie
2025-10-29
phase 3
pivotal
In two replicate Phase 3 studies, upadacitinib (RINVOQ®) achieved the co-primary endpoints of 50% reduction in Total Vitiligo Area Scoring Index (T-VASI 50) from baseline and 75% reduction in...
FDA Accepts BioMarin's PALYNZIQ® (pegvaliase-pqpz) Supplemental Biologics License Application for Priority Review to Expand Use to Adolescents Aged 12-17 with Phenylketonuria
BioMarin Pharmaceutical
2025-10-29
priority review
/PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review...
CTCs Show Promise as Prognostic Biomarker in Newly Diagnosed Multiple Myeloma
Hematology Advisor News
2025-10-29
multiple myeloma
CTC levels may be prognostic in patients with transplant-eligible, newly diagnosed MM who were treated with a standard-of-care quadruplet regimen.
Ferring Highlights New Real-World Research with ADSTILADRIN® (nadofaragene firadenovec-vncg) in Clinical Practice
Ferring Pharmaceuticals
2025-10-29
adstiladrin
Novartis Presents New Data From Potential First Targeted Sjögren’s Treatment
BioSpace
2025-10-29
ianalumab
Late-breaking Phase III data show ianalumab, Novartis’ dual-targeted antibody, reduced disease activity and patient burden in Sjögren’s disease, the pharma reported at the American College of Rheumatology Convergence congress on Wednesday.
FDA Accepts BioMarin's PALYNZIQ® (pegvaliase-pqpz) Supplemental Biologics License Application for Priority Review to Expand Use to Adolescents Aged 12-17 with Phenylketonuria
BioMarin Pharmaceutical
2025-10-29
pdufa
priority review
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the company's PALYNZIQ® (pegvaliase-pqpz) supplemental Biologics License Application (sBLA) to expand treatment to include adolescents aged 12-17 with phenylketonuria (PKU). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2026.
LENZ Therapeutics to Report Third Quarter 2025 Financial Results and Recent Corporate Highlights on November 5, 2025
LENZ Therapeutics
2025-10-29
financial results
Scaling Cell Therapy Development from Research to Commercialization
GenEng News
2025-10-29
cell therapy
In this GEN webinar, Nik Ekman will discuss critical considerations for scaling cell therapy processes from research through commercialization. The post Scaling Cell Therapy Development from Research to Commercialization appeared first on GEN - Genetic Engineering and Biotechnology News .
Will New Data and Approvals for Livdelzi and Trodelvy Change Gilead Sciences' (GILD) Narrative?
Gilead Sciences
2025-10-29
phase 3
In recent weeks, Gilead Sciences announced a series of product-related milestones, including new long-term data for Livdelzi (Seladelpar) in primary biliary cholangitis, positive Phase 3 results for Trodelvy in metastatic triple-negative breast cancer, and Health Canada's conditional approval of LYVDELZI for PBC treatment. The company also presented new research on its HIV, cancer, and hepatitis programs at major medical conferences in October 2025, underscoring advances across its pipeline...
Reduce Staffing Costs and Boost Access with Cell Therapy Automation
GenEng News
2025-10-29
cell therapy
Reducing the number of manual steps involved in cell therapy production using automation would help the industry reduce staffing costs, improve consistency, and help more patients gain access to these potentially lifesaving medicines, say researchers. The post Reduce Staffing Costs and Boost Access with Cell Therapy Automation appeared first on GEN - Genetic Engineering and Biotechnology News .
GSK’s B7-H3-targeted antibody-drug conjugate, GSK’227, receives Orphan Drug Designation in the EU
GSK
2025-10-28
b7-h3
lung cancer
orphan drug
Regulatory designation based on encouraging early clinical data showing potential for GSK’227 in small-cell lung cancer
Next-Gen Lentiviral Vector Manufacturing: Improving Scalability, Control, and Quality
GenEng News
2025-10-28
lentiviral
In this GEN webinar, LV expert Brianna Jayanthi, PhD, will discuss how Asimov's LV Edge Producer and Packaging Systems are leading to higher-quality, higher-productivity cell lines to support clinical and commercial LV therapies. The post Next-Gen Lentiviral Vector Manufacturing: Improving Scalability, Control, and Quality appeared first on GEN - Genetic Engineering and Biotechnology News .
SparingVision Doses First Patient with SPVN20 for Advanced Retinitis Pigmentosa in NYRVANA Trial
Sparing Vision
2025-10-28
retinitis pigmentosa
The post SparingVision Doses First Patient with SPVN20 for Advanced Retinitis Pigmentosa in NYRVANA Trial appeared first on SparingVision .
Adeno-Associated Virus (AAV) Vectors in Gene Therapy: Clinical Insights, Key Companies, Therapeutic Evaluation, Treatment Approaches, and Pipeline Analysis | GenSight Biologics, Ultragenyx Pharmaceuti
GenSight Biologics
2025-10-28
aav
regenxbio
ultragenyx
gene therapy
adeno associated virus
adeno-associated virus
nanoscope therapeutics
rocket pharmaceuticals
Adeno Associated Virus AAV Vectors in Gene Therapy companies are MeiraGTx Nanoscope Therapeutics REGENXBIO Astellas Gene Therapy GeneCradle Therapeutics Asklepios BioPharmaceutical 4D Molecular Therapeutics Adverum Biotechnologies Rocket Pharmaceuticals Innostellar Biotherapeutics Passage Bio Aspa Therapeutics Solid Biosciences Lantu Biopharma Ascidian Therapeutics ...
Ferring Highlights New Real-World Research with ADSTILADRIN® (nadofaragene firadenovec-vncg) in Clinical Practice
Ferring Pharmaceuticals
2025-10-28
adstiladrin
Ferring Highlights New Real-World Research with ADSTILADRIN® (nadofaragene firadenovec-vncg) in Clinical Practice
Gamida Cell Presents Positive Initial Results on Treating Severe Aplastic Anemia (SAA) with Omidubicel
Gamida Cell
2025-10-28
ayrmid ltd.
gamida cell
New data presented at the 2025 Association for the Advancement of Blood & Biotherapies (AABB) Annual Meeting highlights positive outcomes for patients Reports 92.9% of patients achieved rapid neutrophil recovery, with median time to neutrophil recovery of 7 days Ayrmid Ltd.
UK’s first MS patient receives CAR T-cell therapy in landmark trial
Autolus Therapeutics
2025-10-28
car t
cell therapy
Discover how CAR T cell therapy is breaking new ground in treating Multiple Sclerosis for the first time in the UK
Ibrutinib-Rituximab Outperforms Chemoimmunotherapy in Previously Untreated MCL
Hematology Advisor News
2025-10-28
mcl
phase 2
phase 2/3
first-line
Results from the phase 2/3 ENRICH trial support ibrutinib plus rituximab as a “new standard of care option” for first-line treatment of older patients with MCL, researchers wrote.
BioMarin loved its one-shot-and-done fix for hemophilia A when it was approved. But now its looking to sell it
BioMarin Pharmaceutical
2025-10-28
roctavian
gene therapy
hemophilia a
BioMarin plans to divest its hemophilia A gene therapy treatment Roctavian and cut research programs as it faces revenue challenges and potential competition.
Valoctocogene Roxaparvovec May Provide Long-Term Therapeutic Benefit in Hemophilia A
Hematology Advisor News
2025-10-28
phase 1
phase 3
phase 1/2
hemophilia a
The analysis included 141 participants: 134 from the phase 3 GENEr8-1 study and 7 from the 6E13 vg/kg cohort of the phase 1/2 study 270-201.
Novel CLIC5 Gene Therapy Uses AAV Technology to Treat Hearing Loss
Inside Precision Medicine
2025-10-28
aav
gene therapy
adeno-associated virus
The gene therapy targets the CLIC5 gene, responsible for maintaining the structure and stability of sensory hair cells in the inner ear, using an optimized delivery system—a self-complementary adeno-associated virus (scAAV) vector. The post Novel CLIC5 Gene Therapy Uses AAV Technology to Treat Hearing Loss appeared first on Inside Precision Medicine .
Kyowa Kirin and Orchard Therapeutics Announce OTL-200 Granted Orphan Regenerative Medicine Product Designation for Early-onset MLD in Japan
orchard therapeutics
2025-10-28
orphan drug
priority review
– First and only disease-modifying therapy for the treatment of eligible children with MLD also receives Orphan Drug Designation and Priority Review in the Kingdom of Saudi Arabia – TOKYO, LONDON and BOSTON, Oct. 28, 2025 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd., (TSE: 4151, President and COO:
Novartis Continues RNA & Neuroscience Growth Strategy With $12B Avidity Bio Acquisition
MedCity News
2025-10-28
pivotal
Avidity Biosciences brings to Novartis three RNA therapies in pivotal testing for rare neuromuscular diseases along with the technology that produced them. This platform develops drugs in a new class of targeted medicines offering potential to expand the delivery of RNA treatments beyond the liver. The post Novartis Continues RNA & Neuroscience Growth Strategy With $12B Avidity Bio Acquisition appeared first on MedCity News .
Genprex Announces Registered Direct Offering of up to $10.0 Million Priced At-The-Market Under Nasdaq Rules
Genprex
2025-10-28
gene therapy
$3.4 million upfront with up to an additional $6.6 million of potential aggregate gross proceeds upon the exercise in full of short-term warrants AUSTIN, Texas, October 28, 2025 — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that it has […]
Myeloma treatment HDP-101 put on FDA fast track
Rare Cancer News
2025-10-28
fast track
multiple myeloma
The U.S. Food and Drug Administration (FDA) has granted fast track designation to HDP-101 (pamlectabart tismanitin), a multiple myeloma therapy currently in early clinical testing. The FDA gives this designation to promising investigational therapies that, based on early data, have the potential to address unmet medical needs in the management of serious diseases. The designation […] The post Myeloma treatment HDP-101 put on FDA fast track appeared first on Rare Cancer News .
BioMarin Abandons Gene Therapy Roctavian, Cuts Revenue Guidance As Voxzogo Faces Competitors
BioMarin Pharmaceutical
2025-10-28
roctavian
gene therapy
BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending competition for achondroplasia therapy Voxzogo and the divestment of the gene therapy Roctavian.
BioMarin Abandons Gene Therapy Roctavian, Cuts Revenue Guidance as Voxzogo Faces Competitors
BioSpace
2025-10-28
roctavian
gene therapy
BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending competition for achondroplasia therapy Voxzogo and the divestment of the gene therapy Roctavian.
Biology teacher becomes first UK patient to receive 'gamechanger' MS therapy
Autolus Therapeutics
2025-10-28
car t
cell therapy
A biology teacher has become the first UK patient to receive CAR T-cell therapy for multiple sclerosis (MS) as part of a global clinical trial.
Genprex Collaborators Report Positive Preclinical Data on the Use of Reqorsa® Gene Therapy for the Treatment of Lung Cancer at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics
Genprex
2025-10-28
lung cancer
gene therapy
Reqorsa® Gene Therapy is a Potential Treatment for ALK-EML4 Positive Translocated Non-Small Cell Lung Cancer Combining REQORSA with Alectinib Further Increased Apoptosis and Improved Treatment Outcomes in a Mouse Model AUSTIN, Texas — (Oct. 28, 2025) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with […]
‘We should not be waiting’: Early baricitinib therapy benefits teens with alopecia
Healio
2025-10-28
phase 3
More than half of adolescents treated with baricitinib achieved complete or near-complete scalp hair regrowth at 1 year, with those who initiated treatment before completely losing their hair being primary responders, according to a study.The findings from the ongoing phase 3 BRAVE-AA-PEDS study were presented at the 2025 Fall Clinical Dermatology Conference.
Ferring Pharmaceuticals Presents Two New Analyses of Pivotal Trial Data for Follitropin Delta (FE 999049) at ASRM 2025
Ferring Pharmaceuticals
2025-10-28
pivotal
GSK’s Blenrep approved by FDA for multiple myeloma
PMLive Regulatory
2025-10-28
phase 3
multiple myeloma
Approval supported by positive results from phase 3 trial
uniQure: 3-Year Huntington's Data Move AMT-130 From Promising To Probable (Upgrade) (QURE)
uniQure
2025-10-28
amt-130
Discover why uniQure (QURE) is now a Buy after strong AMT-130 data for Huntington’s disease. Explore key financials, clinical results, and investment risks.
BioMarin Looks To Offload Roctavian As Gene Therapy’s Sales Drop
BioMarin Pharmaceutical
2025-10-28
roctavian
gene therapy
hemophilia a
The company said in its third quarter earnings that it would focus on areas that better foster growth for the company as the hemophilia A treatment only brought in $3m in Q3.
Microscope-integrated OCT offers real-time corneal thickness measurements in CXL
Healio
2025-10-28
msc
Microscope-integrated OCT delivers real-time assessment of corneal thickness changes during cross-linking, but it should not be used interchangeably with ultrasound pachymetry, according to a study. While changes in corneal thickness during the procedure can be measured using both methods, “there is little literature on the utility” of microscope-integrated OCT (Mi-OCT), Nandyala Sushma, MSc, of
BioMarin’s ROCTAVIAN Exit and Higher Guidance Could Be a Game Changer for BMRN
BioMarin Pharmaceutical
2025-10-28
roctavian
gene therapy
BioMarin Pharmaceutical recently reported its third-quarter 2025 financial results, showing revenues of US$776.13 million, a rise from the previous year, but also a net loss of US$30.74 million, compared to a profit a year earlier. An important development was the company’s decision to raise its full-year revenue guidance and announce plans to divest the gene therapy ROCTAVIAN, signaling a sharpened focus on its core growth areas such as enzyme therapies and skeletal condition...
Avelumab Does Not Meaningfully Prolong Survival in Advanced Penile Cancer
Hematology Advisor News
2025-10-28
phase 2
Immune checkpoint inhibitors have shown efficacy in other HPV-related cancers, so a phase 2 trial tested avelumab treatment for locally advanced or metastatic penile cancer.
Researchers Develop Ethics-Based Framework for Allocating Gene Therapies to Patients With SCD or TDT
Hematology Advisor News
2025-10-28
scd
gene therapy
gene therapies
Researchers developed an ethical framework for allocating gene therapy to patients with SCD or TDT.
Taysha Gene Therapies to Release Third Quarter 2025 Financial Results and Host Conference Call and Webcast on November 4
Taysha Gene Therapies
2025-10-28
aav
gene therapies
financial results
adeno-associated virus
DALLAS , Oct. 28, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today
Kyowa Kirin and Orchard Therapeutics Announce OTL-200 Granted Orphan Regenerative Medicine Product Designation for Early-onset MLD in Japan
Kyowa Kirin
2025-10-28
orphan drug
priority review
– First and only disease-modifying therapy for the treatment of eligible children with MLD also receives Orphan Drug Designation and Priority Review in the...
BioMarin Beats on Q3 Earnings, Seeks to Divest Hemophilia Gene Therapy
BioMarin Pharmaceutical
2025-10-28
gene therapy
BioMarin Pharmaceutical BMRN reported third-quarter 2025 adjusted earnings per share (EPS) of 12 cents against the Zacks Consensus Estimate of a loss of 15 cents. Quarterly earnings plummeted nearly 87% year over year, primarily due to IPR&D charges of $221 million recorded by the company in connec…
Ultragenyx to Host Conference Call for Third Quarter 2025 Financial Results and Corporate Update—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-10-28
financial results
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Roc in hard place: Biomarin plans divestment to ratchet up numbers
BioMarin Pharmaceutical
2025-10-28
roctavian
gene therapy
Biomarin Pharmaceutical Inc. plans to divest its pioneering gene therapy for hemophilia, Roctavian (valoctocogene roxaparvovec), and remove what had been considered a potential blockbuster from the portfolio in order to grow the company.
BMS, Cabaletta's CAR T Therapy Spurs Remissions in Small Lupus Trial
BioSpace
2025-10-28
car t
Phase I/II data for rese-cel point to its therapeutic potential in systemic lupus erythematosus and lupus nephritis, as well as other autoimmune conditions.
Medherant announces positive phase 1 results for testosterone patch for women
PMLive Research
2025-10-28
phase 1
University of Warwick spin-out develops first-in-class testosterone patch to support women through menopause
Pharmas Hope FDA PreCheck Can Help Avoid Facility-Related Drug Rejections
BioSpace
2025-10-28
complete response
Executives from Eli Lilly, Merck and other companies foresee the FDA's new onshoring proposal being anything from a bureaucratic waste of time to a transformative program that will eliminate inspection-related complete response letters.
BioMarin, following sluggish sales, to offload hemophilia gene therapy
BioMarin Pharmaceutical
2025-10-27
roctavian
gene therapy
The company intends to divest Roctavian and “remove it from our portfolio,” CEO Alexander Hardy said, after nearly three years of slow uptake for a medicine once viewed as a future blockbuster.
Bristol Myers Squibb Presents Two Late-Breaking Presentations Demonstrating Sotyktu (deucravacitinib) Efficacy in Psoriatic Arthritis and Systemic Lupus Erythematosus
Bristol Myers Squibb
2025-10-27
lupus
BioMarin’s Long-Term Study on BMN 270: A Potential Game Changer for Hemophilia A
BioMarin Pharmaceutical
2025-10-27
hemophilia a
Biomarin Pharmaceutical Inc. (($BMRN)) announced an update on their ongoing clinical study. BioMarin Pharmaceutical Inc. is conducting a long-term follow-up study t...
Engene Holdings Inc. Advances in Bladder Cancer Treatment with EG-70 Study
enGene
2025-10-27
phase 1
phase 1/2
bladder cancer
Engene Holdings Inc. (($ENGN)) announced an update on their ongoing clinical study. Study Overview: The LEGEND Study, officially titled A Phase 1/2 Study of EG-70 a...
Intellia Pauses Phase III ATTR Trials for CRISPR Gene Therapy After Life-Threatening Liver Toxicity
BioSpace
2025-10-27
crispr
gene therapy
Intellia earlier this year reported a similar grade 4 liver enzyme elevation associated with the gene therapy nexiguran ziclumeran, though analysts at BMO Capital Markets at the time brushed it off as a “non-concern.”
A Look at uniQure (QURE) Valuation Following Breakthrough Huntington’s Data and Rising Regulatory Optimism
uniQure
2025-10-27
amt-130
gene therapy
uniQure (NasdaqGS:QURE) just released data showing that its AMT-130 gene therapy can actually impact the progression of Huntington’s disease, which is catching the attention of investors across the biotech sector. See our latest analysis for uniQure. uniQure’s breakthrough with AMT-130 has sparked renewed optimism, sending its share price up 11% in just one day and an incredible 25% over the past month. Momentum has been building all year, with a year-to-date share price return of 276%. For...
GSK acquires exclusive rights from Syndivia for antibody-drug conjugate (ADC) in prostate cancer
GSK
2025-10-27
pipeline
prostate cancer
ADC complements GSK’s diverse pipeline in prostate cancer anchored by GSK’227, a B7H3-targeted ADC.
Autolus Therapeutics presents clinical data from CARLYSLE trial
Autolus Therapeutics
2025-10-27
phase 1
Autolus Therapeutics (AUTL) announces that follow-up data from the ongoing CARLYSLE trial, a Phase 1 dose confirmation clinical trial of obe-cel in severe refractor...
CAR T-cell therapy obe-cel begins human trial for progressive MS
Autolus Therapeutics
2025-10-27
car t
phase 1
cell therapy
The first patient has been dosed in a Phase 1 trial testing the experimental CAR T-cell therapy obecabtagene autoleucel for progressive MS.
BioMarin seeks to divest hemophilia A gene therapy Roctavian
BioMarin Pharmaceutical
2025-10-27
roctavian
gene therapy
hemophilia a
BioMarin plans to divest gene therapy Roctavian after poor sales, refocusing on core therapies. Read more here.
Krystal Biotech’s KB707 Study: A New Hope for Advanced Cancer Treatment
Krystal Biotech
2025-10-27
phase 1
phase 1/2
Krystal Biotech, Inc. (($KRYS)) announced an update on their ongoing clinical study. Krystal Biotech, Inc. is conducting a Phase 1/2 clinical study titled ‘A Phase ...
4 Huntington’s Therapies Chasing UniQure
BioSpace
2025-10-27
gene therapy
Last month, “historic positive results” from uniQure’s gene therapy snapped the Huntington’s community out of years of failure. As the biotech prepares to submit for FDA approval, BioSpace looks at four more candidates on the near horizon.
Adjuvant Durvalumab Plus Tremelimumab Improves DFS in Resected RCC
Hematology Advisor News
2025-10-27
phase 3
Investigators presented initial results from a phase 3 trial of adjuvant durvalumab plus tremelimumab for resected primary renal cell carcinoma (RCC).
Blenrep Returns With New FDA Approval in R/R Multiple Myeloma
Hematology Advisor News
2025-10-27
multiple myeloma
The FDA has approved Blenrep (belantamab mafodotin-blmf) with bortezomib and dexamethasone for patients with R/R multiple myeloma who have received at least 2 prior lines of therapy.
Autolus (NASDAQ: AUTL) notes no ICANS, high-grade CRS; 83% in remission and 50M dose set
Autolus Therapeutics
2025-10-27
crs
icans
phase 1
phase 2
Phase 1 CARLYSLE in srSLE: 6 patients at 50x10^6; no DLTs, ICANS or Grade ≥2 CRS; follow-up 8.9 months. Steroids ≤5 mg by month six; Phase 2 start by year-end 2025.
Sarepta Therapeutics to Announce Third Quarter 2025 Financial Results
Sarepta Therapeutics
2025-10-27
sarepta
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Oct. 27, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report third quarter 2025 financial results after the Nasdaq Global Market closes on Monday, Nov. 3, 2025 . Subsequently, at 4:30 p.m. E.T.
Latino patients face greater barriers to timely melanoma care
Healio
2025-10-27
melanoma
Latino patients face multiple obstacles to proper melanoma care, including language barriers and untimely referrals, according to a study.Compared with white patients, Latino patients are more commonly diagnosed with advanced melanoma and have worse survival rates, according to Kimberly A. Miller, PhD, MPH, associate professor in the department of population and public health sciences and
BioMarin Pharmaceutical misses Q3 revenue estimates, revises annual revenue forecast
BioMarin Pharmaceutical
2025-10-27
roctavian
Overview * BioMarin Q3 revenue rose 4% yr/yr, but missed analyst expectations * Adjusted EPS for Q3 missed analyst expectations * Company plans to divest ROCTAVIAN, aligning with...
CG Oncology’s Phase 2 Study: A New Hope for High-Risk NMIBC
CG Oncology
2025-10-27
phase 2
CG Oncology, Inc. (($CGON)) announced an update on their ongoing clinical study. Study Overview: CG Oncology, Inc. is conducting a Phase 2 study titled A Phase 2, M...
Krystal Biotech’s Innovative Approach to Treating Alpha-1 Antitrypsin Deficiency: A Clinical Update
Krystal Biotech
2025-10-27
phase 1
Krystal Biotech, Inc. (($KRYS)) announced an update on their ongoing clinical study. Krystal Biotech, Inc. (KRYS) is conducting a Phase 1 study titled ‘A Phase 1 St...
BioMarin Pharma plans to divest struggling gene therapy
BioMarin Pharmaceutical
2025-10-27
roctavian
gene therapy
BioMarin Pharmaceutical NASDAQ:BMRN said on Monday it plans to divest its gene therapy, a product once expected to be a blockbuster treatment for a type of rare bleeding disorder.The company said it is exploring out-licensing options for the therapy, Roctavian, and will remove it from its portfolio…
Kyowa Kirin’s KK2269 Study: A Potential Game-Changer in Solid Tumor Treatment?
Kyowa Kirin
2025-10-27
solid tumor
Kyowa Kirin Co (($KYKOF)) announced an update on their ongoing clinical study. Kyowa Kirin Co. is conducting a Phase 1 clinical study titled ‘A Pha...
INOVIO to Report Third Quarter 2025 Financial Results on November 10, 2025
Inovio Pharmaceuticals
2025-10-27
financial results
FDA approves Revuforj for AML with NPM1 mutations
Rare Cancer News
2025-10-27
aml
acute myeloid leukemia
The U.S. Food and Drug Administration (FDA) has approved Syndax‘s oral therapy Revuforj (revumenib) as a treatment for certain people with acute myeloid leukemia (AML) who have a susceptible mutation in the gene NPM1. The FDA approval specifically covers Revuforj’s use in AML patients ages 1 and older whose disease is relapsed or refractory — meaning […] The post FDA approves Revuforj for AML with NPM1 mutations appeared first on Rare Cancer News .
UniQure’s Huntington’s Breakthrough Brings Hope to Patients but Experts Urge Caution
BioSpace
2025-10-27
pivotal
gene therapy
Pivotal results from uniQure’s gene therapy for Huntington’s disease have brought new light to patients who have known only disappointment in recent years—but one expert worries that communication of the results is creating “false expectations.”
BioMarin’s Vosoritide Study: A Long-Term Hope for Hypochondroplasia
BioMarin Pharmaceutical
2025-10-27
phase 3
Biomarin Pharmaceutical Inc. (($BMRN)) announced an update on their ongoing clinical study. BioMarin Pharmaceutical Inc. is conducting a Phase 3, open-label, long-t...
Actinium Announces Superior Anti-Tumor Activity of ATNM-400 in Lung Cancer Compared to the Leading First, Second and Third-Line Approved EGFR Mutant Therapies and Mechanistic Synergy with Osimertinib at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics
Actinium Pharma
2025-10-27
aml
cd33
psma
phase 2
pivotal
pipeline
phase 2/3
quarterly
second line
solid tumor
gene therapies
acute myeloid leukemia
ATNM-400 exhibits superior efficacy with 3-5x greater tumor growth inhibition compared to front line therapy osimertinib (EGFR TKI TAGRISSO ® ), second line therapy Dato-DXd (Trop-2 ADC DATROWAY ® ) and third line therapy amivantamab (EGFR-cMET bispecific RYBREVANT ® ) - Combination of ATNM-400 and osimertinib resulted in complete tumor regression in 100% of tumor bearing animals; synergistic mechanism supported by increased ATNM-400 target antigen expression after EGFR inhibition with osimertinib - Improved progression free survival has been demonstrated clinically with the combination of osimertinib and external beam radiotherapy providing strong rationale for a combination with targeted alpha-therapy - Data validates the multi-tumor potential of ATNM-400 in multiple disease and treatment settings that support several blockbuster drugs NEW YORK , Oct. 27, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of differentiated targeted radiotherapies, today announced the presentation of the first ever preclinical data of ATNM-400 in non-small cell lung cancer (NSCLC). ATNM-400 a novel, multi-indication…
Krystal Biotech’s Promising Study on KB801 for Neurotrophic Keratitis: Market Implications
Krystal Biotech
2025-10-27
phase 1
phase 1/2
Krystal Biotech, Inc. (($KRYS)) announced an update on their ongoing clinical study. Krystal Biotech, Inc. is conducting a clinical study titled ‘A Phase 1/2, Multi...
Intellia Therapeutics Provides Update on MAGNITUDE Clinical Trials of Nexiguran Ziclumeran (nex-z)
Intellia Therapeutics Inc.
2025-10-27
crispr
gene editing
Conference call today at 8:30 a.m. ET CAMBRIDGE, Mass. , Oct. 27, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that the company has temporarily
CRISPR biotech Intellia plummets as trial paused for safety
PharmaPhorum
2025-10-27
crispr
phase 3
Intellia lost 45% of its value today as phase 3 trials of one of its priority drug programmes were halted due to a patient being hospitalised.
BioMarin’s Vosoritide Study: A Potential Game-Changer in Pediatric Growth Disorders
BioMarin Pharmaceutical
2025-10-27
phase 2
Biomarin Pharmaceutical Inc. (($BMRN)) announced an update on their ongoing clinical study. BioMarin Pharmaceutical Inc. is conducting a Phase 2 basket study titled...
BridgeBio Pops on Late-Stage Limb-Girdle Muscular Dystrophy Data
BioSpace
2025-10-27
sarepta
gene therapy
New interim data from a Phase III trial puts the company on track to file for FDA approval next year in an indication that not only lacks a disease-modifying treatment but suffered significant setbacks after a patient died in a clinical trial for Sarepta’s investigational gene therapy.
Bristol Myers Squibb Presents Two Late-Breaking Presentations Demonstrating Sotyktu (deucravacitinib) Efficacy in Psoriatic Arthritis and Systemic Lupus Erythematosus
bristol myers squibb
2025-10-27
lupus
Ferring Pharmaceuticals Presents Two New Analyses of Pivotal Trial Data for Follitropin Delta (FE 999049) at ASRM 2025
Ferring Pharmaceuticals
2025-10-27
pivotal
Ferring Pharmaceuticals Presents Two New Analyses of Pivotal Trial Data for Follitropin Delta (FE 999049) at ASRM 2025
Coya Exec Daniel Barvin Is on a Mission To End Genetic ALS and FTD
BioSpace
2025-10-27
amyotrophic lateral sclerosis
The BioSpace 40 Under 40 winner opens up about his very personal career transformation from wealth management to biotech—and what it’s like to develop a drug for amyotrophic lateral sclerosis and frontotemporal dementia as a potential patient himself.
Roivant to Report Financial Results for the Second Quarter Ended September 30, 2025, and Provide Business Update on Monday, November 10, 2025
roivant
2025-10-27
financial results
BASEL, Switzerland and LONDON and NEW YORK, Oct. 27, 2025 (GLOBE NEWSWIRE) -- Roivant (Nasdaq: ROIV) today announced that it will host a live conference call and webcast at 8:00 a.m. ET on Monday, November 10, 2025, to report its financial results for the second quarter ended September 30, 2025,
Autolus Therapeutics Presents Clinical Data Updates at the American College of Rheumatology Convergence 2025
Autolus Therapeutics
2025-10-27
crs
icans
phase 1
phase 2
pivotal
cell therapies
Data from the Phase 1 CARLYSLE study in severe refractory systemic lupus erythematosus (srSLE) suggests obe-cel is well tolerated with no ICANS or high-grade CRS; 50 million cell dose selected for Phase 2 pivotal studyPreliminary efficacy data demonstrate achievement of definition of remission in SLE (DORIS) in 83% of patients and complete renal response (CRR) in 50% of patients; all responses and remissions are ongoing with no evidence of disease activity at a median follow-up of 8.9 monthsAll patients had refractory lupus nephritis; 4/6 patients had significantly impaired kidney function Patients received no other lupus directed therapy; steroids were tapered in all patients to 5mg by month sixCompany remains on track to dose first patient in Phase 2 trial in lupus nephritis (LN) by year-end 2025 LONDON and GAITHERSBURG, Md., Oct. 27, 2025 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, announces that follow-up data from the ongoing CARLYSLE trial, a Phase 1 dose confirmation clinical trial of obe-cel in severe refractory systemic…
AI-based app noninferior to human-led program for preventing diabetes
Healio
2025-10-27
phase 3
Adults with prediabetes and overweight or obesity had similar success in maintaining an HbA1c of less than 6.5% with an AI-powered diabetes prevention program compared with a human-led intervention, according to data published in JAMA. In a phase 3 randomized clinical trial, participants were randomly assigned to use an AI-based program (Sweetch Health) that provided real-time feedback to
BioMarin’s BMN 351 Study: A New Hope for Duchenne Muscular Dystrophy
BioMarin Pharmaceutical
2025-10-27
phase 1
phase 1/2
duchenne muscular dystrophy
Biomarin Pharmaceutical Inc. (($BMRN)) announced an update on their ongoing clinical study. BioMarin Pharmaceutical Inc. is conducting a Phase 1/2 study titled A Ph...
Redefining Clinical Oncology Trials: How Agile Site Networks Accelerate Novel Cancer Therapies
MedCity News
2025-10-26
pivotal
Site networks can play a pivotal role in aligning sponsors, sites, and investigators toward shared goals in oncology clinical trials, but they are not one-size-fits-all. Sponsors must carefully vet site networks to validate that they will deliver strategic value for each individual study. The post Redefining Clinical Oncology Trials: How Agile Site Networks Accelerate Novel Cancer Therapies appeared first on MedCity News .
Study: 96% of patients remain off systemic sclerosis treatment after CAR T-cell therapy
Healio
2025-10-26
cd19
car t
phase 1
cell therapy
chimeric antigen receptor
CHICAGO — CD19 chimeric antigen receptor T-cell therapy improved lung function and skin thickness in patients with severe refractory systemic sclerosis, with 96% remaining off SSc treatment during follow-up, according to a speaker.“Breakfree-1 is a phase 1 basket trial where one-time infusion of BMS-986353 demonstrated a manageable safety profile and meaningful clinical improvements
Could gene therapy rewrite the future of Huntington’s?
uniQure
2025-10-26
gene therapy
Flora Allen explores the treatment offering new hope for a previously incurable disease
Fate Therapeutics Presents New Clinical Data at ACR Convergence 2025 Demonstrating Immune Remodeling and Durable Responses in Patients with Systemic Lupus Erythematosus Treated with FT819 Off-the-Shelf CAR T-Cell Therapy
Fate Therapeutics
2025-10-26
car t
lupus
cell therapy
As of a September 25, 2025 data cut-off-date, 10 patients with treatment-refractory, moderate-to-severe Systemic Lupus Erythematosus (SLE) were treated with a single dose of FT819 with less-intensive or no conditioning chemotherapy Favorable safety profile with no dose-limiting toxicities supports
uniQure (QURE) Is Up 5.2% After Announcing AMT-130 Efficacy and FDA Pre-BLA Meeting Plans
uniQure
2025-10-26
bla
amt-130
gene therapy
uniQure recently announced that its AMT-130 gene therapy has shown credible efficacy in altering the course of Huntington's disease, and the company is now planning a pre-BLA meeting with the FDA in the fourth quarter of 2025, ahead of a potential Biologics License Application in early 2026. This milestone positions uniQure as a potential acquisition target, given the estimated US$15 billion market opportunity and limited treatment options for Huntington's disease. We'll examine how...
Bristol Myers Squibb Presents Encouraging Data from Phase 1 Breakfree-1 Study of CD19 NEX-T™ CAR T Cell Therapy in Three Chronic Autoimmune Diseases at ACR Convergence 2025
Bristol Myers Squibb
2025-10-25
cd19
car t
phase 1
cell therapy
Kyverna Therapeutics Highlights Potential of KYV-101 in Rheumatoid Arthritis with Phase 1 Data from Investigator-Initiated Trial Presented at ACR Convergence 2025
Kyverna Therapeutics
2025-10-25
kyv-101
phase 1
KYV-101 resulted in a profound reduction in disease-associated autoantibodies and impact on disease activity in patients with difficult-to-treat rheumatoid arthritis (RA) KYV-101 continues to demonstrate a well-tolerated profile, consistent with observations from 100 patients treated with KYV-101
Bristol Myers Squibb Presents Encouraging Data from Phase 1 Breakfree-1 Study of CD19 NEX-T™ CAR T Cell Therapy in Three Chronic Autoimmune Diseases at ACR Convergence 2025
bristol myers squibb
2025-10-25
cd19
car t
phase 1
cell therapy
First multiple sclerosis patient in the UK receives CAR T cell therapy in groundbreaking trial
Autolus Therapeutics
2025-10-25
car t
cell therapy
A multiple sclerosis (MS) patient in the UK was the first to receive CAR T cell therapy, invented by UCL researchers, in a clinical trial testing whether this personalized treatment can slow or even halt the progression of the disease.
Biology teacher becomes first UK patient to receive ‘gamechanger’ therapy for MS
Autolus Therapeutics
2025-10-25
multiple sclerosis
Multiple sclerosis is a condition that affects the brain and spinal cord
CG Oncology (CGON) Is Up 5.3% After Advancing BOND-003 Trial and Analyst Upgrades Is the Momentum Sustainable?
CG Oncology
2025-10-25
phase 3
bladder cancer
CG Oncology recently advanced its Phase 3 BOND-003 clinical trial for high-risk bladder cancer, boosting its profile in oncology innovation. The company has attracted unanimous Buy ratings from all analysts covering the stock, reflecting considerable confidence in its clinical approach. We'll explore how progress in the BOND-003 trial adds depth to CG Oncology's investment story at a moment of strong analyst endorsement. We've found 17 US stocks that are forecast to pay a dividend yield of...
Novo Nordisk Submits FDA Application for Hemophilia A Therapy
Hematology Advisor News
2025-10-24
hemophilia a
Denecimig aims to help the body form blood clots more effectively by mimicking the role of FVIIIa and bridging factor IXa and factor X to restore thrombin generation and support normal clot formation.
Gene Expression Test Helps Identify Risk of Melanoma Spreading to Lymph Nodes
Inside Precision Medicine
2025-10-24
melanoma
The test reliably stratified patients into high- and low-risk status for lymph node metastasis, an improvement over sentinel lymph node biopsy (SLNB) that could potentially spare a large number of patients from unnecessary surgery. The post Gene Expression Test Helps Identify Risk of Melanoma Spreading to Lymph Nodes appeared first on Inside Precision Medicine .
Tango plans pivotal trial of vopimetostat for pancreatic cancer
Rare Cancer News
2025-10-24
phase 1
pivotal
phase 1/2
pancreatic cancer
Following new, positive data from a Phase 1/2 clinical study, Tango Therapeutics is planning to launch a pivotal trial to test its therapy candidate vopimetostat (TNG462) in people with pancreatic cancer. The upcoming trial is expected to enroll approximately 300 people with this cancer type who harbor a specific mutation called MTAP-del. To be eligible, […] The post Tango plans pivotal trial of vopimetostat for pancreatic cancer appeared first on Rare Cancer News .
Watertown biotech on the hunt for capital after early-stage cancer data
Roche Group
2025-10-24
phase 1
Remix Therapeutics reported positive preliminary results from the ongoing Phase 1 of its lead drug, REM-422, in treating adenoid cystic carcinoma.
Corcept Therapeutics reports positive phase 3 results in ovarian cancer treatment
PMLive Research
2025-10-24
phase 2
phase 3
ovarian cancer
The company also plans to expand its phase 2 BELLA study to include three new study arms
Actinium Pharmaceuticals Presents New Data Demonstrating Potent and Durable Efficacy of ATNM-400, a First-in-Class Multi-Tumor Actinium-225 Radiotherapy, at the 32nd Annual Prostate Cancer Foundation Scientific Retreat
Actinium Pharma
2025-10-24
aml
cd33
psma
phase 2
clearance
phase 2/3
quarterly
solid tumor
gene therapies
acute myeloid leukemia
ATNM-400 targets a novel, non-PSMA antigen implicated in disease biology with continued expression following androgen receptor pathway inhibitor (ARPI) therapy and 177Lu-PSMA-617 therapy, enabling potent activity independent of PSMA expression - ATNM-400 demonstrated superior efficacy over PSMA-targeted radioligand therapy (177Lu-PSMA-617 and 225Ac-PSMA-617) with enhanced tumor control and prolonged survival in low-PSMA and treatment-resistant models - Demonstrated durable anti-tumor responses, exceeding those achieved with the ARPI enzalutamide - Combination of ATNM-400 with enzalutamide achieved complete tumor regression in 40% of prostate cancer-bearing animals, demonstrating strong mechanistic synergy with AR pathway inhibition NEW YORK , Oct. 24, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of differentiated targeted radiotherapies, today highlighted new preclinical data for ATNM-400, its novel, first-in-class antibody radioconjugate armed with the potent alpha-emitter Actinium-225 (Ac-225) at the 32 nd Annual Prostate Cancer Foundation (PCF) Scientific Retreat being held October 23 – 25,…
Lilly Dives Deeper Into Gene Therapy With up to $262M Adverum Buy
BioSpace
2025-10-24
ixo-vec
gene therapy
wet age-related macular degeneration
The cornerstone of the deal is Ixo-vec, an intravitreal gene therapy currently in Phase III development for wet age-related macular degeneration. Eli Lilly made another foray into genetic medicine in June, picking up Verve Therapeutics for up to $1.3 billion.
GSK Blood Cancer Drug Returns to the U.S. Market, But New FDA Approval Is Still Limited
MedCity News
2025-10-24
multiple myeloma
GSK drug Blenrep, which exited the market in 2022 after failing a confirmatory study, now has a new FDA approval as a third-line multiple myeloma treatment. The U.S. regulatory decision is more limited than the recent European approval for the blood cancer drug. The post GSK Blood Cancer Drug Returns to the U.S. Market, But New FDA Approval Is Still Limited appeared first on MedCity News .
LSM After SVR May Predict HCC Risk in Chronic Hepatitis C
Hematology Advisor News
2025-10-24
hcc
Liver stiffness measurement after success with oral antivirals predicts HCC risk, particularly among patients with advanced chronic liver disease.
GSK's Blenrep's back in US, but with a narrower label
PharmaPhorum
2025-10-24
multiple myeloma
Against the odds, GSK has secured FDA approval for its multiple myeloma therapy Blenrep, three years after it was removed from sale.
Tackling Antimicrobial Resistance and Glioblastoma with Biology-First AI Models
GenEng News
2025-10-24
glioblastoma
In this GEN webinar, Jon Stokes, PhD, and Jeff Skinner, discuss how Stoked Bio’s platform advances the discovery of new antimicrobials and glioblastoma therapies by combining biological insights with cutting-edge computational models. The post Tackling Antimicrobial Resistance and Glioblastoma with Biology-First AI Models appeared first on GEN - Genetic Engineering and Biotechnology News .
FDA approves Blenrep for relapsed or refractory multiple myeloma
Rare Cancer News
2025-10-24
multiple myeloma
The U.S. Food and Drug Administration (FDA) has approved GSK‘s Blenrep (belantamab mafodotin) for adults with relapsed or refractory multiple myeloma. The approval covers the use of Blenrep in combination with dexamethasone and Velcade (bortezomib) in myeloma patients who have received at least two prior lines of treatment, including a proteasome inhibitor and an immunomodulatory […] The post FDA approves Blenrep for relapsed or refractory multiple myeloma appeared first on Rare Cancer News .
A New Era for Cell and Gene Therapy Signals New Market Opportunities
MedCity News
2025-10-24
gene therapy
Recent developments, including a high-profile acquisition and key regulatory changes, point to a paradigm shift. Cell and gene therapy is becoming increasingly commercially viable, with the potential to improve the lives of millions. The post A New Era for Cell and Gene Therapy Signals New Market Opportunities appeared first on MedCity News .
Eli Lilly Strengthens Gene Therapy Portfolio Through Adverum Acquisition
Inside Precision Medicine
2025-10-24
gene therapy
wet age-related macular degeneration
Adverum Biotechnologies has been acquired by Eli Lilly to strengthen its gene therapy portfolio, particularly with a promising Phase III candidate for wet age-related macular degeneration. The post Eli Lilly Strengthens Gene Therapy Portfolio Through Adverum Acquisition appeared first on Inside Precision Medicine .
Orna Therapeutics Announces Encore Presentation at the American College of Rheumatology Annual Meeting Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases
orna therapeutics
2025-10-24
pipeline
WATERTOWN, Mass., October 24, 2025 – Orna Therapeutics, a leading biotechnology company developing a proprietary pipeline of in vivo therapies across a broad range of autoimmune and oncology indications, today announced an upcoming presentation at the American College of Rheumatology (ACR) Annual Meeting being held October 24-29, 2025, in Chicago, Illinois. This dataset is a broader update… The post Orna Therapeutics Announces Encore Presentation at the American College of Rheumatology Annual Meeting Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases appeared first on Orna Therapeutics .
Anixa Biosciences to Present at the Spartan Capital Securities Second Annual Investor Conference
anixa biosciences
2025-10-23
car-t
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
SAN JOSE, Calif. , Oct. 23, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that it will present at the Spartan Capital Securities Second Annual Investor Conference on November 3, 2025, at the Marriott Marquis Hotel in New York City. Mike Catelani, President and CFO of Anixa, will deliver a presentation and will be available for one-on-one meetings during the conference. The conference will showcase more than 60 carefully selected companies across technology, healthcare, consumer, mining, precious metals, and other high-growth sectors. Over 500 institutional and high-net-worth investors are expected to attend. The event will feature main-stage presentations, expert panels, curated one-on-one meetings, and extensive networking sessions, creating meaningful opportunities for dialogue between growth companies and the investment community. For more information about the conference and registration details, please visit Spartan Capital Investor Conference 2025 Registration . A video recording of the company presentation will be available after the…
Blenrep approved by US FDA for use in treatment of relapsed/refractory multiple myeloma
GSK
2025-10-23
bcma
multiple myeloma
Blenrep is the only anti-BCMA accessible in the community setting where 70% of patients receive care.
Marital Status Can Accurately Stratify Survival Risk in CLL/SLL
Hematology Advisor News
2025-10-23
cll
Nomograms demonstrated strong predictive performance, with C-indexes around 0.70 to 0.72 and 5- and 10-year areas under the curve ranging from 0.713 to 0.759 in training and validation cohorts.
Pharmalittle: We're reading about a Moderna setback, CVS playing hardball with Gilead, and more
Gilead Sciences
2025-10-23
phase 3
Moderna's experimental vaccine for cytomegalovirus, a cause of disability in newborns, failed in a Phase 3 trial
Moderna drops mRNA jab for CMV after phase 3 fail
PharmaPhorum
2025-10-23
phase 3
Moderna has decided to halt the development of its mRNA-1647 vaccine for congenital CMV infections, once touted as a future blockbuster.
Genprex Collaborators to Present Positive Preclinical Data on the Use of Reqorsa® Gene Therapy for the Treatment of Lung Cancer at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics
Genprex
2025-10-23
lung cancer
gene therapy
Reqorsa® Gene Therapy is a Potential Treatment for ALK-EML4 Positive Translocated Non-Small Cell Lung Cancer Research Suggests that REQORSA May Be an Effective Treatment in Patients Progressing on Alectinib AUSTIN, Texas — (Oct. 23, 2025) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer […]
CG Oncology (CGON): Assessing Valuation Following Phase 3 Trial Progress and Upbeat Analyst Outlook
CG Oncology
2025-10-23
phase 3
U.S. stocks, Saudi stocks, stock trading and investment platforms
Vertex Presents New Data Across Portfolio of Cystic Fibrosis Medicines Including ALYFTREK® at the North American Cystic Fibrosis Conference
Vertex
2025-10-23
cystic fibrosis
Data presented on outcomes following treatment with CFTR modulators add to growing body of evidence that reduced level of sweat chloride is associated with improved clinical outcomes, particularly in younger people with cystic fibrosis - BOSTON --(BUSINESS WIRE)--Oct.
CG Oncology (CGON): Assessing Valuation Following Phase 3 Trial Progress and Upbeat Analyst Outlook
CG Oncology
2025-10-23
phase 3
bladder cancer
CG Oncology (CGON) has been drawing attention as it advances its Phase 3 clinical trial for BOND-003, which targets high-risk bladder cancer. Progress in this trial is shaping expectations for the company's future direction. See our latest analysis for CG Oncology. Behind the scenes, CG Oncology’s share price return has surged 60% over the past 90 days and climbed 44.7% year-to-date. Optimism around its clinical trial progress and sustained buy ratings are driving momentum. Both short- and...
UK patient first to trial CAR T cell therapy to treat multiple
Autolus Therapeutics
2025-10-23
car t
cell therapy
A multiple sclerosis (MS) patient in the UK was the first to receive CAR T cell therapy, invented by UCL researchers, in a clinical trial testing whether this personalised treatment can slow or even
Novel Gene Therapy Significantly Slows Progression of Huntington Disease
uniQure
2025-10-23
amt-130
gene therapy
huntington disease
AMT-130 demonstrates meaningful reductions in disease progression in the rare and chronic Huntington disease.
BTK Inhibitors Overcome Poor Prognosis of CLL With NOTCH1 Mutations
Hematology Advisor News
2025-10-23
cll
In vitro studies have suggested that BTKi therapy can suppress NOTCH1 activation via B-cell receptor–NOTCH1 interactions, providing a mechanistic rationale for these clinical results.
George's triple therapy for hypertension hits mark in stroke
PharmaPhorum
2025-10-23
phase 3
George Medicines' new triple pill for hypertension delivers a much-needed win in a phase 3 trial in haemorrhagic stroke.
Ipsen to acquire ImCheck Therapeutics in a deal worth up to €1bn
PMLive Research
2025-10-23
phase 1
phase 1/2
The acquisition includes a clinical stage acute myeloid leukaemia treatment currently in phase 1/2 trials
Genprex Announces Registered Direct Offering of up to $8.2 Million Priced At-The-Market Under Nasdaq Rules
Genprex
2025-10-23
gene therapy
$2.7 million upfront with up to an additional $5.5 million of potential aggregate gross proceeds upon the exercise in full of short-term warrants AUSTIN, Texas, October 23, 2025 — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that it has […]
Aviv Regev: The Computational Biologist Remaking Drug Discovery At Genentech
Genentech
2025-10-23
pipeline
Aviv Regev is betting AI-driven drug discovery can transform Genentech's pipeline, with early wins validating her computational biology approach to pharmaceutical R&D.
Guess the Therapy Answer October 23, 2025
CGTLive
2025-10-23
gene therapy
Cell and Gene Therapy Live
Biology teacher becomes first UK patient to receive ‘gamechanger’ therapy for MS
Autolus Therapeutics
2025-10-23
car t
cell therapy
Emily Henders, 37, received her CAR T-cell therapy last week and hopes it will slow down or halt progression of her MS.
Clear Clinical Benefits Identified in Patients With MF on Momelotinib
Hematology Advisor News
2025-10-23
overall response rate
The overall response rate of transfusion-dependent patients was 56% while that of transfusion-independent patients was 46%.
Biology teacher becomes first UK patient to receive ‘gamechanger’ therapy for MS
Autolus Therapeutics
2025-10-23
car t
cell therapy
Emily Henders, 37, received her CAR T-cell therapy last week and hopes it will slow down or halt progression of her MS.
Ventyx Bio Cardiovascular Drug’s Phase 2 Results Set Up Talks With a Big Pharma Firm
MedCity News
2025-10-23
phase 2
In a placebo-controlled mid-stage study, Ventyx Biosciences’ oral NLRP3 inhibitor led to rapid and sustained reductions in levels of a protein that’s an indicator of cardiovascular disease risk. The brain-penetrating properties of this once-daily pill also have potential applications addressing inflammation that drives neurodegenerative disorders. The post Ventyx Bio Cardiovascular Drug’s Phase 2 Results Set Up Talks With a Big Pharma Firm appeared first on MedCity News .
Iovance Biotherapeutics to Host Third Quarter 2025 Financial Results and Corporate Updates Webcast on Thursday, November 6, 2025
Iovance Biotherapeutics
2025-10-23
til
SAN CARLOS, Calif. , Oct. 23, 2025 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, will report its third quarter 2025
Gilead Sciences (GILD) Halts Phase 1 HIV Treatment Trial
Gilead Sciences
2025-10-22
phase 1
Key Takeaways: Gilead Sciences (GILD) halts Phase 1 trial for GS-1219, highlighting challenges in HIV treatment development. Focus shifts to newly approv
Sanofi’s efdoralprin alfa met all primary and key secondary endpoints in alpha-1 antitrypsin deficiency emphysema phase 2 study
sanofi
2025-10-22
phase 2
alpha-1 antitrypsin deficiency
Aqilion announces positive results from eosinophilic esophagitis treatment study
PMLive Research
2025-10-22
phase 2
Phase 2 trials for EoE are now expected to be initiated in Europe, Canada and the US
Krystal Biotech Launches VYJUVEK in Japan
Krystal Biotech
2025-10-22
vyjuvek
dystrophic epidermolysis bullosa
On October 22, 2025, Krystal Biotech announced the launch of VYJUVEK in Japan for treating dystrophic epidermolysis bullosa. Key Highlights: Original SEC Filing: Krystal Biotech, Inc. [ KRYS ] - 8-K - Oct. 22, 2025This is an AI-powered summary. It may contain inaccuracies. Consider verifying impor…
Nanoscope Therapeutics Announces Durable 3-Year Vision Improvements from REMAIN Study of MCO-010 in Retinitis Pigmentosa
Nanoscope Therapeutics
2025-10-22
mco-010
retinitis pigmentosa
REMAIN results showed sustained, 3-line vision gains vs baseline through 152 weeks for MCO-010 in patients having severe vision loss from retinitis pigmentosa, and a favorable safety profile The data form the clinical basis of a rolling Biologics License Application, initiated in June 2025, with the potential to be the first FDA-approved optogenetic therapy MCO […] The post Nanoscope Therapeutics Announces Durable 3-Year Vision Improvements from REMAIN Study of MCO-010 in Retinitis Pigmentosa appeared first on Nanoscope Therapeutics .
uniQure N.V. (QURE): A Bull Case Theory
uniQure
2025-10-22
amt-130
We came across a bullish thesis on uniQure N.V. on Truffle Pigs’s Substack. In this article, we will summarize the bulls’ thesis on QURE. uniQure N.V.’s share was trading at $63.65 as of October 9th. QURE’s forward P/E was 27.93 according to Yahoo Finance. uniQure’s AMT-130 represents a groundbreaking advancement in Huntington’s disease, providing the first credible […]
New Gene Therapy for Huntington’s Shows 75% Decrease in Disease Progression
uniQure
2025-10-22
gene therapy
Small study shows promise; will the FDA move the drug through?
GSK announces positive pivotal phase III data for next-generation low carbon version of Ventolin (salbutamol) metered dose inhaler
GSK
2025-10-22
pivotal
Data confirm therapeutic equivalence and comparable safety profile for Ventolin (salbutamol) containing innovative low carbon propellant.
Improved Outcomes in Hemophilia Patients Linked to a Sustainable Worldwide Registry
Hematology Advisor News
2025-10-22
gene therapy
Hemophilia registries include patient demographics, (sex, age, residence); genotype (ie, factor VIII or factor IX); disease severity; type of treatment (on-demand, prophylaxis, gene therapy); dosing; and infusion history.
Kyverna Therapeutics to Host Conference Call on Interim Phase 2 Data from KYSA-6 Study of KYV-101 in Generalized Myasthenia Gravis
Kyverna Therapeutics
2025-10-22
kyv-101
phase 2
cell therapies
EMERYVILLE, Calif., Oct. 22, 2025 (GLOBE NEWSWIRE) -- Kyverna Therapeutics, Inc. (Nasdaq: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, today announced it will host a conference call on Wednesday, October 29, 2025 at
Nurix Initiates DAYBreak™ Pivotal Study of Bexobrutideg in Relapsed or Refractory Chronic Lymphocytic Leukemia
nurix therapeutics
2025-10-22
cll
phase 2
pivotal
leukemia
accelerated approval
chronic lymphocytic leukemia
600 mg once daily bexobrutideg oral dose cleared by global regulators for pivotal monotherapy trials in relapsed/refractory chronic lymphocytic leukemia (r/r CLL) Phase 2 DAYBreak trial initiated for potential Accelerated Approval New preclinical data support bexobrutideg as potential best-in-class
Rule Out Melanoma Terminology on Pathology Requisitions Has Low Predictive Value
Hematology Advisor News
2025-10-22
melanoma
The use of ROM terminology on pathology requisition forms has a relatively low positive predictive value for a diagnosis of melanoma.
Nkarta Announces Poster Presentation at Upcoming American College of Rheumatology (ACR) Convergence Meeting
nkarta
2025-10-22
cell therapies
SOUTH SAN FRANCISCO, Calif., Oct. 22, 2025 (GLOBE NEWSWIRE) -- Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat autoimmune disease, today announced it will be presenting a poster this Sunday in Chicago at the
Nearly half of endometrial cancer patients respond to Rina-S in trial
Rare Cancer News
2025-10-22
phase 2
Nearly half of women with hard-to-treat endometrial cancer responded to the investigational therapy Rina-S (rinatabart sesutecan) in a Phase 2 clinical trial, according to new data announced by Rina-S’s developer, Genmab. “Women with advanced endometrial cancer are often facing a difficult path, while doctors are confronted with not having enough treatment options,” study investigator Noelle […] The post Nearly half of endometrial cancer patients respond to Rina-S in trial appeared first on Rare Cancer News .
Krystal Biotech Launches VYJUVEK® in Japan
Krystal Biotech
2025-10-22
vyjuvek
The latest update is out from Krystal Biotech ( ($KRYS) ). On October 22, 2025, Krystal Biotech‘s subsidiary in Japan launched VYJUVEK® for treating dystrophic epid...
Sanofi's $1.7B rare disease bet beats CSL's Zemaira in phase 2 face-off
CSL Behring
2025-10-22
phase 2
Sanofi’s $1.7 billion rare disease bet from last year has delivered a phase 2 win. | Sanofi’s $1.7 billion rare disease bet has delivered a phase 2 win. Efdoralprin alfa beat the standard of care on primary and key secondary endpoints, positioning the drugmaker to talk to regulators about the next steps.
Gilead halts early-stage trial for HIV injectable (GILD)
Gilead Sciences
2025-10-22
phase 1
Gilead (GILD) stock is in focus as the company halts a Phase 1 trial for its long-acting HIV injectable GS-1219, spotlighting development hurdles. Read more here.
The Bull Case For Krystal Biotech (KRYS) Could Change Following FDA Platform Technology Designation
Krystal Biotech
2025-10-21
gene therapy
gene therapies
On October 14, 2025, Krystal Biotech announced that the FDA granted platform technology designation to its HSV-1 gene therapy vector, which is used in the redosable KB801 eye drop now in clinical trials for neurotrophic keratitis. This designation may streamline future regulatory pathways for multiple gene therapies built on the same platform, potentially accelerating Krystal Biotech’s product development and review processes for other pipeline candidates. We'll take a closer look at how the...
Krystal Biotech’s FDA Platform Designation Pushes Limits of the Award
Krystal Biotech
2025-10-21
gene therapies
The company’s technology, a modified herpes simplex virus used to deliver gene therapies, was given the FDA’s new designation based on its approved topical skin cream. What this will mean for Krystal’s still-in-development eye drop is unclear.
Cryoport to Report Third Quarter 2025 Financial Results on November 4, 2025
Cryoport Inc
2025-10-21
financial results
Galapagos to Wind Down Cell Therapy Business After Search for a Buyer Comes Up Empty
MedCity News
2025-10-21
cell therapy
cell therapies
Non-binding offers for Galapagos’s cell therapies assets came mostly from financial investors, and none offered sufficient terms or financing to support the business, the company said. Galapagos’s exit from cell therapy work will lead to site closures in Europe, China, and the U.S. The post Galapagos to Wind Down Cell Therapy Business After Search for a Buyer Comes Up Empty appeared first on MedCity News .
Galapagos abandons cell therapy, putting 365 jobs at risk
PharmaPhorum
2025-10-21
cell therapy
Galapagos' difficult year has continued with a decision to wind down its cell therapy business as it seeks 'transformative' deals for its drugs unit.
Tecvayli-Darzalex Faspro combo extends survival in myeloma trial
Rare Cancer News
2025-10-21
top-line results
People with hard-to-treat myeloma given a combination of Tecvayli (teclistamab) and Darzalex Faspro (daratumumab and hyaluronidase-fihj) — both approved cancer therapies from Johnson & Johnson (J&J) — in a clinical trial had significantly longer overall survival than patients receiving standard of care treatment, according to top-line results announced by the company. Additionally, trial participants on the combo […] The post Tecvayli-Darzalex Faspro combo extends survival in myeloma trial appeared first on Rare Cancer News .
Immusoft Receives FDA Fast Track Designation for ISP-001, a First-in-Class Engineered B Cell Therapy for MPS I
Immusoft Corporation
2025-10-21
mps i
fast track
cell therapy
b cell therapy
cell therapies
/PRNewswire/ -- Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, today announced that the U.S. Food and Drug...
Biomarker Can Help Predict Preeclampsia Risk in Women with Sickle Cell Disease
Hematology.org
2025-10-21
sickle cell disease
Biomarker Can Help Predict Preeclampsia Risk in Women with Sickle Cell Disease
Zanubrutinib May Benefit CLL/SLL Patients Intolerant of Other BTK Inhibitors
Hematology Advisor News
2025-10-21
cll
Researchers determined zanubrutinib may be effective for patients with CLL/SLL who were intolerant to other BTK inhibitors.
PTC Therapeutics to Host Conference Call to Discuss Third Quarter 2025 Financial Results
ptc biotherapeutics
2025-10-21
financial results
WARREN, N.J. , Oct. 21, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the company will host a webcast conference call to report its third quarter 2025 financial results and provide an update on the company's business and outlook on Tuesday, November 4 , at 4:30
AskBio Announces Publication in Nature Medicine of 12-month data from Phase 1 Trial of AB-1002 Gene Therapy in Participants with Congestive Heart Failure
askbio
2025-10-21
phase 1
phase 2
gene therapy
dose-escalation
Research Triangle Park, N.C.– October 21, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the publication in peer-reviewed journal Nature Medicine of 12-month data from its Phase 1 trial of AB-1002 investigational gene therapy in participants with congestive heart failure (CHF).1 This non-randomized, sequential dose-escalation trial (NCT04179643) includes escalating dose cohorts to evaluate the safety and preliminary efficacy of investigational gene therapy AB-1002 in participants with New York Heart Association (NYHA) Class III non-ischemic heart failure with reduced ejection fraction (HFrEF).1 It is estimated that 64 million people worldwide are living with heart failure, and despite standard of care, mortality and morbidity remain very high.2,3 The publication, which is available online, confirms that no adverse events were deemed related to AB-1002 in this trial and that clinically meaningful improvements were recorded across several efficacy assessments in participants with non-ischemic CHF.1 The data further support that the AB-1002 capsid may be highly cardiotrophic when administered as…
Ventus Therapeutics Enters into a Collaboration and License Agreement with Genentech to Discover and Develop Small-Molecule Medicines Using Ventus' ReSOLVE® Platform
Genentech
2025-10-21
phase 2
Ventus Therapeutics, a clinical-stage biopharmaceutical company advancing two Phase 2 small-molecule programs for immunological, inflammatory, and neurologic...
FDA Accepts Priority Review of BLA for Orca-T in Hematologic Malignancies
Hematology Advisor News
2025-10-21
bla
phase 3
Phase 3 trial data showed Orca-T led to a significant improvement in survival without moderate-to-severe chronic graft-versus-host disease compared with those receiving alloHSCT.
Ventus Therapeutics Enters into a Collaboration and License Agreement with Genentech to Discover and Develop Small-Molecule Medicines Using Ventus' ReSOLVE® Platform
Genentech
2025-10-21
phase 2
WALTHAM, Mass. & MONTREAL, October 21, 2025--Ventus Therapeutics, a clinical-stage biopharmaceutical company advancing two Phase 2 small-molecule programs for immunological, inflammatory, and neurological disorders, today announced a multi-year collaboration with Genentech, a member of the Roche Group, to discover and optimize novel small-molecule candidates for challenging targets in major disease areas using Ventus’ proprietary drug discovery platform, ReSOLVE®.
V116 Provides Safe, Effective, Broader Pneumococcal Protection in Patients With HIV
Hematology Advisor News
2025-10-21
immunogenicity
Researchers assessed the safety, tolerability, and immunogenicity of the adult-specific 21-valent pneumococcal conjugate vaccine among patients with HIV infection.
First patient dosed in Phase 1 BOBCAT trial — Autolus advances obe-cel for progressive MS (up to 18 patients)
Autolus Therapeutics
2025-10-20
phase 1
Autolus dosed the first patient in the Phase 1 BOBCAT trial at UCLH to evaluate safety/tolerability of obe-cel in refractory progressive MS; trial may enroll up to 18 adults and obe-cel has been studied in >400 patients.
CHMP backs first drug for lung disease bronchiectasis
PharmaPhorum
2025-10-20
cystic fibrosis
Insmed gets EU recommendation for first non-cystic fibrosis bronchiectasis therapy Brinsupri, plus other CHMP decisions.
Autolus Therapeutics Doses First Patient in Phase 1 Trial of Obe-cel for Progressive Multiple Sclerosis
Autolus Therapeutics
2025-10-20
phase 1
Autolus Therapeutics said Monday that the first patient has dosed in its phase 1 trial of obecabtagene autoleucel to treat progressive multiple sclerosis. The patient was treated at University...
First patient dosed in Autolus’ phase 1 trial for multiple sclerosis By Investing.com
Autolus Therapeutics
2025-10-20
phase 1
First patient dosed in Autolus’ phase 1 trial for multiple sclerosis
Genentech Presents New Phase III Pivotal Data for Vamikibart in Uveitic Macular Edema (UME), a Serious Cause of Vision Loss
Genentech
2025-10-20
pivotal
Cell and Gene Therapy Drug Delivery Research Report 2025: Market to Expand Rapidly, Driven by Innovation from Leading Players Including Novartis, Pfizer, Amgen, Gilead Sciences, and Johnson & Johnson - ResearchAndMarkets.com
Gilead Sciences
2025-10-20
gene therapy
DUBLIN--(BUSINESS WIRE)--The "Cell and Gene Therapy Drug Delivery Market - A Global and Regional Analysis: Focus on Therapy Types, Delivery Methods, Country,
First patient dosed in Autolus’ phase 1 trial for multiple sclerosis By Investing.com
Autolus Therapeutics
2025-10-20
phase 1
First patient dosed in Autolus’ phase 1 trial for multiple sclerosis
Opthalmology Times | Video Interview: AAO 2025: Kenneth C. Fan, MD, sees vision gains with MCO-010 in Stargardt Disease
Nanoscope Therapeutics
2025-10-20
mco-010
The post Opthalmology Times | Video Interview: AAO 2025: Kenneth C. Fan, MD, sees vision gains with MCO-010 in Stargardt Disease appeared first on Nanoscope Therapeutics .
ESMO: Datroway, Trodelvy lay claim to frontline TNBC therapy
PharmaPhorum
2025-10-20
first-line
Two TROP2-targeting drugs, Datroway and Trodelvy, have new data suggesting they could become first-line options for triple-negative breast cancer.
FDA Approval for Roche Cancer Drug Brings a New Biologic Option to Lupus Nephritis
Roche Group
2025-10-20
cd20
Roche’s Gazyva, which first reached the market as a blood cancer drug, has expanded its FDA approval to include treatment of the autoimmune disorder lupus nephritis. The CD20-targeting antibody can now compete against a blockbuster GSK drug that is the standard biologic therapy for the debilitating kidney condition.
Vivet Therapeutics Presents Breakthrough Data on AAV Neutralizing Antibody Depletion at ESGCT 2025
Vivet Therapeutics
2025-10-20
aav
cell therapy
Sevilla, October 2025 – At the 32nd Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), Vivet Therapeutics shared new clinical data with its VTX-PID proprietary imlifidase formulation, highlighting the effective reduction of neutralizing antibodies (NAbs) against AAV3B in healthy volunteers. Key Findings: Vivet Therapeutics remains committed to advancing innovative therapies and […] The post Vivet Therapeutics Presents Breakthrough Data on AAV Neutralizing Antibody Depletion at ESGCT 2025 appeared first on Vivet Therapeutics .
Epidermolysis Bullosa Market Dynamics Indicate Upward Trajectory Through 2034, Reports DelveInsight | Amryt Pharma, Krystal Biotech, Abeona Therapeutics, Castle Creek Biosciences, RegeneRx, Lenus
Krystal Biotech
2025-10-20
krystal biotech
abeona therapeutics
The Key Epidermolysis Bullosa Companies in the market include - Berg Pharma, Holostem Terapie Avanzate, Aegle Therapeutics, RegeneRx Biopharmaceuticals, BridgeBio Pharma, InMed Pharmaceuticals, Fibrocell Science, Krystal Biotech, and others. The Epi...
ESMO: AlphaMedix™ phase 2 data support first-in-class potential of new targeted alpha therapy in gastroenteropancreatic neuroendocrine tumors
sanofi
2025-10-20
phase 2
A Look at uniQure (QURE) Valuation Following Promising AMT-130 Huntington’s Disease Trial Results
uniQure
2025-10-20
amt-130
gene therapy
UniQure (QURE) shares have come into focus after promising results from its AMT-130 clinical trials. The trials showed a significant slowdown in Huntington’s disease progression and noted improvements in key biomarkers. See our latest analysis for uniQure. The excitement around AMT-130 has electrified sentiment, following not only strong clinical results but also analyst upgrades and fresh attention on uniQure’s gene therapy pipeline. The share price has surged an extraordinary 318% over the...
Anixa Biosciences Announces Issuance of Chinese Patent Covering Breast Cancer Vaccine Technology
anixa biosciences
2025-10-20
car-t
phase 1
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
Expands global intellectual property coverage in markets with high breast cancer incidence New patent will provide IP protection of Breast Cancer Vaccine in China into 2040 SAN JOSE, Calif. , Oct. 20, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the Chinese National Intellectual Property Administration (CNIPA) has issued Patent Number ZL2020800215666, covering key aspects of the Company's breast cancer vaccine technology. Anixa's breast cancer vaccine, developed in collaboration with Cleveland Clinic, represents a novel approach to the prevention and treatment of breast cancer. Anixa's vaccine is based on immunizing against human α-lactalbumin, a protein associated with lactation that is aberrantly expressed in certain types of breast cancer. This "retired" protein vaccine strategy aims to selectively prime the immune system to prevent tumor formation while avoiding harm to normal tissue. The vaccine was invented at Cleveland Clinic, and this patent—along with others related to this technology—has been exclusively licensed to Anixa Biosciences.…
Hemostemix Adds the treatment of Refractory Angina to its Phase 1 Basket Protocol
Hemostemix
2025-10-20
phase 1
autologous
cell therapy
heart failure
cardiomyopathy
refractory angina
Calgary, Alberta — October 15, 2025 — Hemostemix Inc. (TSXV: HEM; OTCQB: HMTXF; FSE: 2VF0) , the leading autologous (patient's own) stem cell therapy company offering VesCell™ (ACP-01) to no-option individuals suffering from angina, peripheral arterial disease, chronic limb threatening ischemia, ischemic cardiomyopathy, non-ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, in Florida under Florida's SB 1768, is pleased to announce that it has completed the drafting of its clinical trial protocol titled “Treatment of Refractory Angina with Angiogenic Cell Precursors (ACP-01)”.
Gilead Sciences (GILD): Assessing Valuation After Major Trodelvy Breast Cancer Breakthrough and HIV Advances
Gilead Sciences
2025-10-20
phase 3
Gilead Sciences (GILD) has been drawing attention after releasing promising Phase 3 results for Trodelvy. The results show a marked extension in progression-free survival among metastatic triple-negative breast cancer patients. The company also highlighted advances in HIV prevention and treatment. See our latest analysis for Gilead Sciences. All this news comes as Gilead’s share price has surged by nearly 34% year-to-date, with momentum clearly building on the back of major new clinical data,...
20 Oct 2025 CARsgen Presents Preliminary Results on Satri-cel for Adjuvant Therapy of Pancreatic Cancer at ESMO Congress 2025 Oct 20, 2025
Carsgen Therapeutics
2025-10-20
pancreatic cancer
Autolus Therapeutics announces first patient dosed in Phase 1 BOBCAT trial
Autolus Therapeutics
2025-10-20
phase 1
Autolus Therapeutics (AUTL) announces that the first patient has been dosed in its Phase 1 BOBCAT trial of obecabtagene autoleucel in patients with progressive mult...
Groundbreaking Study in the Journal of Clinical Investigation Advances Cell Therapy for Huntington's Disease Treatment
uniQure
2025-10-20
cell therapy
SHANGHAI, Oct. 20, 2025 /PRNewswire/ -- The research team led by Dr. Yuejun Chen, founder of UniXell Biotech, published online an article titled '3D-Cultured Human Medium Spiny Neurons Achieve Functional Integration into Huntingtin Disease Mouse Neural Circuits and Improve Motor Deficits' in the Journal of Clinical Investigation, collaborating closely with Xiong Lab at Fudan University, Shanghai. This study presents a method for generating authentic medium spiny neurons (MSNs) and provides comprehensive mechanistic insights that underpin cell-replacement therapy for Huntington's disease.
Adaptimmune Announces Delisting from Nasdaq
Adaptimmune Therapeutics
2025-10-20
bla
sarcoma
tecelra
solid tumor
cell therapy
cell therapies
synovial sarcoma
letetresgene autoleucel
afamitresgene autoleucel
Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - October 20, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP) ("Adaptimmune" or the "Company") today announced that the Company's Board of Directors (the "Board") has determined to initiate the delisting of its American Depositary Shares ("ADSs"), each representing six ordinary shares, from The Nasdaq Capital Market ("Nasdaq") and deregistration under Section 12(b) of the Securities Exchange Act of 1934, as amended (the "Exchange Act"). Adrian Rawcliffe, Adaptimmune's Chief Executive Officer: "As we noted previously, the transaction with USWM CT, LLC, a subsidiary of US WorldMeds Partners, LLC (collectively, "US WorldMeds") followed an extensive review of strategic alternatives and represented the best path forward for Adaptimmune, our patients and other stakeholders. Since closing the transaction on July 31, 2025, we have been restructuring to support the assets transferred to US WorldMeds, and have continued our efforts to maximize value from our remaining assets including programs targeting PRAME and CD70. The delisting from Nasdaq and deregistration should facilitate cost reductions and further support…
ESMO: Ivonescimab beats BeOne PD-1 in first-line lung cancer
PharmaPhorum
2025-10-20
first-line
Akeso/Summit's PD-1xVEGF drug ivonescimab bests a PD-1 inhibitor as first-line therapy in Chinese NSCLC patients, but a bigger test is yet to come.
Immatics Highlights Compelling Anti-Tumor Activity of Anzu-cel PRAME Cell Therapy in Metastatic Uveal Melanoma at the ESMO 2025 Presidential Symposium
Immatics Biotechnologies
2025-10-20
melanoma
cell therapy
One-time infusion of anzu-cel (anzutresgene autoleucel, IMA203) PRAME cell therapy in 16 patients with metastatic uveal melanoma in the ongoing Phase 1b trial continues to show strong clinical benefit with more mature data: cORR of 67%, mDOR of 11.0 months, mPFS of 8.5 months and mOS not reached at
ProQR Receives CTA Authorization for AX-0810 and Announces Virtual Investor and Analyst Event on November 3, 2025
proqr therapeutics
2025-10-20
cta
phase 1
LEIDEN, Netherlands & CAMBRIDGE, Mass., Oct. 20, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that following review under the new European Medicines Agency (EMA) centralized review process, the Central Committee on Research Involving Human Subjects (CCMO) has authorized ProQR’s Clinical Trial Application (CTA) for a Phase 1 study of AX-0810 in healthy volunteers. AX-0810 is the Company’s lead investigational editing oligonucleotide (EON) targeting NTCP, which is being developed for the treatment of cholestatic diseases like primary sclerosing cholangitis and biliary atresia.
Autolus Therapeutics Announces First Patient Dosed in Phase 1 Trial of Obecabtagene Autoleucel (Obe-Cel) in Patients with Progressive Multiple Sclerosis
Autolus Therapeutics
2025-10-20
car t
phase 1
cell therapies
LONDON and Gaithersburg, Md., Oct. 20, 2025 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, announces that the first patient has been dosed in its Phase 1 BOBCAT trial of obecabtagene autoleucel (obe-cel) in patients with progressive multiple sclerosis (PMS). The patient was treated at University College London Hospitals NHS Foundation Trust (UCLH), a leading CAR T and neurological disorders treatment center in the United Kingdom and, with UCL, development partner of Autolus for obe-cel.
Genentech wins approval of lupus drug obinutuzumab
Genentech
2025-10-20
phase 3
The US FDA approved Gazyva (obinutuzumab), a lupus nephritis treatment from Roche-owned (OTCQX:RHHBY) Genentech. Gazyva is given via infusion four times in the first year, followed by twice a year after that. In a phase 3 study, 46.4% of patients on Gazyva and standard therapy achieved a complete renal response vs. 33.1% on standard therapy alone. ...
All 3 patients in trial to date see benefits with treatment for AML
Rare Cancer News
2025-10-20
aml
phase 1
phase 1/2
acute myeloid leukemia
All three adults with high-risk acute myeloid leukemia (AML) who had already been given 3-4 lines of cancer treatment responded to a combination of Venclexta (venetoclax) and AB Science‘s AB8939 after the first treatment cycle, with one patient achieving complete remission. That’s according to early data from the AB18001 Phase 1/2 (NCT05211570) clinical trial, which […] The post All 3 patients in trial to date see benefits with treatment for AML appeared first on Rare Cancer News .
Genentech Adds Lupus Nephritis To Cancer Indications On Gazyva’s Label
Genentech
2025-10-20
cd20
Seeking to compete with GSK’s Benlysta, the Roche unit obtains the first FDA approval of an anti-CD20 agent in lupus nephritis, a complication of lupus that can lead to end-stage kidney disease.
Autolus Therapeutics Announces First Patient Dosed in Phase 1 Trial of Obecabtagene Autoleucel (Obe-Cel) in Patients with Progressive Multiple Sclerosis
syncona
2025-10-20
multiple sclerosis
Retinal Physician | Article and Video Interview with Allen C. Ho, MD: Optogenetic Therapy Offers Renewed Hope in Retinitis Pigmentosa
Nanoscope Therapeutics
2025-10-19
retinitis pigmentosa
The post Retinal Physician | Article and Video Interview with Allen C. Ho, MD: Optogenetic Therapy Offers Renewed Hope in Retinitis Pigmentosa appeared first on Nanoscope Therapeutics .
PSMAddition data show Novartis Pluvicto™ delays progression to end-stage prostate cancer
Novartis
2025-10-19
prostate cancer
19 Oct 2025 Case Report | Preliminary Clinical Data of CARsgen’s Allogeneic BCMA CAR-T Product CT0596 for the Treatment of Primary Plasma Cell Leukemia Oct 19, 2025
Carsgen Therapeutics
2025-10-19
bcma
car-t
leukemia
allogeneic
IZERVAY™ (avacincaptad pegol intravitreal solution) Showed Increased Benefit in Reducing Geographic Atrophy Progression Over Time and Consistent Long-Term Safety
astellas
2025-10-19
open
In GATHER2 open-label extension study, IZERVAY reduced GA growth 37-40.5% vs. projected sham, with earlier intervention resulting in greater protection of retinal tissue area Study results found...
PADCEV™ Plus KEYTRUDA™, Given Before and After Surgery, Cuts the Risk of Recurrence, Progression or Death by 60% and the Risk of Death by 50% for Certain Patients with Bladder Cancer
astellas
2025-10-19
bladder cancer
First and only combination regimen to improve survival when used before and after surgical cystectomy in cisplatin-ineligible patients with muscle-invasive bladder cancer Unprecedented survival...
Quarterly Activities/Appendix 4C Cash Flow Report
Mesoblast
2025-10-19
quarterly
BioCryst’s $700M Astria Acquisition Gives It a Shot to Beat Larger Rivals in Rare Disease HAE
MedCity News
2025-10-19
phase 3
hereditary angioedema
BioCryst Pharmaceuticals’ Astria Therapeutics acquisition brings navenibart, a drug in Phase 3 testing for the rare disease hereditary angioedema. The injectable antibody has the potential to match the efficacy of the blockbuster Takeda Pharmaceutical drug that currently dominates the market, but with less burdensome dosing that could encourage HAE patients to switch. The post BioCryst’s $700M Astria Acquisition Gives It a Shot to Beat Larger Rivals in Rare Disease HAE appeared first on MedCity News .
Health matters by alice park - 18 Oct 2025 - Time Magazine Europe
uniQure
2025-10-18
gene therapy
Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first...
Aurion Biotech Achieves All Primary, Secondary, and Exploratory Endpoints in AURN001 Phase 1/2 CLARA Trial at 12 Months
aurion biotech
2025-10-18
phase 1
phase 3
pivotal
phase 1/2
cell therapy
Data strongly supports advancing high dose AURN001 regenerative cell therapy for patients with corneal endothelial disease to pivotal Phase 3 trial in H1 2026 Seattle, WA; Cambridge, MA; Tokyo, Japan – 18 October – Aurion Biotech, a clinical-stage regenerative medicine company with a mission to restore vision to millions of patients, today announced positive 12-month […]
A Look at Krystal Biotech's Valuation Following FDA Platform Technology Designation for Gene Therapy Vector
Krystal Biotech
2025-10-18
gene therapy
U.S. stocks, Saudi stocks, stock trading and investment platforms
Does QURE's High Short Interest Reveal Deeper Questions About Its Gene Therapy Partnership Strategy?
uniQure
2025-10-18
gene therapy
U.S. stocks, Saudi stocks, stock trading and investment platforms
ESMO: Boehringer, Bayer seek first-line HER2+ NSCLC role
PharmaPhorum
2025-10-18
her2
first-line
New trial data back the use of Boehringer's new oral HER2 inhibitor Hernexeos in front-line treatment of HER2+ NSCLC, but Bayer is in hot pursuit.
ESMO: Grail's data on cancer blood test firms up
PharmaPhorum
2025-10-18
phase 3
Data from a new phase 3 trial of Grail's blood test for multiple cancers show improved efficacy and a lower rate of false alarms than a prior study.
Vyjuvek treatment helps infant with DEB achieve motor milestones
Epidermolysis Bullosa News
2025-10-17
vyjuvek
gene therapy
dystrophic epidermolysis bullosa
The gene therapy Vyjuvek healed wounds and led to motor skill development of an infant with dystrophic epidermolysis bullosa, a study shows.
Vertex Announces Progress in Povetacicept Development Program and Presentation of New Data at American Society of Nephrology Kidney Week
Vertex
2025-10-17
pivotal
Food and Drug Administration grants rolling review of Biologics License Application for povetacicept in IgA nephropathy; Vertex to submit first module before end of year - - Second pivotal development program of povetacicept underway with Phase 2b/3 trial initiation in primary membranous
AbbVie Completes Acquisition of Gilgamesh Pharmaceuticals' Bretisilocin
Abbvie
2025-10-17
phase 2
Acquisition expands AbbVie's psychiatry pipeline with the addition of a next-generation psychedelic compound currently in Phase 2 development for the treatment of major depressive disorder (MDD)....
Ferring Expands Growing Body of Real-World Evidence Reinforcing Use of ADSTILADRIN® (nadofaragene firadenovec-vncg) in Urology Practices
Ferring Pharmaceuticals
2025-10-17
adstiladrin
Pelage Pharmaceuticals’ alopecia treatment moves forward to phase 3 trial
PMLive Regulatory
2025-10-17
phase 3
The news follows positive phase 2a results for the androgenetic alopecia treatment
Iovance Biotherapeutics Reports Inducement Grants under NASDAQ Listing Rule 5635(c)(4)
Iovance Biotherapeutics
2025-10-17
til
SAN CARLOS, Calif. , Oct. 17, 2025 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA) ("Iovance" or the “Company”), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (“TIL”) therapies for patients with cancer,
Autolus Therapeutics Plc (AUTL) Stock Analysis: Exploring A 472.75% Potential Upside In T Cell Therapy Innovation
Autolus Therapeutics
2025-10-17
cell therapy
Autolus Therapeutics plc (AUTL) Stock Analysis: Exploring a 472.75% Potential Upside in T Cell Therapy Innovation
Novartis Kisqali® 5-year NATALEE data demonstrate 28% reduction in risk of recurrence in the broadest early breast cancer patient population
Novartis
2025-10-17
breast cancer
BMS acquires Orbital Therapeutics in $1.5bn deal
PMLive Research
2025-10-16
cell therapy
The deal includes several next-gen cell therapy candidates for autoimmune diseases
From data to decisions: Prime’s GLP-1 insights inform clinical strategy
Prime Therapeutics
2025-10-16
persistence
In the latest episode of the “Pharmacy Friends” podcast, join health outcomes experts for a discussion about Prime’s industry-leading research into persistence and adherence of glucagon-like peptide-1 (GLP-1) agonist medications.
Taysha Gene Therapies Regains Full Rights to Lead TSHA-102 Program in Clinical Evaluation for the Treatment of Rett Syndrome
Taysha Gene Therapies
2025-10-16
phase 1/2
rett syndrome
gene therapies
The 2022 Option Agreement between Astellas and Taysha has expired Regaining full rights to TSHA-102 Rett syndrome program enables Taysha to focus on driving its long-term value with full strategic flexibility and optionality TSHA-102 has demonstrated compelling Part A REVEAL Phase 1/2 safety and
OpenEvidence and Veeva Announce Open Vista Partnership
PharmaVoice
2025-10-16
open
News for pharmacy pros
Adicet Bio Announces First Patient Dosed in Phase 1 Clinical Trial of ADI-001 in Treatment-Refractory Rheumatoid Arthritis (RA)
Adicet Bio
2025-10-16
phase 1
allogeneic
cell therapies
gamma delta t cell
Preliminary clinical data from the Phase 1 trial expected in 2H/2026 REDWOOD CITY, Calif. --(BUSINESS WIRE)--Oct. 16, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer,
Cellectis’ R&D Day Highlights Lasme-cel’s Potential to Address Significant Unmet Need for Patients with r/r B-ALL
cellectis
2025-10-16
bla
crs
orr
cd19
cd22
b-all
car-t
icans
phase 1
phase 2
pivotal
allogeneic
autologous
first line
persistence
second line
cell therapy
gene editing
gene therapy
neurotoxicity
gene therapies
complete response
overall response rate
hematopoietic stem cell
cytokine release syndrome
b-cell acute lymphoblastic leukemia
immune effector cell–associated neurotoxicity syndrome
Efficacy: ORR of 68% with lasme-cel Process 2 (n=22), 83% at RP2D (n=12) and 100% in the target Phase 2 population (n=9) Safety: in Phase 1 (n=40), lasme-cel was generally well-tolerated (including 1 case of grade 2 IEC-HS which resolved) Durability: in patients who achieved MRD-negative CR/CRi, median OS was 14.8 months In the target Phase 2 population, CR/CRi rate of 56% with ~80% of patients achieving MRD-negative status In the target Phase 2 population, 100% patients became transplant eligible with 78% proceeding to transplant Among 11 patients previously treated with all 3 targeted therapies (inotuzumab, blinatumomab, and CD19 CAR-T), 8 responded and 7 achieved MRD-negative status BALLI-01 pivotal Phase 2 in r/r B-ALL initiated Potential peak gross sales of up to ~$700 million across the U.S., EU4, UK New York, NY – October 16, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies , today hosted an R&D Day providing promising clinical data from the Phase 1 BALLI-01 study of lasme-cel (UCART22) for transplant ineligible patients…
Arcellx to Participate at the 2025 Morgan Stanley Global Healthcare Conference
arcellx, inc
2025-10-16
cell therapy
cell therapies
Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, today announced that management will participate in a fireside chat at the 2025 Morgan Stanley Global Healthcare Conference on Wednesday, September 10, 2025 at 1:05 p.m. ET. A live webcast of this discussion will be accessible from Arcellx's website at www.arcellx.com in the Investors section. A replay of the webcast will be archived and available for 30 days following the event. About Arcellx, Inc. Arcellx, Inc. is a clinical-stage biotechnology company reimagining cell therapy by engineering innovative immunotherapies for patients with cancer and other incurable diseases. Arcellx believes that cell therapies are one of the forward pillars of medicine and Arcellx's mission is to advance humanity by developing cell therapies that are safer, more effective, and more broadly accessible. For more
Cellectis Hosts R&D Day Today Showcasing Pipeline Progress and Long-Term Value Drivers
cellectis
2025-10-16
b-all
phase 1
phase 2
pivotal
pipeline
gene editing
gene therapies
Event features full Phase 1 data and pivotal Phase 2 strategy for lasme-cel (UCART22) in r/r B-ALL New York, NY – October 16, 2025 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, today hosts a R&D Day in New York City. The Company’s leadership team and key opinion leaders will present the full Phase 1 dataset and outline the pivotal Phase 2 trial design and commercial opportunity for lasme-cel in r/r B-ALL. Details of the Event: Date: Today, October 16, 2025 Time: 08:30 – 10:30 a.m. ET Format: In-person and live webcast Webcast: Join live via https://us06web.zoom.us/j/84630848404 A replay will be available after the event on the Cellectis website
OTC-HOPE: The first in vivo, liver directed, AAV-mediated, gene insertion clinical trial in infants with Ornithine Transcarbamylase Deficiency
Iecure
2025-10-16
aav
ornithine transcarbamylase
ornithine transcarbamylase deficiency
American Society of Human Genetics (ASHG) Annual Meeting October 16, 2025 The post OTC-HOPE: The first in vivo, liver directed, AAV-mediated, gene insertion clinical trial in infants with Ornithine Transcarbamylase Deficiency first appeared on iECURE .
Axogen, Inc. to Report 2025 Third Quarter Financial Results on October 29, 2025
axogen
2025-10-15
financial results
BMS Deepens ALS Alliance With Insitro, Puts $2B+ On the Line
BioSpace
2025-10-15
amyotrophic lateral sclerosis
Bristol Myers Squibb and insitro first partnered in 2020 to develop induced pluripotent stem cell models of amyotrophic lateral sclerosis and frontotemporal dementia. Last December, BMS exercised its option for an ALS target.
AstraZeneca to invest $4.5bn in US manufacturing facility
PMLive Healthcare
2025-10-15
pipeline
The investment will further support the company’s growing oncology pipeline
Prime’s strategic vision for smarter, member-centered breast cancer care
Prime Therapeutics
2025-10-15
breast cancer
As breast cancer treatment evolves, Prime stays ahead of emerging therapies and technologies — helping members access breakthrough care while supporting clients in managing cost and complexity
Sarepta’s Not Done With Gene Therapy Yet as LGMD Asset Yields Promising Data
BioSpace
2025-10-15
sarepta
gene therapy
Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene therapy: its limb-girdle muscular dystrophy candidate. But the treatment’s path forward, analysts say, is highly uncertain.
MIMEDX to Host Third Quarter 2025 Operating and Financial Results Conference Call on October 29
mimedx
2025-10-15
financial results
MARIETTA, Ga., Oct. 15, 2025 (GLOBE NEWSWIRE) -- MiMedx Group, Inc. (Nasdaq: MDXG) (“MIMEDX” or the “Company”) today announced that it will report its operating and financial results for the third quarter ended September 30, 2025 after the market close on Wednesday, October 29, 2025.
Vanessa Almendro-Navarro: Baby KJ and The Promise of Custom Gene Editing
Inside Precision Medicine
2025-10-15
gene editing
Today, Behind the Breakthroughs turns one year old. To celebrate, the episode will be a deep dive into the biggest precision medicine news of the year: the story of Baby KJ. The post Vanessa Almendro-Navarro: Baby KJ and The Promise of Custom Gene Editing appeared first on Inside Precision Medicine .
Landmark Bio Forges New Collaboration to Accelerate Early-Stage Translational Cell and Gene Therapies
landmark bio
2025-10-15
cell therapy
gene therapies
Agreement establishes framework for advancing Mass General Brigham-originated cell and gene therapies. Landmark Bio to support early-stage development and manufacturing through collaboration with Mass General Brigham’s Gene and Cell Therapy Institute. The post Landmark Bio Forges New Collaboration to Accelerate Early-Stage Translational Cell and Gene Therapies first appeared on Landmark Bio .
Lilly says oral GLP-1 could be 'foundational' diabetes drug
PharmaPhorum
2025-10-15
phase 3
Eli Lilly has built the case for its oral GLP-1 orforglipron with two more phase 3 readouts, including a head-to-head win over Farxiga in diabetes.
Q3 Earnings Are Here, Novo Ditches Cell Therapy but Buys Akero, Gov’t Shutdown Hits CDC
BioSpace
2025-10-15
cell therapy
Johnson & Johnson has yet to make a drug pricing deal with Trump; Novo makes more moves under new CEO; more than 1,000 laid off from CDC, though many immediately hired back; the BIOSECURE Act is back and more.
MRD-Guided Anti-CD20 Retreatment May Delay CLL Relapse
Hematology Advisor News
2025-10-15
cll
cd20
Time to next treatment ranged from 0 months to 15.8 years, with a median of 2.3 years.
FDA’s Second Platform Designation Goes to Krystal After Sarepta Withdrawal
Krystal Biotech
2025-10-15
sarepta
krystal biotech
sarepta therapeutics
After the FDA revoked Sarepta Therapeutics’ platform designation in July, Krystal Biotech is “poised to be the first major beneficiary” of this program, according to analysts at William Blair.
Insider Confidence Shines: Krystal Biotech and Growth Stocks with Strong Management Stakes Navigate Volatile Markets
Krystal Biotech
2025-10-15
hsv vector
gene therapy
The U.S. stock market is currently a landscape of contrasting forces, with robust bank earnings providing a bedrock of stability against the backdrop of persistent trade tensions with China and intermittent volatility. In this nuanced environment, a compelling narrative is emerging around growth stocks exhibiting significant insider ownership. This trend suggests a strong alignment between company leadership and shareholder interests, potentially signaling resilience and long-term conviction in an otherwise uncertain market. Krystal Biotech (NASDAQ: KRYS), alongside other promising growth companies, stands out in this regard, offering investors a glimpse into firms where those at the helm have a substantial personal stake in their success.Insider Stakes: A Deep Dive into Krystal Biotech, Upstart Holdings, and Atour Lifestyle HoldingsThe current financial climate, marked by fluctuating indices and a CBOE Volatility Index (VIX) that signals ongoing market choppiness, places a premium on indicators of corporate strength and stability. While major U.S. banks like JPMorgan Chase (NYSE: JPM) and Bank of America (NYSE: BAC) have reported stronger-than-expected Q3 2025 earnings, providing…
Brexucabtagene Autoleucel Unlikely to Yield Complete Response in Relapsed, Refractory CLL
Hematology Advisor News
2025-10-15
cll
complete response
Researchers determined brexucabtagene autoleucel may not yield high response rates among patients with relapsed or refractory CLL.
FDA’s Second Platform Designation Goes to Krystal After Sarepta Withdrawal
BioSpace
2025-10-15
sarepta
After the FDA revoked Sarepta Therapeutics’ platform designation in July, Krystal Biotech is “poised to be the first major beneficiary” of this program, according to analysts at William Blair.
BioCryst Snaps Up Fellow Angiodema Player Astria for $700M
BioSpace
2025-10-15
hereditary angioedema
The acquisition will give BioCryst an investigational injectable drug for hereditary angioedema, potentially complementing its FDA-approved oral drug Orladeyo.
Does FDA’s Platform Technology Designation Redefine the Innovation Thesis for Krystal Biotech (KRYS)?
Krystal Biotech
2025-10-14
gene therapy
viral vector
Krystal Biotech recently announced that the FDA has granted platform technology designation to its genetically modified, non-replicating HSV-1 viral vector used in the KB801 redosable eye drop gene therapy, which is under evaluation for neurotrophic keratitis. This regulatory recognition is designed to streamline development and review processes for Krystal’s genetic medicines, potentially accelerating its broader pipeline and reinforcing confidence in the company’s technology...
Precision BioSciences Announces Late-Breaking Oral Presentation for PBGENE-HBV at AASLD The Liver Meeting® 2025
precision biosciences
2025-10-14
gene editing
DURHAM, N.C. --(BUSINESS WIRE)--Oct. 14, 2025-- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS ® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that it has been selected to
Krystal Biotech wins second-ever FDA platform designation for viral vector
Krystal Biotech
2025-10-14
sarepta
viral vector
Krystal Biotech receives FDA platform technology designation for HSV-1 viral vector, becoming second company after Sarepta to receive this status.
Boehringer, Click DTx for schizophrenia clears pivotal trial
PharmaPhorum
2025-10-14
pivotal
Boehringer Ingelheim and Click Therapeutics have revealed the data from their pivotal trial of a digital therapeutic for schizophrenia.
How we prepared our child with AADC deficiency for gene therapy
AADC News/BioNews
2025-10-14
gene therapy
For many children with aromatic l-amino acid decarboxylase (AADC) deficiency, gene therapy holds opportunities for the future. It’s a chance for them to move, speak, and experience life in ways that once seemed impossible. I have often written about the life-changing benefits of gene therapy. Yet, the unfortunate truth remains that some children still haven’t […] The post How we prepared our child with AADC deficiency for gene therapy appeared first on AADC News .
FDA Platform Designation: Krystal Biotech's HSV‑1 Viral Vector for KB801 to Advance Neurotrophic Keratitis Treatment
Krystal Biotech
2025-10-14
vyjuvek
viral vector
FDA grants platform technology designation to Krystal Biotech's non‑replicating HSV‑1 vector used in KB801, which is in a randomized placebo‑controlled trial for neurotrophic keratitis; designation may allow leveraging VYJUVEK data and earlier FDA engagement.
Roche moves SC against Natco’s launch of generic Risdiplam in India
Roche Group
2025-10-14
spinal muscular atrophy
F. Hoffmann-La Roche AG has approached the Supreme Court. This follows a Delhi High Court decision permitting Natco Pharma to introduce a generic version of Risdiplam. Risdiplam treats spinal muscular atrophy. The High Court rejected Roche's plea to halt Natco's manufacturing. Natco plans to sell the drug at a significantly lower price.
Blood-Based Protein Biomarkers Linked to Acute Pain in Sickle Cell Disease
Hematology Advisor News
2025-10-14
sickle cell disease
Twenty-eight blood-based protein biomarkers, including substance P and CRP, are elevated during sickle cell vaso-occlusive episodes, but not chronic pain.
What a gene therapy win means for the Huntington’s disease pipeline
uniQure
2025-10-14
gene therapy
Recent results for a Huntington’s gene therapy confer a hopeful sign for R&D in the space.
Monoclonal antibody effects persist for 6 months in patients
Healio
2025-10-14
phase 1
Patients with mild to moderate asthma receiving one dose of an anti-thymic stromal lymphopoietin monoclonal antibody experienced decreases in biomarkers that lasted through 6 months, according to interim results from a phase 1 trial.These results on GB-0895 (Generate:Biomedicines) were presented at the European Respiratory Society International Congress.
Krystal Biotech Receives FDA Platform Technology Designation for HSV-1 Viral Vector Used in KB801 for the Treatment of Neurotrophic Keratitis
Krystal Biotech
2025-10-14
viral vector
PITTSBURGH, Oct. 14, 2025 -- Krystal Biotech, Inc. announced today that the United States Food and Drug Administration granted platform technology designation to the genetically modified,...
Fate Therapeutics Expands Leadership Team with Appointment of Kamal Adawi as Chief Financial Officer
Fate Therapeutics
2025-10-14
pipeline
SAN DIEGO , Oct. 14, 2025 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE) (the Company), a clinical-stage biopharmaceutical company dedicated to bringing a pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients, today announced the
Kailera Secures $600M to Advance Dual-Targeting Obesity Drug to Global Phase 3 Tests
MedCity News
2025-10-14
phase 3
Bain Capital Private Equity led Kailera Therapeutics’ Series B financing, which will support global Phase 3 tests of KAI-9531, a weekly injectable drug that hits the same targets as Eli Lilly’s blockbuster obesity medication, Zepbound. The capital will also support development of other oral and injectable metabolic medicines in Kailera’s pipeline. The post Kailera Secures $600M to Advance Dual-Targeting Obesity Drug to Global Phase 3 Tests appeared first on MedCity News .
FDA Grants Platform Technology Status to Krystal Biotech's Gene Therapy
Krystal Biotech
2025-10-14
gene therapy
viral vector
Key Takeaways: Krystal Biotech (KRYS) has received platform technology designation from the U.S. FDA for its viral vector technology. The company's KB801
Latest Novo Cuts Affect Almost 250 Cell Therapy Employees
BioSpace
2025-10-14
cell therapy
Follow along as BioSpace tracks job cuts and restructuring initiatives.
Krystal Biotech Gets US FDA Platform Technology Designation for HSV-1 Viral Vector Used in KB801
Krystal Biotech
2025-10-14
viral vector
Krystal Biotech said Tuesday that the US Food and Drug Administration has granted platform technology designation to the genetically modified, non-replicating herpes simplex virus type 1, or...
Astellas suffers pancreatic cancer failure with Vyloy
PharmaPhorum
2025-10-14
claudin 18.2
pancreatic cancer
Astellas' effort to expand the reach of its first-in-class claudin 18.2 drug Vyloy into pancreatic cancer has had a major setback.
$600m round will help Kailera push obesity drug into phase 3
PharmaPhorum
2025-10-14
phase 3
pivotal
Kailera Therapeutics has raised an eye-watering $600 million in Series B financing to take its lead drug for weight loss into pivotal trials.
Single Nucleotide Edit Fixes Heart Muscle Disease
Inside Precision Medicine
2025-10-14
gene editing
Meticulous gene editing involving a single nucleotide base change in the genome could alleviate some cardiomyopathies, a proof-of-concept study suggests. The post Single Nucleotide Edit Fixes Heart Muscle Disease appeared first on Inside Precision Medicine .
Layoff Tracker: Latest Novo Cuts Affect Almost 250 Cell Therapy Employees
Ferring Pharmaceuticals
2025-10-14
cell therapy
Follow along as BioSpace tracks job cuts and restructuring initiatives.
Celularity Announces Peer-Reviewed Publication of Phase 2 Clinical Trial Results Demonstrating the Safety and Efficacy of Human Placenta-Derived Cells (PDA-002) for Diabetic Foot Ulcers Complicated by Peripheral Artery Disease
celularity
2025-10-14
phase 2
Rocket Pharmaceuticals Announces FDA Acceptance of BLA Resubmission of KRESLADI™ for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)
Rocket Pharmaceuticals
2025-10-14
bla
pdufa
kresladi
Prescription Drug User Fee Act (PDUFA) target action date is March 28, 2026 All primary and secondary endpoints were met, and KRESLADI™ was well tolerated Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher, should KRESLADI ™ be approved CRANBURY, N.J. --(BUSINESS WIRE)--Oct.
Novo Retreats From Cell Therapy, Axes Hundreds as Restructuring Rolls On
BioSpace
2025-10-13
cell therapy
cell therapies
Novo had around 250 employees working on cell therapies, all of whom will be laid off, though a spokesperson declined to reveal which offices and locations will be affected.
Regeneron Readies FDA Run for Hearing Loss Gene Therapy That Could 'Transform Patients’ Lives'
BioSpace
2025-10-13
gene therapy
Regeneron is aiming to file a regulatory application for DB-OTO by the end of the year.
Abeona Therapeutics® Announces ABO-503 Gene Therapy for X-linked Retinoschisis (XLRS) Selected by FDA for Rare Disease Endpoint Advancement (RDEA) Pilot Program
Abeona Therapeutics
2025-10-13
gene therapy
Moderna Claims Early Victory for Cancer Antigen in Melanoma
BioSpace
2025-10-13
melanoma
Moderna’s mRNA-4359, when used with Keytruda, achieves a 24% overall objective response rate in patients with melanoma, with efficacy increasing to 67% in those positive for PD-L1.
Actinium Pharmaceuticals to Unveil the Multi-Tumor Potential of ATNM-400, a First-in-Class Actinium-225 Radiotherapy, with Data in Non-Small Cell Lung Cancer at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics
Actinium Pharma
2025-10-13
aml
cd33
psma
phase 2
pivotal
phase 2/3
quarterly
solid tumor
gene therapies
acute myeloid leukemia
ATNM-400 is advancing as a first-in-class, multi-tumor Actinium-225 radiotherapy candidate with activity across prostate and lung cancers, two of the largest cancer indications globally New preclinical findings demonstrate ATNM-400 overcomes resistance to the EGFR inhibitor osimertinib in EGFR-mutated NSCLC, addressing a major unmet clinical need NEW YORK , Oct. 13, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM), a leader in the development of differentiated targeted radiotherapies, today announced that the first-ever preclinical data from its ATNM-400 program in non-small cell lung cancer (NSCLC) has been accepted for presentation at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics being held October 22 – 26, 2025, at the Hynes Convention Center in Boston, Massachusetts. ATNM-400 is a highly innovative, first-in-class, Actinium-225 (Ac-225) antibody radioconjugate with preclinical data in both prostate cancer and NSCLC. Together, these two indications represent more than 500,000 new cases annually in the U.S. alone. ATNM-400 in NSCLC: Addressing Resistance to EGFR TKIs NSCLC accounts for approximately 85% of lung…
Consistency in the Chaos: FDA Approvals Within Average Range as Q4 Kicks Off
BioSpace
2025-10-13
gene therapy
Heading into the final quarter of a year that has seen dramatic upheaval at the FDA—from the exodus of numerous senior leaders to unclear policy changes and a safety saga that engulfed the gene therapy space—drug approvals appear roughly on par with recent years.
Gilead Sciences/Arcus Biosciences' Investigational Cancer Drug Shows Overall Survival Of Around 27 Months - Arcus Biosciences (NYSE:RCUS), Gilead Sciences (NASDAQ:GILD)
Gilead Sciences
2025-10-13
phase 2
Arcus Biosciences reports 26.7-month median survival and 59% response rate in Phase 2 cancer study with domvanalimab combination therapy.
Genentech Data at ESMO 2025 Showcase Advances in Science and Cancer Care Across Multiple Tumor Types
Genentech
2025-10-13
her2
pivotal
allogeneic
first-line
bladder cancer
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that it will present more than 30 abstracts across more than 10 cancer types at the European Society for Medical Oncology (ESMO) Congress 2025, held October 17-21, 2025 in Berlin, Germany. The data underscore Genentech’s commitment to deliver transformative medicines for some of the most challenging cancer types, including breast cancers, lung cancers, gastrointestinal and genitourinary cancers. Key presentations include: Giredestrant: Primary results from the Phase III evERA Breast Cancer study, the first positive head-to-head Phase III trial investigating an all-oral selective estrogen receptor (ER) degrader-containing regimen versus a standard of care combination in the post-cyclin-dependent kinase inhibitor setting for people with ER-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer. The study met both co-primary endpoints, demonstrating a statistically significant and clinically meaningful improvement in progression-free survival in both the intention-to-treat and ESR1-mutated populations. Data will be presented as a late-breaking…
Kernal Bio awarded up to $48M to develop blood cancer treatments
Rare Cancer News
2025-10-13
car t
cell therapies
The Advanced Research Projects Agency for Health (ARPA-H) has awarded up to $48 million to Kernal Bio to support the development of more efficient, cheaper CAR T-cell therapies for the treatment of some types of blood cancer. ARPA-H is an independent entity within the National Institutes of Health, created in 2022 to support health research […] The post Kernal Bio awarded up to $48M to develop blood cancer treatments appeared first on Rare Cancer News .
Verismo Therapeutics’ Trial for CAR-T SynKIR-310 Doses First Patient With Follicular Lymphoma
CGTLive
2025-10-13
car-t
follicular lymphoma
Verismo emphasized that the milestone strengthens its relationship with the Institute for Follicular Lymphoma Innovation.
AI in CAR T Development: Lessons From the Front Lines
BioSpace
2025-10-13
car t
cell therapy
While the benefits of AI are clear, the amount data sets needed for effective AI integration is proving to be a challenge. This is particularly true for cell therapy companies as they are eagerly seeking ways to reduce development costs. Two experts at Charles River Laboratories provide insights by giving their takeaways from their own AI integrations.
Actinium to Present ATNM-400 Preclinical Data Highlighting Durable Tumor Control and Ability to Overcome Resistance to Standard-of-Care Prostate Cancer Therapies at the 32nd Annual Prostate Cancer Foundation Scientific Retreat
Actinium Pharma
2025-10-13
aml
cd33
psma
phase 2
phase 2/3
quarterly
solid tumor
gene therapies
acute myeloid leukemia
ATNM-400 targets a highly differentiated, non-PSMA antigen associated with the development and progression of prostate cancer, exhibiting potent therapeutic activity independent of PSMA expression levels - Extended follow-up in prostate cancer models demonstrated durable anti-tumor responses with ATNM-400, exceeding those achieved with the androgen receptor pathway inhibitor (ARPI), enzalutamide NEW YORK , Oct. 13, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of differentiated targeted radiotherapies, today announced that compelling preclinical data for ATNM-400, its novel, first-in-class antibody radioconjugate armed with the potent alpha-emitter Actinium-225 (Ac-225), has been accepted for presentation at the 32 nd Annual Prostate Cancer Foundation (PCF) Scientific Retreat being held on October 23 – 25, 2025 in Carlsbad, CA. The presentation will highlight ATNM-400's ability to induce robust, durable anti-tumor activity in metastatic castration-resistant prostate cancer (mCRPC) models, including tumors resistant to standard-of-care therapies such as the androgen receptor pathway inhibitor…
Gilead, Arcus keep TIGIT dream alive with 27-month OS data from phase 2 cancer cohort
Gilead Sciences
2025-10-13
phase 2
Gilead Sciences and Arcus Biosciences’ anti-TIGIT antibody domvanalimab has been tied to median overall survival of close to 27 months, keeping alive one of the last remaining hopes for this once-h | Gilead and Arcus’ anti-TIGIT antibody domvanalimab has been tied to median overall survival of almost 27 months, keeping alive one of the last remaining hopes for this once-hyped modality.
FDA approves labeling changes for Janssen’s Carvykti
Janssen Global Services
2025-10-13
car-t
carvykti
cell therapy
The US Food and Drug Administration (FDA) on Friday revealed it has received reports of immune effector cell-associated enterocolitis (IEC-EC) in patients who received treatment with Carvykti (ciltacabtagene autoleucel), a CAR-T cell therapy from Johnson & Johnson subsidiary Janssen.
BMS Joins the In Vivo Cell Therapy Chase With $1.5B Orbital Therapeutics Acquisition
MedCity News
2025-10-13
cell therapy
Startup Orbital Therapeutics brings Bristol Myers Squibb an in vivo cell therapy in development for treating autoimmune disease. With the deal, BMS joins AstraZeneca, AbbVie, and Gilead Sciences in turning to M&A as a way to get in vivo cell therapy assets and technologies. The post BMS Joins the In Vivo Cell Therapy Chase With $1.5B Orbital Therapeutics Acquisition appeared first on MedCity News .
Gene Therapy in Ophthalmology Market Revenue to Expand Significantly by 2034, States DelveInsight Report | REGENXBIO, GenSight Biologics, Neurophth Therapeutics
GenSight Biologics
2025-10-13
gene therapy
The Key Gene Therapy in Ophthalmology Companies in the market include - REGENXBIO, GenSight Biologics, Neurophth Therapeutics, and others. DelveInsight’s “Gene Therapy in Ophthalmology Market Insights, Epidemiology, and Market Forecast-2034″ report...
Gene Therapy in Ophthalmology Market Revenue to Expand Significantly by 2034, States DelveInsight Report | REGENXBIO, GenSight Biologics, Neurophth Therapeutics
GenSight Biologics
2025-10-13
gene therapy
The Key Gene Therapy in Ophthalmology Companies in the market include - REGENXBIO, GenSight Biologics, Neurophth Therapeutics, and others. DelveInsight’s “Gene Therapy in Ophthalmology Market Insights, Epidemiology, and Market Forecast-2034″ report...
FDA approves labeling changes for Janssen’s Carvykti
Janssen Global Services
2025-10-13
car
carvykti
cell therapy
The US Food and Drug Administration (FDA) on Friday revealed it has received reports of immune effector cell-associated enterocolitis (IEC-EC) in patients who received treatment with Carvykti (ciltacabtagene autoleucel), a CAR-T cell therapy from Johnson & Johnson subsidiary Janssen.
CG Oncology (CGON) Is Up 8.6% After Analysts Spotlight Late-Stage Bladder Cancer Therapy Data – Has the Bull Case Changed?
CG Oncology
2025-10-11
phase 3
bladder cancer
complete response
In recent days, several analysts including Guggenheim initiated coverage of CG Oncology, highlighting promising late-stage clinical trial data for its lead bladder cancer therapy, cretostimogene. This analyst attention comes as the company reported a 41.8% complete response rate at 24 months for high-risk patients in Phase 3 trials, reflecting growing optimism around upcoming FDA review prospects. Let's explore how analyst focus on cretostimogene's efficacy is influencing CG Oncology's...
What Is Graft vs Host Disease?
Hematology Advisor Features
2025-10-10
hsc
Graft vs host disease (GVHD) is a side effect of blood, marrow, or HSC transplantation that can affect patients receiving cancer treatment.
How Small-Scale Chromatography Models Transform mAb Viral Clearance and Process Characterization
GenEng News
2025-10-10
clearance
In this GEN webinar, our expert speaker, Matt Taylor, discusses how pre-packed chromatography columns can provide reliable small-scale models of the downstream mAb purification process. The post How Small-Scale Chromatography Models Transform mAb Viral Clearance and Process Characterization appeared first on GEN - Genetic Engineering and Biotechnology News .
FDA Approves Labeling Changes that Include a Boxed Warning for Immune Effector Cell-associated Enterocolitis Following Treatment with Ciltacabtagene Autoleucel (CARVYKTI, Janssen Biotech, Inc.)
FDA MedWatch
2025-10-10
carvykti
The FDA has received reports of immune effector cell-associated enterocolitis (IEC-EC) in patients who received treatment with CARVYKTI.
CRISPR Therapeutics Presents New Preclinical Data for CTX460™ Demonstrating In Vivo Gene Correction of Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase™ Editing Platform
CRISPR
2025-10-10
crispr
cell therapy
Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) highlight a potential best-in-class profile- -The AATD program, CTX460, is the first investigational candidate to emerge from the SyNTase editing platform and the Company expects to initiate a clinical trial with
Bristol Myers Squibb Strengthens and Diversifies Cell Therapy Portfolio with Acquisition of Orbital Therapeutics
Bristol Myers Squibb
2025-10-10
cell therapy
Bristol Myers Squibb Strengthens and Diversifies Cell Therapy Portfolio with Acquisition of Orbital Therapeutics
bristol myers squibb
2025-10-10
cell therapy
CG Oncology (CGON): Assessing Valuation Following Analyst Coverage and Positive Phase 3 Trial Results
CG Oncology
2025-10-10
phase 3
bladder cancer
CG Oncology (CGON) is in the spotlight after several analysts, including Guggenheim, initiated coverage. This attention is supported by promising late-stage trial results for its lead bladder cancer therapy and anticipation of an FDA review next year. See our latest analysis for CG Oncology. Momentum has been strongly building for CG Oncology, as the stock has climbed over 66% in the past 90 days, capped by an 11.55% one-week share price return following analyst bullishness and positive...
‘From Rs 18 lakh to Rs 6 lakh a year, I can finally afford therapy within my salary’: Delhi HC allows Natco to manufacture Roche’s rare disease drug
Roche Group
2025-10-10
spinal muscular atrophy
Natco Pharmaceuticals wins case against international pharmaceutical giant Roche; patients of spinal muscular atrophy heave a sigh of relief.
Chiesi’s Rare Disease Strategy Expands to Genetic Meds via Alliance With CRISPR Startup Arbor
MedCity News
2025-10-10
crispr
Chiesi Global Rare Diseases is committing up to $115 million to begin a collaboration on Arbor Biotechnologies’ in vivo gene-editing therapy for primary hyperoxaluria type 1, an inherited liver disorder. It’s the rare disease company’s first foray into genetic medicines. The post Chiesi’s Rare Disease Strategy Expands to Genetic Meds via Alliance With CRISPR Startup Arbor appeared first on MedCity News .
All main goals hit in trial testing AlphaMedix for pancreatic cancer
Rare Cancer News
2025-10-10
phase 2
pancreatic cancer
A Phase 2 clinical trial testing Sanofi‘s therapy candidate AlphaMedix (212Pb-DOTAMTATE) in people with a certain type of pancreatic cancer has hit all its main goals, according to new data announced by the company. Treatment with AlphaMedix led to tumor shrinkage, as well as gains in survival, among cancer patients taking part in the small trial, […] The post All main goals hit in trial testing AlphaMedix for pancreatic cancer appeared first on Rare Cancer News .
Hemophilia B gene therapy Hemgenix to be covered in Canada
CSL Behring
2025-10-10
hemgenix
gene therapy
hemophilia b
The cost of Hemgenix, a one-time hemophilia B gene therapy, will be covered for patients living in Canada, except in Quebec.
Rocket Pharmaceuticals Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Rocket Pharmaceuticals
2025-10-10
pipeline
CRANBURY, N.J. --(BUSINESS WIRE)--Oct. 10, 2025-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that on October 6, 2025 , the
Roche loses Risdiplam patent appeal as Indian court cites 'obviousness' | MLex | Specialist news and analysis on legal risk and regulation
Roche Group
2025-10-10
spinal muscular atrophy
In a landmark win for drug accessibility in India, a court upheld findings that Roche's Risdiplam patent was "obvious" and invalid, thereby allowing Natco Pharma to sell a cheaper generic for spinal muscular atrophy.
AskBio Presents Interim Safety Results from Phase 1/Phase 2 LION-CS101 Clinical Trial of AB-1003 in Participants with Limb-Girdle Muscular Dystrophy 2I/R9
askbio
2025-10-10
aav
lgmd
phase 1
phase 2
gene therapy
dose-escalation
Research Triangle Park, N.C. – October 10, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced it will present initial safety data from the first cohort of participants from its Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in participants with limb-girdle muscular dystrophy (LGMD) 2I/R9 at the 30th Annual International Congress of the World Muscle Society, taking place October 7–11, 2025, in Vienna, Austria. The presentation represents interim, blinded Cohort 1 safety data. Participants enrolled in Cohort 1 received a single intravenous infusion of AB-1003 or placebo and were followed for 52 weeks post-treatment during the main trial before entering a planned four-year long-term follow-up period. Safety assessments included adverse event monitoring, laboratory testing, physical exams, vital signs, electrocardiograms, and echocardiograms.1 There were no dose-limiting toxicities or serious adverse events reported up to 52 weeks post-treatment. Commonly reported (>2 participants) treatment-emergent adverse events were mild-to-moderate in severity and included…
Bristol Myers Squibb Moves Into In Vivo CAR T with $1.5 Billion Orbital Buy
Inside Precision Medicine
2025-10-10
car t
abecma
breyanzi
cell therapy
cell therapies
chimeric antigen receptor
BMS already owns two approved ex vivo , chimeric antigen receptor (CAR) T cell therapies, Breyanzi and Abecma, for treatment of blood cancers, but wants to expand its cell therapy pipeline. The post Bristol Myers Squibb Moves Into In Vivo CAR T with $1.5 Billion Orbital Buy appeared first on Inside Precision Medicine .
BMS builds in 'in vivo' cell therapy with $1.5bn Orbital buy
PharmaPhorum
2025-10-10
cell therapy
cell therapies
Bristol Myers Squibb has ended the week with an agreement to buy Orbital Therapeutics, a developer of in vivo cell therapies, for $1.5bn in cash.
Editas Medicine Reports In Vivo Proof-of-Concept Data for EDIT-401 at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress
Editas
2025-10-10
cell therapy
AviadoBio Looks to China for Eye-Based Gene Therapy in $413M UgeneX Pact
aviadobio
2025-10-10
gene therapy
The post AviadoBio Looks to China for Eye-Based Gene Therapy in $413M UgeneX Pact appeared first on AviadoBio .
No Elevated Risk for Renal Dysfunction Seen in Hemophilia B Patients Without Inhibitors
Hematology Advisor News
2025-10-10
hemophilia b
In the B-Natural study, patients with severe hemophilia (ie, factor IX
Data from GNT-018-IDES trial supports feasibility of imlifidase as pretreatment in gene therapy treatment for patients with Crigler–Najjar syndrome who are immune to AAV
Genethon
2025-10-10
aav
aav vector
gene therapy
PARIS, France (October 10, 2025) – Genethon, a worldwide pioneer and leader in research and development of gene therapy for rare genetic diseases, and Hansa Biopharma, a Sweden-based leader in IgG cleaving enzyme technology announced today that a patient with a rare liver disease and immunity to the AAV vector has been successfully treated with … Continued
BMS Makes $1.5B Cell Therapy Play With Orbital Takeover
BioSpace
2025-10-10
cell therapy
Investor reaction to the deal was muted, with BMO Capital Markets analysts saying they "continue to look for more" from Bristol Myers Squibb before they can "get excited about the near term turnaround story."
Statins significantly improve OS for certain prostate cancers treated with apalutamide
Healio
2025-10-10
phase 3
Statin use significantly improved OS for men with metastatic hormone-sensitive and nonmetastatic castrate-resistant prostate cancer treated with apalutamide, based on combination of individual patient data from two randomized phase 3 trials.A meta-analysis of more than 2,000 patients found statin exposure improved OS between 8% and 14% in these patient populations.
HC bins Roche's injunction plea, allows generic version of only drug for SMA | India News - The Times of India
Roche Group
2025-10-10
sma
India News: A division bench of Delhi high court Thursday rejected the appeal of pharma giant, Roche, seeking an injunction against Indian pharma company Natco Ph.
Lilly’s Omvoh shows improved outcomes in ulcerative colitis
PMLive Research
2025-10-10
phase 3
The phase 3 trial results demonstrated improved symptomatic, clinical and quality-of-life measures
Children's Hospital Colorado set to provide Zevaskyn for RDEB
Epidermolysis Bullosa News
2025-10-10
zevaskyn
dystrophic epidermolysis bullosa
recessive dystrophic epidermolysis bullosa
Children's Hospital Colorado has become a qualified treatment center for Zevaskyn for recessive dystrophic epidermolysis bullosa.
uniQure (QURE): Examining Valuation Following Positive Huntington’s Disease Gene Therapy Results
uniQure
2025-10-10
amt-130
gene therapy
uniQure (NasdaqGS:QURE) is attracting attention after reporting positive Phase I/II trial results for its Huntington’s disease gene therapy candidate, AMT-130. The data showed a significant slowing in disease progression. See our latest analysis for uniQure. uniQure’s story has quickly shifted from steady to headline-grabbing, thanks to the promising AMT-130 update. The stock’s recent 30-day share price return of 252.15% and a remarkable 1-year total shareholder return nearing 990% show...
Nobel Prizes Announced and Breakthroughs for Huntington’s and Rare Diseases
GenEng News
2025-10-10
gene editing
gene therapy
In this episode of GEN 's Touching Base , we discuss the Nobel prizes, positive early results from a clinical trial testing a gene therapy for Huntington’s disease, and plans to apply gene editing to rare liver diseases. The post Nobel Prizes Announced and Breakthroughs for Huntington’s and Rare Diseases appeared first on GEN - Genetic Engineering and Biotechnology News .
Natco Pharma up 6% as Delhi HC clears SMA drug, rejects Roche appeal
Roche Group
2025-10-10
sma
The buying on the counter came after the Delhi High Court dismissed an appeal by F. Hoffmann-La Roche AG, removing the last legal hurdle for Natco Pharma to launch a generic version of Risdiplam
Hemostemix Files for Ethics Approval of its Study of Vascular Dementia
Hemostemix
2025-10-09
phase 1
Calgary, Alberta, October 9, 2025 - Hemostemix (TSXV: HEM) (OTC: HMTXF) (FSE: 2VF0) (" Hemostemix " or the " Company ") is pleased to announce it has filed its Institutional Review Board (IRB) application for approval of its Phase 1 clinical trial titled “Treatment of Vascular Cognitive Impairment and Dementia with Angiogenic Cell Precursors (ACP-01)” . The 102-page submission marks a critical milestone in expanding the therapeutic reach of ACP-01.
Disrupting Gene Therapy: Inside Jim Wilson’s Rare Disease Revolution
Inside Precision Medicine
2025-10-09
gene therapy
The gene therapy pioneer describes the guiding principles of his recently founded business, RareTx, which develops globally accessible, reasonably priced medications for ultra orphan diseases. The post Disrupting Gene Therapy: Inside Jim Wilson’s Rare Disease Revolution appeared first on Inside Precision Medicine .
Taysha Gene Therapies Presents New Supplemental Data Analysis from Part A of the REVEAL Phase 1/2 Trials for TSHA-102 in Rett Syndrome at the 54th CNS Annual Meeting
Taysha Gene Therapies
2025-10-09
pivotal
phase 1/2
rett syndrome
gene therapies
Previously disclosed 100% response rate across all 10 patients in Part A for pivotal trial primary endpoint of gain/regain of ≥ one natural history defined developmental milestone assessed via video-evidenced review by independent central raters New supplemental analysis of validated, structured
Nurix Therapeutics Reports Third Quarter 2025 Financial Results and Provides a Corporate Update
nurix therapeutics
2025-10-09
cll
pivotal
Announced clinical development plans for bexobrutideg, including the initiation of pivotal trials in relapsed/refractory CLL in H2 2025 Presented preclinical data at EADV 2025 for GS-6791 (NX-0479), a novel IRAK4 degrader in collaboration with Gilead, showing potent pathway inhibition and efficacy
Legend Biotech (NasdaqGS:LEGN): Evaluating Valuation Following New Carvykti Agreement With Janssen Pharmaceuticals
Janssen Global Services
2025-10-09
carvykti
U.S. stocks, Saudi stocks, stock trading and investment platforms
Isatuximab-VRd Regimen Effective in Transplant-Ineligible Multiple Myeloma
Hematology Advisor News
2025-10-09
multiple myeloma
Subcutaneous isatuximab plus bortezomib, lenalidomide, and dexamethasone is effective in transplant-ineligible, newly diagnosed multiple myeloma.
Delhi HC allows Natco Pharma to sell generic Risdiplam, rejects Roche plea
Roche Group
2025-10-09
sma
The division bench upheld the single-judge bench’s findings, which cited public interest in ensuring affordable access to SMA treatment, allowing Natco Pharma to launch a lower-cost generic despite Roche’s patent claims.
Precision BioSciences Announces Publication in Nucleic Acids Research Elucidating the Mechanisms for High Efficiency Gene Insertion in Dividing and Non-Dividing Cells Using ARCUS Nucleases
precision biosciences
2025-10-09
base editing
gene editing
Publication describes how the unique qualities of ARCUS nucleases contribute to precise and efficient gene editing for gene insertion, single base editing, specific small and large deletions, and replacement of large stretches of genomic DNA - DURHAM, N.C. --(BUSINESS WIRE)--Oct.
Natco Pharma to launch generic of SMA drug Risdiplam with Delhi HC ruling on Roche’s appeal
Roche Group
2025-10-09
sma
Natco Pharma launches generic SMA drug at ₹15,900, following court ruling against Roche's unaffordable pricing.
Neurogene Announces Positive Regulatory Update for NGN-401 Gene Therapy in Rett Syndrome with Plans to Initiate Dosing in Embolden™ Registrational Trial in Q4 2025
neurogene
2025-10-09
gene therapy
rett syndrome
Completed discussions with FDA on registrational protocol; 13 sites to allow for rapid enrollment New preclinical data at ESGCT Congress highlight NGN-401 best-in-class potential and add to body of evidence that ICV administration is superior to IT-L in reaching key brain regions underlying Rett
CG Oncology (CGON): Valuation in Focus After Analyst Coverage and Promising Cretostimogene Phase 3 Results
CG Oncology
2025-10-09
phase 3
bladder cancer
CG Oncology (CGON) has come into focus following fresh analyst coverage and new clinical trial data. Recent updates highlight promising results for cretostimogene in high-risk non-muscle invasive bladder cancer, which is drawing investor attention to upcoming milestones. See our latest analysis for CG Oncology. After releasing encouraging Phase 3 data and landing fresh positive analyst coverage, CG Oncology’s share price momentum has shifted firmly upward, notching a 50% return over the past...
SparingVision Highlights Advances in Gene-Agnostic Gene Therapies at ESGCT 2025
Sparing Vision
2025-10-09
gene therapy
gene therapies
SparingVision Highlights Advances in Gene-Agnostic Gene Therapies at ESGCT 2025 Paris, 9 October 2025 – SparingVision (“the Company”), a clinical-stage genomic medicine company transforming the treatment of retinal disease, today announces the presentation of data on its two gene-agnostic gene therapy programs, SPVN06 and SPVN20, and related research, at the 32nd Annual Meeting of… The post SparingVision Highlights Advances in Gene-Agnostic Gene Therapies at ESGCT 2025 appeared first on SparingVision .
Will EnGene Join The Ranks Of Merck, Ferring, And ImmunityBio In Bladder Cancer Treatment?
enGene
2025-10-09
pivotal
bladder cancer
detalimogene voraplasmid
enGene Holdings Inc. (ENGN), which reported promising initial efficacy and safety data from the pivotal cohort of its LEGEND trial of Detalimogene voraplasmid last year, is expected to provide an update this quarter.
Delhi HC allows Natco Pharma to launch risdiplam, rejects Roche plea
Roche Group
2025-10-09
spinal muscular atrophy
The Delhi High Court has permitted Natco Pharma to launch its affordable generic version of risdiplam, a crucial drug for spinal muscular atrophy. This decision overturns a previous stay, allowing Natco to offer the medication at a significantly lower price than Swiss multinational Roche's imported Evrysdi. Roche expressed disappointment, emphasizing the importance of patent protection for innovation.
Gene Therapy Reverses SYNGAP1 Brain Disorder Symptoms in Mice
Inside Precision Medicine
2025-10-09
gene therapy
The gene therapy represents the first demonstration that SYNGAP1 gene supplementation can restore neurological function after symptoms have appeared. The post Gene Therapy Reverses SYNGAP1 Brain Disorder Symptoms in Mice appeared first on Inside Precision Medicine .
CG Oncology: Upgraded, Rising, But Should We Still Be Optimistic? (NASDAQ:CGON)
CG Oncology
2025-10-09
phase 3
bladder cancer
CG Oncology stands out with strong phase 3 data and a robust pipeline in bladder cancer. Click here to read my most recent analysis of CGON stock now.
Duchenne Muscular Dystrophy Drugs Market Global Forecast 2025-2032: Sarepta, Pfizer, BioMarin, Roche, Bayer, Solid Biosciences, Cytokinetics, and NS Pharma Drive Innovation and Commercial Expansion
BioMarin Pharmaceutical
2025-10-09
sarepta
duchenne muscular dystrophy
The Duchenne Muscular Dystrophy Drugs Market presents opportunities in advanced genetic therapies, digital health, and adaptive models amid rising global...
CSL (ASX:CSL): Evaluating Valuation After Canadian Access Win for HEMGENIX Gene Therapy
CSL Behring
2025-10-09
hemgenix
gene therapy
hemophilia b
CSL (ASX:CSL) has secured a new agreement that will give more Canadians access to HEMGENIX, its innovative gene therapy for hemophilia B. The Letter of Intent with Canadian health authorities represents a meaningful step for patient access. See our latest analysis for CSL. While this Canadian access win marks a bright spot for CSL, the bigger picture has been more challenging for shareholders. The past year’s -28.2% total shareholder return reflects both broad market pressures and investor...
FDA grants orphan drug designation to glioblastoma treatment
Rare Cancer News
2025-10-09
orphan drug
glioblastoma
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to an experimental therapy called cintredekin besudotox (IL13-PE38QQR), which Precision Neuromed is developing to treat glioblastoma, a form of glioma. The FDA gives this designation to investigational medicines that are designed to treat diseases affecting fewer than 200,000 people in the U.S. The […] The post FDA grants orphan drug designation to glioblastoma treatment appeared first on Rare Cancer News .
Is uniQure's Stock Poised for a Comeback?
uniQure
2025-10-09
amt-130
uniQure N.V.’s stock jumped 6.09% on promising clinical milestone, reinforcing investor confidence and market optimism. Analyst Upgrades Drive Momentum Wells Fargo raised its target price for uniQure due to AMT-130’s impressive results, suggesting a much higher valuation than before. Guggenheim has now set a much loftier price target for uniQure after positive developments in AMT-130’s…
REGENXBIO Announces Presentation at the American Academy of Ophthalmology 2025 Annual Meeting
REGENXBIO
2025-10-09
abbv-rgx-314
ROCKVILLE, Md. , Oct. 9, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present interim data from the Phase II ALTITUDE ® trial evaluating suprachoroidal delivery of surabgene lomparvovec (ABBV-RGX-314, sura-vec) for the treatment of diabetic retinopathy (DR) at the
Genethon Confirms 2-year Efficacy in Duchenne Muscular Dystrophy Patients Treated with Its Low Dose Micro-Dystrophin Gene Therapy (GNT0004) at the ESGCT 32nd Annual Congress
Genethon
2025-10-09
dmd
phase 1/2
gene therapy
duchenne muscular dystrophy
The Duchenne muscular dystrophy (DMD) patients experiencing long-term efficacy were treated with the therapeutic 3×10¹³ vg/kg dose of GNT0004 in the Phase 1/2 dose escalation portion of Genethon’s all-in-one Phase 1/2/3 European trial.The Phase 3 trial using the 3×10¹³ vg/kg dose, which is lower than doses used in other DMD gene therapy clinical trials and … Continued
CRISPR gene-editing therapy may benefit patients with amyloidosis, polyneuropathy
Healio
2025-10-09
cas9
crispr
phase 1
crispr-cas9
A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin greatly reduced transthyretin levels in patients with hereditary amyloidosis and polyneuropathy, according to the results of a phase 1 trial. As Healio previously reported, the therapy, nexiguran ziclumeran (nex-z, Intellia/Regeneron), reduced serum transthyretin levels in patients with cardiac amyloidosis by 89% at 28
CG Oncology (CGON): Valuation in Focus After Analyst Coverage and Promising Cretostimogene Phase 3 Results
CG Oncology
2025-10-09
phase 3
U.S. stocks, Saudi stocks, stock trading and investment platforms
With $4.7B Akero Acquisition, Novo Nordisk Maps Out Broader Strategy in MASH and Beyond
MedCity News
2025-10-09
phase 2
Novo Nordisk is acquiring Akero Therapeutics for drug candidate efruxifermin, which executive say could be first and best in the emerging class of FGF21 analogs for the fatty liver disease MASH. The deal comes months after Novo’s internal FGF21 drug candidate failed a Phase 2 trial. The post With $4.7B Akero Acquisition, Novo Nordisk Maps Out Broader Strategy in MASH and Beyond appeared first on MedCity News .
AviadoBio and UgeneX Therapeutics Announce Exclusive Option-License Agreement to Develop Next Generation Optogenetics Gene Therapy to Restore Vision in Patients with Retinal Disease
aviadobio
2025-10-09
gene therapy
The post AviadoBio and UgeneX Therapeutics Announce Exclusive Option-License Agreement to Develop Next Generation Optogenetics Gene Therapy to Restore Vision in Patients with Retinal Disease appeared first on AviadoBio .
AviadoBio secures option to rare eye disease gene therapy that could bring Chinese biotech $413M
aviadobio
2025-10-09
gene therapy
The post AviadoBio secures option to rare eye disease gene therapy that could bring Chinese biotech $413M appeared first on AviadoBio .
NKGen Biotech Provides Clarification on Quotation Display and Trading Status on the OTC Expert Market
nkgen biotech
2025-10-09
allogeneic
autologous
SANTA ANA, Calif., October 9, 2025 – NKGen Biotech, Inc. (OTC: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer (“NK”) cell therapeutics, today issued the following statement in response to recent shareholder inquiries regarding its current trading status on the OTC […] The post NKGen Biotech Provides Clarification on Quotation Display and Trading Status on the OTC Expert Market first appeared on NKGen Biotech .
In vivo gene therapy: lentiviral vectors into the spotlight
Fondazione Telethon
2025-10-08
lentiviral
gene therapy
gene therapies
ex vivo gene therapy
in vivo gene therapy
Lentiviral vectors, long used in ex vivo gene therapy, are gaining attention for in vivo use. Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy and a pioneer in the field, emphasizes their untapped potential across tissues and diseases. Rising academic and industry interest marks a new era for scalable, versatile gene therapies.
How Takeda Built Its Cell Therapy Portfolio, Only To Walk Away
BioSpace
2025-10-08
cell therapy
Takeda wanted to create something new in the cell therapy world by combining the technology with T cell engagers. A series of acquisitions in 2021 started the process.
Jim Cramer on uniQure: “This One Has Too Many Questions for Me Up Here”
uniQure
2025-10-08
gene therapy
uniQure N.V. (NASDAQ:QURE) is one of the stocks Jim Cramer recently talked about. A caller asked whether to buy, hold, or take profits in the stock after the release of clinical data, which indicated its gene therapy candidate significantly slowed the progression of Huntington’s disease. Cramer said: “Yeah, I saw that news and it was […]
Scientists may have discovered first gene therapy for incurable brain disease
uniQure
2025-10-08
amt-130
gene therapy
Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with FDA approval application planned for early next year.
Abeona Therapeutics® and Children's Hospital Colorado Announce Newest Treatment Center for ZEVASKYN® Gene Therapy
Abeona Therapeutics
2025-10-08
zevaskyn
gene therapy
Chiesi, Arbor Target Rare Liver Diseases in Up-to-$2.1B Gene Editing Collaboration
GenEng News
2025-10-08
gene editing
The partnership marks the first effort by Chiesi to develop a gene editing therapy and comes as the Italian biopharma is building a pipeline of rare disease treatments as one of its three therapeutic areas of focus. The post Chiesi, Arbor Target Rare Liver Diseases in Up-to-$2.1B Gene Editing Collaboration appeared first on GEN - Genetic Engineering and Biotechnology News .
Sanofi Comes For Novartis’ Radiopharma Crown With ‘Clinically Meaningful’ Pancreatic Tumor Data
BioSpace
2025-10-08
overall response rate
Sanofi’s Orano Med-partnered radioligand therapy AlphaMedix achieved all primary efficacy endpoints, which included a measure of overall response rate, in the mid-stage ALPHAMEDIX-02 study.
Lexeo Therapeutics Announces Progress in FDA Discussions for Accelerated Approval Pathway and Positive Interim Clinical Data for LX2006 in Friedreich Ataxia Cardiomyopathy
Lexeo Therapeutics
2025-10-08
cardiomyopathy
accelerated approval
Despite mRNA Vaccine Pullback, HHS Awards $48M for Kernal’s mRNA CAR T Program
BioSpace
2025-10-08
car t
The $48 million award, granted through the Advanced Research Projects Agency for Health, will help Kernal take its in vivo mRNA-encoded CAR T therapy forward.
Individuals with Sickle Cell Disease Face Long Delays to Pain Care in Emergency Department
Hematology.org
2025-10-08
sickle cell disease
Individuals with Sickle Cell Disease Face Long Delays to Pain Care in Emergency Department
Sickle Cell Disease Market to Witness Promising Upswing by 2034, DelveInsight Forecasts | Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA
CSL Behring
2025-10-08
sickle cell disease
The Key Sickle Cell Disease Companies in the market include - BRL Medicine, Oryzon Genomics, GlaxoSmithKline, Agios Pharmaceuticals, Beam Therapeutics Inc., Graphite Bio, Roche, Quercis Pharma, Editas Medicin, Pfizer, Emmaus Medical, Inc, Vertex Phar...
Sickle Cell Disease Market to Witness Promising Upswing by 2034, DelveInsight Forecasts | Sanofi, CSL Behring, ITB-Med LLC, Novo Nordisk A/S, Fortrea, Inc., Novartis, Afimmune, Pfizer, ADDMEDICA SASA
CSL Behring
2025-10-08
sickle cell disease
The Key Sickle Cell Disease Companies in the market include - BRL Medicine, Oryzon Genomics, GlaxoSmithKline, Agios Pharmaceuticals, Beam Therapeutics Inc., Graphite Bio, Roche, Quercis Pharma, Editas Medicin, Pfizer, Emmaus Medical, Inc, Vertex Phar...
Evolocumab Reduces CV Events In High-Risk Adults Without Prior Stroke, Heart Attack
Hematology Advisor News
2025-10-08
phase 3
Topline data were announced from a phase 3 trial evaluating evolocumab in adults at high cardiovascular risk without prior heart attack or stroke.
Shutdown Pauses New Drug Reviews, CDC Issues New COVID Guidance, CGT Meets on Mesa
BioSpace
2025-10-08
gene therapy
The U.S. government remains shut down, with the FDA closed for new drug applications until further notice; cell and gene therapy leaders gather for the annual meeting in Phoenix with the field in a state of flux; Pfizer and Amgen will make drugs available at a discount as President Donald Trump’s tariffs still loom; and new regulatory documents show how Pfizer beat out the competition for Metsera.
Glioblastoma Affects Skull Bone Marrow to Alter Immune Response
Inside Precision Medicine
2025-10-08
glioblastoma
The researchers found that glioblastoma dramatically increased the presence of pro-inflammatory myeloid cells, including neutrophils, while nearly eliminating B cell subsets involved in antibody production. The post Glioblastoma Affects Skull Bone Marrow to Alter Immune Response appeared first on Inside Precision Medicine .
Melanoma risk higher among adults with one tattoo, lower for those with several
Healio
2025-10-08
melanoma
A population-based study designed to examine the impact tattoos may have on a person’s risk for melanoma revealed a surprising disparate pattern.Adults who had only one tattoo session exhibited a slightly elevated risk for melanoma — particularly in situ disease — compared with tattoo-free individuals.
Around the Helix: Cell and Gene Therapy Company Updates – October 8, 2025
CGTLive
2025-10-08
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Higher Tattoo Exposure Linked to Reduced Melanoma Risk
Hematology Advisor News
2025-10-08
melanoma
Overall melanoma risk lower for those who had received four or more tattoo sessions, had three or more large tattoos.
On Course: Intellia CSO Offers Clinical Update of In Vivo Gene Editing Therapies at ESGCT
GenEng News
2025-10-08
gene editing
Birgit Schultes, PhD, shared an update on three of the company’s ongoing clinical programs, which offer several advantages to viral vectors—including efficient liver delivery, transient expression, redosing capability, scalability, biodegradability, and a large cargo capacity. The post On Course: Intellia CSO Offers Clinical Update of In Vivo Gene Editing Therapies at ESGCT appeared first on GEN - Genetic Engineering and Biotechnology News .
Kebilidi gene therapy delivers big quality-of-life boost, study finds
AADC News/BioNews
2025-10-08
kebilidi
gene therapy
A new analysis makes a case for the value of Kebilidi (eladocagene exuparvovec-tneq), the first gene therapy approved for aromatic L-amino acid decarboxylase (AADC) deficiency. While the one-time treatment has a high cost, it leads to significant improvements in a patient’s quality of life and survival compared to the best standard of care. Researchers determined […] The post Kebilidi gene therapy delivers big quality-of-life boost, study finds appeared first on AADC News .
Rituximab Regimens Safe for Acquired Hemophilia A, Standard Dose Acts Faster
Hematology Advisor News
2025-10-08
hemophilia a
In a cohort of 11 patients with acquired hemophilia A, all patients experienced significant clinical improvement, with the majority (81.8%) achieving complete remission and the remainder (18.2%) showing partial remission.
Experimental Glioblastoma Trial Findings Could Lead to Change in Patient Monitoring and Therapy
GenEng News
2025-10-08
glioblastoma
Multiomics analysis of serial tumor biopsies from glioblastoma involved in an immunotherapy trial allowed scientists to safely monitor tumor progression and therapeutic response, capturing details not discernible by advanced imaging methods. The post Experimental Glioblastoma Trial Findings Could Lead to Change in Patient Monitoring and Therapy appeared first on GEN - Genetic Engineering and Biotechnology News .
vMiX™: A novel AAV-based RNA interference platform demonstrating consistent gene silencing efficacy in mouse studies
aviadobio
2025-10-07
aav
The post vMiX™: A novel AAV-based RNA interference platform demonstrating consistent gene silencing efficacy in mouse studies appeared first on AviadoBio .
Baxdrostat lowers 24-hour ambulatory blood pressure in resistant hypertension
Healio
2025-10-07
phase 3
Baxdrostat improved ambulatory 24-hour average systolic blood pressure at 12 weeks compared with placebo in patients with treatment-resistant hypertension, AstraZeneca reported.The positive topline results of the Bax24 phase 3 trial of baxdrostat 2 mg, an aldosterone synthase inhibitor, were announced in a company press release.
uniQure’s Huntington’s gene therapy holds blockbuster potential, says analyst
uniQure
2025-10-07
amt-130
pivotal
gene therapy
Dutch biotech uniQure recently announced that its novel gene therapy, AMT-130 (ifezuntirgene inilparvovec), demonstrated statistically-significant disease slowing in a Huntington’s disease (HD) pivotal trial. The results indicate that AMT-130 holds blockbuster potential in the disease space.
In a Major Breakthrough for Cell and Gene Therapy, Immusoft Has Re-dosed a Patient with ISP-001 in Its Lead Program for MPS I
Immusoft Corporation
2025-10-07
mps i
gene therapy
/PRNewswire/ -- Immusoft of CA, a clinical stage, engineered B cell company, today announced a historic achievement: the safe and well-tolerated re-dosing of a...
Orca Bio’s BLA for Hematologic Malignancy Cell Therapy Orca-T Accepted by FDA With Priority Review
CGTLive
2025-10-07
bla
cell therapy
AZ chalks up another positive trial for hypertension drug
PharmaPhorum
2025-10-07
phase 3
AstraZeneca's baxdrostat has hit the mark in another phase 3 trial in resistant hypertension, keeping it on track for filing later this year.
Anixa Biosciences Announces Completion of Final Patient Visit in Breast Cancer Vaccine Clinical Trial
anixa biosciences
2025-10-07
car-t
phase 1
quarterly
cell therapies
first-in-human
ovarian cancer
tumor infiltrating lymphocytes
Final Clinical Visit Marks Major Milestone in First-in-Human Evaluation of Breast Cancer Vaccine Comprehensive Immune Response and Safety Data to Be Presented at the San Antonio Breast Cancer Symposium in December SAN JOSE, Calif. , Oct. 7, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced the completion of the final patient visit in its breast cancer vaccine clinical trial. This novel vaccine, invented at Cleveland Clinic, is being developed in partnership with Cleveland Clinic, and the Phase 1 trial is fully funded by a grant from the U.S. Department of Defense. The vaccine is designed to stimulate the immune system to recognize and target breast cancer before it can recur or develop. A total of 35 women received the vaccine in the study, spanning three distinct patient cohorts: TNBC Group : Women who have completed treatment for triple-negative breast cancer, are currently cancer-free, and are at risk of recurrence. Prevention Group : Women who are cancer-free but carry genetic mutations associated with elevated breast cancer risk, and who elected to…
Editas Medicine Announces EDIT-401 Oral Presentation at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress and Participation in Upcoming Investor Conferences
Editas
2025-10-07
cell therapy
Affinia Therapeutics Announces Series C Financing to Advance New Class of Gene Therapies Intended to Provide Improved Safety and Efficacy
Affinia Therapeutics
2025-10-07
gene therapies
Affinia Therapeutics Appoints Hideo Makimura, M.D., Ph.D., as Chief Medical Officer The post Affinia Therapeutics Announces Series C Financing to Advance New Class of Gene Therapies Intended to Provide Improved Safety and Efficacy appeared first on AffiniaTX .
Precision BioSciences Announces Opening of First Clinical Trial Site in U.S. for ELIMINATE-B Trial
precision biosciences
2025-10-07
phase 1
Massachusetts General Hospital , Harvard Medical School , actively recruiting chronic hepatitis B patients along with multiple global clinical trial sites - - Phase 1 ELIMINATE-B trial for PBGENE-HBV is progressing and on track to report additional data from higher dose cohorts in 2025 - DURHAM,
AMD more likely among divorced, white, heterosexual individuals
Healio
2025-10-07
msc
Being divorced, white and heterosexual are among the social determinants of health that raise the odds of having age-related macular degeneration, according to a study published in Retina. “The progressive nature of AMD poses a significant burden on both patients and health care systems, with profound psychosocial and economic impacts,” Ryan S. Huang, MSc, MD(C), of Temerty Faculty of Medicine at
Baby Steps: Kiran Musunuru Lays Out the Path Beyond KJ at ESGCT
GenEng News
2025-10-07
gene editing
Musunuru presented thousands of ESGCT delegates with a promising roadmap to advance N-of-1 gene editing therapies, building on lessons learned from the diagnosis and treatment of Baby KJ over the past year. The post Baby Steps: Kiran Musunuru Lays Out the Path Beyond KJ at ESGCT appeared first on GEN - Genetic Engineering and Biotechnology News .
Adicet Bio, Inc. Announces $80 Million Registered Direct Offering
Adicet Bio
2025-10-07
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Oct. 7, 2025-- Adicet Bio, Inc. (“Adicet”) (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced the pricing of an underwritten
Join us at ESGCT 2025 in Sevilla
Vivet Therapeutics
2025-10-07
aav
gene therapy
gene therapies
Join us at ESGCT 2025 in Sevilla for an important presentation by Jean-Philippe Combal from Vivet Therapeutics on October 9th at 10:00am CET during the session entitled Immune responses to gene therapy. https://lnkd.in/enSxNwzFLearn how VTX-PID (imlifidase) enables optimal depletion of anti-AAV3B neutralizing antibodies in human subjects, expanding eligibility for future systemic AAV-based gene therapies through innovative dose-ranging and […] The post Join us at ESGCT 2025 in Sevilla appeared first on Vivet Therapeutics .
MeiraGTx Announces Two Posters at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress
MeiraGTx
2025-10-07
cell therapy
Multiple Posters Highlight the Breadth of Company’s Novel Genetic Medicine and Cell Therapy Platforms LONDON and NEW YORK, Oct. 07, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the Company will
GenSight Biologics Reports Cash Position as of September 30, 2025
GenSight Biologics
2025-10-07
gene therapies
PARIS, October 07, 2025--Regulatory News: GenSight Biologics ("GenSight Biologics" or the "Company") (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported its cash position as of September 30, 2025.
CSL Behring Signs Letter of Intent with pan-Canadian Pharmaceutical Alliance (pCPA) for Public Reimbursement of HEMGENIX® (etranacogene dezaparvovec), the First Gene Therapy for Hemophilia B
CSL Behring
2025-10-07
hemgenix
gene therapy
hemophilia b
Legend Biotech (LEGN) Partners with Janssen for Global Carvykti Supply
Janssen Global Services
2025-10-07
car-t
carvykti
Key Takeaways: Legend Biotech (LEGN) has secured a strategic supply agreement with Janssen Pharmaceuticals for the production of Carvykti, a CAR-T cell ther
Two poster presentations at ESGCT 2025 in Sevilla
Vivet Therapeutics
2025-10-07
gene therapy
Vivet Therapeutics is excited to announce two poster presentations at ESGCT 2025 in Sevilla, showcasing innovative advances in liver-directed gene therapy. Catch us on Thursday, October 9th, from 14:00 to 15:30 CEST: The post Two poster presentations at ESGCT 2025 in Sevilla appeared first on Vivet Therapeutics .
Psychological well-being improved with psoriasis clearance
Healio
2025-10-07
clearance
Sustained two-year treatment with tildrakizumab improved the psychological well-being of people with moderate to severe plaque psoriasis, as well as their partners, according to a study. The POSITIVE study is a prospective, observational, real-world phase 4 trial that evaluated the overall well-being of people who are being treated with tildrakizumab (Ilumetri, Almirall/Ilumya, Sun
Cellectis to Present Data on Non-Viral Gene Therapy and TALE Base Editors at the ESGCT Annual Congress
cellectis
2025-10-07
aav
base editing
gene editing
gene therapy
gene therapies
New York, NY – October 7, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that findings highlighting the strong potential of circular single-stranded DNA (CssDNA) as a universal, efficient non-viral template for gene therapy, along with a comprehensive study of TALE base editors (TALEB) off-targets in the nuclear genome, will be presented at the European Society of Cell and Gene Therapy (ESGCT) annual congress, that will take place on October 7-10, 2025, in Sevilla, Spain. Poster presentation: Title: Circularization of Single-Stranded DNA Donor Template Unleashes the Power of Non-Viral Gene Delivery for Long-Term HSCs editing Presenter : Julien Valton, Ph.D., Vice President Gene Therapy at Cellectis Date/Time: Wednesday, October 8, 2025 from 2:00 p.m. to 3:30 p.m. CET Poster number: P0439 Over the past decade, non-viral DNA template delivery has been used with engineered nucleases to target single-stranded DNA sequences in hematopoietic stem and progenitor cells (HSPCs). While developed for gene therapy…
Mytos Launches Automated CDMO for Manufacturing Stem-Cell Derived Therapies
GenEng News
2025-10-07
gene therapy
The Cell and Gene Therapy Catapult site is the first of several planned global sites aimed at building the manufacturing backbone needed to bring regenerative medicines to millions of patients worldwide. The post Mytos Launches Automated CDMO for Manufacturing Stem-Cell Derived Therapies appeared first on GEN - Genetic Engineering and Biotechnology News .
Acepodia Announces IND Clearance by Japan PMDA for ACE1831 in the Treatment of IgG4-Related Disease; China IND Review in Progress-Acepodia® | POWERFUL, ACCESSIBLE CELL THERAPIES FOR PATIENTS WITH CANCER
Acepodia
2025-10-07
ind
clearance
cell therapies
Acepodia Announces IND Clearance by Japan PMDA for ACE1831 in the Treatment of IgG4-Related Disease; China IND Review in ProgressALAMEDA, Calif. & TAIPE, October 7, 2025 — Acepodia (6976: TT) today announced that the Investigational New Drug (IND) application for ACE1831 in the treatment of IgG4-related disease (IgG4-RD) has been cleared by Japan’s Pharmaceuticals and Medical Devices Agency (PMD..
VIDEO: Combination treatment ‘vast improvement’ in DLBCL care
Healio
2025-10-07
dlbcl
In this video, Tycel Phillips, MD, discusses initial results of a phase 1b clinical trial focused on treating diffuse large B-cell lymphoma with a bispecific antibody and antibody-drug conjugate combination.The findings were presented at the Society of Hematologic Oncology’s Annual Meeting.
Developers partner on cemsidomide plus Elrexfio to treat myeloma
Rare Cancer News
2025-10-07
multiple myeloma
C4 Therapeutics and Pfizer are teaming up to evaluate whether combining C4’s investigational oral therapy cemsidomide with Pfizer’s approved multiple myeloma treatment Elrexfio (elranatamab) can strengthen the immune system’s ability to fight the blood cancer. Under the agreement, Pfizer will provide Elrexfio at no cost for C4’s planned Phase 1b clinical trial, set to begin […] The post Developers partner on cemsidomide plus Elrexfio to treat myeloma appeared first on Rare Cancer News .
Legend Biotech (LEGN) Partners with Janssen for Global Carvykti Supply
Janssen Global Services
2025-10-07
car-t
carvykti
Key Takeaways: Legend Biotech (LEGN) has secured a strategic supply agreement with Janssen Pharmaceuticals for the production of Carvykti, a CAR-T cell ther
Legend Biotech, J&J ink Carvykti supply agreement
Janssen Global Services
2025-10-07
car-t
carvykti
Legend Biotech and Janssen sign global Carvykti supply deal, sharing production costs for CAR-T therapy. Read more here.
AMT Designation Takes First Steps Amid Uncertain Benefits
BioSpace
2025-10-07
gene therapy
Cell and gene therapy experts question where the FDA designation fits in an environment that features a range of intersecting regulatory perks.
Glofitamab May Improve Outcomes in Patients With Relapsed or Refractory DLBCL
Hematology Advisor News
2025-10-07
dlbcl
complete response
Researchers determined glofitamab may induce a complete response rate in 27% of real-world cases of relapsed or refractory DLBCL.
Legend Biotech (LEGN) Partners with Janssen for Global Carvykti Supply
Janssen Global Services
2025-10-07
car-t
carvykti
Key Takeaways: Legend Biotech (LEGN) has secured a strategic supply agreement with Janssen Pharmaceuticals for the production of Carvykti, a CAR-T cell ther
CSL Behring Signs Letter of Intent with pan-Canadian Pharmaceutical Alliance (pCPA) for Public Reimbursement of HEMGENIX® (etranacogene dezaparvovec), the First Gene Therapy for Hemophilia B
CSL Behring
2025-10-06
hemgenix
gene therapy
hemophilia b
/CNW/ -- CSL Behring Canada Inc., a business unit of global biopharma leader CSL, today announced that it has signed a Letter of Intent (LOI) with the...
Pompe Disease Market Research 2025: Treatment, Route of Administration, and Region Analysis and Fore
BioMarin Pharmaceutical
2025-10-06
pompe disease
DUBLIN--(BUSINESS WIRE)--The "Pompe Disease Market - A Global and Regional Analysis: Focus on Treatment Type, Route of Administration, and Region - Analysis and Forecast, 2025-2035" report has been added to ResearchAndMarkets.com's offering.The global Pompe disease treatment market is steadily gr...
Why uniQure (QURE) Is Up After Strong AMT-130 Data and $475 Million in New Funding
uniQure
2025-10-06
amt-130
pivotal
uniQure N.V. recently announced positive topline results from its pivotal Phase I/II study of AMT-130 for Huntington’s disease, meeting its prespecified primary endpoint and demonstrating a statistically significant slowing of disease progression in high-dose patients over three years compared to an external control. This milestone was accompanied by a successful US$300 million follow-on equity offering and a new US$175 million term loan, positioning the company to advance regulatory...
VIDEO: Spinal muscular atrophy trials focus on combating muscle atrophy, loss
Healio
2025-10-06
spinal muscular atrophy
Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, highlights recent advancements and ongoing clinical trials in the spinal muscular atrophy space.
VIDEO: Role of personalized medicine in spinal muscular atrophy treatment
Healio
2025-10-06
spinal muscular atrophy
Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, discusses the role of personalized medicine in spinal muscular atrophy.
Immix Biopharma to Present Abstract at the upcoming ASH 2025 Annual Meeting
Immix Biopharma
2025-10-06
nxc-201
Los Angeles, CA, Oct. 06, 2025 (GLOBE NEWSWIRE) — Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), the global leader in relapsed/refractory AL Amyloidosis, today announced that it will present a NXC-201 abstract at the upcoming American Society of Hematology (ASH) 67th Annual Meeting and Exposition to be held December 6-9, 2025, in […] The post Immix Biopharma to Present Abstract at the upcoming ASH 2025 Annual Meeting first appeared on IMMIX BIOPHARMA INC .
VIDEO: Lack of head-to-head trials presents challenge in selecting SMA treatment
Healio
2025-10-06
sma
spinal muscular atrophy
Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, discusses how physicians position different therapies for patients with spinal muscular atrophy.
Meeting on the Mesa Arrives Amid Mixed Signals for Cell and Gene Therapy
BioSpace
2025-10-06
gene therapy
As industry leaders gather at the annual event in Phoenix, the cell and gene therapy space remains in a state of flux, with M&A activity and regulatory support signaling momentum while commercialization challenges continue to hinder broader investor interest.
NKGen Biotech to Present on the Use of Troculeucel for Neurodegenerative Diseases at the XXVII World Congress of Neurology
nkgen biotech
2025-10-06
allogeneic
autologous
SANTA ANA, Calif., October 6, 2025 – NKGen Biotech, Inc. (OTC: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer (“NK”) cell therapeutics, today announced that Paul Y. Song, M.D., Chairman and Chief Executive Officer of NKGen, will present at the XXVII […] The post NKGen Biotech to Present on the Use of Troculeucel for Neurodegenerative Diseases at the XXVII World Congress of Neurology first appeared on NKGen Biotech .
Inflammation During Menstrual Cycle May Be Associated With VOEs in Females With SCD
Hematology Advisor News
2025-10-06
scd
Female adults with SCD may experience a pattern of inflammation related to the menstrual cycle that could potentially be associated with VOEs.
Rocket Pharmaceuticals Pulls BLA for Fanconi Anemia Gene Therapy RP-L102
CGTLive
2025-10-06
bla
rp-l102
gene therapy
fanconi anemia
The company cited strategic prioritization as the reason for its decision.
VIDEO: Current therapies available in spinal muscular atrophy
Healio
2025-10-06
spinal muscular atrophy
Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, highlights the current FDA-approved therapies for spinal muscular atrophy.
VIDEO: Patients with spinal muscular atrophy urged to stay in neurology care for lifetime
Healio
2025-10-06
spinal muscular atrophy
Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, highlights why it’s important patients with spinal muscular atrophy stay in neurology care throughout their entire lifetime.
Why uniQure (QURE) Is Up After Strong AMT-130 Data and $475 Million in New Funding
uniQure
2025-10-06
amt-130
U.S. stocks, Saudi stocks, stock trading and investment platforms
AbbVie Announces Positive Topline Results from Phase 2 ELATE Trial Evaluating OnabotulinumtoxinA (BOTOX®) for the Treatment of Upper Limb Essential Tremor
Abbvie
2025-10-06
phase 2
OnabotulinumtoxinA (BOTOX®) met the primary endpoint in the Phase 2 trial, demonstrating a statistically significant improvement from baseline in the Tremor Disability Scale-Revised (TREDS-R)...
Gene Therapy’s Future Hinges on the Economics of Safety
BioSpace
2025-10-06
gene therapy
Smarter design through targeted delivery and human-relevant testing can save the industry from costly safety failures.
CSL Behring Signs Letter of Intent with pan-Canadian Pharmaceutical Alliance (pCPA) for Public Reimbursement of HEMGENIX® (etranacogene dezaparvovec), the First Gene Therapy for Hemophilia B
CSL Behring
2025-10-06
hemgenix
gene therapy
hemophilia b
CSL Behring Canada Inc., a business unit of global biopharma leader CSL, today announced that it has signed a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for the public reimbursement of HEMGENIX® (etranacogene dezaparvovec). HEMGENIX is the first one-time gene therapy authorized by Health Canada for the treatment of adults with hemophilia B who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes. The successful completion of the LOI
Is gene therapy for Huntington’s the miracle we’ve been waiting for?
uniQure
2025-10-06
gene therapy
Columnist Carlos Briceño is cautiously optimistic about recent results from clinical trials of a gene therapy for Huntington's disease.
Vertex to Announce Third Quarter 2025 Financial Results on November 3rd
Vertex
2025-10-06
financial results
BOSTON --(BUSINESS WIRE)--Oct. 6, 2025-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its third quarter 2025 financial results on Monday, November 3, 2025 , after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET .
Skye Bio Obesity Drug Fails in Phase 2; Hopes Now Ride on Higher Doses and Combo Treatment
MedCity News
2025-10-06
phase 2
While Skye Bioscience said its drug nimacimab may yet achieve better weight loss results with higher doses, the company and analysts are interested in the drug’s potential to be combined with Novo Nordisk’s Wegovy. Skye’s drug is an antibody designed to preferentially target CB1 receptors in the gut, offering an alternative approach to activating GLP-1 receptors. The post Skye Bio Obesity Drug Fails in Phase 2; Hopes Now Ride on Higher Doses and Combo Treatment appeared first on MedCity News .
VIDEO: Genetic factors help determine treatment plan in spinal muscular atrophy
Healio
2025-10-06
spinal muscular atrophy
Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, discusses selecting a treatment plan for patients with spinal muscular atrophy.
Ryoncil Revenues Increase 66% in Second Quarter Post Launch
Mesoblast
2025-10-06
ryoncil
CSL Ltd - Unit signs LoI with pan-Canadian pharmaceutical alliance for Hemgenix reimbursement
CSL Behring
2025-10-06
hemgenix
CSL Ltd: CSL Ltd - unit signs LoI with pan-Canadian pharmaceutical alliance for Hemgenix reimbursement CSL Ltd - LoI for public reimbursement of Hemgenix ...
uniQure’s AMT-130: Promising Huntington’s Disease Therapy with Strong Clinical Efficacy and Favorable Safety Profile
uniQure
2025-10-06
amt-130
H.C. Wainwright analyst Patrick Trucchio has reiterated their bullish stance on QURE stock, giving a Buy rating on October 2. Patrick Trucchio has given his Buy rat...
Taysha Snags FDA Breakthrough Therapy Designation for Rett Syndrome Gene Therapy TSHA-102
CGTLive
2025-10-06
gene therapy
breakthrough therapy
FDA Lifts Clinical Hold, Clearing Neurizon’s ALS Drug for Human Trials
BioSpace
2025-10-06
clinical hold
The hold was placed earlier this year when the FDA asked for more preclinical data, but the agency was slow to respond due to ‘strain’ on its capacity, according to Neurizon.
CSL Behring Signs Letter of Intent with pan-Canadian Pharmaceutical Alliance (pCPA) for Public Reimbursement of HEMGENIX® (etranacogene dezaparvovec), the First Gene Therapy for Hemophilia B
CSL Behring
2025-10-06
hemgenix
gene therapy
hemophilia b
CSL Behring Canada Inc., a business unit of global biopharma leader CSL, today announced that it has signed a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for the public reimbursement of HEMGENIX® (etranacogene dezaparvovec). HEMGENIX is the first one-time gene therapy authorized by Health Canada for the treatment of adults with hemophilia B who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes. The successful completion of the LOI
Pompe Disease Market Research 2025: Treatment, Route of Administration, and Region Analysis and Forecast to 2035 Featuring Amicus Therapeutics, Astellas, BioMarin, Maze Therapeutics, Oxyrane Parasail
BioMarin Pharmaceutical
2025-10-06
pompe disease
DUBLIN--(BUSINESS WIRE)--Oct 6, 2025--
BrYet US, Inc. Receives Approval to Initiate in Australia a First-in-Human Phase 1/2 Trial of ML-016 in Advanced Cancers with Lung and/or Liver Involvement
PharmaVoice
2025-10-06
phase 1
phase 1/2
first-in-human
News for pharmacy pros
Oculis takes second eye disease therapy into pivotal trials
PharmaPhorum
2025-10-06
phase 2
pivotal
Oculis is poised to start registration trials for privosegtor, its drug candidate for neuro-ophthalmic diseases, in the wake of positive phase 2 data.
Rocket Aborts FDA Filing for Rare Blood Disease Gene Therapy
BioSpace
2025-10-06
gene therapy
fanconi anemia
Rocket Pharmaceuticals' strategic realignment initiative in July pulled funding from fanca-cel, which the biotech was developing for Fanconi anemia.
VIDEO: Caring for patients with spinal muscular atrophy involves ‘entire community'
Healio
2025-10-06
spinal muscular atrophy
Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, discusses the importance of multidisciplinary care in spinal muscular atrophy.
Krystal Biotech (KRYS): Evaluating Valuation After Vyjuvek FDA Label Expansion and Market Opportunity Broadening
Krystal Biotech
2025-10-04
vyjuvek
dystrophic epidermolysis bullosa
Krystal Biotech (KRYS) just received an FDA label update for Vyjuvek, which opens up expanded use for more patients, including at-home administration. This move could rapidly change the landscape for treating dystrophic epidermolysis bullosa. See our latest analysis for Krystal Biotech. Krystal Biotech’s FDA label expansion for Vyjuvek comes on the heels of notable milestones, including over 575 reimbursement approvals and brisk sales in 2025. The momentum around DEB treatments has been...
Should Positive Phase 3 PALYNZIQ Results Prompt Action From BioMarin (BMRN) Investors?
BioMarin Pharmaceutical
2025-10-04
phase 3
U.S. stocks, Saudi stocks, stock trading and investment platforms
Should Positive Phase 3 PALYNZIQ Results Prompt Action From BioMarin (BMRN) Investors?
BioMarin Pharmaceutical
2025-10-04
phase 3
In September 2025, BioMarin Pharmaceutical announced positive Phase 3 results for its PALYNZIQ® therapy in teens with phenylketonuria, showing significant reductions in dangerous phenylalanine levels compared to diet alone. Despite headwinds from industry competition and index removal, these robust clinical data have prompted multiple analyst earnings upgrades and renewed focus on the company's rare disease pipeline. We'll explore how the latest PALYNZIQ® clinical results could reshape...
ESGCT Sevilla
Vivet Therapeutics
2025-10-03
aav
cell therapy
Vivet Therapeutics is delighted to announce its participation in the European Society of Gene & Cell Therapy (ESGCT) Annual Congress, taking place in Sevilla, on October 7–10, 2025 (https://www.esgctcongress.com). On Thursday, October 9, Vivet will present new data on VTX-PID, highlighting compelling clinical results that demonstrate a reduction of neutralizing antibodies against AAV in naturally immunized populations. These findings have […] The post ESGCT Sevilla appeared first on Vivet Therapeutics .
Can BioMarin (BMRN) Turn PALYNZIQ Success Into a Stronger Rare Disease Growth Story?
BioMarin Pharmaceutical
2025-10-03
phase 3
In September 2025, BioMarin Pharmaceutical Inc. was removed from the FTSE All-World Index following share price pressure, despite announcing positive Phase 3 results for its PALYNZIQ® therapy in teens with phenylketonuria. While the company faces industry headwinds and increased competition, its robust rare disease pipeline and encouraging clinical outcomes have renewed investor interest in its long-term growth prospects. We’ll assess how BioMarin’s strong Phase 3 results for PALYNZIQ® could...
NMIBC Market to Experience Notable Growth in Forecast Span by 2034, DelveInsight Predicts | Ferring Pharma, FKD Therapies Oy, ImmunityBio, CG Oncology, Pfizer, UroGen Pharma, Janssen R&D, Protara
Ferring Pharmaceuticals
2025-10-03
cg oncology
bladder cancer
ferring pharmaceuticals
The Key Non Muscle Invasive Bladder Cancer Companies in the market include Ferring Pharmaceuticals FKD Therapies Oy ImmunityBio CG Oncology Pfizer UroGen Pharma Janssen Research Development LLC Protara Therapeutics Pfizer AstraZeneca ImmunityBio Inc Janssen Research Development Tollys Aura Biosciences Vaxiion ...
Sarepta Therapeutics to Present New Data from its Neuromuscular Portfolio at 2025 World Muscle Society Congress
Sarepta Therapeutics
2025-10-03
sarepta
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Oct. 3, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the 30 th Annual Congress of the World Muscle Society (WMS) 2025 Congress , taking place Oct. 7-11, 2025 , in Vienna, Austria .
GLP-1s from Novo, Lilly 'should be first-line in obesity'
PharmaPhorum
2025-10-03
first-line
European doctors have said GLP-1 drugs are so effective in obesity that they should be the first choice drug treatment in almost all cases.
Exposure to dogs during infancy linked to reduced childhood asthma risks
Healio
2025-10-03
msc
Babies who were exposed to dog allergens were less likely to develop asthma by age 5 years compared with babies who were not, according to an abstract presented at the European Respiratory Society International Congress. However, exposure to cat allergens had no similar protective effect, Jacob McCoy, MD, asthma fellow and MSc candidate, division of respiratory medicine, The Hospital for Sick
Dianthus Therapeutics Highlights Claseprubart Data Presentations Planned for 2025 AANEM Annual MeetingPDF VersionRead More
Dianthus Therapeutics
2025-10-03
myasthenia gravis
Positive results from Phase 2 MaGic trial including additional analyses, and new preclinical data highlighting potential benefits of upstream inhibition, to be presented for claseprubart in generalized Myasthenia Gravis NEW YORK and WALTHAM, Mass., Oct. PDF Version Read More
Intellia Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Intellia Therapeutics Inc.
2025-10-03
crispr
gene editing
CAMBRIDGE, Mass. , Oct. 03, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that on October 1, 2025 , it awarded inducement grants to six new employees
Ryoncil Receives CMS (Medicaid) J-Code for Reimbursement
Mesoblast
2025-10-03
ryoncil
Hope Is On The Horizon For Huntington’s Patients
uniQure
2025-10-03
gene therapy
New data from a gene therapy trial provide the first credible evidence that the progression of Huntington’s disease may be slowed by a single, targeted intervention.
Study Identifies Determinants of Desmopressin Response in Female Hemophilia A Carriers
Hematology Advisor News
2025-10-03
hemophilia a
Basal von Willebrand factor antigen levels influenced FVIII kinetics, with lower von Willebrand factor antigen associated with higher postdesmopressin FVIII peaks and recovery.
Rocket Pharmaceuticals Pulls FDA Filing for Rare Blood Disorder Gene Therapy
MedCity News
2025-10-03
gene therapy
fanconi anemia
Rocket Pharmaceuticals voluntarily withdrew its FDA submission for its Fanconi anemia gene therapy. The move follows a corporate restructuring over the summer that refocused the company on its cardiovascular programs. The post Rocket Pharmaceuticals Pulls FDA Filing for Rare Blood Disorder Gene Therapy appeared first on MedCity News .
Rocket aborts FDA filing for Fanconi gene therapy
PharmaPhorum
2025-10-03
gene therapy
Rocket Pharma has withdrawn a filing in the US for its experimental gene therapy for Fanconi anaemia, but may consider partnering the programme.
Our Pilot study of Cu64 data in Heterozygous Wilson’s Disease patients
Vivet Therapeutics
2025-10-03
wilson disease
We are excited to share our paper on Pilot clinical study showing abnormal copper metabolism in healthy Wilson disease heterozygote subjects, accepted to CTSJournal. See it here : https://lnkd.in/e-rzZnXp #ASCPTJournalFamily – Wilson Disease Association 64Cu has been used for decades to study WD, however clear data are lacking to document the rate of fecal elimination […] The post Our Pilot study of Cu64 data in Heterozygous Wilson’s Disease patients appeared first on Vivet Therapeutics .
Utidelone now FDA orphan drug after promising cancer trial results
Rare Cancer News
2025-10-03
orphan drug
pancreatic cancer
Biostar Pharma’s experimental therapy utidelone has been granted orphan drug status for pancreatic cancer by the U.S. Food and Drug Administration (FDA). The designation is intended to incentivize the development of treatments for rare diseases that affect fewer than 200,000 people in the U.S. This status provides Biostar with regulatory benefits like tax breaks, fee […] The post Utidelone now FDA orphan drug after promising cancer trial results appeared first on Rare Cancer News .
QURE: Why Analysts See Up to 63% Upside After 250% Single-Day Pop
uniQure
2025-10-03
amt-130
In one of the most incredible single-day moves this year, biotechnology stock uniQure NASDAQ:QURE surged by 284% on Sept. 24. This came as the firm released fantastic clinical results for its developmental medicine, AMT-130.With such a massive spike, uniQure is turning the heads of Wall Street anal…
Cold AIHA May Signal Future Risk of CLL: A Case Report
Hematology Advisor News
2025-10-03
cll
Molecular analysis revealed a hypermutation in the immunoglobulin heavy chain region, indicating a more indolent form of CLL and a better prognosis.
First Human Trial Results Show Enzyme Technology Could Enable Universal Donor Organs
GenEng News
2025-10-03
first-in-human
In a first-in-human trial, a type A kidney was transformed into the universal O type using specialized enzymes, transplanted, and functioned for days with limited signs of rejection. The post First Human Trial Results Show Enzyme Technology Could Enable Universal Donor Organs appeared first on GEN - Genetic Engineering and Biotechnology News .
Umoja Biopharma's In Situ CAR-T Therapy UB-VV111 Fast Tracked for Several R/R B-Cell Malignancy Indications
CGTLive
2025-10-03
car-t
FDA approves lurbinectedin, atezolizumab combination for small cell lung cancer
Healio
2025-10-03
first-line
The FDA approved the combination of lurbinectedin and atezolizumab as first-line maintenance treatment for certain adults with extensive-stage small cell lung cancer.This indication of lurbinectedin (Zepzelca; Jazz Pharmaceuticals, Pharma Mar) applies to adults whose disease has not progressed after first-line induction therapy with atezolizumab (Tecentriq, Genentech), carboplatin and etoposide.
Generative AI Designs Synthetic Gene Editing Proteins Better than Nature
Inside Precision Medicine
2025-10-03
piggybac
gene editing
Their use of generative AI focused on PiggyBac transposases, naturally occurring enzymes that have long been used for gene delivery and genetic engineering, and uncovered more than 13,000 previously unidentified PiggyBac sequences. The post Generative AI Designs Synthetic Gene Editing Proteins Better than Nature appeared first on Inside Precision Medicine .
Evolocumab effective for primary prevention in patients with atherosclerosis, diabetes
Healio
2025-10-03
phase 3
Evolocumab, a PCSK9 inhibitor, reduced risk for major adverse CV events in people with no history of heart attack or stroke already taking a statin or other LDL-lowering therapy, according to topline results released by Amgen.In the phase 3 VESALIUS-CV trial of more than 12,000 patients with atherosclerosis or diabetes and no prior heart attack or stroke, evolocumab (Repatha) met both primary
What Comes Next? Industry Takeaways from FDA’s Cell and Gene Therapy Listening Session
GenEng News
2025-10-03
gene therapy
In this GEN webinar, an outstanding panel of CGT experts will unpack some of the key themes raised during the recent FDA Listening Session and examine their implications for the future of product development and regulatory strategy. The post What Comes Next? Industry Takeaways from FDA’s Cell and Gene Therapy Listening Session appeared first on GEN - Genetic Engineering and Biotechnology News .
A Look at uniQure (QURE) Valuation After Pivotal AMT-130 Results and FDA Breakthrough Designation
uniQure
2025-10-03
rmat
amt-130
pivotal
breakthrough therapy
uniQure (NasdaqGS:QURE) just announced statistically significant results from its pivotal Phase I/II trial of AMT-130 in Huntington’s disease. The company also closed a $300 million follow-on equity offering and secured Breakthrough Therapy and RMAT designations from the FDA. See our latest analysis for uniQure. After unveiling pivotal clinical results and bolstering its balance sheet with new financings, uniQure’s story has started to capture more investors’ attention. The share price has...
Will uniQure’s Results Pressure PTC Therapeutics’ (PTCT) Rare Disease Leadership and Revenue Ambitions?
uniQure
2025-10-03
gene therapy
Recently, clinical trial data from uniQure revealed a 75% reduction in Huntington's disease progression, increasing competition for PTC Therapeutics’ own gene therapy pipeline as insider selling and questions around revenue growth raised new concerns among investors. This development intensifies the spotlight on PTC's late-stage programs and heightens stakeholder scrutiny of its potential to maintain a competitive edge in rare disease treatments. To better understand the impact of uniQure’s...
FDA’s New Cell and Gene Therapy Guidances Finally Go a 'Click Further' to 'Game Changing'
BioSpace
2025-10-03
gene therapy
gene therapies
Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways for cell and gene therapies ‘for small populations’ is receiving a warmer welcome—but experts warn it will take more to turn the tide for the fraught therapeutic space.
A Look at uniQure (QURE) Valuation Following Breakthrough Huntington’s Disease Data and Fresh Capital Raises
uniQure
2025-10-03
amt-130
pivotal
uniQure (NasdaqGS:QURE) grabbed attention after reporting pivotal Phase I/II results for its experimental Huntington’s disease therapy, AMT-130. The clinical data signaled a meaningful slowdown in disease progression, which sparked renewed investor enthusiasm. See our latest analysis for uniQure. uniQure’s recent breakthroughs have fueled strong momentum, with the positive AMT-130 trial data and successful $300 million equity raise sending the stock to all-time highs. Investors have taken...
Gene Therapy Targeting Incontinence Shows Promise in Patients
Inside Precision Medicine
2025-10-03
gene therapy
A precise, localized gene therapy to treat incontinence caused by nerve damage associated with spinal cord injury reduced urinary incontinence by 88% at the lowest dose after 12 weeks. The post Gene Therapy Targeting Incontinence Shows Promise in Patients appeared first on Inside Precision Medicine .
Krystal Biotech's Heavy Reliance on Vyjuvek: Growth Driver or Risk Factor?
Krystal Biotech
2025-10-03
vyjuvek
KRYS gains momentum with Vyjuvek's expanded FDA label and global approvals, while advancing a pipeline led by oncology candidate KB707.
Dissecting the CAR T-Cell Membrane Using the Proximity Network Assay
GenEng News
2025-10-03
car t
Pixelgen’s technology provides novel functional biology insights.. The post Dissecting the CAR T-Cell Membrane Using the Proximity Network Assay appeared first on GEN - Genetic Engineering and Biotechnology News .
Iberdomide Shows Promising Phase 3 Results in R/R Multiple Myeloma
Hematology Advisor News
2025-10-02
phase 3
multiple myeloma
Topline data were announced from a phase 3 trial evaluating iberdomide in combination with standard therapies in patients with RRMM.
KURA ONCOLOGY AND KYOWA KIRIN LAUNCH CLINICAL TRIAL EVALUATING DUAL INHIBITION OF NPM1 AND FLT3 MUTATIONS IN PATIENTS WITH NEWLY DIAGNOSED ACUTE MYELOID LEUKEMIA (AML)
Kyowa Kirin Co.
2025-10-02
aml
acute myeloid leukemia
Strimvelis receives European marketing authorisation to treat ADA-SCID
Fondazione Telethon
2025-10-02
ada-scid
gene therapy
The European Commission has approved the first ex-vivo stem cell gene therapy to treat patients with a very rare disease of the immune system
BLA for the gene therapy for the treatment of Wiskott-Aldrich syndrome submitted to FDA
Fondazione Telethon
2025-10-02
bla
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
Fondazione Telethon announced the submission of the Biologic License Application (BLA) for the gene therapy for the treatment of patients with Wiskott-Aldrich Syndrome (WAS).
Takeda Exits Cell Therapy Amid Strategic Pivot, Dealing Blow to Once Promising Modality
BioSpace
2025-10-02
cell therapy
Takeda is looking to offload its cell therapy platform and preclinical assets to a yet-unidentified external partner. 137 employees will be let go as part of the move.
Tigem and SR-Tiget involved in UPGRADE project, funded by the European Union
Fondazione Telethon
2025-10-02
gene therapies
Fondazione Telethon announces the launch of the UPGRADE project, funded by the European Union with 15 million euros, aiming at developing more precise and effective gene therapies approaches.
ERC Awards €2.5M to TIGEM scientist for project on programmable genetic circuits
Fondazione Telethon
2025-10-02
gene therapy
di Bernardo’s DIMERCIRCUITS integrates AI and synthetic biology to advance precision gene therapy.
Investigational Therapy Offers New Hope for Huntington
uniQure
2025-10-02
gene therapy
huntington disease
An investigational gene therapy delivered directly to the brain via a single surgical procedure is linked to a slowing of Huntington disease progression, early data show.
Strimvelis receives European marketing authorisation to treat very rare disease, ADA-SCID
Fondazione Telethon
2025-10-02
ada-scid
gene therapy
GlaxoSmithKline (GSK), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) today announced that the European Commission has approved Strimvelis, the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID
Taysha Gene Therapies Announces FDA Breakthrough Therapy Designation and Provides Positive Regulatory Update on TSHA-102 in Rett Syndrome
Taysha Gene Therapies
2025-10-02
pivotal
phase 1/2
rett syndrome
gene therapies
breakthrough therapy
Breakthrough Therapy designation granted based on FDA’s review of available clinical evidence of safety and efficacy from all 12 patients treated in Part A of the REVEAL Phase 1/2 trials Finalized FDA alignment on REVEAL pivotal trial protocol and SAP following resolution of remaining clinical and
Clinical Evidence Confirms Timing as Crucial in Gene Therapy for MLD
Fondazione Telethon
2025-10-02
gene therapy
The study was conducted at Ospedale San Raffaele in Milan and is the result of more than 20 years of research by the San Raffaele-Telethon Institute for Gene Therapy and the commitment of Fondazione Telethon to transforming research into therapies available for patients.
AskBio Announces 6 Presentations at the European Society of Gene and Cell Therapy 32nd Annual Meeting
askbio
2025-10-02
aav
phase 2
pipeline
cell therapy
gene therapy
pompe disease
gene therapies
adeno-associated virus
Research Triangle Park, N.C.– OCTOBER 2, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will deliver 6 presentations offering insights into the research and development of adeno-associated virus (AAV) therapies for a range of diseases as well as advancements in manufacturing technologies, at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Meeting taking place October 7–10, 2025, in Seville, Spain. “ESGCT brings together leading researchers, clinicians, and industry experts to drive forward the field of gene and cell therapy and, as a company with a significant presence both in U.S. and Europe, this meeting is a special opportunity for us to interact with these prestigious stakeholders,” said Gustavo Pesquin, Chief Executive Officer, AskBio. “This year, alongside Viralgen, AskBio is contributing six presentations that highlight not only our latest clinical milestones but also our advanced manufacturing capabilities. As we share new data spanning discovery, pre-clinical, and clinical stages, we reaffirm our commitment to delivering therapies at scale, with robust production and translational…
Sanofi’s Neovascular Age-Related Macular Degeneration Gene Therapy SAR402663 Garners FDA Fast Track Designation
CGTLive
2025-10-02
fast track
gene therapy
SAR402663 comprises a onetime intravitreal treatment and is intended to inhibit VEGF by providing a gene for soluble FLT01.
A hope twelve thousand kilometres long
Fondazione Telethon
2025-10-02
gene therapy
The story of Amari, who came from Trinidad to Milan to receive gene therapy for his rare genetic disorder of the immune system.
AbbVie to Host Third-Quarter 2025 Earnings Conference Call
Abbvie Investors
2025-10-02
financial results
NORTH CHICAGO, Ill. , Oct. 2, 2025 /PRNewswire/ -- AbbVie (NYSE: ABBV) will announce its third-quarter 2025 financial results on Friday, October 31, 2025 , before the market opens. AbbVie will host a live webcast of the earnings conference call at 8 a.m. Central time .
Teclistamab-Based Induction Regimens Produce “Very High” MRD Negativity Rates
Hematology Advisor News
2025-10-02
phase 2
multiple myeloma
Teclistamab-based induction regimens have demonstrated efficacy in a phase 2 trial of patients with newly diagnosed multiple myeloma.
MAA for the Gene Therapy for the treatment of Wiskott-Aldrich Syndrome submitted to EMA
Fondazione Telethon
2025-10-02
maa
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
Fondazione Telethon announced that it has submitted the MAA for the gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS).
Data Roundup: September 2025 Features Updates from Huntington Disease Gene Therapy Trial, Pivotal MPS II Gene Therapy Study, and More
CGTLive
2025-10-02
mps ii
pivotal
gene therapy
huntington disease
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive® team.
A platform dedicated to Duchenne Muscular Dystrophy
Fondazione Telethon
2025-10-02
dmd
duchenne muscular dystrophy
It will make available hundreds of DMD patients' data and will lay the foundations for collecting new data in the future according to homogeneous standards.
Mucopolysaccharidosis 6 and gene therapy: present and future
Fondazione Telethon
2025-10-02
gene therapy
FDA Approves Genentech’s Tecentriq Plus Lurbinectedin as First-Line Maintenance Therapy for Extensive-Stage Small Cell Lung Cancer
Genentech
2025-10-02
first-line
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Oct 2, 2025--
MavriX Bio’s Angelman Syndrome Gene Therapy MVX-220 Garners FDA Fast Track Designation
CGTLive
2025-10-02
fast track
gene therapy
MVX-220 is intended to provide a functional copy of UBE3A, the disease-targeted gene, to the neurons.
CAR T Cells Engineered with Fusion Proteins Improve Solid Tumor Response
Inside Precision Medicine
2025-10-02
car t
solid tumor
ovarian cancer
Engineering CAR T cells to produce fusion proteins combining a cytokine and an antibody has shown to enhance efficacy and safety in animal models of prostate and ovarian cancer. The post CAR T Cells Engineered with Fusion Proteins Improve Solid Tumor Response appeared first on Inside Precision Medicine .
Lack of Standardized Fatigue Assessment Impairs SCD Management
Hematology Advisor News
2025-10-02
scd
Current assessments use inconsistent definitions of fatigue dimensions, scoring methods, and reporting.
Verismo Therapeutics Treats First Follicular Lymphoma Patient in CELESTIAL-301 Trial, Strengthening Collaboration with IFLI
Verismo Therapeutics
2025-10-02
follicular lymphoma
The post Verismo Therapeutics Treats First Follicular Lymphoma Patient in CELESTIAL-301 Trial, Strengthening Collaboration with IFLI appeared first on Verismo .
Phase 1/2 Trial for uniQure's Huntington Disease Gene Therapy AMT-130 Hits Primary End Point
CGTLive
2025-10-01
amt-130
phase 1
phase 1/2
gene therapy
huntington disease
The study also met a key secondary end point.
Immix Biopharma to Present at the 37th Annual Piper Sandler Healthcare Conference
Immix Biopharma
2025-10-01
cell therapies
Los Angeles, CA, Oct. 01, 2025 (GLOBE NEWSWIRE) — Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and other serious diseases, today announced that it will present and host institutional investor meetings at the 37th Annual Piper Sandler Healthcare Conference being held on […] The post Immix Biopharma to Present at the 37th Annual Piper Sandler Healthcare Conference first appeared on IMMIX BIOPHARMA INC .
Novo Breaks $598M Heartseed Pact Amid ‘Decisive Restructuring’ by New CEO
BioSpace
2025-10-01
cell therapy
Novo Nordisk and Heartseed first partnered in 2021 to develop an investigational cell therapy for heart failure.
Adicet Bio Reports Inducement Grant under Nasdaq Listing Rule 5635(c)(4)
Adicet Bio
2025-10-01
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Sep. 30, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced it granted an inducement award on September
Ultragenyx's Glycogen Storage Disease Gene Therapy DTX401 Continues to Enable Decreases in Reductions in Daily Cornstarch Intake at 96 Weeks
CGTLive
2025-10-01
dtx401
gene therapy
With regard to safety, DTX401’s safety profile was characterized as “acceptable and expected.”
Roger Hajjar, MD, on the Commercial Viability of Gene Therapy for Ultra Rare Diseases
CGTLive
2025-10-01
gene therapy
Genmab to Acquire Merus and Novel Advanced Cancer Prospect for $8 Billion
Inside Precision Medicine
2025-10-01
breakthrough therapy
Petosemtamab, with pembrolizumab, is in Phase III development for head and neck squamous cel and has two breakthrough therapy designations. The post Genmab to Acquire Merus and Novel Advanced Cancer Prospect for $8 Billion appeared first on Inside Precision Medicine .
enGene Appoints Hussein Sweiti, M.D., MSc, as Chief Medical Officer
enGene
2025-10-01
msc
Roger Hajjar, MD, on Broader Trends in Cell and Gene Therapy
CGTLive
2025-10-01
gene therapy
Quarterly Drug Approvals: October 2025
Prime Therapeutics
2025-10-01
quarterly
M&A Picks Up, Walmsley Moves On, Pfizer’s MFN Deal and Hope for Huntington’s
BioSpace
2025-10-01
gene therapy
M&A headlined for a second straight week as Genmab acquired Merus for $8 billion; Pfizer strikes most-favored-nation deal with White House; CDER Director George Tidmarsh caused a stir with a now-deleted LinkedIn post; GSK CEO Emma Walmsley will step down from her role; and uniQure’s gene therapy offers new hope for patients with Huntington’s disease.
Umoja's in vivo cell therapy fast-tracked by FDA
PharmaPhorum
2025-10-01
cell therapy
A pioneer of in vivo cell therapy, Umoja has cleared another regulatory milestone by getting fast-track status for its lead blood cancer programme.
Trastuzumab Deruxtecan Followed by THP Under Review for HER2+ Early Breast Cancer
Hematology Advisor News
2025-10-01
her2
Findings from DESTINY-Breast11 showed T-DXd followed by THP demonstrated a statistically significant and clinically meaningful improvement in pCR rate vs ddAC-THP.
Phase 1/2 Trial for Immix Biopharma’s AL Amyloidosis CAR T NXC-201 Hits Over 50% Enrollment
CGTLive
2025-10-01
car t
phase 1
phase 1/2
The trial, which is taking place in the United States, is expected to enroll 40 patients in total.
AI Tool Strengthens Precision Care for Patients with CNS Cancers
Inside Precision Medicine
2025-10-01
glioblastoma
PICTURE, a Harvard-led AI tool, helps surgeons distinguish glioblastoma from similar brain tumors during surgery, offering near-perfect accuracy and guiding real-time treatment choices. The post AI Tool Strengthens Precision Care for Patients with CNS Cancers appeared first on Inside Precision Medicine .
Symptom Provocation May Improve TMS Outcomes in OCD and Nicotine Dependence
Hematology Advisor News
2025-10-01
clearance
Symptom provocation prior to transcranial magnetic stimulation (TMS) may enhance clinical outcomes for patients with obsessive-compulsive disorder (OCD) and nicotine dependence, according to findings published in JAMA Psychiatry. TMS is a noninvasive brain stimulation technique with FDA clearance for several psychiatric conditions, including OCD and nicotine use disorder. While current protocols for these two indications…
Bristol Myers Squibb’s Anti-MTBR-Tau-Targeting Antibody, BMS-986446, Granted Fast Track Designation by U.S. FDA for the Treatment of Alzheimer’s Disease
Bristol Myers Squibb
2025-10-01
fast track
Radiation matches proton therapy for treatment of oropharyngeal cancer
Healio
2025-10-01
phase 3
Intensity-modulated radiation therapy did not increase the risk for feeding tube dependence for patients with oropharyngeal squamous cell carcinoma compared with proton therapy, according to results of a randomized phase 3 trial.
Quarterly Drug Pipeline: October 2025
Prime Therapeutics
2025-10-01
pipeline
quarterly
Marginal Zone Lymphoma Market Growth Projections 2024-2034: DelveInsight Analysis | Gilead Sciences, AstraZeneca, Nordic Nanovector, Christian Buske, AbbVie, Pharmacyclics LLC, Hutchison Medipharma
Gilead Sciences
2025-10-01
marginal zone lymphoma
GetNews Press Release.
enGene Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4)
enGene
2025-10-01
msc
BOSTON & MONTREAL--(BUSINESS WIRE)--enGene Holdings Inc. (Nasdaq: ENGN, “enGene” or the “Company”), a clinical-stage, non-viral genetic medicines company, today reported the grant of inducement equity awards to four newly-hired employees, including Hussein Sweiti, M.D., MSc, Chief Medical Officer...
Around the Helix: Cell and Gene Therapy Company Updates – October 1, 2025
CGTLive
2025-10-01
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
iECURE to Present OTC-HOPE Clinical Trial Data of ECUR-506 in Ornithine Transcarbamylase Deficiency at the European Society of Gene & Cell Therapy Annual Congress and the American Society of Human Genetics Annual Meeting
Iecure
2025-10-01
cell therapy
ornithine transcarbamylase
ornithine transcarbamylase deficiency
October 2, 2025 The post iECURE to Present OTC-HOPE Clinical Trial Data of ECUR-506 in Ornithine Transcarbamylase Deficiency at the European Society of Gene & Cell Therapy Annual Congress and the American Society of Human Genetics Annual Meeting first appeared on iECURE .
CRISPR Therapeutics to Present Preclinical Data on Alpha-1 Antitrypsin Deficiency (AATD) Utilizing Novel SyNTase Gene Editing Technology at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress
CRISPR
2025-10-01
crispr
cell therapy
gene editing
ZUG, Switzerland and BOSTON , Oct. 01, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced the acceptance of an abstract for oral presentation at the European Society
Novartis Nabs FDA Approval for First-of-Its-Kind Drug for Chronic Hives
MedCity News
2025-10-01
second-line
Novartis drug Rhapsido is now approved as a second-line treatment for chronic spontaneous urticaria, a rare inflammatory skin disease. Additional clinical trials are ongoing to support potential expansion of the twice-daily pill to other immunological disorders. The post Novartis Nabs FDA Approval for First-of-Its-Kind Drug for Chronic Hives appeared first on MedCity News .
Weighing the pros and cons of paying off a mortgage: A strategic guide for physicians
Healio
2025-10-01
pivotal
For many physicians, financial success often leads to a pivotal question: Should I pay off my mortgage early?
Timely Opioid Administration in Pediatric Sickle Cell Pain Linked to Fewer Hospitalizations
Hematology Advisor News
2025-10-01
sickle cell disease
New research shows that giving opioids to pediatric patients with sickle cell disease within 60 minutes of ED arrival reduces hospitalization risk.
FDA Activity Recap: September 2025 Features New Draft Guidance Documents, RMAT Designation, and More
CGTLive
2025-10-01
rmat
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.
Capricor Therapeutics Testing DMD Cell Therapy Deramiocel’s Mettle in Phase 3 HOPE-3 Clinical Trial
CGTLive
2025-10-01
dmd
phase 3
deramiocel
cell therapy
In honor of Duchenne Action Month and World Duchenne Day, held every year in September, CGTLive is taking a closer look at this ongoing study.
The Importance of Addressing Gene Therapy's Financial Viability in Ultrarare Diseases
CGTLive
2025-10-01
cell therapy
gene therapy
Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, discussed a trend of physicians and scientists working on models to make gene therapy products commercially viable.
Bristol Myers Squibb’s Anti-MTBR-Tau-Targeting Antibody, BMS-986446, Granted Fast Track Designation by U.S. FDA for the Treatment of Alzheimer’s Disease
bristol myers squibb
2025-10-01
fast track
Jainu Jogani on the Potential to Address CDKL5 Deficiency With Gene Therapy
CGTLive
2025-09-30
gene therapy
Aurion Biotech Expands Leadership Team, Promoting Andrew Torres, Ph.D., to Chief Manufacturing Officer and Sterling Chung to Chief Regulatory & Quality Officer
aurion biotech
2025-09-30
cell therapy
Strategic appointments fuel global clinical and commercial development of AURN001, a transformational cell therapy with the potential to restore vision Seattle, WA; Cambridge, MA; Tokyo, Japan – 30 September – Aurion Biotech, a clinical-stage regenerative medicine company with a mission to restore vision to millions of patients, today announced the promotions of Andrew Torres, Ph.D., […]
Krystal Biotech's Heavy Reliance on Vyjuvek: Growth Driver or Risk Factor?
Krystal Biotech
2025-09-30
vyjuvek
KRYS gains momentum with Vyjuvek's expanded FDA label and global approvals, while advancing a pipeline led by oncology candidate KB707.
Neurotech Granted Permanent J-Code (J3403) for ENCELTO (revakinagene taroretcel-lwey), Effective October 1, 2025
Neurotech Pharmaceuticals
2025-09-30
j-code
encelto
GenSight Biologics Reports Interim Financial Results for the First Half of 2025
GenSight Biologics
2025-09-30
financial results
Opus Genetics Reports Positive Pediatric Data from OPGx-LCA5 Phase 1/2 Trial in Leber Congenital Amaurosis Type 5 (LCA5)
opus genetics
2025-09-30
phase 1
phase 1/2
leber congenital amaurosis
Data backs Vertanical's cannabis therapy for back pain
PharmaPhorum
2025-09-30
phase 3
A phase 3 trial of Vertanical's cannabis extract in back pain has been published, and the results bode well for marketing applications under review.
Ernexa Therapeutics President & CEO Sanjeev Luther to Present on Expert Panel at 5th Annual iPSC Drug Development Summit
Ernexa Therapeutics
2025-09-30
ipsc
J&J's Bladder Cancer Development Leader Who Secured FDA Approval Joins enGene as Chief Medical Officer
enGene
2025-09-30
bla
bladder cancer
Hussein Sweiti, former J&J Global Medical Head for Oncology, joins biotech enGene to lead clinical development. Key hire ahead of 2H 2026 BLA submission for detalimogene in bladder cancer.
CDKL5 and Gene Therapy
CGTLive
2025-09-30
gene therapy
Jainu Jogani, the cofounder of Child’s Cure Genetic Research, discussed his daughter’s rare genetic disease and the need for new treatment options.
FDA rejects biologics license application for apitegromab
Healio
2025-09-30
complete response
spinal muscular atrophy
The FDA has rejected a biologics license application for apitegromab to treat spinal muscular atrophy, according to manufacturer Scholar Rock. In a press release, Scholar Rock stated that the FDA’s Complete Response Letter (CRL) cited “adverse conditions” discovered during an inspection of Catalent Indiana LLC, a third-party fill-finish facility.
Receiving Gene Therapy May Cause Anxiety in Patients With Hemophilia
Hematology Advisor News
2025-09-30
gene therapy
A psychosocial model is needed that provides guidance for designing comprehensive care that addresses the psychosocial needs of patients and their families at all phases of gene therapy.
FDA Publishes 3 New Draft Guidance for Industry Documents Aimed at Cell and Gene Therapy Development
CGTLive
2025-09-30
gene therapy
The documents cover topics including efficient trial design, expedited review programs, and postapproval data collection.
First Patient Treated in Capsida Biotherapeutics’ Trial for Encephalopathy Gene Therapy CAP-002 Passes Away
CGTLive
2025-09-30
gene therapy
Capsida has made the move to pause the trial voluntarily.
uniQure N.V. (QURE) Reports Positive Phase I/II Results, Prepares FDA BLA Submission
uniQure
2025-09-30
bla
amt-130
hemgenix
gene therapy
hemophilia b
We recently compiled a list of the 12 Best Performing Healthcare Stocks to Buy Right Now. uniQure N.V. is one of them. uniQure N.V. (NASDAQ:QURE) is a leading gene therapy company focused on developing transformative treatments for severe medical conditions. Its pipeline includes HEMGENIX for hemophilia B and AMT-130, a gene therapy candidate for Huntington’s […]
Duchenne Action Month 2025: Looking Back at News and Expert Insights
CGTLive
2025-09-30
dmd
cell therapy
In observance of Duchenne Action Month and World Duchenne Day, held annually in September, we took a look back at recent news in gene and cell therapy for DMD.
Anti-HBc Positivity Linked to More Severe ITP
Hematology Advisor News
2025-09-30
complete response
Treatment response differed significantly between the groups, with patients in the anti-HBc positive group being less likely to achieve a complete response to initial treatment.
Sarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Sarepta Therapeutics
2025-09-30
sarepta
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Sep. 30, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, granted equity awards on September 30, 2025 (the “Grant Date”) that were approved by the Compensation Committee of its Board of Directors under
News in Cell and Gene Therapy for Rare Cancer Day 2025
CGTLive
2025-09-30
cell therapy
gene therapy
In observance of Rare Cancer Day, held annually on September 30, catch up on the past few months’ news related to cell therapy for such indications.
Iberdomide helps eliminate myeloma cells, trial data show
Rare Cancer News
2025-09-30
phase 3
multiple myeloma
Iberdomide can help eliminate cancer cells in people with relapsed or refractory multiple myeloma, according to clinical trial results announced by Bristol Myers Squibb (BMS). The finding comes from an interim analysis of data from the Phase 3 clinical trial EXCALIBER-RRMM (NCT04975997). The study enrolled hundreds of myeloma patients whose cancer had come back (relapsed) […] The post Iberdomide helps eliminate myeloma cells, trial data show appeared first on Rare Cancer News .
Around the Helix: Cell and Gene Therapy Company Updates – September 24, 2025
CGTLive
2025-09-30
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Top News in Lymphoma Cell Therapy for World Lymphoma Awareness Day 2025
CGTLive
2025-09-30
cell therapy
For World Lymphoma Awareness Day, held annually on September 15, take a look at the stories that have stood out in lymphoma cell therapy in recent months.
Precision BioSciences Announces Late-Breaking Poster Presentation at the 30th Annual International Congress of the World Muscle Society
precision biosciences
2025-09-30
cta
dmd
ind
duchenne muscular dystrophy
Data to highlight latest long-term preclinical efficacy and durability data supporting PBGENE-DMD for the treatment of Duchenne Muscular Dystrophy - - On track to file an IND and/or CTA by the end of 2025; clinical data anticipated in 2026- DURHAM, N.C. --(BUSINESS WIRE)--Sep.
Atsena Therapeutics’ Trial for X-Linked Retinoschisis Gene Therapy ATSN-201 Finishes Dosing Adult Patients in Part B
CGTLive
2025-09-30
gene therapy
Part B of LIGHTHOUSE enrolled 9 patients and is expected to enroll 3 pediatric patients.
FDA Approves Beremagene Geperpavec-svdt (Vyjuvek) Label Update in DEB
Krystal Biotech
2025-09-30
vyjuvek
dystrophic epidermolysis bullosa
The label update expands the treatment eligible population to include dystrophic epidermolysis bullosa patients from birth and allows at-home application.
AbbVie Submits Biologics License Application (BLA) to U.S. FDA for Pivekimab sunirine (PVEK) - an Investigational Antibody-Drug Conjugate (ADC) to Treat Rare Cancer with Limited Treatment Options
Abbvie
2025-09-30
bla
phase 1
phase 1/2
– BLA based on data from the global Phase 1/2 CADENZA trial NORTH CHICAGO, Ill., Sept. 30, 2025 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced submission of a new Biologics License...
GenSight Biologics Reports Interim Financial Results for the First Half of 2025
GenSight Biologics
2025-09-30
financial results
enGene appoints Sweiti as Chief Medical Officer
enGene
2025-09-30
msc
enGene (ENGN) announced that Hussein Sweiti, M.D., MSc, was appointed Chief Medical Officer, effective September 29, 2025. Dr. Sweiti most recently served as Global...
Shorter radiation course for prostate cancer improves quality of life, not disease control
Healio
2025-09-30
phase 3
A shorter radiation course improved quality of life among men with intermediate-risk, localized prostate cancer, according to results of a randomized phase 3 trial.
Zilganersen Improves Functional Mobility in Patients With Alexander Disease
Hematology Advisor News
2025-09-30
pivotal
Topline data were announced from a pivotal study evaluating zilganersen in children and adults living with Alexander disease.
uniQure completes $345 million public offering to fund gene therapy pipeline By Investing.com
uniQure
2025-09-29
gene therapy
uniQure completes $345 million public offering to fund gene therapy pipeline
Enhertu scores again in early breast cancer
PharmaPhorum
2025-09-29
phase 3
AstraZeneca and Daiichi Sankyo are eyeing regulatory filings for Enhertu in early breast cancer after a second phase 3 trial delivers.
$345 Million Raised: Gene Therapy Leader uniQure Completes Upsized Offering for AMT-130 Launch Plans
uniQure
2025-09-29
amt-130
gene therapy
Gene therapy company uniQure closes public offering of 6.7M shares at $47.50/share plus pre-funded warrants. Proceeds to fund AMT-130 commercialization, clinical pipeline, and business development.
Around the Helix: Cell and Gene Therapy Company Updates – September 17, 2025
CGTLive
2025-09-29
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
HSCT Appears Effective for Transformed Severe Aplastic Anemia
Hematology Advisor News
2025-09-29
aml
Patients with transformed severe aplastic anemia who underwent HSCT for MDS, AML, or an isolated cytogenetic abnormality demonstrated good clinical outcomes.
New hope for Huntington’s families as gene therapy shows remarkable results
uniQure
2025-09-29
gene therapy
Hope grows for Huntington’s patients, but scientific validation is still pending.
World Heart Day 2025: Looking Back at Recent Progress in Cardiology Cell and Gene Therapy
CGTLive
2025-09-29
gene therapy
In observance of World Heart Day, held annually on September 29, we took a look back at news in cell/gene therapy for cardiovascular disease from the past few months.
KURA ONCOLOGY AND KYOWA KIRIN ANNOUNCE FIRST PATIENT DOSED IN PIVOTAL PHASE 3 KOMET-017 TRIAL OF ZIFTOMENIB FOR FRONTLINE ACUTE MYELOID LEUKEMIA (AML)
Kyowa Kirin Co.
2025-09-29
aml
phase 3
pivotal
acute myeloid leukemia
uniQure Announces Closing of Upsized Public Offering and Full Exercise by Underwriters of Option to Purchase Additional Shares
uniQure
2025-09-29
gene therapy
LEXINGTON, Mass. and AMSTERDAM, Sept. 29, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the closing of its previously announced underwritten public offering of 6,736,841 ordinary shares at a public offering price of $47.50 per share, which includes 947,368 ordinary shares issued pursuant to the exercise in full by the underwriters of their option to purchase additiona
Neurotech Granted Permanent J-Code (J3403) for ENCELTO™ (revakinagene taroretcel-lwey), Effective October 1, 2025
Neurotech Pharmaceuticals
2025-09-29
encelto
Neurotech Pharmaceuticals, Inc., a private biotech company focused on developing transformative therapies for chronic eye diseases, today announced that the ...
Roche to Acquire 89bio and Phase 3 MASH Candidate in Potential $3.5B Deal
Roche Group
2025-09-29
phase 3
Pegozafermin offers a distinct mechanism of action with potential for enhanced efficacy and tolerability and opportunities for combination development with incretins.
Project aims to improve AML patients’ access to clinical trials
Rare Cancer News
2025-09-29
aml
acute myeloid leukemia
A new research project aims to make it easier for people with acute myeloid leukemia (AML) to participate in clinical trials. The project is led by Tara Lin, MD, director of the University of Kansas Cancer Center’s adult leukemia program, and funded by a nearly $680,000 award from the National Cancer Institute (NCI), a part […] The post Project aims to improve AML patients’ access to clinical trials appeared first on Rare Cancer News .
Induced Mesenchymal Stem Cells: A New Frontier in Ovarian Cancer Treatment
MedCity News
2025-09-29
allogeneic
ovarian cancer
mesenchymal stem cells
Ovarian cancer urgently needs bold, transformative treatment approaches – not just small, incremental improvements. Synthetic, allogeneic iMSCs as tumor-targeting delivery vehicles are an example of how cell biology and engineering may unite and overcome longstanding oncology challenges. The post Induced Mesenchymal Stem Cells: A New Frontier in Ovarian Cancer Treatment appeared first on MedCity News .
Sanofi's DM1 Gene Therapy SAR446268 Snags Fast Track Designation
CGTLive
2025-09-29
fast track
orphan drug
gene therapy
SAR446268 has previously received orphan drug designations from the FDA and European Medicines Agency.
Ferring Pharmaceuticals
Ferring Pharmaceuticals
2025-09-29
gene therapy
Ferring Pharmaceuticals is a privately-owned, research-driven, specialty biopharmaceutical group committed to building families and helping people live better lives. In the United States, Ferring is a leader in reproductive medicine, and in areas of gastroenterology and orthopaedics. We are at the forefront of innovation in microbiome-based therapeutics and uro-oncology intravesical gene therapy. The company was founded in 1950 and is headquartered in Saint-Prex, Switzerland. Ferring employs more than 7,000 people worldwide and markets its medicines in over 100 countries.
Lisata Therapeutics Announces Encouraging Preliminary Results from the Pancreatic Cancer Cohort of the CENDIFOX Trial
Lisata Therapeutics
2025-09-29
pancreatic cancer
Results to be presented at the AACR Special Conference in Cancer Research: Advances in Pancreatic Cancer Research—Emerging Science Driving Transformative Solutions BASKING RIDGE, N.J., Sept. 29, 2025 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a
Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline 2025: Innovations by 180+ Global Leaders - DelveInsight | Featuring GenSight Biologics, Ultragenyx Pharmaceutical, MeiraGTx
GenSight Biologics
2025-09-29
aav
regenxbio
ultragenyx
gene therapy
adeno associated virus
adeno-associated virus
nanoscope therapeutics
rocket pharmaceuticals
Adeno Associated Virus AAV Vectors in Gene Therapy companies are Nanoscope Therapeutics REGENXBIO Astellas Gene Therapy GeneCradle Therapeutics Asklepios BioPharmaceutical 4D Molecular Therapeutics Adverum Biotechnologies Rocket Pharmaceuticals Innostellar Biotherapeutics Passage Bio Aspa Therapeutics Solid Biosciences Lantu Biopharma Ascidian Therapeutics Decibel ...
Roche’s Lunsumio receives positive CHMP opinion for follicular lymphoma
PMLive Regulatory
2025-09-29
follicular lymphoma
The recommendation for the subcutaneous formulation follows encouraging trial data
GenSight Biologics Reports Interim Financial Results for the First Half of 2025
GenSight Biologics
2025-09-29
gene therapies
PARIS, September 29, 2025--Regulatory News: GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported its interim financial results for the first half of 2025.
UniQure Announces Closing of Upsized Public Offering and Full Exercise by Underwriters of Option to Purchase Additional Shares
uniQure
2025-09-29
gene therapy
LEXINGTON, Mass. and AMSTERDAM, Sept. 29, 2025 -- uniQure N.V. , a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the...
Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline 2025: Innovations by 180+ Global Leaders – DelveInsight | Featuring GenSight Biologics, Ultragenyx Pharmaceutical, MeiraGTx
GenSight Biologics
2025-09-29
aav
gene therapy
adeno-associated virus
GetNews Press Release.
Genmab to Pay $8B to Get Its Hands on Merus Head & Neck Cancer Drug With Blockbuster Potential
MedCity News
2025-09-29
pivotal
Merus brings Genmab a bispecific antibody in two pivotal studies for head and neck cancer. Genmab, which had long developed drugs under partnerships with larger companies, is now pursuing a strategy of developing and commercializing medications it fully owns. The post Genmab to Pay $8B to Get Its Hands on Merus Head & Neck Cancer Drug With Blockbuster Potential appeared first on MedCity News .
PTC Therapeutics (PTCT): Valuation Update Following uniQure's Positive Huntington’s Gene Therapy Results
uniQure
2025-09-28
phase 1
phase 1/2
gene therapy
For investors eyeing PTC Therapeutics (PTCT) this week, attention is focused on uniQure’s latest clinical update. The biotech sector just saw a shakeup as uniQure reported impressive Phase 1/2 results for its AMT130 gene therapy, with the data showing a 75% drop in Huntington’s disease progression over three years. With both companies active in rare, neurodegenerative conditions, this development could directly impact how markets view the promise of PTCT’s competing therapies. PTC...
StockWatch: uniQure Shares Reach Five-Year High on "Game Changing" Huntington's Data
uniQure
2025-09-28
amt-130
gene therapy
The dazzling 36-month clinical data announced by uniQure showing a significant slowing of Huntington’s disease (HD) progression in patients treated with its gene therapy candidate AMT-130 was just one of several reasons why the company’s stock more than tripled this past week on the better-than-expected news, reaching a five-year high.
Around the Helix: Cell and Gene Therapy Company Updates – September 10, 2025
CGTLive
2025-09-28
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Patients With DMD Treated With Avidity Biosciences’ Antibody-Oligonucleotide Conjugate Del-Zota Show Functional Improvements
CGTLive
2025-09-28
dmd
phase 1
phase 2
phase 1/2
The findings come from patients with DMD44 treated in the phase 1/2 EXPLORE44 clinical trial and the phase 2 EXPLORE44OLE clinical trial.
Patients Treated With REGENXBIO's MPS II Gene Therapy RGX-121 Sustain 82% Median Reduction in CSF HS D2S6 Levels Through 1 Year
CGTLive
2025-09-28
mps ii
rgx-121
gene therapy
According to REGENXBIO, HS D2S6 is “a key biomarker of MPS II brain disease that is reasonably likely to predict clinical benefit.”
Noah Stansfield | Authors
CGTLive
2025-09-28
gene therapy
Noah Stansfield | Authors | Cell and Gene Therapy Live
uniQure (QURE) Is Up 291.6% After AMT-130’s Breakthrough Phase I/II Huntington’s Results and FDA Designations – Has The Bull Case Changed?
uniQure
2025-09-27
rmat
amt-130
pivotal
gene therapy
breakthrough therapy
Earlier this week, uniQure N.V. announced positive topline results from its pivotal Phase I/II study of AMT-130 in Huntington's disease, revealing statistically significant slowing of disease progression over 36 months and receipt of Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. This marks a potentially groundbreaking moment for Huntington's disease treatment and positions uniQure as a frontrunner in the gene therapy space, supported by...
Gilead Sciences (GILD) Stock Sees Positive Outlook Amid HIV, Liver, and Cancer Drug Momentum
Gilead Sciences
2025-09-27
clearance
Gilead Sciences, Inc. (NASDAQ:GILD) ranks among the most undervalued NASDAQ stocks to buy now. Citing momentum in Gilead Sciences, Inc. (NASDAQ:GILD)’s product pipeline, Moody’s updated the company’s outlook from stable to positive on September 18, maintaining the company’s A3 senior unsecured ratings. The forecast takes into account Gilead’s recent clearance by regulators and introduction of […]
Why uniQure (QURE) Is Up After Positive AMT-130 Trial Results and Major New Funding Announcements
uniQure
2025-09-27
amt-130
pivotal
uniQure N.V. recently announced positive topline results from its pivotal Phase I/II trial of AMT-130, demonstrating statistically significant slowing of Huntington's disease progression, and entered into a US$175 million senior secured term loan facility with Hercules Capital, alongside launching a US$200 million underwritten equity offering. The regulatory designations for AMT-130 and the implementation of non-dilutive debt financing, together with equity issuance, highlight both the...
'Best news' for Huntington's disease community comes with unanswered questions
uniQure
2025-09-27
gene therapy
An experimental gene therapy seems to show potential to slow the debilitating progression of Huntington's disease, a European biotech company that developed the new treatment says.
Dawn's reflection: hope from Tigem research
Fondazione Telethon
2025-09-27
gene therapy
The child, born with mucopolysaccharidosis type 6, was treated with gene therapy in 2020 and to date her liver is able to produce the missing enzyme that causes the disease.
In Response to FDA’s Release of Full CRL for Deramiocel, Capricor Publishes Full Response to the Agency
CGTLive
2025-09-27
deramiocel
Capricor stated that it was not notified in advance by the FDA that the latter would be publishing the CRL publicly on its website.
uniQure Announces Pricing of Upsized $300 Million Public Offering
uniQure
2025-09-26
gene therapy
LEXINGTON, Mass. and AMSTERDAM, Sept. 25, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the pricing of its previously announced underwritten public offering of 5,789,473 ordinary shares at a public offering price of $47.50 per share, and, in lieu of ordinary shares to certain investors, pre-funded warrants to purchase 526,316 of its ordinary shares at the public offer
A Huntington’s Disease Treatment Is Closer Than Ever
uniQure
2025-09-26
gene therapy
An experimental gene therapy may slow progression of the disease by up to 75%.
Standardizing Gene Therapy Care in Duchenne: An Overview of Consensus Guidelines
CGTLive
2025-09-26
gene therapy
Mind Moments, a podcast from our sister site Neurology Live, held an exclusive interview with Barry Byrne, MD, PhD.
uniQure’s gene therapy makes significant breakthrough for Huntington’s disease
uniQure
2025-09-26
amt-130
pivotal
gene therapy
On 24 September, uniQure announced positive topline results from its pivotal Phase I/II studies (HD-GENE-TRX1, HD-GENE-TRX2) for its novel gene therapy AMT-130 in Huntington’s disease (HD).
Atebimetinib yields ‘extraordinary’ results in pancreatic cancer trial
Rare Cancer News
2025-09-26
first-line
pancreatic cancer
An experimental cancer therapy known as atebimetinib is delivering “extraordinary” results for patients with pancreatic cancer. New data from an ongoing Phase 2a clinical trial shows that 86% of patients who received the first-line treatment are still alive nine months later — a higher survival rate than the 47% expected from standard chemotherapy alone, according […] The post Atebimetinib yields ‘extraordinary’ results in pancreatic cancer trial appeared first on Rare Cancer News .
The Therapeutic Potential of Circular RNA
CGTLive
2025-09-26
cell therapy
Alex Wesselhoeft, PhD, the director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute, discussed the Institute's work in exploring the new modality.
Trends in RNA Therapeutic Research
CGTLive
2025-09-26
cell therapy
Robert Alexander Wesselhoeft, PhD, the director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute, expressed optimism for RNA therapy's future.
KURA ONCOLOGY AND KYOWA KIRIN ANNOUNCE PUBLICATION OF PIVOTAL ZIFTOMENIB DATA IN RELAPSED/REFRACTORY NPM1 MUTATED AML IN THE JOURNAL OF CLINICAL ONCOLOGY
Kyowa Kirin Co.
2025-09-26
aml
pivotal
AbbVie Submits New Drug Application to U.S. FDA for Tavapadon for the Treatment of Parkinson's Disease
Abbvie
2025-09-26
phase 3
Submission supported by data from the Phase 3 TEMPO program that demonstrated symptomatic improvement across the Parkinson's disease spectrum Positive results across all three Phase 3 TEMPO trials...
With FDA Nod in Rare Hormone Disease, Crinetics Can Challenge Blockbuster Meds From Novartis, Ipsen
MedCity News
2025-09-26
first-line
FDA approval of Crinetics Pharmaceuticals’ Palsonify makes it the first oral first-line therapy for acromegaly, a rare endocrine disorder. The once-daily pill will compete with blockbuster acromegaly drugs from Novartis and Ipsen that are administered as injections. The post With FDA Nod in Rare Hormone Disease, Crinetics Can Challenge Blockbuster Meds From Novartis, Ipsen appeared first on MedCity News .
New Treatment Shows Promise for Huntington’s Disease
uniQure
2025-09-26
gene therapy
A gene therapy injected into patients’ brains slowed progression of the disease by 75 percent, preliminary findings reported.
Mesoblast Cell Therapy Products Not Subject to U.S. Tariffs
Mesoblast
2025-09-26
cell therapy
Friday briefing: The Huntington’s treatment is a ray of light for victims of a brutal disease
uniQure
2025-09-26
gene therapy
In today’s newsletter: A gene therapy trial has successfully treated Huntington’s for the first time – one of several recent groundbreaking medical advances bringing hope in gloomy times
MiNK Therapeutics Dr. Jennifer Buell to Deliver Featured Plenary at 10th Annual CAR-TCR Summit in Boston, MA
Mink Therapeutics
2025-09-26
cell therapies
NEW YORK , Sept. 25, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (Nasdaq: INKT), a clinical-stage biopharmaceutical company pioneering invariant natural killer T (iNKT) cell therapies, today announced that its President and Chief Executive Officer, Dr.
Follicular Lymphoma Market to Experience Notable Growth in Forecast Span by 2034, DelveInsight Predicts | Roche, MEI Pharma, Novartis, Innovent Biologics, Regeneron, Genentech, Xynomic Pharma
Genentech
2025-09-26
follicular lymphoma
Key Follicular Lymphoma Companies in the market include Roche Xynomic Pharmaceuticals MEI Pharma Novartis Innovent Biologics Regeneron Genentech Xynomic Pharma Eli Lilly and Company ADC Therapeutics S A BeiGene Cephalon Epizyme Inc Pharmacyclics LLC Gemin X AbbVie Millennium Pharma and ...
Vertex Announces Key Advancements Across Kidney Portfolio
Vertex
2025-09-25
phase 2
phase 2/3
accelerated approval
breakthrough therapy
Food and Drug Administration granted Breakthrough Therapy Designation for povetacicept in IgA nephropathy; on track to file for accelerated approval in the U.S. in H1 2026 if 36-week interim analysis data positive - - Enrollment completed for interim analysis cohort of AMPLITUDE global Phase 2/3
Intellia Therapeutics Announces Positive Longer-Term Phase 1 Data for Nexiguran Ziclumeran (nex-z) in Patients with Hereditary Transthyretin (ATTR) Amyloidosis with Polyneuropathy
Intellia Therapeutics Inc.
2025-09-25
phase 1
One dose of nex-z led to rapid, deep and durable TTR reductions, with mean reductions of at least 90% from baseline sustained through three years Stabilization or improvement in disease-related clinical measures observed at 24 months Continue to observe generally favorable safety data with no new
Celyad Oncology reports first half year 2025 financial results
Celyad
2025-09-25
financial results
Mont-Saint-Guibert, Belgium; September 25, 2025, 6:30 pm CET; regulated information – Celyad Oncology (Euronext: CYAD) (the “Company”), today announced its […] The post Celyad Oncology reports first half year 2025 financial results appeared first on .
A gene therapy success in Huntington's and Trump's autism announcement
uniQure
2025-09-25
gene therapy
This week on "The Readout LOUD" podcast: a gene therapy success in Huntington's, Trump's autism announcement, and more.
CAR T-Cell Therapy Market will Reach USD 11.49 Billion by 2034 | Autolus Therapeutics; 2Seventy Bio, Inc.; Bristol-Myers Squibb; Pfizer; Gilead Sciences
Autolus Therapeutics
2025-09-25
car t
cell therapy
The global CAR T cell therapy market is poised for significant expansion valued at USD 2 25 billion in 2024 and projected to reach USD 11 49 billion by 2034 reflecting a robust compound annual growth rate CAGR of 17 ...
Leber’s Hereditary Optic Neuropathy (LHON) Pipeline 2025: Pioneering Clinical Developments by 6+ Global Leaders – DelveInsight | Featuring GenSight Biologics, Neurophth Therapeutics, Mitotech, Stealth
GenSight Biologics
2025-09-25
pipeline
"Leber's Hereditary Optic Neuropathy (LHON) Pipeline 2025"DelveInsight’s, “Leber’s Hereditary Optic Neuropathy - Pipeline Insight, 2025,” report provides comprehensive insights about 6+ companies and 7+ pipeline drugs in Leber’s Hereditary Optic Neur...
Leber's Hereditary Optic Neuropathy (LHON) Pipeline 2025: Pioneering Clinical Developments by 6+ Global Leaders - DelveInsight | Featuring GenSight Biologics, Neurophth Therapeutics, Mitotech, Stealth
GenSight Biologics
2025-09-25
pipeline
DelveInsight s Leber s Hereditary Optic Neuropathy Pipeline Insight 2025 report provides comprehensive insights about 6 companies and 7 pipeline drugs in Leber s Hereditary Optic Neuropathy pipeline landscape It covers the pipeline drug profiles including clinical and nonclinical stage ...
Conversion of Unresectable Into Resectable Pancreatic Cancer: Chemoradiotherapy vs Chemotherapy
ASCO Post Journals
2025-09-25
pancreatic cancer
In a German phase III trial (CONKO-007) reported in the Journal of Clinical Oncology,1 Rainer Fietkau, MD, of the Department of Radiation Oncology, Friedrich-Alexander-Universität Erlangen-Nürnberg (F...
New NIH-Funded Study Identifies Urine-Based Assay for Prostate Cancer
ASCO Post Journals
2025-09-25
prostate cancer
Researchers have developed a novel method to test for prostate cancer using biomarkers present in urine. This approach may significantly reduce the need for invasive, often painful biopsies, the resea...
Capricor Therapeutics Provides Regulatory Update on Deramiocel Program for Duchenne Muscular Dystrophy Following Type A Meeting
Capricor Therapeutics
2025-09-25
deramiocel
duchenne muscular dystrophy
Acadia, Harmony record late-stage rare disease failures
PharmaPhorum
2025-09-25
phase 3
Biotechs Acadia Pharma and Harmony Biosciences both suffered setbacks in phase 3 trials of drugs for rare diseases, in one case ending the programme.
New Approvals for Thrombocytopenia, Lung Cancer, Brain Cancer, and Multiple Myeloma
ASCO Post FDA
2025-09-25
multiple myeloma
BTK Inhibitor for Chronic Immune Thrombocytopenia: On September 2, 2025, the U.S. Food and Drug Administration (FDA) approved the Bruton’s tyrosine kinase (BTK) inhibitor rilzabrutinib (Wayrilz) table...
Kyverna Therapeutics Highlights Potential of KYV-101 in Multiple Sclerosis with Data from Phase 1 Investigator-Initiated Trials to be Presented at ECTRIMS
Kyverna Therapeutics
2025-09-24
car t
kyv-101
phase 1
KYV-101 IIT data demonstrate promising clinical activity, including robust CAR T penetration into the central nervous system (CNS) and improved expanded disability status scale scores (EDSS) KYV-101 continues to demonstrate a tolerable safety profile, consistent with observations from the first 100
Chronic Periodontitis Pipeline 2025: Innovative Clinical Developments from Over 10 Global Leaders – DelveInsight | Highlighting Amyndas Pharmaceuticals, Takeda, CSL Behring, Vaxcyte, Noveome, and Peri
CSL Behring
2025-09-24
pipeline
"Chronic Periodontitis Pipeline 2025"DelveInsight’s, “Chronic Periodontitis – Pipeline Insight, 2025,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Chronic Periodontitis pipeline landscape. It covers the pipeli...
FDA knocks back Biogen's high-dose Spinraza
PharmaPhorum
2025-09-24
sma
Biogen's efforts to defend its SMA business have been dealt a setback after the FDA declined to approve a high-dose version of Spinraza.
UniQure Gene Therapy’s Clinical Trial Results Keep It on Path to Become First Huntington’s Treatment
MedCity News
2025-09-24
amt-130
gene therapy
UniQure plans a first quarter 2026 FDA submission for AMT-130 as a treatment for Huntington’s disease. This gene therapy delivers microRNA designed to silence the mutated gene that produces a protein driving this disorder, which so far has no FDA-approved therapies. The post UniQure Gene Therapy’s Clinical Trial Results Keep It on Path to Become First Huntington’s Treatment appeared first on MedCity News .
BMS eyes filings for myeloma 'celmod' iberdomide
PharmaPhorum
2025-09-24
phase 3
multiple myeloma
Bristol Myers Squibb has said it hopes to be ready soon to file for approval of its multiple myeloma drug iberdomide after a positive phase 3 trial.
Roche pushes a novel myeloma target
Roche Group
2025-09-24
phase 3
The company will be the first to take a FcRH5-targeting project into phase 3.
Joy as uniQure Huntington's therapy clears pivotal trial
PharmaPhorum
2025-09-24
amt-130
pivotal
gene therapy
uniQure's gene therapy AMT-130 has cleared a trial in Huntington's, setting up filings and raising the prospect of a first treatment for the disease.
Manufacturing issues block FDA nod for Scholar Rock SMA drug
PharmaPhorum
2025-09-23
sma
complete response
Unresolved issues at a manufacturing plant operated by Novo Nordisk have led to a complete response letter for Scholar Rock's SMA drug apitegromab.
Roche's oral SERD comes good in 2nd-line breast cancer trial
PharmaPhorum
2025-09-23
second-line
Roche has staked a claim to use its oral SERD giredestrant as a second-line therapy for breast cancer, regardless of a patient's ESR1 mutation status.
Ionis Pharma’s Drug for Rare Disease With No Approved Therapies Meets Goals of Pivotal Study
MedCity News
2025-09-23
pivotal
Ionis Pharmaceuticals plans a 2026 FDA submission for zilganersen as a treatment for Alexander disease, an ultra-rare inherited neurological disorder. The antisense oligonucleotide developer has been moving toward commercializing drugs on its own rather than seeking partners. The post Ionis Pharma’s Drug for Rare Disease With No Approved Therapies Meets Goals of Pivotal Study appeared first on MedCity News .
CG Oncology (CGON): Assessing Valuation After New Clinical Milestones and Regulatory Pipeline Updates
CG Oncology
2025-09-23
phase 3
pivotal
CG Oncology (CGON) just dropped a string of updates that will make investors pause and take notice. The company announced encouraging results from the BOND-003 Cohort C trial, kicked off a new clinical study (CORE-008 Cohort CX), and set timelines for wrapping up enrollment in its pivotal Phase 3 PIVOT-006 trial. Management also flagged an upcoming Biologics License Application submission for cretostimogene by the end of the year. All these signals point to a packed regulatory calendar and...
Bristol Myers Squibb Announces Phase 3 EXCALIBER-RRMM Study Evaluating Iberdomide in Combination with Standard Therapies Demonstrated a Significant Improvement in Minimal Residual Disease Negativity Rates in Relapsed or Refractory Multiple Myeloma
bristol myers squibb
2025-09-23
phase 3
multiple myeloma
Evaluating Roche After Shipments Resume for Key Duchenne Muscular Dystrophy Therapy
Roche Group
2025-09-23
duchenne muscular dystrophy
If you’re staring at Roche Holding’s ticker and wondering what to do next, you’re in good company. Over the past week, the stock has nudged up 2.6%, and it is up 3.4% for the year to date, even after a stretch of underwhelming long-term returns. It has been a bit of a wild ride, with shares down 5.3% over the last three years, hinting at shifting market sentiment and possibly some uncertainty around the broader pharmaceutical sector. Roche’s recent price moves have not happened in a vacuum...
Krystal Biotech (KRYS) Is Up 6.2% After FDA Expands VYJUVEK Eligibility to All Ages Has the Bull Case Changed?
Krystal Biotech
2025-09-23
vyjuvek
dystrophic epidermolysis bullosa
Earlier this month, Krystal Biotech announced that the FDA approved a label update for VYJUVEK®, expanding its eligibility to include dystrophic epidermolysis bullosa patients from birth and allowing greater flexibility in wound care management based on real-world safety and efficacy data. This expansion meaningfully broadens VYJUVEK's treatable patient base in the US and may strengthen the company's competitive position in global genetic dermatology markets. To assess how this FDA approval...
Hemostemix Files FDA Submission for Basket Protocol (Filing 1517) to Advance ACP-01 (VesCell™) Across Multiple Ischemic and Vascular Indications Simultaneously
Hemostemix
2025-09-23
ind
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta – September 23, 2025 – Hemostemix Inc. (TSXV: HEM; OTCQB: HMTXF; FSE: 2VF0), the leading autologous (patient’s own) stem cell therapy company offering VesCell™ (ACP-01) to no-option individuals suffering from peripheral arterial disease, chronic limb threatening ischemia, angina, ischemic cardiomyopathy, non-ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, in Florida under Florida’s SB 1768, is pleased to announce that it has filed FDA Pre-IND Application 1517 , seeking regulatory feedback on a basket protocol Phase I clinical trial of its lead therapy, ACP-01 (VesCell™) .
Bristol Myers Squibb Announces Phase 3 EXCALIBER-RRMM Study Evaluating Iberdomide in Combination with Standard Therapies Demonstrated a Significant Improvement in Minimal Residual Disease Negativity Rates in Relapsed or Refractory Multiple Myeloma
Bristol Myers Squibb
2025-09-23
phase 3
multiple myeloma
Nanoscope Nabs RMAT Designation for Retinitis Pigmentosa Gene Therapy MCO-010
CGTLive
2025-09-23
rmat
mco-010
orphan drug
gene therapy
retinitis pigmentosa
Notably, the European Medicines Agency also granted MCO-010 orphan drug designation for 5 retinal dystrophy categories.
Atsena Therapeutics Announces Dosing Complete for Adults in Part B of the Phase I/II/III LIGHTHOUSE Trial Evaluating ATSN-201 to Treat X-linked Retinoschisis
atsena therapeutics
2025-09-23
x-linked
x-linked retinoschisis
Atsena Therapeutics Announces Dosing Complete for Adults in Part B of the Phase I/II/III LIGHTHOUSE Trial Evaluating ATSN-201 to Treat X-linked Retinoschisis Pediatric dosing expected ... The post Atsena Therapeutics Announces Dosing Complete for Adults in Part B of the Phase I/II/III LIGHTHOUSE Trial Evaluating ATSN-201 to Treat X-linked Retinoschisis appeared first on Atsena Therapeutics .
Cleveland Clinic to Present Final Results of Phase 1 Clinical Trial for Anixa Biosciences' Breast Cancer Vaccine at 2025 San Antonio Breast Cancer Symposium
anixa biosciences
2025-09-22
car-t
phase 1
phase 2
pivotal
quarterly
cell therapies
immunogenicity
ovarian cancer
tumor infiltrating lymphocytes
Phase 1 Data Highlights Potential of First-in-Class Vaccine to Transform Breast Cancer Treatment and Prevention Comprehensive Results, Supported by U.S. Department of Defense Funding, to Inform FDA Discussions and Advance Phase 2 Development Plans SAN JOSE, Calif. , Sept. 22, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that final results from the Phase 1 clinical trial of its breast cancer vaccine will be presented at the 2025 San Antonio Breast Cancer Symposium (SABCS) on Thursday, December 11, 2025. The trial, conducted at Cleveland Clinic and funded by a grant from the U.S. Department of Defense, evaluated the safety and immunogenicity of an alpha-lactalbumin (aLA) vaccine for breast cancer. The presentation, titled "Final Results of a Phase I Trial of alpha-lactalbumin (aLA) Vaccine for Breast Cancer ," will be delivered by Justin Johnson, Ph.D., Program Manager at Cleveland Clinic and co-inventor of the vaccine technology. With the trial now complete and comprehensive analyses of blood and tissue samples underway, this presentation at SABCS…
First European Participants Randomized in AskBio Phase 2 Gene Therapy Trial of AB-1005 for Parkinson’s Disease
askbio
2025-09-22
bla
rmat
phase 1
phase 2
gene therapy
gene therapies
Research Triangle Park, N.C. – September 22, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first European participants have been randomized in REGENERATE-PD, a Phase 2 clinical trial in participants with moderate-stage Parkinson’s disease (PD). “I believe the randomization of the first European participants in REGENERATE-PD, which makes this the first neurosurgical gene therapy program for Parkinson’s to successfully randomize patients from both the United States and Europe into a single Phase 2 trial, is positive news for people living with Parkinson’s disease and the physicians treating them,” said Alan Whone, MD, PhD, REGENERATE-PD Europe Lead and Principal Investigator. “There is a significant need for neurorestorative therapies in Parkinson’s and seeing the advancement of an important investigational gene therapy in a Phase 2 clinical trial will give hope to patients and the medical community alike.” PD is a progressive, neurodegenerative disorder affecting 1.2 million people in Europe, and this number is expected to double by 2030.1 With the worldwide unmet medical need in…
CRISPR Therapeutics and Sirius Therapeutics Announce First Patient Dosed in Phase 2 Trial of SRSD107 for Thromboembolic Disorders in Europe
CRISPR
2025-09-22
crispr
phase 2
ZUG , Switzerland and BOSTON and SAN DIEGO and SHANGHAI, Sept. 22, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Sirius Therapeutics, a clinical stage biotech company
Bayer starts pivotal Parkinson's cell therapy trial
PharmaPhorum
2025-09-22
phase 3
pivotal
cell therapy
Bayer's much-anticipated stem cell therapy for Parkinson's disease is gathering momentum, with dosing in phase 3 trials now underway.
Cystic Fibrosis Pipeline 2025: Therapies Under Investigation, Clinical Trials Milestones, and FDA Approvals by DelveInsight | Insmed Inc, AstraZeneca, Zambon, CSL Behring, Chiesi Farmaceutici
CSL Behring
2025-09-22
cystic fibrosis
Las Vegas Nevada United States As per DelveInsight s assessment globally Cystic Fibrosis pipeline constitutes 50 key companies continuously working towards developing 55 Cystic Fibrosis treatment therapies analysis of Clinical Trials Therapies Mechanism of Action Route of Administration and Developments ...
Preclinical Evidence for PH1 Gene Therapy
CGTLive
2025-09-22
gene therapy
John Murphy, PhD, and Tia DiTommaso, PhD, of Arbor Biotechnologies, discussed ABO-101, the company’s gene therapy for primary hyperoxaluria type 1.
First Successful Phase 3 Trial: Genentech's Oral Therapy Breakthrough for ER+ Breast Cancer Treatment
Genentech
2025-09-22
phase 3
Giredestrant plus everolimus achieved significant PFS improvement in ER+ breast cancer post-CDK therapy. First positive Phase 3 trial for oral SERD regimen vs standard care. Safety profile consistent with expectations.
Genentech to file for oral SERD approval on Phase III breast cancer win
Genentech
2025-09-22
her2
Genentech will be heading to regulators after the Phase III success of its oral SERD and full antagonist therapy in oestrogen receptor (ER)-positive, HER2-negative, locally advanced or metastatic breast cancer.
Roche moves obesity asset CT-388 into phase 3
Roche Group
2025-09-22
phase 3
Roche advances CT-388, a dual GLP-1/GIP receptor agonist, to phase 3 for obesity. Read more here.
Benitec Biopharma Releases Full Year 2025 Financial Results and Provides Operational Update
benitec biopharma
2025-09-22
financial results
Cystic Fibrosis Pipeline 2025: Therapies Under Investigation, Clinical Trials Milestones, and FDA Approvals by DelveInsight | Insmed Inc, AstraZeneca, Zambon, CSL Behring, Chiesi Farmaceutici
CSL Behring
2025-09-22
cystic fibrosis
GetNews Press Release.
Positive Phase III Results Show Genentech’s Giredestrant Significantly Improved Progression-Free Survival in ER-positive Advanced Breast Cancer
Genentech
2025-09-22
her2
SOUTH SAN FRANCISCO, Calif., September 22, 2025--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today positive results from the Phase III evERA study evaluating investigational giredestrant in combination with everolimus in people with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer previously treated with a cyclin-dependent kinase (CDK) 4/6 inhibitor and endocrine therapy. The
Intellia Therapeutics to Present Longer-Term Data from the Ongoing Phase 1 Clinical Trial of Nexiguran Ziclumeran (nex-z) for the Treatment of Hereditary Transthyretin (ATTR) Amyloidosis with Polyneuropathy
Intellia Therapeutics Inc.
2025-09-22
phase 1
Longer-term data to be presented at the 5th International ATTR Amyloidosis Meeting for Patients and Doctors on September 25 highlighting up to three years of patient follow-up and additional insight into ATTRv-PN disease-relevant measures CAMBRIDGE, Mass. , Sept.
Broader Trends in Cell and Gene Therapy
CGTLive
2025-09-22
car-t
cell therapy
gene therapy
Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, discussed innovations in viral vectors, CAR-T, and more.
Around the Helix: Cell and Gene Therapy Company Updates – September 3, 2025
CGTLive
2025-09-21
gene therapy
gene therapies
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
CG Oncology (CGON) Is Up 10.7% After Positive Phase 3 Results and Insider Buying
CG Oncology
2025-09-21
phase 3
U.S. stocks, Saudi stocks, stock trading and investment platforms
Krystal Biotech (KRYS): Valuation in Focus After FDA Expands Vyjuvek Use to All Ages and Home Application
Krystal Biotech
2025-09-21
vyjuvek
gene therapy
dystrophic epidermolysis bullosa
Krystal Biotech (KRYS) just got a green light from the FDA for a label update on Vyjuvek, its lead gene therapy for dystrophic epidermolysis bullosa (DEB). Now, Vyjuvek can be used in patients from birth, and, more importantly, can be applied at home by families and patients themselves. This shift away from clinic-only application is not just about convenience; it potentially opens access to a broader group of patients, streamlines the treatment journey for families, and signals that...
John Murphy, PhD; and Tia DiTommaso, PhD, on Preclinical Evidence for PH1 Gene Therapy
CGTLive
2025-09-20
gene therapy
FDA Activity Recap: August 2025 Features Papillomatosis Gene Therapy Approval, Eli-Cel Labelling Update, and More
CGTLive
2025-09-19
gene therapy
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.
Dual-Vector AAV Gene Therapy for GM2 Gangliosidosis Shows Promising Early Outcomes
CGTLive
2025-09-19
aav
phase 1
phase 1/2
gene therapy
The rAAVrh8 gene therapy delivers HEXA and HEXB genes and has posted positive phase 1/2 results in infantile GM2 gangliosidosis, with enhancements in enzymatic activity and neurological outcomes.
NetworkNewsAudio Announces Audio Press Release (APR) on Innovation in Cancer, Rare Disease Treatment Space
Autolus Therapeutics
2025-09-19
pipeline
AUSTIN, Texas, Sept. 19, 2025 (GLOBE NEWSWIRE) -- via IBN – NetworkNewsAudio announces the Audio Press Release (APR) titled “Innovative Therapeutics Pipeline Positions Industry for Strong Market Growth,” featuring Oncotelic
Iovance Biotherapeutics Reports Inducement Grants under NASDAQ Listing Rule 5635(c)(4)
Iovance Biotherapeutics
2025-09-19
til
SAN CARLOS, Calif. , Sept. 19, 2025 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA) ("Iovance" or the “Company”), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (“TIL”) therapies for patients with cancer,
FDA Grants Accelerated Approval to First Treatment for Barth Syndrome
FDA Press
2025-09-19
accelerated approval
The U.S. Food and Drug Administration granted accelerated approval to Forzinity (elamipretide) injection as the first treatment for Barth syndrome, in patients weighing at least 30 kg.
FDA update allows at-home gene therapy for all DEB patients
Krystal Biotech
2025-09-19
vyjuvek
gene therapy
The FDA approved an update to the prescribing information of gene therapy Vyjuvek expanding access to all patients and allowing at-home use.
MiNK Therapeutics Appoints Dr. Terese C. Hammond to Accelerate iNKT Pipeline to Pivotal Development
Mink Therapeutics
2025-09-19
pivotal
Imminent launch of grant-funded clinical trial in graft-versus-host disease (GVHD) Advancing clinical program in severe pulmonary inflammatory disease NEW YORK , Sept. 18, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (Nasdaq: INKT), a clinical-stage biopharmaceutical company pioneering
NKGen Biotech to Present on the Use of Troculeucel for Neurodegenerative Diseases at the 3rd China Great Bay Cell and Gene Therapy Forum
nkgen biotech
2025-09-18
gene therapy
[Updated September 24, 2025]: Due to severe weather conditions and Typhoon-related disruptions affecting the Guangzhou area, Dr. Song will no longer be speaking at the 3rd China Great Bay Cell and Gene Therapy Forum as originally scheduled. We thank the organizers and wish everyone in the region safety during this time. [Updated September 18, 2025, […] The post NKGen Biotech to Present on the Use of Troculeucel for Neurodegenerative Diseases at the 3rd China Great Bay Cell and Gene Therapy Forum first appeared on NKGen Biotech .
Immix Biopharma Announces 50% Enrollment Milestone Surpassed in its ongoing relapsed/refractory AL Amyloidosis Clinical Trial, NEXICART-2
Immix Biopharma
2025-09-18
bla
cell therapy
cell therapies
– On track for first FDA Biologics License Application (BLA) approved cell therapy in orphan indication relapsed/refractory AL Amyloidosis – Los Angeles, CA, Sept. 18, 2025 (GLOBE NEWSWIRE) — Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and other serious diseases, today announced […] The post Immix Biopharma Announces 50% Enrollment Milestone Surpassed in its ongoing relapsed/refractory AL Amyloidosis Clinical Trial, NEXICART-2 first appeared on IMMIX BIOPHARMA INC .
Andembry wins NICE nod
CSL Behring
2025-09-18
hereditary angioedema
The National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending Andembry (garadacimab) for the prevention of recurrent attacks of hereditary angioedema (HAE) in people aged 12 years and older, if they have two or more attacks a month.
Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Transfusion-Dependent Beta Thalassemia and Sickle Cell Disease in Italy
Vertex
2025-09-18
scd
casgevy
gene editing
beta thalassemia
sickle cell disease
CASGEVY is the first, and only, gene editing therapy approved for the treatment of transfusion-dependent beta thalassemia ( TDT) and sickle cell disease (SCD) in Europe - - Italy has the largest population of people living with TDT in Europe - LONDON --(BUSINESS WIRE)--Sep.
BioMarin Pharmaceutical Inc.’s (BMRN) Phenylketonuria Candidate Drug Impresses in Phase 3 Trials
BioMarin Pharmaceutical
2025-09-18
phase 3
pivotal
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the best beginner stocks to buy, according to analysts. On September 6, the company delivered positive pivotal Phase 3 data for PALYNZIQ (pegvaliase-pqpz) in Adolescents with Phenylketonuria. Clinical data showed that the candidate drug successfully triggered a 49.7% decrease in mean blood phenylalanine (Phe) levels in adolescents aged […]
Intellia Therapeutics Completes Enrollment in the Global Phase 3 HAELO Study of Lonvoguran Ziclumeran (lonvo-z) for Hereditary Angioedema
Intellia Therapeutics Inc.
2025-09-18
bla
phase 3
hereditary angioedema
Completed patient enrollment within nine months with nearly half enrolled from the U.S. Expect to report Phase 3 topline data in the first half of 2026 On track to submit BLA in the second half of 2026 for an anticipated U.S. launch in the first half of 2027 Plan to present additional data from the
United States Hemophilia A Treatment Market Growth Fueled by Gene Therapy & FDA Approvals | Major Players: Takeda Pharmaceutical Company Limited, SANOFI CORPORATION, Genentech USA, Inc., Novo Nordisk, Pfizer Inc., CSL Behring LLC.
CSL Behring
2025-09-18
gene therapy
hemophilia a
Global Hemophilia A Treatment Market reached US 9 1 billion in 2022 and is expected to reach US 13 3 billion by 2031 growing with a CAGR of 5 during the forecast period 2024 2031 The Hemophilia A Treatment market ...
BioMarin Pharmaceutical Inc.’s (BMRN) Phenylketonuria Candidate Drug Impresses in Phase 3 Trials
BioMarin Pharmaceutical
2025-09-18
phase 3
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the best beginner stocks to buy, according to analysts.
Hemostemix to Present at Life Science Investor Forum
Hemostemix
2025-09-17
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta – September 17, 2025 – Hemostemix Inc. (TSXV: HEM; OTCQB: HMTXF; FSE: 2VF0), the leading autologous (patient’s own) stem cell therapy company offering VesCell™ (ACP-01) to individuals suffering from peripheral arterial disease, chronic limb threatening ischemia, angina, ischemic cardiomyopathy, non-ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, in Florida under Florida’s SB 1768, is pleased to announce that Chief Executive Officer Thomas Smeenk will present live at the Life Science Investor Forum on Thursday, September 18, 2025, at 12:00 p.m. ET .
Hemostemix to Present its Clinical Data of the Treatment of Severe Angina
Hemostemix
2025-09-17
autologous
cell therapy
heart failure
cardiomyopathy
Calgary, Alberta – September 16, 2025 – Hemostemix Inc. (TSXV: HEM; OTCQB: HMTXF; FSE: 2VF0), the leading autologous (patient’s own) stem cell therapy company offering VesCell™ (ACP-01) to individuals suffering from peripheral arterial disease, chronic limb threatening ischemia, angina, ischemic cardiomyopathy, non-ischemic dilated cardiomyopathy, congestive heart failure, and total body ischemia, in Florida under Florida’s SB 1768, announces that Croom Lawrence will present Hemostemix’s data on the treatment of Severe Angina Pectoris at the company’s weekly webinar on Thursday, September 18, 2025 .
AskBio Announces Completion of Enrollment in Phase 1 Clinical Trial of AB-1005 Gene Therapy for Multiple System Atrophy-Parkinsonian Type (MSA-P)
askbio
2025-09-17
aav2
rmat
phase 1
phase 2
gene therapy
viral vector
Research Triangle Park, N.C. – SEPTEMBER 17, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the completion of enrollment for REGENERATE MSA-101, its Phase 1 clinical trial of AB-1005, an investigational gene therapy being developed as a treatment for multiple system atrophy-parkinsonian type (MSA-P).1 The completion of enrollment marks a significant milestone in the development of AB-1005, an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, and it brings this potential treatment option one step closer to reaching the many patients affected by MSA-P. MSA-P can initially be difficult to distinguish from Parkinson’s disease and is marked by slow movement, lack of coordination, imbalance, dizziness, and fainting, among other symptoms.2 Individuals experience increasing difficulty with movement and autonomic dysfunction.2 This is a result of progressive loss of nerve cells in the brain and spinal cord.2 Affecting approximately 400,000 people worldwide, MSA is a rare disease that…
Dystrophic Epidermolysis Bullosa Pipeline Insight 2025: Krystal Biotech's FDA Label Expansion for VYJUVEK, Emerging Gene Therapies and Regenerative Approaches Drive Innovation
Krystal Biotech
2025-09-17
vyjuvek
gene therapies
dystrophic epidermolysis bullosa
Dystrophic epidermolysis bullosa is a rare and debilitating genetic skin disorder characterized by extreme skin fragility painful blistering chronic wounds and risk of scarring and squamous cell carcinoma For decades treatment options were largely limited to supportive wound care pain ...
Advancing Cancer Research Brings New Hope for Patients Worldwide
Autolus Therapeutics
2025-09-17
glioblastoma
pancreatic cancer
Progress in RNA-based medicines, immunotherapies and targeted treatments is not simply desirable, it is a critical necessity. Deadly cancers such as glioblastoma and pancreatic cancer remain among the hardest to treat, while rare pediatric disorders still take a tragic toll on young patients each year. Against this backdrop of significant unmet need, Oncotelic Therapeutics Inc. (OTCQB: OTLC) (Profile) distinguishes itself. Guided by the forward-looking leadership of chair and CEO Dr. Vuong Trieu
Nurix Therapeutics Presents Preclinical Data from Novel IRAK4 Degrader, GS-6791, Demonstrating Potent Inhibition of IL-1 and IL-36 in Vitro and Efficacy in a Model of Dermatitis
nurix therapeutics
2025-09-17
phase 1
first-in-human
Data support advancement of GS-6791 into clinical testing in patients with inflammatory diseases; first-in-human Phase 1 trial in healthy volunteers is ongoing Data are being presented at the European Academy of Dermatology and Venereology (EADV) Congress SAN FRANCISCO, Sept.
VarmX and CSL to collaborate in research deal worth up to $2.1bn
PMLive Research
2025-09-17
fast track
VarmX's lead candidate, VMX-C001, recently received US FDA Fast Track Designation
Dystrophic Epidermolysis Bullosa Pipeline Insight 2025: Krystal Biotech’s FDA Label Expansion for VYJUVEK, Emerging Gene Therapies and Regenerative Approaches Drive Innovation
Krystal Biotech
2025-09-17
vyjuvek
gene therapies
dystrophic epidermolysis bullosa
Dystrophic epidermolysis bullosa is a rare and debilitating genetic skin disorder characterized by extreme skin fragility, painful blistering, chronic wounds, and risk of scarring and squamous cell carcinoma. For decades, treatment options were large...
Navigating the anxiety of change while notching the improvements
AADC News/BioNews
2025-09-16
gene therapy
As parents, Judy and I have learned that change can mean very different things to different children. For many, change brings excitement, curiosity, and growth. For our daughter, Rylae-Ann, change once meant fear, uncertainty, and overwhelming anxiety because of her rare neurotransmitter disease, aromatic l-amino acid decarboxylase (AADC) deficiency. Before she received gene therapy in […] The post Navigating the anxiety of change while notching the improvements appeared first on AADC News .
Zero Grade 2+ Side Effects: Autolus' Revolutionary CAR-T Treatment Shows Promise in Lupus Patients
Autolus Therapeutics
2025-09-16
crs
car-t
Autolus' CARLYSLE study of obe-cel CAR-T therapy demonstrates clean safety profile in srSLE patients with no DLTs or severe CRS. All patients show SLEDAI-2K improvement with 3 complete renal responses.
Autolus Therapeutics Announces Upcoming Presentation at ACR Convergence 2025 on CARLYSLE Study Results for Severe Refractory Systemic Lupus Erythematosus
Autolus Therapeutics
2025-09-16
cell therapies
Autolus Therapeutics will present a study on obe-cel for systemic lupus erythematosus at ACR Convergence 2025.Quiver AI SummaryAutolus Therapeutics plc, a biopharmaceutical company focused on programmed T cell therapies, has announced the online publication of an abstract for the American College of Rheumatology Convergence 2025, scheduled for October 24-29 in Chicago. The company will present a poster featuring updated results from the CARLYSLE
Krystal Biotech Stock Up on FDA Nod to Vyjuvek Label Update
Krystal Biotech
2025-09-16
vyjuvek
KRYS shares jump after the FDA expands Vyjuvek's label to include DEB patients from birth, with added at-home use flexibility.
Amgen and Kyowa Kirin announce rocatinlimab results for atopic dermatitis
PMLive Research
2025-09-16
phase 3
The phase 3 study included around 2,600 patients
Reflecting On Therapeutics Stocks’ Q2 Earnings: BioMarin Pharmaceutical (NASDAQ:BMRN)
BioMarin Pharmaceutical
2025-09-16
cystic fibrosis
sickle cell disease
spinal muscular atrophy
The end of an earnings season can be a great time to discover new stocks and assess how companies are handling the current business environment. Let’s take a look at how BioMarin Pharmaceutical (NASDAQ: BMRN) and the rest of the therapeutics stocks fared in Q2. Over the next few years, therapeutic companies, which develop a wide variety of treatments for diseases and disorders, face strong tailwinds from advancements in precision medicine (including the use of AI to improve hit rates) and growing demand for treatments targeting rare diseases. However, headwinds such as rising scrutiny over drug pricing, regulatory unknowns, and competition from larger, more resourced pharmaceutical companies could weigh on growth. The 9 therapeutics stocks we track reported a strong Q2. As a group, revenues beat analysts’ consensus estimates by 5.1%. Luckily, therapeutics stocks have performed well with share prices up 11% on average since the latest earnings results. BioMarin Pharmaceutical (NASDAQ: BMRN) Pioneering treatments for conditions that often had no previous therapeutic options, BioMarin Pharmaceutical (NASDAQ: BMRN) develops and commercializes therapies that address the root causes of…
Dystrophic Epidermolysis Bullosa: FDA Approves Labeling Changes for Topical Gene Therapy
Krystal Biotech
2025-09-16
gene therapy
dystrophic epidermolysis bullosa
The indication has been expanded to include use in newborns and allows patients and caregivers to apply the treatment at home.
InspiroGene by McKesson Selected as the Specialty Pharmacy Partner for Iovance’s Amtagvi®—First Cell Therapy Approved for Solid Tumors
Iovance Biotherapeutics
2025-09-15
amtagvi
cell therapy
Major DEB Treatment Breakthrough: VYJUVEK Now Available From Birth, Patients Can Self-Administer at Home
Krystal Biotech
2025-09-15
vyjuvek
FDA approves expanded VYJUVEK label for DEB treatment from birth, enabling at-home application by patients/caregivers. Update allows flexible wound dressing management, based on real-world data.
Kalaris Therapeutics Now Enrolling Phase 1b/2 Multiple Ascending Dose Study of TH103 in Neovascular Age-Related Macular Degeneration | Mon, 09/15/2025
Kalaris Therapeutics
2025-09-15
phase 3
Recently initiated Phase 1b/2 multiple ascending dose study intended to assess safety and preliminary efficacy in nAMD patients receiving up to four initial monthly doses of TH103 Phase 1b/2 study to inform dose selection for potential Phase 3 development program, with initial data expected in 2H
Krystal Biotech gets expanded FDA approval for Vyjuvek
Krystal Biotech
2025-09-15
vyjuvek
gene therapy
dystrophic epidermolysis bullosa
Krystal Biotech (NASDAQ:KRYS) announced that the FDA has approved an updated label for its gene therapy, Vyjuvek (beremagene geperpavec). The new approval broadens the patient eligibility for Vyjuvek to include individuals with dystrophic epidermolysis bullosa from birth. It also allows patients greater flexibility in how they apply Vyjuvek and man...
Krystal Biotech Announces FDA Approval of VYJUVEK Label Update to Include DEB Patients from Birth and Allow Self-Application
Krystal Biotech
2025-09-15
vyjuvek
dystrophic epidermolysis bullosa
FDA approves VYJUVEK label update, allowing DEB patients from birth to apply treatment at home, enhancing flexibility and compliance.Quiver AI SummaryKrystal Biotech, Inc. announced that the FDA has approved a label update for its genetic medicine VYJUVEK® (beremagene geperpavec-svdt), allowing its use for dystrophic epidermolysis bullosa (DEB) patients from birth. The updated label enables patients and caregivers to apply VYJUVEK at home and off
Kyverna Therapeutics to Highlight Interim Phase 2 Data from KYV-101 KYSA-6 Study in Myasthenia Gravis at AANEM 2025
Kyverna Therapeutics
2025-09-15
kyv-101
phase 2
phase 3
Oral presentation to include topline efficacy and safety data for 6 patients with up to 9 months of follow up Enrollment for registrational Phase 3 portion of KYSA-6 trial in MG on track to initiate by year-end 2025 EMERYVILLE, Calif., Sept. 15, 2025 (GLOBE NEWSWIRE) -- Kyverna Therapeutics,
FDA expands label for Krystal’s gene therapy; The latest on Novo Nordisk’s Wegovy
Krystal Biotech
2025-09-15
gene therapy
Plus, news about Revvity, Ocugen and Kwangdong.
FDA expands B-VEC label to include patients from birth with dystrophic epidermolysis bullosa
Krystal Biotech
2025-09-15
dystrophic epidermolysis bullosa
The updated label is based on real-world data collected since B-VEC was launched in the United States, following its approval in May of 2023.
Buy Rating for Krystal Biotech Driven by Vyjuvek’s Expanded Label and Market Potential
Krystal Biotech
2025-09-15
vyjuvek
William Blair analyst Sami Corwin has maintained their bullish stance on KRYS stock, giving a Buy rating on September 9. Sami Corwin has given his Buy rating due to...
FDA Expands Krystal Biotech Vyjuvek's Label Allowing Newborns Access To Gene Therapy For Blistering Skin
Krystal Biotech
2025-09-15
vyjuvek
gene therapy
U.S. stocks, Saudi stocks, stock trading and investment platforms
Krystal Biotech gets expanded FDA approval for Vyjuvek (KRYS:NASDAQ)
Krystal Biotech
2025-09-15
vyjuvek
dystrophic epidermolysis bullosa
FDA expands Vyjuvek's use for dystrophic epidermolysis bullosa from birth, offering new treatment flexibility. Read more here.
Krystal Biotech announces FDA approval of updated Vyjuvek label
Krystal Biotech
2025-09-15
vyjuvek
Krystal Biotech Inc: Krystal Biotech announces FDA approval of updated Vyjuvek® label ...
UPDATE – Kalaris Therapeutics Now Enrolling Phase 1b/2 Multiple Ascending Dose Study of TH103 in Neovascular Age-Related Macular Degeneration | Mon, 09/15/2025
Kalaris Therapeutics
2025-09-15
phase 3
Recently initiated Phase 1b/2 multiple ascending dose study intended to assess safety and efficacy in nAMD patients receiving four initial monthly doses of TH103 Phase 1b/2 study to inform dose selection for potential Phase 3 development program, with initial data expected in 2H 2026 TH103’s
Innovative Therapeutics Pipeline Positions Industry for Strong Market Growth
Autolus Therapeutics
2025-09-15
sarepta
Detailed price information for Sarepta Therapeutics (SRPT-Q) from The Globe and Mail including charting and trades.
Ryoncil Launch Highlighted at Global Healthcare Conferences
Mesoblast
2025-09-14
ryoncil
Kyverna Therapeutics to Present at Upcoming Investor Conferences in September
Kyverna Therapeutics
2025-09-14
cell therapies
EMERYVILLE, Calif., Aug. 25, 2025 (GLOBE NEWSWIRE) -- Kyverna Therapeutics, Inc. (Nasdaq: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, today announced that Warner Biddle, Chief Executive Officer of Kyverna, will
Will Cretostimogene’s Strong 24-Month Results Reshape CG Oncology's (CGON) Immuno-Oncology Narrative?
CG Oncology
2025-09-13
phase 3
bladder cancer
complete response
CG Oncology announced updated long-term results from its Phase 3 BOND-003 trial, showing cretostimogene achieved a 41.8% complete response rate at 24 months in high-risk, heavily pretreated non-muscle invasive bladder cancer patients, with no grade 3 or greater treatment-related adverse events or deaths reported. This marks one of the most durable long-term response rates observed in this difficult patient population, with 90% of 12-month responders maintaining response at two years. We’ll...
Phase 3 Pancreatic Cancer Drug Leads Oncotelic's 6-Program Pipeline Expansion in $50B+ Markets
Autolus Therapeutics
2025-09-12
aml
phase 3
pancreatic cancer
Biotech firm Oncotelic advances OT-101 to Phase 3 in pancreatic cancer, plus 5 late-stage programs including AML/MDS and Parkinson's treatments. Pipeline targets multiple billion-dollar markets.
Precision BioSciences Presents Data from the Phase 1 ELIMINATE-B Trial of PBGENE-HBV at the 6th International Coalition to Eliminate HBV Cure Symposium
precision biosciences
2025-09-12
phase 1
gene editing
DURHAM, N.C. --(BUSINESS WIRE)--Sep. 12, 2025-- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that the Company presented
Vertex Scientists Awarded the 2025 Lasker~DeBakey Clinical Medical Research Award for Pioneering Discoveries in Cystic Fibrosis
Vertex
2025-09-11
cystic fibrosis
BOSTON --(BUSINESS WIRE)--Sep. 11, 2025-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Senior Vice President Paul Negulescu has been named as one of the winners of this year’s Lasker~DeBakey Clinical Medical Research Award for his role “in the development of a novel,
Ferring Japan announces PMDA Acceptance of NDA Filing for nadofaragene firadenovec
Ferring Pharmaceuticals
2025-09-11
phase 3
adenovirus
first-line
gene therapy
bladder cancer
complete response
Nadofaragene firadenovec is the first and only non-replicating gene therapy administered intravesically, and it is already approved and launched in the US This represents the first filing of a novel non-chemotherapy approach in non-muscle invasive bladder cancer (NMIBC) treatment In the Japanese Phase 3 clinical trial, 75% complete response rate was achieved with single quarterly dosing, without requiring re-induction protocols1 This acceptance highlights Ferring's ongoing commitment to establish the new standard of care for BCG-unresponsive NMIBC Ferring Pharmaceuticals Co., Ltd. announced that the PMDA has accepted the NDA for nadofaragene firadenovec for review, following submission on August 27th, 2025. This non-replicating gene therapy, administered intravesically, offers patients with NMIBC a bladder-sparing treatment option. Nadofaragene firadenovec's quarterly dosing eliminates the burden of frequent treatments, while delivering a non-chemotherapy mechanism of action through interferon gene therapy. The NDA acceptance further highlights Ferring's ongoing commitment to establish the new standard of care for high-risk BCG-unresponsive NMIBC. Professor Keiji Inoue, M.D.,…
enGene Reports Third Quarter 2025 Financial Results and Provides Business Update
enGene
2025-09-11
rmat
pivotal
Achieved target enrollment milestone for LEGEND trial pivotal cohort in high-risk BCG unresponsive NMIBC with carcinoma in situLEGEND’s pivotal cohort data update planned for 4Q 2025Detalimogene granted Regenerative Medicine Advanced Therapy (RMAT) designation by FDABiologic License Application (...
Ferring Pharmaceuticals / Foster + Partners
Ferring Pharmaceuticals
2025-09-11
pivotal
Completed in 2022 in København, Denmark. Images by Nigel Young. Ferring Pharmaceuticals A/S home in Copenhagen. The site occupies a pivotal waterfront position alongside the Øresund crossing, just north of...
enGene Holdings Achieves Key Milestone in LEGEND Trial and Secures RMAT Designation
enGene
2025-09-11
rmat
pivotal
enGene Holdings ( ($ENGN) ) has provided an announcement. enGene Holdings has reached a significant milestone by achieving target enrollment for its pivotal LEGEND ...
100 Patients Enrolled: enGene's Breakthrough Bladder Cancer Gene Therapy Gains Special FDA Status
enGene
2025-09-11
rmat
pivotal
gene therapy
bladder cancer
Clinical-stage biotech enGene reaches full enrollment in pivotal LEGEND trial for bladder cancer therapy, secures RMAT designation. Q3 loss $28.9M with $224.9M cash runway into 2027.
enGene Posts 78% Expense Jump in Q3
enGene
2025-09-11
rmat
pivotal
Key PointsenGene achieved its target enrollment milestone of 100 patients in its pivotal LEGEND trial and secured Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA.
Nancy T. Chang, Proven Biotechnology Leader, Joins Immix Biopharma Board of Directors
Immix Biopharma
2025-09-11
cell therapies
– Dr. Chang led XOLAIR® from invention through FDA approval and commercialization, exceeding $5 billion in sales so far – Los Angeles, CA, Sept. 11, 2025 (GLOBE NEWSWIRE) — Immix Biopharma, Inc. (“Company”, “We” or “Us”), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and other serious diseases, today announced that Nancy T. […] The post Nancy T. Chang, Proven Biotechnology Leader, Joins Immix Biopharma Board of Directors first appeared on IMMIX BIOPHARMA INC .
Ferring Japan announces PMDA Acceptance of NDA Filing for nadofaragene firadenovec
Ferring Pharmaceuticals
2025-09-11
gene therapy
[spb_boxed_content type=”coloured” box_link_target=”_self” padding_vertical=”0″ padding_horizontal=”0″ width=”1/1″ el_position=”first last”] Ferring Japan announces PMDA Acceptance of NDA Filing for nadofaragene firadenovec [/spb_boxed_content] [spb_blank_spacer height=”30px” width=”1/1″ el_position=”first last”] [spb_boxed_content type=”coloured” box_link_target=”_self” padding_vertical=”0″ padding_horizontal=”0″ width=”1/1″ el_position=”first last”] Nadofaragene firadenovec is the first and only non-replicating gene therapy administered intravesically, and it is already approved and launched in the US This represents the first filing of […]
Instil Bio Announces ImmuneOnco’s Presentation of ‘2510 Monotherapy Data in Patients with 2L+ Squamous NSCLC at the 2025 World Conference on Lung Cancer (WCLC)
Instil Bio
2025-09-10
orr
lung cancer
ORR of 35% in previously treated squamous NSCLC patients with responses across PD-L1 TPS scores Differentiated structure of ‘2510 potentially results in best-in-class monotherapy activity in 2L+ NSCLC for PD-(L)1xVEGF bispecifics DALLAS, Sept. 10, 2025 (GLOBE NEWSWIRE) -- Instil Bio, Inc.
FDA nod for Janssen’s Inlexzo in NMIBC
Janssen Global Services
2025-09-10
bladder cancer
The US Food and Drug Administration has approved Janssen Biotech’s Inlexzo (gemcitabine) intravesical system for adults with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.
FDA nod for Janssen’s Inlexzo in NMIBC
Janssen Global Services
2025-09-10
bladder cancer
The US Food and Drug Administration has approved Janssen Biotech’s Inlexzo (gemcitabine) intravesical system for adults with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.
AMGEN AND KYOWA KIRIN ANNOUNCE TOP-LINE RESULTS FROM ROCATINLIMAB PHASE 3 ASCEND LONG-TERM EXTENSION STUDY IN ADULTS WITH MODERATE TO SEVERE ATOPIC DERMATITIS
Kyowa Kirin Co.
2025-09-09
phase 3
top-line results
Germany: CSL’s Andembry HTA Submission Offers Critical Indirect Comparison Lessons
CSL Behring
2025-09-09
hereditary angioedema
Health technology assessment authorities in Germany have concluded that CSL Behring’s Andembry offers significant additional benefits for hereditary angioedema patients, which could strengthen the company’s position during pricing negotiations.
CG Oncology (CGON): Assessing Valuation Following Landmark 24-Month BOND-003 Results for Bladder Cancer Therapy
CG Oncology
2025-09-09
bladder cancer
complete response
If you have been tracking CG Oncology (CGON), you probably noticed a surge in the spotlight following its latest clinical update. The company just shared new long-term data from its BOND-003 Cohort C study, revealing a strong 24-month complete response rate for cretostimogene monotherapy in high-risk, heavily pretreated non-muscle invasive bladder cancer patients. The absence of new safety concerns, along with durable patient responses in such a tough disease setting, will likely catch the...
Germany: CSL’s Andembry HTA Submission Offers Critical Indirect Comparison Lessons
CSL Behring
2025-09-09
hereditary angioedema
Health technology assessment authorities in Germany have concluded that CSL Behring’s Andembry offers significant additional benefits for hereditary angioedema patients, which could strengthen the company’s position during pricing negotiations.
CRISPR Therapeutics to Present Late-Breaking Data at the American Heart Association (AHA) Scientific Sessions 2025
CRISPR
2025-09-09
crispr
phase 1
ZUG, Switzerland and BOSTON , Sept. 09, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that a late-breaking oral presentation highlighting the Company's Phase 1
Dianthus Therapeutics Announces Positive Data for Claseprubart (DNTH103) from the Phase 2 MaGic Trial in Generalized Myasthenia Gravis, Supporting Its Potential Best-In-Class ProfilePDF VersionRead More
Dianthus Therapeutics
2025-09-09
myasthenia gravis
Claseprubart 300mg and 600mg Q2W doses both achieved statistically significant and clinically meaningful improvements in Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores at Week 13 Claseprubart demonstrated a rapid and sustained onset of action, PDF Version Read More
Hemophilia Market Surges as Gene Therapy and Novel Biologics Transform Patient Outcomes 2025 | Top key players - Bayer AG, BioMarin Pharmaceutical, Inc., CSL Behring.
CSL Behring
2025-09-09
gene therapy
Market Size and Growth The Hemophilia Market reached US 13 03 Billion in 2024 and is expected to reach US 21 82 Billion by 2033 growing at a CAGR of 5 9 during the forecast period 2025 2033 The Hemophilia ...
AMGEN AND KYOWA KIRIN ANNOUNCE TOP-LINE RESULTS FROM ROCATINLIMAB PHASE 3 ASCEND LONG-TERM EXTENSION STUDY IN ADULTS WITH MODERATE TO SEVERE ATOPIC DERMATITIS
Kyowa Kirin
2025-09-09
top-line results
/PRNewswire/ -- Amgen (NASDAQ: AMGN) and Kyowa Kirin Co., Ltd. (TSE: 4151) today announced preliminary top-line results from the ASCEND study evaluating...
NKGen Biotech, Inc. Announces Completion of NKMax Co., Ltd. Acquisition; Gains Full Control of Global Manufacturing and Intellectual Property Assets
nkgen biotech
2025-09-09
pivotal
cell therapy
NKGen acquires controlling stake in NKMax, marking a pivotal moment in its transformation into a fully integrated, self-sustaining, cell therapy company with global manufacturing and IP control. Approximately $16.9 million paid to acquire 65% equity stake in recapitalized debt-free NKMax, with NKMax operations now expected to be funded through 2026. Funding provided by AlpineBrook Capital […] The post NKGen Biotech, Inc. Announces Completion of NKMax Co., Ltd. Acquisition; Gains Full Control of Global Manufacturing and Intellectual Property Assets first appeared on NKGen Biotech .
Capricor Therapeutics Responds to FDA Posting of Complete Response Letter (CRL) for Deramiocel
Capricor Therapeutics
2025-09-09
deramiocel
complete response
ImmunityBio’s ANKTIVA® Reverses Lymphopenia and Extends Overall Survival in Patients With Advanced Non-Small Cell Lung Cancer Resistant to Checkpoint Therapy
Immunity Bio
2025-09-08
lung cancer
The post ImmunityBio’s ANKTIVA® Reverses Lymphopenia and Extends Overall Survival in Patients With Advanced Non-Small Cell Lung Cancer Resistant to Checkpoint Therapy appeared first on ImmunityBio .
ImmunityBio’s ANKTIVA® Reverses Lymphopenia and Extends Overall Survival in Patients With Advanced Non-Small Cell Lung Cancer Resistant to Checkpoint Therapy
Immunity Bio
2025-09-08
lung cancer
The post ImmunityBio’s ANKTIVA® Reverses Lymphopenia and Extends Overall Survival in Patients With Advanced Non-Small Cell Lung Cancer Resistant to Checkpoint Therapy appeared first on ImmunityBio .
First Disclosure of Global Interim Phase 2 Data for BioNTech and Bristol Myers Squibb PD-L1xVEGF-A Bispecific Antibody Pumitamig (BNT327/BMS986545) in Patients with Extensive-Stage Small Cell Lung Cancer Shows Encouraging Antitumor Activity
bristol myers squibb
2025-09-08
phase 2
lung cancer
Anixa Biosciences and Moffitt Cancer Center Complete Dosing of Fourth Cohort in Ovarian Cancer CAR-T Clinical Trial; Multiple Patients Surpassing Median Expected Survival
anixa biosciences
2025-09-08
car-t
phase 1
quarterly
neurotoxicity
cell therapies
first-in-human
ovarian cancer
cytokine release syndrome
tumor infiltrating lymphocytes
Continue to observe a positive safety profile—no dose limiting toxicities, cytokine release syndrome or immune effector cell-associated neurotoxicity SAN JOSE, Calif. , Sept. 8, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced it has completed dosing of the fourth cohort in its ongoing Phase 1 clinical trial ( ClinicalTrials.gov NCT05316129 ) evaluating its novel FSHR-targeted CAR-T/CER-T therapy for recurrent ovarian cancer. The study is being conducted through a research partnership with Moffitt Cancer Center ("Moffitt"). The fifth cohort is expected to commence following a routine 30-day safety verification that no adverse effects have been observed in the fourth cohort. To date several treated patients remain alive beyond disease-specific median survival benchmarks. Notably, one patient from the 1 st cohort remains alive 28 months post-treatment. These observations are preliminary and from a small number of patients and dose levels. The fourth cohort in the trial received 3×10⁶ CAR-positive cells per kilogram of body weight—approximately a 30-fold…
A2 Biotherapeutics Presents Data from Ongoing Phase 1/2 EVEREST-2 Study During the International Association for the Study of Lung Cancer 2025 World Conference on Lung Cancer
A2 Bio
2025-09-08
phase 1
phase 1/2
The post A2 Biotherapeutics Presents Data from Ongoing Phase 1/2 EVEREST-2 Study During the International Association for the Study of Lung Cancer 2025 World Conference on Lung Cancer appeared first on A2 Biotherapeutics .
Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-09-08
aav
dtx401
phase 3
gene therapy
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
First Disclosure of Global Interim Phase 2 Data for BioNTech and Bristol Myers Squibb PD-L1xVEGF-A Bispecific Antibody Pumitamig (BNT327/BMS986545) in Patients with Extensive-Stage Small Cell Lung Cancer Shows Encouraging Antitumor Activity
Bristol Myers Squibb
2025-09-08
phase 2
Lineage Cell Therapeutics Initiates Cell Transplant Program In Type 1 Diabetes
lineage cell therapeutics
2025-09-08
allogeneic
cell therapies
Initial Program Focus Will Be On Addressing Manufacturing Scale CARLSBAD, Calif. --(BUSINESS WIRE)--Sep. 8, 2025-- Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel allogeneic, or “off the shelf”, cell therapies for serious
First FDA-approved islet transplant at UI Health ends insulin dependence
CellTrans Inc.
2025-09-05
lantidra
Doctors at UI Health performed the first islet transplant with Lantidra, the only therapy approved by the U.S. Food and Drug Administration to treat brittle type 1 diabetes.
REGENXBIO Presents Positive Twelve-Month Pivotal Data from Phase I/II/III CAMPSIITE® Trial of RGX-121 for Treatment of MPS II
REGENXBIO
2025-09-05
mps ii
pivotal
rgx-121
12-month pivotal data further demonstrate the ability of one-time RGX-121 treatment to improve outcomes for patients with MPS II >80% reduction in CSF levels of HS D2S6, key biomarker of MPS II brain disease, sustained through 1 year Pivotal patients demonstrate continued skill acquisition or
New Data for Genentech’s Vabysmo Reinforce Its Efficacy, Safety and Durability in Wet Age-Related Macular Degeneration (AMD)
Genentech
2025-09-05
wet age-related macular degeneration
SOUTH SAN FRANCISCO, Calif., September 05, 2025--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new data from the AVONELLE-X and SALWEEN studies of Vabysmo® (faricimab-svoa), presented at the 25th Euretina Congress in Paris, France. Data from the open-label AVONELLE-X study reinforce the efficacy, safety and durability of Vabysmo over four years in wet age-related macular degeneration (AMD), a leading cause of vision loss. In the single-arm SALWEEN study, Va
Vyjuvek found similarly effective for Japanese vs. US DEB patients
Epidermolysis Bullosa News
2025-09-05
vyjuvek
gene therapy
In a small Japanese trial, topical gene therapy Vyjuvek showed similar safety and efficacy to that seen in studies involving U.S. patients.
Sarepta Therapeutics Announces Recipients of Route 79, The Duchenne Scholarship Program, for the 2025-2026 Academic Year
Sarepta Therapeutics
2025-09-05
sarepta
– Recipients include 20 individuals living with Duchenne and five siblings of individuals living with the condition – Now in its 8 th year, program has awarded more than 150 scholarships CAMBRIDGE, Mass. --(BUSINESS WIRE)--Sep. 5, 2025-- Sarepta Therapeutics, Inc.
Doctors perform first islet cell transplant with Lantidra
CellTrans Inc.
2025-09-05
lantidra
Doctors at UI Health performed the first islet transplant with Lantidra, the only therapy approved by the U.S. Food and Drug Administration to treat brittle type 1 diabetes. A 69-year-old man from Illinois ...
UI Health Performs First Islet Cell Transplant with Lantidra
CellTrans Inc.
2025-09-05
lantidra
Doctors at UI Health performed the first islet transplant with Lantidra, the only therapy approved by the U.S. Food and Drug Administration to treat brittle type 1 diabetes. A 69-year-old man from Illinois received the therapy on Aug. 26 and subsequently was able to stop taking daily, life-saving insulin injections. Lantidra became available exclusively at UI Health last November.
FDA Announces Real-Time Release of Complete Response Letters, Posts Previously Unpublished Batch of 89
FDA Press
2025-09-04
complete response
The U.S. Food and Drug Administration today announced that it will release future complete response letters (CRLs) promptly after they are issued to sponsors.
Beacon Therapeutics Announces Positive Interim 9+ Month Results from DAWN Trial and 36-Month Phase 2 SKYLINE Trial Data for Laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) at EURETINA 2025
Beacon Therapeutics
2025-09-04
phase 2
retinitis pigmentosa
x-linked retinitis pigmentosa
The post Beacon Therapeutics Announces Positive Interim 9+ Month Results from DAWN Trial and 36-Month Phase 2 SKYLINE Trial Data for Laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) at EURETINA 2025 appeared first on Beacon Therapeutics .
Sangamo Therapeutics Presents Detailed Data from Registrational STAAR Study in Fabry Disease at International Congress of Inborn Errors of Metabolism 2025
Sangamo BioSciences
2025-09-04
fabry disease
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Prostate Cancer Pipeline Analysi, 2025 by DelveInsight | Pfizer, Janssen Pharmaceutical, POINT Biopharma, Tavanta Therapeutics, Exelixis, Astellas Pharma Inc., Antev, ORCA Therapeutics, Regeneron Phar
Janssen Global Services
2025-09-03
prostate cancer
"Prostate Cancer Pipeline Analysis"DelveInsight’s, “Prostate Cancer - Pipeline Insight, 2025,” report provides comprehensive insights about 140+ companies and 150+ pipeline drugs in Prostate Cancer pipeline landscape. It covers the pipeline drug prof...
Lyell Immunopharma Announces the Initiation of a Phase 3 Head-to-Head CAR T-Cell Therapy Clinical Trial in Aggressive Large B-Cell Lymphoma and Formation of Expert Steering Committee
lyell immunopharma
2025-09-03
cd19
cd20
car t
phase 3
second line
cell therapy
cell therapies
b-cell lymphoma
large b-cell lymphoma
PiNACLE - H2H will evaluate rondecabtagene autoleucel (ronde-cel) versus investigator’s choice of approved CD19 CAR T-cell therapies in patients with aggressive large B-cell lymphoma receiving treatment in the second line (2L) setting Ronde-cel is Lyell’s next-generation, dual-targeting CD19/CD20
enGene Achieves Target Enrollment Milestone for LEGEND Trial Pivotal Cohort
enGene
2025-09-03
phase 2
pivotal
gene therapy
bladder cancer
detalimogene voraplasmid
BOSTON & MONTREAL, September 03, 2025--enGene Holdings Inc. (Nasdaq: ENGN, "enGene" or the "Company"), a clinical-stage, non-viral gene therapy company, today announced it has achieved its target enrollment milestone of 100 patients for the pivotal cohort of its ongoing, open-label, multi-cohort Phase 2 LEGEND trial of detalimogene voraplasmid ("detalimogene" and previously EG-70) in patients with high-risk, non-muscle invasive bladder cancer (NMIBC). LEGEND’s pivotal Cohort 1 is studying detali
Prostate Cancer Pipeline Analysi, 2025 by DelveInsight | Pfizer, Janssen Pharmaceutical, POINT Biopharma, Tavanta Therapeutics, Exelixis, Astellas Pharma Inc., Antev, ORCA Therapeutics, Regeneron Phar
Janssen Global Services
2025-09-03
pipeline
"Prostate Cancer Pipeline Analysis"DelveInsight’s, “Prostate Cancer - Pipeline Insight, 2025,” report provides comprehensive insights about 140+ companies and 150+ pipeline drugs in Prostate Cancer pipeline landscape. It covers the pipeline drug prof...
Celularity Announces Filing of Form 10-Q Quarterly Reports for the First Quarter and the Second Quarter 2025, Confirmation of Nasdaq Listing Rule 5250(c)(1) Compliance
celularity
2025-09-03
quarterly
Lyell Immunopharma Announces Participation in September Investor Conferences
lyell immunopharma
2025-09-02
car t
cell therapies
SOUTH SAN FRANCISCO, Calif., Sept. 02, 2025 (GLOBE NEWSWIRE) -- Lyell Immunopharma, Inc. (Nasdaq: LYEL), Lyell Immunopharma, Inc. (Nasdaq: LYEL), a late-stage clinical company advancing next-generation CAR T-cell therapies for patients with cancer, announced today that members of its senior
Nanoscope Strengthens Global Regulatory Pathways for MCO-010 with FDA RMAT and Five EMA Orphan Designations
Nanoscope Therapeutics
2025-09-02
rmat
mco-010
fast track
orphan drug
European Medicines Agency grants MCO-010 five Orphan designations across a wide spectrum of retinal diseases involving photoreceptor loss, including syndromic and non-syndromic, rod- and cone-dominant, and macular dystrophies. FDA grants MCO-010 Regenerative Medicine Advanced Therapy (RMAT) designation, in addition to current Orphan Drug and Fast Track designations, for Stargardt disease. Nanoscope is redefining the standard […] The post Nanoscope Strengthens Global Regulatory Pathways for MCO-010 with FDA RMAT and Five EMA Orphan Designations appeared first on Nanoscope Therapeutics .
Johnson & Johnson Innovative Medicine Partner with Multiple Myeloma Ireland to launch the 2025 "Miles for Myeloma" Challenge
Janssen Global Services
2025-09-02
multiple myeloma
Multiple Myeloma Ireland is the only charitable organisation in Ireland working to empower people affected by multiple myeloma
Genprex to Present and Participate at Upcoming September Investor and Industry Conferences
Genprex
2025-09-02
gene therapy
gene therapies
Presentation to Highlight the Company’s Gene Therapies for Cancer and Diabetes AUSTIN, Texas — (Sept. 2, 2025) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced the Company’s participation in the following upcoming investor and industry conferences being held in […]
CRISPR Therapeutics to Participate in Upcoming Investor Conferences
CRISPR
2025-09-02
crispr
ZUG , Switzerland and BOSTON, Sept. 02, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that members of its senior management team are scheduled to participate in
Anixa Biosciences Announces Time Change for Presentation at the H.C. Wainwright 27th Annual Global Investment Conference
anixa biosciences
2025-09-02
car-t
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
SAN JOSE, Calif. , Sept. 2, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the time of management's presentation at the H.C. Wainwright 27 th Annual Global Investment Conference, being held September 8 th – 10 th , 2025 in New York City, has been changed to 10:30 AM ET on September 9. Mike Catelani, President and CFO of Anixa, will deliver a presentation and will be available for one-on-one meetings during the conference. Details of the presentation are as follows: Event: H.C. Wainwright 27 th Annual Global Investment Conference Date: September 9, 2025 Time: 10:30 AM ET Location: Kennedy I – 4 th Floor, Lotte New York Palace Hotel Webcast: https://journey.ct.events/view/3bd15396-cb90-4cb0-916e-a546bf1f5c10 About Anixa Biosciences, Inc. Anixa is a clinical-stage biotechnology company focused on the treatment and prevention of cancer. Anixa's therapeutic portfolio consists of an ovarian cancer immunotherapy program being developed in collaboration with Moffitt Cancer Center, which uses a novel type of CAR-T, known as chimeric endocrine receptor-T…
Nkarta to Participate in a September Investor Conference
nkarta
2025-09-02
cell therapies
SOUTH SAN FRANCISCO, Calif., Sept. 02, 2025 (GLOBE NEWSWIRE) -- Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies, today announced its participation in the following investor conference: H.C.
Adicet Bio Reports Second Quarter 2025 Financial Results and Provides Business Updates
Adicet Bio
2025-08-29
phase 1
Actively enrolling patients with lupus nephritis (LN), systemic lupus erythematosus (SLE) and systemic sclerosis (SSc) in Phase 1 clinical trial of ADI-001 in autoimmune diseases On track to report preliminary clinical data for ADI-001 in 2H/2025 Enrollment now open for patients with idiopathic
GenSight Biologics Postpones Release of 2025 Half-Year Financial Results
GenSight Biologics
2025-08-29
financial results
GenSight Biologics Postpones Release of 2025 Half-Year Financial Results
GenSight Biologics
2025-08-29
financial results
Iovance Biotherapeutics to Present at Upcoming Conferences
Iovance Biotherapeutics
2025-08-29
til
SAN CARLOS, Calif. , Aug. 29, 2025 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, today announced that senior
GenSight Biologics Postpones Release of 2025 Half-Year Financial Results
GenSight Biologics
2025-08-28
gene therapies
PARIS, August 28, 2025--Regulatory News: GenSight Biologics ("GenSight Biologics" or the "Company") (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that the release of its half-year financial results, originally scheduled for September 19, 2025, has been postponed to September 29, 2025, after the market close
Kyverna Therapeutics Highlights Neuroimmunology CAR T Franchise and Registrational Phase 3 Trial Design in Myasthenia Gravis at Virtual KOL Event
Kyverna Therapeutics
2025-08-28
bla
car t
kyv-101
phase 3
stiff person syndrome
KYV-101 has the potential to deliver durable, drug-free, disease-free remission in myasthenia gravis and set a new treatment standard for stiff person syndrome Innovative FDA-aligned KYSA-6 Phase 3 trial design for myasthenia gravis supports clear and rapid path to BLA Webcast today August 28th, at
Clonogenic hepatocytes drive postnatal liver growth and unlock new avenues for pediatric gene therapy
Fondazione Telethon
2025-08-28
gene therapy
A new study of the San Raffaele Telethon Institute for Gene Therapy show that only 15–20% of hepatocytes in newborn mice are responsible for generating over 90% of the adult liver mass: these findings have major implications for pediatric gene therapy.
Zemcelpro® (UM171 Cell Therapy) receives EC authorization as the first and only cell therapy for blood cancer patients without access to suitable donor cells
Excellthera
2025-08-28
allogeneic
cell therapy
myelodysplastic
Zemcelpro® is conditionally authorized by the European Commission for adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available Zemcelpro® will increase access to allogeneic stem cell transplantation to treat life-threatening haematologic malignancies, including leukemias and myelodysplastic syndromes Timing of […]
SparingVision to Showcase its Gene-Agnostic Gene Therapy Programs at Upcoming Conferences
Sparing Vision
2025-08-28
gene therapy
SparingVision to Showcase its Gene-Agnostic Gene Therapy Programs at Upcoming Conferences Paris, 28 August 2025 – SparingVision (“the Company”), a clinical-stage genomic medicine company transforming the treatment of retinal diseases, today announces that it will showcase its gene-agnostic gene therapy programs, SPVN06 and SPVN20, and research on retinal diseases at upcoming scientific conferences. Conference… The post SparingVision to Showcase its Gene-Agnostic Gene Therapy Programs at Upcoming Conferences appeared first on SparingVision .
Gene Therapy Improves Quality of Life for SCD, Beta Thalassemia
Hematology.org
2025-08-27
scd
gene therapy
beta thalassemia
Gene Therapy Improves Quality of Life for SCD, Beta Thalassemia
Wugen Secures $115 Million to Advance Pivotal Study of First-in-Class Allogeneic CAR-T Therapy, WU-CART-007
Wugen Inc.
2025-08-27
car-t
pivotal
allogeneic
— Financing led by Fidelity Management & Research Company with participation from RiverVest Venture Partners, Lightchain Capital, LYZZ Capital, Abingworth, Intermediate Capital Group (ICG), Tybourne Capital Management, Aisling Capital Management, and other top-tier life sciences investors — — Proceeds will fund the ongoing pivotal T-RRex trial in relapsed/refractory T-ALL/T-LBL — — WU-CART-007 achieved 91% overall... The post Wugen Secures $115 Million to Advance Pivotal Study of First-in-Class Allogeneic CAR-T Therapy, WU-CART-007 appeared first on Wugen .
Allogene Therapeutics Announces Participation in Upcoming Investor Conference
Allogene
2025-08-27
car t
allogeneic
SOUTH SAN FRANCISCO, Calif. , Aug. 27, 2025 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™) products for cancer and autoimmune disease, today announced that it will participate in one
Nanoscope Announces Groundbreaking 3-Year REMAIN Trial Data for Patients with Retinitis Pigmentosa at 2025 Euretina Congress and Retina Society Annual Scientific Meeting
Nanoscope Therapeutics
2025-08-26
retinitis pigmentosa
Talks highlight efficacy, durability, and safety of Nanoscope’s MCO (multi-characteristic opsin) optogenetic therapy platform Data forms the foundation for recent rolling Biologics License Application submission with the FDA for retinitis pigmentosa DALLAS, Aug. 26, 2025 — Nanoscope Therapeutics Inc., a biotechnology company committed to developing and commercializing novel disease-agnostic therapies for patients with photoreceptor loss and vision impairment from […] The post Nanoscope Announces Groundbreaking 3-Year REMAIN Trial Data for Patients with Retinitis Pigmentosa at 2025 Euretina Congress and Retina Society Annual Scientific Meeting appeared first on Nanoscope Therapeutics .
Initial Data Shows 100% Disease Control in 5 Out of 5 Patients With Recurrent Glioblastoma With Two Patients in Near Complete Response Treated With ImmunityBio’s ANKTIVA®, NK Cell Therapy Plus Optune Gio® Device
Immunity Bio
2025-08-26
cell therapy
glioblastoma
nk cell therapy
complete response
The post Initial Data Shows 100% Disease Control in 5 Out of 5 Patients With Recurrent Glioblastoma With Two Patients in Near Complete Response Treated With ImmunityBio’s ANKTIVA®, NK Cell Therapy Plus Optune Gio® Device appeared first on ImmunityBio .
Brinsupri ™ (brensocatib) for the Treatment of Non-Cystic Fibrosis Bronchiectasis
CSL Behring
2025-08-26
cystic fibrosis
Doreen Addrizzo-Harris, MD, FCCP discusses Brinsupri™ (brensocatib), a new FDA-approved treatment for non-cystic fibrosis bronchiectasis. Dr
Brensocatib Approval Highlights Potential of DPP1 Inhibitors for Neutrophil-Mediated Diseases: James D. Chalmers, MBChB, PhD
CSL Behring
2025-08-25
cystic fibrosis
Brensocatib, the first FDA-approved dipeptidyl peptidase 1 (DPP1) inhibitor for non–cystic fibrosis bronchiectasis, may have broader potential for other neutrophil-driven conditions, according to James D. Chalmers, MBChB, PhD.
Non-cystic Fibrosis Bronchiectasis Market Ready to Shift Gears with the Entry of First DPP1 Blocker
CSL Behring
2025-08-25
pivotal
cystic fibrosis
Insmed’s BRINSUPRI (brensocatib) has reached a significant regulatory milestone, earning FDA approval as the first-ever therapy for non-cystic fibrosis bronchiectasis and the first drug targeting DPP1. As the only available treatment in the non-cystic fibrosis bronchiectasis market, this represents both a scientific breakthrough and a pivotal shift in a market long defined by high unmet medical needs.New York, USA, Aug. 25, 2025 (GLOBE NEWSWIRE) -- Non-cystic Fibrosis Bronchiectasis Market Ready
Beam Therapeutics to Participate in Upcoming September 2025 Investor Conferences
beam therapeutics
2025-08-25
base editing
CAMBRIDGE, Mass., Aug. 25, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that management will participate in fireside chats at the following upcoming investor conferences: Citi’s
Caribou Biosciences to Participate in the Citi 2025 Biopharma Back to School Conference
caribou biosciences
2025-08-25
crispr
BERKELEY, Calif., Aug. 25, 2025 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today announced Rachel Haurwitz, PhD, Caribou’s president and chief executive officer, will participate in a fireside chat at the
Fate Therapeutics to Present at Upcoming Investor Conferences
Fate Therapeutics
2025-08-25
pipeline
SAN DIEGO , Aug. 25, 2025 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients, announced that the
Anixa Biosciences to Present at the H.C. Wainwright 27th Annual Global Investment Conference
anixa biosciences
2025-08-25
car-t
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
SAN JOSE, Calif. , Aug. 25, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that management will participate in the H.C. Wainwright 27 th Annual Global Investment Conference being held September 8 th – 10 th , 2025 at the Lotte New York Palace Hotel in New York City. Mike Catelani, President and CFO of Anixa, will deliver a presentation and will be available for one-on-one meetings during the conference. Details of the presentation are as follows: Event: H.C. Wainwright 27 th Annual Global Investment Conference Date: September 8, 2025 Time: 4:30 PM ET Location: Lotte New York Palace Hotel Webcast: https://journey.ct.events/view/3bd15396-cb90-4cb0-916e-a546bf1f5c10 About Anixa Biosciences, Inc. Anixa is a clinical-stage biotechnology company focused on the treatment and prevention of cancer. Anixa's therapeutic portfolio consists of an ovarian cancer immunotherapy program being developed in collaboration with Moffitt Cancer Center, which uses a novel type of CAR-T, known as chimeric endocrine receptor-T cell (CER-T) technology. This technology is…
Ayrmid Ltd. Announces FDA Acceptance and Priority Review for Omidubicel for the Treatment of Severe Aplastic Anemia (SAA)
Gamida Cell
2025-08-25
pdufa
ayrmid ltd.
gamida cell
cell therapy
priority review
FDA assigns Prescription Drug User Fee Act (PDUFA) target date of December 10, 2025 LONDON--Ayrmid Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., a cell therapy pioneer working to turn cells into powerful therapeutics, today announced that the U.S.
iECURE to Present Initial OTC-HOPE Clinical Trial Data of ECUR-506 in Ornithine Transcarbamylase Deficiency at 6th International Symposium on UCDs and 15th International Congress of Inborn Errors of Metabolism
Iecure
2025-08-24
ornithine transcarbamylase
ornithine transcarbamylase deficiency
August 25, 2025 The post iECURE to Present Initial OTC-HOPE Clinical Trial Data of ECUR-506 in Ornithine Transcarbamylase Deficiency at 6th International Symposium on UCDs and 15th International Congress of Inborn Errors of Metabolism first appeared on iECURE .
Janssen Pharmaceutica reports new NLRP3 inflammasome inhibitors
Janssen Global Services
2025-08-23
amyotrophic lateral sclerosis
Several recent Janssen Pharmaceutica NV patents describe new NLRP3 inflammasome inhibitors reported to be useful for the treatment of traumatic brain injury, multiple sclerosis, amyotrophic lateral sclerosis, dementia, Huntington’s, Parkinson’s, Alzheimer’s and motor neuron diseases.
FDA Approves New Option for Non-Cystic Fibrosis Bronchiectasis
CSL Behring
2025-08-22
cystic fibrosis
Oral tablets in two dosages target exacerbations.
1st RDEB patient in US to soon receive gene therapy Zevaskyn
Epidermolysis Bullosa News
2025-08-22
zevaskyn
gene therapy
Abeona is ramping up for Zevaskyn dosing to start, with the first person expected to receive treatment in the next few months.
Beacon Therapeutics Announces EURETINA 2025 Presentations on 9+-Month Phase 2 DAWN and 36-Month Phase 2 SKYLINE Trial Data for laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP)
Beacon Therapeutics
2025-08-21
phase 2
retinitis pigmentosa
x-linked retinitis pigmentosa
The post Beacon Therapeutics Announces EURETINA 2025 Presentations on 9+-Month Phase 2 DAWN and 36-Month Phase 2 SKYLINE Trial Data for laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) appeared first on Beacon Therapeutics .
Sarepta Therapeutics Announces Refinancing of Approximately $700 Million of 1.25% Convertible Senior Notes due 2027
Sarepta Therapeutics
2025-08-21
sarepta
Sarepta entered into exchange agreements with a limited number of holders of its Convertible Senior Notes due 2027 that will extend maturity to 2030 while also de-levering the balance sheet - The transaction further strengthens Sarepta’s capital structure and reflects its proactive approach to
U.S. FDA Approves World's First Cell Therapy (Lantidra) for Type 1 Diabetes
CellTrans Inc.
2025-08-21
lantidra
allogeneic
cell therapy
A historic milestone was set on June 28, 2023, when the U.S. Food and Drug Administration (FDA) granted approval for Lantidra to CellTrans, Inc., a company headquartered in Chicago, Illinois. Lantidra is a groundbreaking allogeneic pancreatic islet cellular therapy derived from the pancreatic cells of deceased donors. This landmark decision made it the world’s first […]
World Conference on Lung Cancer
Immunity Bio
2025-08-20
lung cancer
The post World Conference on Lung Cancer appeared first on ImmunityBio .
Rocket Pharmaceuticals Announces FDA Has Lifted the Clinical Hold on the Pivotal Phase 2 Trial of RP-A501 for the Treatment of Danon Disease
Rocket Pharmaceuticals
2025-08-20
phase 2
pivotal
clinical hold
CRANBURY, N.J. --(BUSINESS WIRE)--Aug. 20, 2025-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S.
Anixa Biosciences Receives Notice of Allowance from Chinese National Intellectual Property Administration for Patent Covering Breast Cancer Vaccine Technology
anixa biosciences
2025-08-20
car-t
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
Notice of Allowance marks first Chinese patent to be issued on Anixa Breast Cancer Vaccine Expands global intellectual property coverage in markets with high breast cancer incidence New patent will provide IP protection of Breast Cancer Vaccine in China into at least the 2040s SAN JOSE, Calif. , Aug. 20, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the Chinese National Intellectual Property Administration (CNIPA) has issued a Notice of Allowance for a new patent related to its breast cancer vaccine technology. This patent, exclusively licensed from Cleveland Clinic, will provide composition of matter protection for the Company's novel approach to breast cancer treatment and prevention in China. With this allowance, Anixa continues to expand the international scope of its intellectual property portfolio, reinforcing its leadership in the field of cancer immunotherapy. The Chinese patent complements patents issued in the United States and other key global jurisdictions, and represents an important step toward future regulatory and commercial…
ImmunityBio Announces Phase 2 Study of ANKTIVA® in Patients with Long COVID
Immunity Bio
2025-08-19
phase 2
The post ImmunityBio Announces Phase 2 Study of ANKTIVA® in Patients with Long COVID appeared first on ImmunityBio .
PTC Therapeutics Receives Complete Response Letter for Vatiquinone NDA
PTC Therapeutics
2025-08-19
complete response
WARREN, N.J. , Aug. 19, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) related to the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living
Genprex Receives Notice of Allowance for Multiple Patents for Reqorsa® Gene Therapy with PD-L1 and PD-1 Antibodies to Treat Cancers
Genprex
2025-08-18
gene therapy
Strengthens Intellectual Property Estate for REQORSA Oncology Program with Immune Checkpoint Inhibitors AUSTIN, Texas — (Aug. 18, 2025) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that the U.S. Patent and Trademark Office has issued a Notice of Allowance […]
Ultragenyx Initiates Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-08-18
aav
bla
dtx401
gene therapy
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
REGENXBIO Announces FDA Review Extension of BLA for RGX-121 to Treat Patients with MPS II
REGENXBIO
2025-08-18
bla
mps ii
pivotal
rgx-121
RGX-121 would be the first and only potential one-time commercially-available therapy designed to directly address the underlying genetic cause of Hunter syndrome, if approved Commercial launch plans remain on track REGENXBIO plans to present updated pivotal data during the ICIEM meeting in
Anixa Biosciences Announces Treatment of Second Patient in Fourth Cohort of Ovarian Cancer CAR-T Clinical Trial
anixa biosciences
2025-08-18
car-t
phase 1
quarterly
cell therapies
first-in-human
ovarian cancer
chimeric antigen receptor
tumor infiltrating lymphocytes
Fourth Cohort Receiving 30x Initial Dose with No Dose-Limiting Toxicities Observed to Date SAN JOSE, Calif. , Aug. 18, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the second patient in the fourth dose cohort has been treated in the ongoing Phase 1 clinical trial evaluating the Company's novel chimeric antigen receptor-T cell (CAR-T) therapy for recurrent ovarian cancer. The study is being conducted through a research partnership with Moffitt Cancer Center ("Moffitt"). Patients in the fourth cohort are receiving a dose of three million CAR-positive cells per kilogram of body weight—representing a 30-fold increase over the initial dose level. To date, no dose-limiting toxicities (DLTs) have been observed in the fourth cohort. Anixa's proprietary CAR-T program targets the follicle-stimulating hormone receptor (FSHR), which preclinical research indicates is selectively expressed on ovarian cells, tumor vasculature, and certain cancer cells—but not in healthy tissue. The ongoing first-in-human clinical trial ( NCT05316129 ) is enrolling adult women…
Izalontamab Brengitecan (EGFRxHER3 ADC) Granted Breakthrough Therapy Designation by U.S. FDA for Patients with Previously Treated Advanced EGFR-Mutated Non-Small Cell Lung Cancer
bristol myers squibb
2025-08-18
lung cancer
breakthrough therapy
Non-Cystic Fibrosis Bronchiectasis Pipeline 2025: Therapies Under Investigation, Clinical Trials Milestones, and FDA Approvals by DelveInsight | Insmed Inc, AstraZeneca, Zambon, CSL Behring
CSL Behring
2025-08-18
cystic fibrosis
(Las Vegas, Nevada, United States) As per DelveInsight’s assessment, globally, Non-Cystic Fibrosis Bronchiectasis pipeline constitutes 15+ key companies continuously working towards developing 15+ Non-Cystic Fibrosis Bronchiectasis treatment therapie...
How the Hereditary Angioedema Market Will Evolve by 2032 - Growth, Segments, and Revenue Trends| Takeda Pharmaceuticals (Japan/U.S.), CSL Behring (U.S.)
CSL Behring
2025-08-18
hereditary angioedema
The latest report on the Hereditary Angioedema Market by Worldwide Market Reports delivers a comprehensive and detailed evaluation of global industry dynamics uncovering the core trends that shape the competitive landscape By analyzing key growth drivers market constraints emerging opportunities ...
Capricor Therapeutics Announces First Subjects Dosed in Phase 1 Clinical Trial of Novel Exosome-Based Vaccine
Capricor Therapeutics
2025-08-18
phase 1
Izalontamab Brengitecan (EGFRxHER3 ADC) Granted Breakthrough Therapy Designation by U.S. FDA for Patients with Previously Treated Advanced EGFR-Mutated Non-Small Cell Lung Cancer
Bristol Myers Squibb
2025-08-18
breakthrough therapy
Iovance’s Amtagvi® (lifileucel) Receives Health Canada Approval for Advanced Melanoma
Iovance Biotherapeutics
2025-08-18
amtagvi
melanoma
solid tumor
cell therapy
First T cell therapy for a solid tumor cancer and first treatment option approved in Canada for advanced melanoma after anti-PD-1 and targeted therapy SAN CARLOS, Calif. , Aug. 18, 2025 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on
MiNK Therapeutics Reports Clinical and Strategic Milestones and Second Quarter 2025 Results
Mink Therapeutics
2025-08-15
gastric cancer
Cash runway extended to deliver clinical program in GVHD and Ph2 results in 2L Gastric Cancer Department of Defense STTR Grant to advance INKTs in GVHD announced; program advancing New clinical grant awarded to launch AgenT-797 in GvHD clinical trial; target initiation 2H2025 NEW YORK , Aug.
Lexeo Therapeutics Reports Second Quarter 2025 Financial Results and Operational Highlights
Lexeo Therapeutics
2025-08-15
financial results
Beam Therapeutics Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to BEAM-101 for the Treatment of Sickle Cell Disease
beam therapeutics
2025-08-14
rmat
base editing
sickle cell disease
CAMBRIDGE, Mass., Aug. 14, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced
The Lancet | Safety and efficacy of MCO-010 optogenetic therapy in patients with Stargardt disease in USA (STARLIGHT): an open-label multi-center Ph2 trial
Nanoscope Therapeutics
2025-08-14
mco-010
Byron L. Lam, Vitaliy Zak, Victor H. Gonzalez, Ninel Z. Gregori, Sai H. Chavala, Subrata Batabyal, Michael Carlson, Sanghoon Kim, Ananta Ayyagari, Jean Chang, Hayley Lane, Nozhat Choudry, John Koester, Mark Von Tress, Jody Piltz-Seymour, Najam A. Sharif, Stephen H. Tsang, Vinit B. Mahajan, David S. Boyer, Allen C. Ho, Samarendra K. Mohanty The post The Lancet | Safety and efficacy of MCO-010 optogenetic therapy in patients with Stargardt disease in USA (STARLIGHT): an open-label multi-center Ph2 trial appeared first on Nanoscope Therapeutics .
Century Therapeutics Reports Second Quarter 2025 Financial Results and Provides Business Update
Century Therapeutics
2025-08-14
financial results
Nanoscope Therapeutics Announces Publication of STARLIGHT Phase 2 Study Evaluating MCO-010 Gene-Agnostic Therapy in Patients with Stargardt Disease
Nanoscope Therapeutics
2025-08-14
mco-010
phase 2
The STARLIGHT study is the first and only clinical trial to demonstrate vision improvement in patients with Stargardt disease. MCO-010 demonstrated mean visual acuity improvement of 12 ETDRS letters over 48 weeks in patients with atrophy confined to the macula -with no reports of serious adverse events. Nanoscope has completed an end of phase 2 meeting with the FDA […] The post Nanoscope Therapeutics Announces Publication of STARLIGHT Phase 2 Study Evaluating MCO-010 Gene-Agnostic Therapy in Patients with Stargardt Disease appeared first on Nanoscope Therapeutics .
MeiraGTx Reports Second Quarter 2025 Financial and Operational Results
MeiraGTx
2025-08-14
bla
phase 2
pivotal
Gained alignment with U.S. Food and Drug Administration (FDA) on the ongoing Phase 2 AQUAx2 randomized double-blind, placebo-controlled pivotal study in Grade 2/3 radiation-induced xerostomia (RIX) to support a potential Biologics License Application (BLA) filing; on track for potential data
Abeona Therapeutics® Reports Second Quarter 2025 Financial Results and Corporate Updates
Abeona Therapeutics
2025-08-14
financial results
BrainStorm Cell Therapeutics Announces Second Quarter 2025 Financial Results and Provides Corporate Update
BrainStorm Cell Therapeutics
2025-08-14
financial results
Kalaris Reports Second Quarter 2025 Financial Results and Provides Business Updates | Wed, 08/13/2025
Kalaris Therapeutics
2025-08-13
phase 1
Continuing to enroll nAMD patients in a Phase 1 trial of TH103, a novel, differentiated anti-VEGF agent engineered to potentially provide longer-lasting and increased anti-VEGF activity to treat neovascular and exudative diseases of the retina; initial clinical data is expected in Q4 2025 Cash and
Instil Bio Reports Second Quarter 2025 Financial Results and Provides Corporate Update
Instil Bio
2025-08-13
ind
clearance
lung cancer
With the clearance of the U.S. IND, initiation of the U.S. clinical trial of AXN-2510/IMM2510 (“‘2510”) anticipated before the end of 2025 Updated ‘2510 monotherapy data in squamous-NSCLC to be presented at IASLC’s 2025 World Conference on Lung Cancer (WCLC) by ImmuneOnco DALLAS, Aug.
Sarepta Therapeutics Announces Advancement of siRNA Collaboration and Sale of Arrowhead Equity Investment
Sarepta Therapeutics
2025-08-13
phase 1
sarepta
phase 1/2
dose escalation
Safety review leading to dose escalation and achievement of first predetermined enrollment target in Phase 1/2 study of SRP-1003 for DM1 triggers $100 million milestone payment - Sarepta sells at least $174 million of Arrowhead common stock for cash proceeds, Arrowhead will redeem $50 million of
Tourmaline Bio Reports Second Quarter 2025 Financial Results and Recent Business Highlights
Talaris Therapeutics
2025-08-13
quarterly
financial results
– Reported positive topline results from the ongoing Phase 2 TRANQUILITY trial of pacibekitug in May 2025, demonstrating rapid, deep, and durable reductions in high-sensitivity C-reactive protein with quarterly dosing – — Additional data from the ongoing Phase 2 TRANQUILITY trial to be presented at
Allogene Therapeutics Reports Second Quarter 2025 Financial Results and Business Update
Allogene
2025-08-13
phase 2
pivotal
first line
Pivotal Phase 2 ALPHA3 Trial with Cemacabtagene Ansegedleucel (Cema-Cel) in First Line (1L) Consolidation in Large B-Cell Lymphoma (LBCL) ALPHA3 Proceeding as a Two-Arm Randomized Trial Comparing Cema-cel After Standard Fludarabine and Cyclophosphamide (FC) Lymphodepletion vs.
Avista Therapeutics and Forge Biologics Announce AAV Development and cGMP Manufacturing Partnership
Forge Biologics
2025-08-13
aav
“Forge’s AAV-specific manufacturing expertise and platform technologies give us confidence in a smooth path forward in the development of AVST-101,” said Robert Lin, Ph.D., Chief Executive Officer of Avista. The post Avista Therapeutics and Forge Biologics Announce AAV Development and cGMP Manufacturing Partnership appeared first on Forge Biologics .
ImmunityBio Reports Complete Responses in Non-Hodgkin Waldenstrom Lymphoma Patients with Chemotherapy-Free, First-In-Class CD19 CAR-NK Immunotherapy
Immunity Bio
2025-08-13
cd19
car-nk
The post ImmunityBio Reports Complete Responses in Non-Hodgkin Waldenstrom Lymphoma Patients with Chemotherapy-Free, First-In-Class CD19 CAR-NK Immunotherapy appeared first on ImmunityBio .
Adaptimmune Reports Q2 Financial Results and Provides Business Update
Adaptimmune Therapeutics
2025-08-13
tecelra
afami-cel
solid tumor
cell therapy
cell therapies
Q2 Tecelra sales of $11.1m with 16 patients invoiced represents >150% growth vs Q1 2025 Entered into a definitive agreement for the sale of TECELRA, lete-cel, afami-cel, and uza-cel cell therapies to US WorldMeds for $55 million upfront with up to $30 million in future milestone payments Following the transaction, Adaptimmune has repaid its debt and is restructuring to maximize value from remaining assets, including PRAME and CD70 directed T-cell therapies Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - August 13, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP) today reported financial results and provided a business update for the second quarter ended June 30, 2025. Adrian Rawcliffe, Adaptimmune's Chief Executive Officer: "The launch of TECELRA continued to accelerate through Q2 with an increase of over 150% in patients invoiced and in revenue. The full network of ATCs is close to completion with 30 now accepting referrals. Our manufacturing organization continues to deliver with a 100% commercial manufacturing success rate through to the end of Q2. The transaction with US WorldMeds will ensure that patient access to TECLRA continues and also places…
Taysha Gene Therapies Reports Second Quarter 2025 Financial Results and Provides Corporate Update
Taysha Gene Therapies
2025-08-12
pivotal
gene therapies
financial results
Commenced site activation for REVEAL pivotal trial in accordance with previously aligned upon key design elements, following receipt of No Objection Letter (NOL) from Health Canada and feedback from the FDA; patient enrollment anticipated to begin in Q4 2025 High dose and low dose TSHA-102 continue
Novartis ianalumab Phase III trial meets primary endpoint in ITP, demonstrating statistically significant improvement in time to treatment failure
Novartis
2025-08-12
ianalumab
Adagene Reports Six Months 2025 Financial Results and Provides Corporate Updates
AdaGene
2025-08-12
phase 2
phase 3
Muzastotug (ADG126) Phase 1b/2 in MSS CRC shows 19.4-month median OS (mOS) in 10 mg/kg dose cohorts; mOS for 20 mg/kg cohorts has not yet been reached Alignment with FDA on Phase 2 and Phase 3 trial design elements. Company expects to begin enrolling patients in Phase 2 in 2H 2025 SAFEbody option
Kyverna Therapeutics Provides Business Update and Reports Second Quarter 2025 Financial Results
Kyverna Therapeutics
2025-08-12
bla
kyv-101
phase 2
phase 3
stiff person syndrome
Topline data for registrational Phase 2 trial of KYV-101 in stiff person syndrome (SPS) and BLA submission anticipated in 1H 2026 Registrational Phase 3 KYV-101 trial in myasthenia gravis (MG) to include ~60 patients with enrollment to initiate by year-end 2025; interim Phase 2 data expected in Q4
Eureka Therapeutics Appoints Dr. Reginald Seeto and William S. Solari III to Board of Directors
Eureka Therapeutics
2025-08-12
cell therapies
Emeryville, Calif., August 12th, 2025 – Eureka Therapeutics, Inc., a clinical-stage biotechnology company pioneering safer and more effective T-cell therapies, today announced the appointment of Dr. Reginald Seeto and William S. Solari III to its Board of Directors. Dr. Seeto is a global leader in biopharmaceuticals and diagnostics with expertise in corporate strategy, business development, The post Eureka Therapeutics Appoints Dr. Reginald Seeto and William S. Solari III to Board of Directors appeared first on Eureka .
Lineage Cell Therapeutics Reports Second Quarter 2025 Financial Results and Provides Business Update
lineage cell therapeutics
2025-08-12
phase 1
Positive RG6501 (OpRegen ® ) Phase 1/2a Clinical Study 36 Month Results Featured at Clinical Trials at the Summit 2025 Successfully Reduced-to-Practice Critical Aspect of Commercial-Scale, Cell-Based, GMP Production Processes First Chronic Patient Treated in New Clinical Study of OPC1 in Patients
Caribou Biosciences Reports Second Quarter 2025 Financial Results and Provides Business Update
caribou biosciences
2025-08-12
financial results
Two robust clinical datasets from CB-010 and CB-011 expected to be disclosed in H2 2025 -- -- $184 million in cash, cash equivalents, and marketable securities expected to fund the Company’s current operating plan into H2 2027 -- BERKELEY, Calif., Aug.
Passage Bio Reports Second Quarter 2025 Financial Results and Provides Recent Business Highlights
passage bio
2025-08-12
financial results
Lyell Immunopharma Reports Business Highlights and Financial Results for the Second Quarter 2025
lyell immunopharma
2025-08-12
phase 1
pivotal
phase 1/2
b-cell lymphoma
large b-cell lymphoma
Presented positive new clinical data demonstrating high rates of durable complete responses from the Phase 1/2 trial of LYL314 for the treatment of aggressive large B-cell lymphoma Initiated the PiNACLE pivotal trial of LYL314 in patients with large B-cell lymphoma receiving treatment in the third‑
Anixa Biosciences Announces Issuance of Additional U.S. Patent for CAR-T Technology
anixa biosciences
2025-08-12
car-t
quarterly
solid tumor
cell therapies
ovarian cancer
chimeric antigen receptor
tumor infiltrating lymphocytes
Newly issued patent extends protection of proprietary solid tumor CAR-T platform to 2045 SAN JOSE, Calif. , Aug. 12, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent Number 12,384,826 covering its chimeric antigen receptor-T cell (CAR-T) technology. This new patent extends protection of Anixa's CAR-T technology to 2045. The allowed claims in this patent encompass core methods and compositions that are fundamental to Anixa's innovative CAR-T approach. Anixa's CAR-T platform is specifically designed to address the long-standing challenges of applying CAR-T therapies to solid tumors, positioning the program as a potential breakthrough in immuno-oncology. This newly issued patent builds on Anixa's growing portfolio of CAR-T intellectual property, collectively designed to protect the platform's use across multiple tumor types. This patent, along with others, was granted to The Wistar Institute and exclusively licensed to Anixa Biosciences. Anixa's CAR-T technology is currently…
Nkarta Reports Second Quarter 2025 Financial Results and Corporate Highlights
nkarta
2025-08-12
nkx019
Initial update for NKX019 in multiple autoimmune indications expected in second half of 2025 Cash balance of $334.0 million on June 30, 2025, including cash, cash equivalents and investments, expected to fund operations into 2029 SOUTH SAN FRANCISCO, Calif., Aug.
Kyverna Therapeutics Provides Business Update and Reports Second Quarter 2025 Financial Results
Kyverna Therapeutics
2025-08-12
bla
kyv-101
phase 2
phase 3
stiff person syndrome
Topline data for registrational Phase 2 trial of KYV-101 in stiff person syndrome (SPS) and BLA submission anticipated in 1H 2026 Registrational Phase 3 KYV-101 trial in myasthenia gravis (MG) to include ~60 patients with enrollment to initiate by year-end 2025; interim Phase 2 data expected in Q4
Fate Therapeutics Reports Second Quarter 2025 Financial Results and Business Updates
Fate Therapeutics
2025-08-12
car t
lupus
First patient treated with FT819 off-the-shelf CAR T-cell product candidate following fludarabine-free conditioning for severe lupus nephritis demonstrated durability of response with drug-free definition of remission in systemic lupus erythematosus (DORIS) at 12-month follow-up Held initial
Novartis announces both ianalumab Phase III clinical trials met primary endpoint in patients with Sjögren's disease
Novartis
2025-08-11
ianalumab
As gene therapy sales sputter, one biotech aims to defy the odds
Ferring Pharmaceuticals
2025-08-11
gene therapy
bladder cancer
With a strong launch underway for a bladder cancer gene therapy, Ferring is finding the kind of commercial success that’s eluded many of its peers.
Capricor Therapeutics Reports Second Quarter 2025 Financial Results and Provides Corporate Update
Capricor Therapeutics
2025-08-11
financial results
Atara Biotherapeutics Announces Second Quarter Financial Results and Operational Progress
Atara Biotherapeutics
2025-08-11
financial results
Protara Therapeutics Announces Second Quarter 2025 Financial Results and Provides Business Update
protara therapeutics
2025-08-11
financial results
Presentation of interim analysis from approximately 25 six-month evaluable BCG-Unresponsive patients in the ongoing ADVANCED-2 trial expected at a medical conference in 1Q 2026 Dosing of first patient in THRIVE-3 registrational trial of IV Choline Chloride in patients dependent on parenteral
Neurogene Reports Second Quarter 2025 Financial Results and Highlights Recent Updates
neurogene
2025-08-11
phase 1
phase 1/2
gene therapy
rett syndrome
Announced design of Embolden™ registrational clinical trial of NGN-401 gene therapy for Rett syndrome; trial initiation activities underway Completed dosing in Phase 1/2 NGN-401 trial, and remains on track to report updated clinical efficacy and safety data in the second half of 2025 Cash runway
Gepotidacin accepted for priority review by the US FDA for the oral treatment of uncomplicated urogenital gonorrhoea
GSK
2025-08-11
priority review
Submission supported by positive phase III data in patients with uncomplicated urogenital gonorrhoea in EAGLE 1 trial
Roivant Reports Financial Results for the First Quarter Ended June 30, 2025, and Provides Business Update
roivant
2025-08-11
phase 3
BASEL, Switzerland and LONDON and NEW YORK, Aug. 11, 2025 (GLOBE NEWSWIRE) -- Roivant (Nasdaq: ROIV) today reported its financial results for the first quarter ended June 30, 2025, and provided a business update. Brepocitinib VALOR Phase 3 study in dermatomyositis (DM) remains on track for topline
As gene therapies falter on the market, this biopharma is aiming to defy the odds
Ferring Pharmaceuticals
2025-08-08
gene therapy
gene therapies
After a strong launch for its cancer gene therapy, Ferring Pharmaceuticals is setting the stage for long-term growth.
Anixa Biosciences Announces Dr. Robert Wenham will speak about Immunotherapy in surviving Ovarian Cancer at the 13th Annual Ovarcoming Cancer Conference
anixa biosciences
2025-08-08
car-t
phase 1
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
Dr. Wenham is Principal investigator on Anixa's CAR-T therapy for Ovarian Cancer SAN JOSE, Calif. , Aug. 8, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced Dr. Robert Wenham, Chair of the Gynecologic Oncology Program at Moffitt Cancer Center and principal investigator for the Company's ongoing ovarian cancer CAR-T therapy Phase 1 clinical trial, will be presenting at the 13 th Annual Ovarcoming Cancer Conference, being held September 18 – 19, 2025, virtually and in-person at the Briar Club in Houston, Texas. Dr. Wenham will join leading experts in the field to discuss advancements in ovarian cancer research and therapies, including Anixa's CAR-T therapy. The presentation is scheduled for Thursday, September 18, 2025, from 12:05 PM – 12:30 PM CT. Ovarcome is an ovarian cancer foundation envisioned to end the scourge of ovarian cancer. The Ovarcoming Cancer Conference brings together survivors, caregivers, and medical professionals to learn from world renowned experts on topics of greatest importance in the ovarian cancer landscape. For more information…
BrainStorm to Announce Second Quarter Financial Results and Provide a Corporate Update
BrainStorm Cell Therapeutics
2025-08-08
financial results
REGENXBIO Reports Second Quarter 2025 Financial Results and Operational Highlights
REGENXBIO
2025-08-07
bla
pivotal
rgx-121
gene therapy
clemidsogene lanparvovec
duchenne muscular dystrophy
RGX-202 in Duchenne muscular dystrophy on track for topline results 1H 2026 and BLA submission mid-2026 Pivotal trial enrollment accelerated, expected to complete in October 2025 ahead of previous guidance Clemidsogene lanparvovec (RGX-121) on track to be first gene therapy and one-time treatment
Intellia Therapeutics Announces Second Quarter 2025 Financial Results and Highlights Recent Company Progress
Intellia Therapeutics Inc.
2025-08-07
phase 3
Enrollment in the global Phase 3 MAGNITUDE trial of nexiguran ziclumeran (nex-z) in ATTR with cardiomyopathy (ATTR-CM) continues to track ahead of projections; Tracking to enroll at least 650 patients cumulatively by year-end Expanding total enrollment of the MAGNITUDE study to approximately 1,200
Lisata Therapeutics Reports Second Quarter 2025 Financial Results and Provides Business Update
Lisata Therapeutics
2025-08-07
financial results
Cash runway extending into the fourth quarter of 2026 with no debt, funding current clinical programs through to their next data milestone Conference call scheduled for today at 4:30 p.m. Eastern Time BASKING RIDGE, N.J., Aug. 07, 2025 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc.
ProQR Announces Second Quarter 2025 Operating and Financial Results
proqr therapeutics
2025-08-07
cta
phase 1
pipeline
rett syndrome
Submitted CTA for lead program AX-0810, targeting NTCP for cholestatic diseases Advancing AX-2402 program toward clinical candidate selection, targeting MECP2 (R270X) for Rett Syndrome Hosting fall Analyst and Investor Event (virtual), featuring detailed AX-0810 Phase 1 trial design and 2025 data expectations, plus updates across differentiated liver and CNS pipeline € 119.8 million cash and cash equivalents as of end Q2 – providing runway into mid-2027, not including additional potential milestones from Lilly partnership LEIDEN, Netherlands & CAMBRIDGE, Mass., Aug. 07, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V.
IN8bio Reports Second Quarter 2025 Financial Results and Recent Business Highlights
In8Bio
2025-08-07
financial results
Delivered an oral presentation at ASCO 2025 Annual Meeting, demonstrating extended median progression-free survival (mPFS) in patients receiving multiple doses of INB-200, including a patient remaining progression-free for over four years. Received the Host Region USA East Abstract Award at ISCT
PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2025 Financial Results
ptc biotherapeutics
2025-08-07
financial results
– European and FDA approval of Sephience™ (sepiapterin) with broad labeling for PKU – – Global launch underway in Europe and U.S. – – Total Q2 Revenue of $179M – WARREN, N.J. , Aug. 7, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. , (NASDAQ: PTCT) today announced a corporate update and financial
Iovance Biotherapeutics Reports Financial Results and Corporate Updates for Second Quarter and First Half 2025
Iovance Biotherapeutics
2025-08-07
amtagvi
$60.0M in 2Q25 Total Product Revenue More than 100 Patients Treated with Amtagvi ® in 2Q25 Strategic Restructuring Extends Cash Runway into 4Q26 FY25 Total Product Revenue Guidance of $250M-$300M Reiterated SAN CARLOS, Calif. , Aug. 07, 2025 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc.
Rocket Pharmaceuticals Reports Second Quarter 2025 Financial Results and Highlights Recent Progress
Rocket Pharmaceuticals
2025-08-07
aav
rmat
pivotal
gene therapy
Prioritizing development of RP-A501 (Danon disease), RP-A601 (PKP2-ACM), and RP-A701 (BAG3-DCM) to advance AAV cardiovascular gene therapy platform for sustained value creation FDA RMAT designation awarded to RP-A601 for PKP2-ACM; Engaging with FDA on pivotal trial design following encouraging
Cabaletta Bio Reports Second Quarter 2025 Financial Results and Provides Business Update
cabaletta bio
2025-08-07
financial results
REGENXBIO Announces Pivotal Program for Surabgene Lomparvovec in Diabetic Retinopathy
REGENXBIO
2025-08-07
pivotal
A pivotal Phase IIb/III clinical trial using suprachoroidal delivery for treatment of diabetic retinopathy will be initiated New Phase II ALTITUDE ® trial data demonstrate a durable safety and efficacy profile observed through two years with a single, in-office injection of surabgene lomparvovec in
Capricor Therapeutics to Present Second Quarter 2025 Financial Results and Recent Corporate Update on August 13
Capricor Therapeutics
2025-08-06
financial results
NKGen Biotech To Present on Allogeneic Enhanced NK Cells without Lymphodepletion in Solid Tumors at the 13th Annual Immuno-Oncology Summit
nkgen biotech
2025-08-06
allogeneic
autologous
SANTA ANA, Calif., August 6, 2025 – NKGen Biotech, Inc. (OTC: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer (“NK”) cell therapeutics, today announced that Paul Y. Song, M.D., Chairman and Chief Executive Officer of NKGen, will present at the 13th […] The post NKGen Biotech To Present on Allogeneic Enhanced NK Cells without Lymphodepletion in Solid Tumors at the 13th Annual Immuno-Oncology Summit first appeared on NKGen Biotech .
Allogene Therapeutics to Report Second Quarter 2025 Financial Results and Provide Business Update
Allogene
2025-08-06
car t
allogeneic
Conference Call and Webcast Scheduled for August 13, 2025 at 2:00 p.m. PT / 5:00 p.m. ET SOUTH SAN FRANCISCO, Calif. , Aug. 06, 2025 (GLOBE NEWSWIRE) -- Allogene Therapeutics , Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™)
Sarepta Therapeutics Announces Second Quarter 2025 Financial Results and Recent Corporate Developments
Sarepta Therapeutics
2025-08-06
sarepta
elevidys
ELEVIDYS shipments have resumed for ambulatory individuals living with Duchenne following notification from the U.S. Food and Drug Administration (FDA); continuing to work with FDA on safety labeling process and risk-mitigation approach for non-ambulatory individuals Net product revenues for the
Adicet Bio to Participate in a Fireside Chat at the 45th Annual Canaccord Genuity Growth Conference
Adicet Bio
2025-08-05
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Aug. 5, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced that Chen Schor , President and Chief
Beam Therapeutics Reports Second Quarter 2025 Financial Results and Provides Update on BEAM-302 Development Progress in Alpha-1 Antitrypsin Deficiency (AATD)
beam therapeutics
2025-08-05
phase 1
phase 1/2
With 17 Patients Dosed in the Phase 1/2 Trial, BEAM-302 Continues to Demonstrate Durable Correction of the Disease-causing Mutation, Restoration of AAT Physiology, and a Well Tolerated Safety Profile BEAM-302 Expanded Dose Exploration Underway in Part A and Enrollment Initiated in Part B; Data from
Ultragenyx Reports Second Quarter 2025 Financial Results and Corporate Update—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-08-05
financial results
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Axogen, Inc. Reports Second Quarter 2025 Financial Results
axogen
2025-08-05
financial results
Cryoport Reports Second Quarter 2025 Financial Results
Cryoport Inc
2025-08-05
financial results
Eureka Therapeutics Adds Stanford Liver Cancer Expert Dr. Samuel So to Scientific Advisory Board
Eureka Therapeutics
2025-08-05
cell therapies
Emeryville, Calif., August 5th, 2025 – Eureka Therapeutics, Inc., a clinical-stage biotechnology company pioneering safer and more effective T-cell therapies, today announced the appointment of Samuel So, MBBS, FACS, a globally recognized leader in liver cancer surgery, prevention, and health policy at Stanford University, to its Scientific Advisory Board. Dr. So will provide strategic guidance The post Eureka Therapeutics Adds Stanford Liver Cancer Expert Dr. Samuel So to Scientific Advisory Board appeared first on Eureka .
Lineage Cell Therapeutics to Report Second Quarter 2025 Financial Results and Provide Business Update on August 12, 2025
lineage cell therapeutics
2025-08-05
allogeneic
cell therapies
CARLSBAD, Calif. --(BUSINESS WIRE)--Aug. 5, 2025-- Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for serious neurological conditions, today announced that it will report its second quarter 2025 financial
CRISPR Therapeutics Provides Business Update and Reports Second Quarter 2025 Financial Results
CRISPR
2025-08-04
crispr
casgevy
CASGEVY ® momentum building; >75 authorized treatment centers (ATCs) activated globally, achieving the target goal and ~115 patients have had cells collected across all regions; positioning the program for strong future growth- -Clinical trial ongoing for CTX310™, targeting ANGPTL3, with
Genprex Issues Stockholder Letter and Provides 2025 Corporate Update
Genprex
2025-08-04
lung cancer
gene therapy
Company Achieves Multiple Clinical Development Milestones in 2025 Patient Treatment Continues in Two Lung Cancer Clinical Trials AUSTIN, Texas — (Aug. 4, 2025) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that it has issued a stockholder letter and […]
Cellectis Reports Second Quarter 2025 Financial Results & Business Updates
cellectis
2025-08-04
cd19
b-all
car-t
phase 1
phase 2
pivotal
pipeline
allogeneic
first-line
gene editing
gene therapy
gene therapies
in vivo gene therapy
hematological malignancies
b-cell acute lymphoblastic leukemia
Cellectis to host an Investor R&D Day in New York City on October 16, 2025: Phase 1 dataset and late-stage development strategy for lasme-cel (UCART22) in r/r B-ALL to be presented End-of-Phase 1 meetings with FDA & EMA for lasme-cel (UCART22) in r/r B-ALL completed in July 2025; on track to launch pivotal Phase 2 in H2 2025 Servier arbitration: arbitral decision expected to be rendered on or before December 15, 2025 eti-cel (UCART20x22): Phase 1 study in r/r NHL ongoing with readout expected in late 2025 AstraZeneca partnership: R&D activities are continuing to advance for the three programs initiated Appointment of Mr. André Muller as Director to Cellectis’ Board of Directors Cash, cash equivalents and fixed-term deposits of $230 million as of June 30, 2025 [1] provides runway into H2 2027 Conference call and webcast scheduled for tomorrow, August 5, 2025 at 8:00AM ET / 2:00PM CET New York, NY – August 4, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, today provided financial results for the second quarter 2025 ending June…
Bristol Myers Squibb’s Application for Breyanzi (lisocabtagene maraleucel) Accepted for Priority Review by U.S. Food and Drug Administration (FDA) in Fifth Cancer Type for Relapsed or Refractory Marginal Zone Lymphoma (MZL)
Bristol Myers Squibb
2025-08-04
breyanzi
marginal zone lymphoma
Anixa Biosciences Announces Commencement of US FDA Approved IND Transfer to Support Upcoming Phase 2 Breast Cancer Vaccine Trial
anixa biosciences
2025-08-04
ind
car-t
phase 1
phase 2
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
Progress Follows Positive Immune Response Observed in Phase 1 Study SAN JOSE, Calif. , Aug. 4, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that, in collaboration with Cleveland Clinic, it has initiated the transfer of the Investigational New Drug (IND) application that supported the Phase 1 clinical trial of its breast cancer vaccine. With enrollment completed and encouraging immune response data observed in the Phase 1 trial, Anixa plans to advance the vaccine into a Phase 2 clinical trial and will assume full sponsorship of the IND. The IND, currently held by Cleveland Clinic, is in the process of being transferred to Anixa. To oversee this process, Anixa has engaged Advyzom, a leading regulatory consulting firm specializing in strategic FDA interactions, to act as its U.S. regulatory agent regarding the assigned application. Anixa's breast cancer vaccine, developed in collaboration with Cleveland Clinic, targets α-lactalbumin—a lactation-associated protein that is typically expressed only in breast tissue during lactation, but which re-emerges…
Bristol Myers Squibb’s Application for Breyanzi (lisocabtagene maraleucel) Accepted for Priority Review by U.S. Food and Drug Administration (FDA) in Fifth Cancer Type for Relapsed or Refractory Marginal Zone Lymphoma (MZL)
bristol myers squibb
2025-08-04
breyanzi
priority review
marginal zone lymphoma
Vertex Reports Second Quarter 2025 Financial Results
Vertex
2025-08-04
casgevy
— Total revenue of $2.96 billion , a 12% increase compared to Q2 2024; reiterated full year 2025 financial guidance, including revenue guidance of $11.85 to $12 billion — — Continued strong execution of CASGEVY ® , ALYFTREK ® and JOURNAVX ® launches — — Rapid advancement of next wave of clinical
Lineage Announces Dosing of First Patient in New Clinical Study of OPC1 for Subacute and Chronic Spinal Cord Injury
lineage cell therapeutics
2025-08-04
first-in-human
Successful First-In-Human Use of New Parenchymal Spinal Delivery System First-ever Administration of OPC1 to a Chronic Injury Patient CARLSBAD, Calif. --(BUSINESS WIRE)--Aug. 4, 2025-- Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing
Novartis twice-yearly* Leqvio® (inclisiran) receives FDA approval for new indication enabling first-line use
Novartis
2025-08-01
first-line
Intellia Therapeutics Reports Inducement Grants under Nasdaq Listing Rule 5635(c)(4)
Intellia Therapeutics Inc.
2025-08-01
crispr
gene editing
CAMBRIDGE, Mass. , Aug. 01, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that on August 1, 2025 , it awarded an inducement grant to five new
Allogene Therapeutics Moves Forward with Standard Fludarabine and Cyclophosphamide (FC) Lymphodepletion Regimen in the ALPHA3 Trial for Cemacabtagene Ansegedleucel (Cema-Cel) in First-Line Consolidation for Large B-Cell Lymphoma
Allogene
2025-08-01
first-line
Lymphodepletion Regimen Selection Made in Conjunction with the ALPHA3 Data and Safety Monitoring Board (DSMB) and Steering Committee , and in Consultation with the FDA Following an ALLO-647-Related Death in the FCA (FC plus ALLO-647) Lymphodepletion Arm Unplanned Review of Safety and Biomarker Data
MiNK Therapeutics to Provide Corporate Update and Second Quarter 2025 Financial Report
Mink Therapeutics
2025-08-01
allogeneic
cell therapies
NEW YORK , July 31, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering the discovery, development, and commercialization of allogeneic, off-the-shelf, invariant natural killer T (iNKT) cell therapies to treat cancer and other
Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-07-31
phase 3
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Diagnosis of Frontotemporal Dementia is Often in Non-Specialist Settings and Complicated by Non-FTD Dementia and Psychiatric Diagnoses: Patient and Provider Findings from Analysis of a Large US Real-World Dataset
aviadobio
2025-07-31
frontotemporal dementia
The post Diagnosis of Frontotemporal Dementia is Often in Non-Specialist Settings and Complicated by Non-FTD Dementia and Psychiatric Diagnoses: Patient and Provider Findings from Analysis of a Large US Real-World Dataset appeared first on AviadoBio .
ASPIRE-FTD Study Update: A Phase 1/2 Clinical Study to Evaluate AVB-101 in FTD with GRN Mutations
aviadobio
2025-07-31
phase 1
phase 1/2
The post ASPIRE-FTD Study Update: A Phase 1/2 Clinical Study to Evaluate AVB-101 in FTD with GRN Mutations appeared first on AviadoBio .
Sarepta Therapeutics to Announce Second Quarter 2025 Financial Results
Sarepta Therapeutics
2025-07-31
sarepta
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jul. 31, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report second quarter 2025 financial results after the Nasdaq Global Market closes on Wednesday, August 6, 2025 .
AbbVie Reports Second-Quarter 2025 Financial Results
Abbvie Investors
2025-07-31
financial results
Reports Second-Quarter Diluted EPS of $0.52 on a GAAP Basis, a Decrease of 32.5 Percent; Adjusted Diluted EPS of $2.97 , an Increase of 12.1 Percent; These Results Include an Unfavorable Impact of $0.42 Per Share Related to Acquired IPR&D and Milestones Expense Delivers Second-Quarter Net
REGENXBIO to Host Conference Call on August 7 to Discuss Second Quarter 2025 Financial Results and Operational Highlights
REGENXBIO
2025-07-31
financial results
ROCKVILLE, Md. , July 31, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Thursday, August 7 , at 8:00 a.m. ET to discuss its financial results for the second quarter ended June 30, 2025 , and operational highlights.
IGRT Shows No Reduction in Serious Infections for Patients with CLL
Hematology.org
2025-07-31
cll
IGRT Shows No Reduction in Serious Infections for Patients with CLL
Actinium Presents Data Supporting Paradigm Changing Potential of ATNM-400 in Prostate Cancer Demonstrating Its Superior Efficacy and Improved Survival in Treatment Resistant Tumor Models versus Pluvicto and ARPI Therapy, and Also Enhanced Efficacy in Combination with ARPI Therapy at the 4th Annual Targeted Radiopharmaceuticals Summit
Actinium Pharma
2025-07-31
aml
cd33
psma
phase 2
pivotal
clearance
phase 2/3
quarterly
solid tumor
gene therapies
acute myeloid leukemia
ATNM-400 demonstrates robust efficacy in prostate cancer tumor models with acquired resistance to Pluvicto ® and enzalutamide, follow-up continues - ATNM-400 significantly improved survival compared to the approved prostate cancer treatment Pluvicto ® , the first blockbuster radiotherapy - In combination with enzalutamide, an approved ARPI therapy, ATNM-400 produced enhanced efficacy with 40% of prostate cancer tumor-bearing animals achieving complete cures NEW YORK , July 31, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today reported additional preclinical data supporting its ATNM-400 radiotherapy prostate cancer candidate at the 4 th Annual Targeted Radiopharmaceuticals Summit (TRP) being held July 29 – 31, 2025 in San Diego, CA. ATNM-400 is a novel, first-in-class targeted radiotherapy designed to deliver potent Actinium-225 (Ac-225), an alpha-emitter radioisotope, to prostate cancer cells by targeting a non-Prostate Specific Membrane Antigen (PSMA), disease-driving protein overexpressed in advanced and treatment-resistant disease. Unlike PSMA-targeted agents that…
MIMEDX Announces Record Second Quarter 2025 Operating and Financial Results
mimedx
2025-07-30
quarterly
Highest Quarterly Net Sales in MIMEDX History Second Quarter Net Sales of $99 million Grew 13% Year-Over-Year Second Quarter GAAP Net Income and Diluted Earnings Per Share were $10 Million and $0.06 , Respectively Second Quarter Adjusted EBITDA was $24 Million, or 25% of Net Sales Raises 2025 Net
Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-07-30
financial results
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Be Bio Announces First Participant Dosed in BeCoMe-9 Trial of BE-101 in Hemophilia B
be biopharma
2025-07-30
phase 1
phase 1/2
hemophilia b
CAMBRIDGE, Mass., July 30, 2025 – Be Biopharma, Inc. (“Be Bio” or “the Company”), a clinical-stage company pioneering the discovery and development of engineered B Cell Medicines (BCMs), today announced the first participant was treated with BE-101 in the BeCoMe-9 Phase 1/2 clinical trial. BE-101 was administered without any preconditioning or immunosuppression, supported by preclinical […] The post Be Bio Announces First Participant Dosed in BeCoMe-9 Trial of BE-101 in Hemophilia B appeared first on Be Biopharma .
LENZ Therapeutics Reports Second Quarter 2025 Financial Results and Recent Corporate Highlights
LENZ Therapeutics
2025-07-30
financial results
Sumitomo Pharma America Announces that the European Medicines Agency Has Granted Orphan Drug Designation to Nuvisertib (TP-3654) for the Treatment of Myelofibrosis
Enzyvant Therapeutics
2025-07-30
orphan drug
Anixa Biosciences Receives Notice of Allowance from Canadian Intellectual Property Office for Patent Covering Breast Cancer Vaccine Technology
anixa biosciences
2025-07-30
car-t
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
Patent Strengthens International Protection for First-of-Its-Kind Immunopreventive Platform Expands Global IP Coverage in Markets with High Incidence of Breast Cancer SAN JOSE, Calif. , July 30, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the Canadian Intellectual Property Office (CIPO) has issued a Notice of Allowance for a new patent related to its breast cancer vaccine technology. This patent, exclusively licensed from Cleveland Clinic, will provide composition-of-matter protection for the Company's novel immunogenic approach to breast cancer prevention and treatment in Canada. With this allowance, Anixa continues to expand the international scope of its intellectual property portfolio, reinforcing its leadership in the field of cancer immunoprevention. The Canadian patent complements issued and pending patents in the United States and other key global jurisdictions, and represents an important step toward future regulatory and commercial efforts outside the U.S. "This newly allowed patent further illustrates the international recognition…
Kyverna Therapeutics to Host Virtual KOL Event to Highlight Neuroimmunology CAR T Franchise
Kyverna Therapeutics
2025-07-29
car t
cell therapies
Webcast to be held August 28, 2025 , 11:00am ET EMERYVILLE, Calif. , July 29, 2025 /PRNewswire/ -- Kyverna Therapeutics, Inc. (Kyverna, Nasdaq: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, today announced that the
Capricor Therapeutics and Parent Project Muscular Dystrophy to Host Webinar to Review Latest Developments in the Deramiocel Program for the Treatment of Duchenne Muscular Dystrophy
Capricor Therapeutics
2025-07-28
deramiocel
duchenne muscular dystrophy
Genethon to Launch Pivotal Trial in Europe of GNT0004 a Low-Dose Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
Genethon
2025-07-28
dmd
pivotal
phase 1/2
gene therapy
gene therapies
duchenne muscular dystrophy
Phase 3 trial clearance is based on the Phase 1/2 results demonstrating safety and efficacy of the 3×10¹³ vg/kg dose of microdystrophin, which is lower than doses used in other gene therapies for Duchenne muscular dystrophy (DMD).The double blind trial will initiated in the UK and France beginning in August and September, and will enroll … Continued
PTC Therapeutics Announces FDA Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)
PTC Therapeutics
2025-07-28
phenylketonuria
Broad labeling inclusive of all disease subtypes for individuals 1 month of age and older - - PTC will host a conference call on Monday, July 28 th at 5:00 pm ET - WARREN, N.J. , July 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S.
Roivant to Report Financial Results for the First Quarter Ended June 30, 2025, and Provide Business Update on Monday, August 11, 2025
roivant
2025-07-28
financial results
BASEL, Switzerland and LONDON and NEW YORK, July 28, 2025 (GLOBE NEWSWIRE) -- Roivant (Nasdaq: ROIV) today announced that it will host a live conference call and webcast at 8:00 a.m. ET on Monday, August 11, 2025, to report its financial results for the first quarter ended June 30, 2025, and
FDA Informs Sarepta That It Recommends That Sarepta Remove Its Pause and Resume Shipments of ELEVIDYS for Ambulatory Individuals With Duchenne Muscular Dystrophy
Sarepta Therapeutics
2025-07-28
sarepta
elevidys
duchenne muscular dystrophy
Company commends FDA for swift and comprehensive review of available information - Shipments to sites of care will resume imminently - Sarepta and FDA will continue dialogue on next steps in the safety labeling process and risk-mitigation approach for non-ambulatory patients CAMBRIDGE, Mass.
Cellectis to Report Second Quarter 2025 Financial Results on August 4, 2025
cellectis
2025-07-28
gene therapies
New York, NY – July 28, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the second quarter 2025 ending June 30, 2025 on Monday August 4, 2025 after the close of the US market. The publication will be followed by an investor conference call and webcast on Tuesday August 5, 2025 at 8:00 AM ET / 2:00 PM CET. The call will include the Company’s second quarter results and an update on business activities. Details for the call are as follows: Dial in information: Domestic: +1-800-343-5172 International: +1-203-518-9856 Conference ID: CLLSQ2 Webcast Link: https://viavid.webcasts.com/starthere.jsp?ei=1727030&tp_key=10a62ee950
NKGen Biotech Presents Troculeucel Mechanism of Action with Corresponding Phase 1 Biomarker Data at the Alzheimer’s Association International Conference 2025
nkgen biotech
2025-07-28
phase 1
autologous
Troculeucel, a cryopreserved autologous, non-genetically modified NK cell product, appears to cross the blood brain barrier (BBB) via CXCR3, to reduce neuroinflammation via a simple IV administration. Troculeucel was shown to internalize and digest amyloid and α-synuclein proteins in vitro and to improve CSF levels of amyloid, α-synuclein, and p-tau in Phase 1 patients in […] The post NKGen Biotech Presents Troculeucel Mechanism of Action with Corresponding Phase 1 Biomarker Data at the Alzheimer’s Association International Conference 2025 first appeared on NKGen Biotech .
Nanoscope Therapeutics Data to be Presented at 2025 American Society of Retina Specialists Annual Scientific Meeting
Nanoscope Therapeutics
2025-07-28
mco-010
retinitis pigmentosa
126-week results from RESTORE follow-up study (REMAIN) to highlight the extended efficacy and safety of Nanoscope’s MCO-010 optogenetic therapy for Retinitis Pigmentosa DALLAS, July 28, 2025 /PRNewswire/ — Nanoscope Therapeutics Inc., a biotechnology company committed to developing and commercializing novel gene-agnostic therapies for patients suffering severe vision loss from retinal degenerative diseases, announced that its multi-characteristic opsin (MCO) gene […] The post Nanoscope Therapeutics Data to be Presented at 2025 American Society of Retina Specialists Annual Scientific Meeting appeared first on Nanoscope Therapeutics .
Adaptimmune Announces Entry into Definitive Agreement for Sale of TECELRA, lete-cel, afami-cel and uza-cel Cell Therapies to US WorldMeds
Adaptimmune Therapeutics
2025-07-28
tcr t
tecelra
afami-cel
allogeneic
solid tumor
cell therapy
cell therapies
Tecelra will continue to be available to patients without interruption US WorldMeds plans to bring lete-cel to market and will continue development of uza-cel in collaboration with Galapagos Purchase price is $55m in cash to be paid upon consummation of the sale with up to $30m in potential future payments upon achievement of certain milestones Adaptimmune will retain rights to its preclinical assets including PRAME, CD70, and its allogeneic program Adaptimmune will restructure to support the transition of these therapies to US WorldMeds and to maximize value from its remaining assets Adaptimmune's Board of Directors determined this transaction is in the best interest of all stakeholders following a comprehensive review of all strategic alternatives Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - July 28, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company redefining the treatment of solid tumor cancers with cell therapy, today announced its entry into a definitive agreement for the sale of TECELRA, lete-cel, afami-cel, and uza-cel cell therapies to US WorldMeds. Consummation of the sale is expected to occur before the end of the week. Adrian…
Long-Term Follow-Up of SCD Patients Following Curative Treatment
Hematology Advisor Features
2025-07-25
scd
Researchers developed a road map for long-term follow-up and monitoring after curative-intent treatment for SCD patients.
Lyell Immunopharma Announces up to $100 Million Equity Private Placement
lyell immunopharma
2025-07-25
car t
cell therapies
SOUTH SAN FRANCISCO, Calif., July 25, 2025 (GLOBE NEWSWIRE) -- Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical-stage company advancing next-generation CAR T-cell therapies for patients with cancer, today announced that it has entered into a securities purchase agreement for a private placement
Sarepta Therapeutics Provides Clarifying Statement on ELEVIDYS
Sarepta Therapeutics
2025-07-25
sarepta
elevidys
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jul. 25, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement: Just before 6:00 p.m. ET today, the U.S. Food and Drug Administration (FDA) issued a press release
Anixa Biosciences Awarded New U.S. Patent Extending Breast Cancer Vaccine IP Protection into 2040s
anixa biosciences
2025-07-24
car-t
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
Patent Expands Immunogenic Coverage of α-Lactalbumin-Based Vaccine Platform Technology Addresses Substantial Unmet Need in Preventive Oncology with Multi-Billion Dollar Market Potential SAN JOSE, Calif. , July 24, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the United States Patent and Trademark Office (USPTO) will issue U.S. Patent Number 12,370,244 on July 29, 2025, covering key aspects of the Company's breast cancer vaccine technology. The patent protects novel methods of immunizing patients against breast cancer by administering an immunogenic composition containing human α-lactalbumin protein—a protein typically found in breast tissue during lactation but also expressed in certain breast cancers, making it an attractive target for immunoprevention strategies. Anixa's breast cancer vaccine, developed in collaboration with Cleveland Clinic, represents a novel approach to the prevention and treatment of breast cancer. The vaccine was invented at Cleveland Clinic, and this patent—along with others related to this technology—has been…
Atara Biotherapeutics Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application for Tabelecleucel (Tab-cel®) for the Treatment of Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease
Atara Biotherapeutics
2025-07-24
tab-cel
tabelecleucel
post-transplant lymphoproliferative disease
Iovance Biotherapeutics to Host Second Quarter and First Half 2025 Financial Results and Corporate Updates Webcast on Thursday, August 7, 2025
Iovance Biotherapeutics
2025-07-24
til
SAN CARLOS, Calif. , July 24, 2025 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, will report its second quarter and
Adicet Bio Announces First Systemic Sclerosis (SSc) Patient Dosed in Ongoing Phase 1 Clinical Trial of ADI-001 in Autoimmune Diseases
Adicet Bio
2025-07-24
lupus
Phase 1 clinical trial of ADI-001 in autoimmune diseases actively enrolling patients with lupus nephritis (LN), systemic lupus erythematosus (SLE) and SSc Preliminary clinical data from Phase 1 trial of ADI-001 in autoimmune diseases expected in 2H/2025 REDWOOD CITY, Calif.
Rocket Pharmaceuticals Announces Strategic Corporate Reorganization and Pipeline Prioritization of Cardiovascular Programs
Rocket Pharmaceuticals
2025-07-24
aav
kresladi
Restructuring pipeline to focus on AAV cardiovascular platform (Danon disease, PKP2-ACM, BAG3-DCM) and regulatory activities for KRESLADI ™ (severe LAD-I), setting foundation for near- and long-term value creation Organizational restructuring expected to reduce headcount by approximately 30% and
Blenrep (belantamab mafodotin) combinations approved in EU for treatment of relapsed/refractory multiple myeloma
GSK
2025-07-24
multiple myeloma
Sixth regulatory approval for Blenrep combinations with applications under review in all major markets
Nkarta to Participate in the H.C. Wainwright “HCW@Home” Series
nkarta
2025-07-23
cell therapies
SOUTH SAN FRANCISCO, Calif., July 23, 2025 (GLOBE NEWSWIRE) -- Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies, today announced its participation in the H.C. Wainwright “HCW@Home” Series.
GSK announces extension of US Food and Drug Administration review period for Blenrep (belantamab mafodotin-blmf) in relapsed/refractory multiple myeloma
GSK
2025-07-23
pdufa
multiple myeloma
New PDUFA date scheduled for 23 October 2025.
Axogen, Inc. to report 2025 second quarter financial results on August 5, 2025
axogen
2025-07-22
financial results
NKGen Biotech to Present at the Alzheimer’s Association International Conference 2025
nkgen biotech
2025-07-22
allogeneic
autologous
cell therapy
nk cell therapy
SANTA ANA, Calif., July 22, 2025 — NKGen Biotech, Inc. (OTC: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer (“NK”) cell therapeutics, today announced that it will present on the mechanism of action for troculeucel, enhanced non-genetically modified NK cell therapy, […] The post NKGen Biotech to Present at the Alzheimer’s Association International Conference 2025 first appeared on NKGen Biotech .
Cryoport to Report Second Quarter 2025 Financial Results on August 5, 2025
Cryoport Inc
2025-07-22
financial results
Nanoscope Therapeutics Publishes Mechanism of Action Details for Proprietary Broadband and Fast Multi-Characteristic Opsin Technology
Nanoscope Therapeutics
2025-07-22
gene therapies
New research paper highlights the unique structure and function of Nanoscope’s synthetic opsin, supporting its potential in vision restoration DALLAS, July 22, 2025 — Nanoscope Therapeutics Inc., a biotechnology company committed to developing and commercializing novel gene therapies for patients suffering vision loss from retinal degenerative diseases, today announced the publication of a paper “Topology and Mechanism of Broadband […] The post Nanoscope Therapeutics Publishes Mechanism of Action Details for Proprietary Broadband and Fast Multi-Characteristic Opsin Technology appeared first on Nanoscope Therapeutics .
PTC Therapeutics to Host Conference Call to Discuss Second Quarter 2025 Financial Results
ptc biotherapeutics
2025-07-21
financial results
WARREN, N.J. , July 21, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the company will host a webcast conference call to report its second quarter 2025 financial results and provide an update on the company's business and outlook on Thursday, August 7 , at 4:30
NKGen Receives FDA Expanded Access Protocol Authorization for Landmark Treatment of Several Neurodegenerative Diseases
nkgen biotech
2025-07-21
autologous
cell therapy
natural killer cell therapy
Troculeucel, a cryopreserved, autologous enhanced natural killer cell therapy, has demonstrated preliminary clinical benefit without any drug-related adverse events in two Phase I clinical trials in Alzheimer’s Disease. Company receives expanded access protocol authorization from the U.S. Food and Drug Administration (FDA) for troculeucel granted to treat patients with known diagnoses of multiple neurodegenerative diseases. […] The post NKGen Receives FDA Expanded Access Protocol Authorization for Landmark Treatment of Several Neurodegenerative Diseases first appeared on NKGen Biotech .
Bristol Myers Squibb Announces Topline Results from Phase 3 INDEPENDENCE Trial for Reblozyl® (luspatercept-aamt) in Adult Patients with Myelofibrosis-Associated Anemia
bristol myers squibb
2025-07-18
phase 3
GenSight Biologics Announces Publication on Predictors of Final Visual Outcome in Patients Treated with LUMEVOQ® Gene Therapy
GenSight Biologics
2025-07-18
gene therapy
Bristol Myers Squibb Announces Topline Results from Phase 3 INDEPENDENCE Trial for Reblozyl® (luspatercept-aamt) in Adult Patients with Myelofibrosis-Associated Anemia
Bristol Myers Squibb
2025-07-18
phase 3
Center in California now offering Zevaskyn gene therapy for RDEB
Epidermolysis Bullosa News
2025-07-18
zevaskyn
gene therapy
Lucile Packard Children’s Hospital Stanford, a center in the San Francisco bay area, is ready to accept patients for treatment with Zevaskyn.
Eliminating Barriers to Allo-HCT When Treating AML
Hematology Advisor Features
2025-07-18
aml
Researchers determined lower education and higher poverty are associated with decreased access to allo-HCT for patients with AML.
Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-A601 Gene Therapy for PKP2-Arrhythmogenic Cardiomyopathy
Rocket Pharmaceuticals
2025-07-17
rmat
gene therapy
CRANBURY, N.J. --(BUSINESS WIRE)--Jul. 17, 2025-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S.
GenSight Biologics Raises Approx. EUR 500K
GenSight Biologics
2025-07-17
gene therapies
GenSight Biologics, a Paris, France-based biopharma company focused on developing and commercializing gene therapies for retinal neurodegenerative diseases and central nervous system disorders, raised approximately EUR 500K
GSK provides update on US FDA advisory committee review of Blenrep (belantamab mafodotin-blmf) combinations for patients with relapsed/refractory multiple myeloma
GSK
2025-07-17
multiple myeloma
The US FDA Oncologic Drugs Advisory Committee voted against the proposed dosage of Blenrep (belantamab mafodotin-blmf) combinations.
NKGen Biotech and HekaBio Enter Strategic Partnership to Bring Novel NK Cell Therapy to Japan
nkgen biotech
2025-07-17
autologous
cell therapy
cell therapies
nk cell therapy
In connection with HekaBio’s recently announced strategic investment in NKGen, the partnership will expedite the regulatory, manufacturing, and commercial development of NKGen’s autologous NK cell therapy, troculeucel, in Japan. Under Japan’s Regenerative Medicine guidelines, cell therapies enjoy a regulatory environment with multiple paths for expedited commercialization. Troculeucel is well positioned to be the first-in-class forerunner […] The post NKGen Biotech and HekaBio Enter Strategic Partnership to Bring Novel NK Cell Therapy to Japan first appeared on NKGen Biotech .
MiNK Therapeutics Announces Frontiers in Immunology Publication Highlighting iNKT Cells as a Dual-Function Platform Key to Overcoming Barriers in Solid Tumor Cell Therapy
Mink Therapeutics
2025-07-16
allogeneic
solid tumor
cell therapy
iNKT cells uniquely remodel the tumor microenvironment, overcome immune resistance, and enable scalable off-the-shelf cell therapy NEW YORK , July 15, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering allogeneic invariant natural
Adagene Announces Regulatory Update on Clinical Development Plan for Muzastotug in Microsatellite Stable Colorectal Cancer Following Productive Type B (End of Phase 1) Meeting with FDA
AdaGene
2025-07-15
phase 1
phase 2
Alignment gained on Phase 2 inclusion/exclusion criteria, primary and secondary endpoints, and study design, including sample size and dosing regimen - Company expects to begin enrolling patients in Phase 2 in 2H 2025 SAN DIEGO and SUZHOU, China , July 15, 2025 (GLOBE NEWSWIRE) -- Adagene Inc.
ZEVASKYN™ Gene Therapy Now Available at New Qualified Treatment Center in San Francisco Bay Area
Abeona Therapeutics
2025-07-15
zevaskyn
gene therapy
Pie Magazine | Nanoscope Kickstarts BLA Submission for Restorative RP Therapy, Adds to Advisory Board
Nanoscope Therapeutics
2025-07-15
bla
The post Pie Magazine | Nanoscope Kickstarts BLA Submission for Restorative RP Therapy, Adds to Advisory Board appeared first on Nanoscope Therapeutics .
Modern Retina | FDA grants Nanoscope rolling BLA submission of MCO-010
Nanoscope Therapeutics
2025-07-14
bla
mco-010
The post Modern Retina | FDA grants Nanoscope rolling BLA submission of MCO-010 appeared first on Nanoscope Therapeutics .
Nanoscope Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for MCO-010, the First Gene-Agnostic Therapy to Treat Retinitis Pigmentosa
Nanoscope Therapeutics
2025-07-14
bla
mco-010
retinitis pigmentosa
First modules of BLA submitted to FDA under rolling review, with full submission anticipated in early 2026 MCO-010 BLA for retinitis pigmentosa is eligible for priority review based on fast-track designation If approved, MCO-010 has the potential to be the standard of care for retinitis pigmentosa, delivered via a one-time, in-office intravitreal injection with the […] The post Nanoscope Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for MCO-010, the First Gene-Agnostic Therapy to Treat Retinitis Pigmentosa appeared first on Nanoscope Therapeutics .
Atara Biotherapeutics Provides Regulatory and Business Updates on Tabelecleucel (Tab-cel®)
Atara Biotherapeutics
2025-07-14
tab-cel
tabelecleucel
Eyewire News | Nanoscope Initiates Rolling Submission of BLA to FDA for MCO-010, the First Gene-Agnostic Therapy to Treat RP
Nanoscope Therapeutics
2025-07-14
bla
mco-010
The post Eyewire News | Nanoscope Initiates Rolling Submission of BLA to FDA for MCO-010, the First Gene-Agnostic Therapy to Treat RP appeared first on Nanoscope Therapeutics .
Healio Ocular Surgery News | Nanoscope begins rolling submission of biologics license application for RP gene therapy
Nanoscope Therapeutics
2025-07-14
gene therapy
The post Healio Ocular Surgery News | Nanoscope begins rolling submission of biologics license application for RP gene therapy appeared first on Nanoscope Therapeutics .
MiNK Therapeutics Announces Publication of Complete Remission Following Allogeneic iNKT Cell Therapy in Metastatic Testicular Cancer
Mink Therapeutics
2025-07-12
allogeneic
cell therapy
cell therapies
New report adds to growing evidence of iNKT cell therapy’s potential in solid tumors NEW YORK , July 11, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering allogeneic, off-the-shelf invariant natural killer T (iNKT) cell therapies,
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy
Capricor Therapeutics
2025-07-11
bla
deramiocel
duchenne muscular dystrophy
Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-07-11
aav
ux111
gene therapy
complete response
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Should High ED Utilization Be Redefined for Patients With SCD?
Hematology Advisor Features
2025-07-11
scd
Emergency department utilization trends for patients with SCD call into question how high utilization is defined for this population.
REGENXBIO Announces Publication of Preclinical Results Demonstrating Functional Benefits of Novel Microdystrophin Construct in RGX-202 Investigational Gene Therapy for Duchenne Muscular Dystrophy
REGENXBIO
2025-07-10
gene therapy
duchenne muscular dystrophy
Construct including CT domain demonstrated higher levels of microdystrophin protein, increased muscle force, and improved resistance to damage in mice lacking dystrophin REGENXBIO's next-generation investigational gene therapy, RGX-202, is the only microdystrophin construct that includes the CT
Quell Therapeutics Initiates CHILL Phase 1/2 Trial of Novel QEL-005 CAR Treg Therapy in Refractory Rheumatoid Arthritis and Systemic Sclerosis Following UK CTA Approval
syncona
2025-07-09
car
Atsena Therapeutics Announces Alignment with FDA on Regulatory Pathway to Approval for ATSN-201 in X-Linked Retinoschisis (XLRS)
atsena therapeutics
2025-07-09
rmat
Atsena Therapeutics Announces Alignment with FDA on Regulatory Pathway to Approval for ATSN-201 in X-Linked Retinoschisis (XLRS) Outcome of Regenerative Medicine Advanced Therapy (RMAT) meeting ... The post Atsena Therapeutics Announces Alignment with FDA on Regulatory Pathway to Approval for ATSN-201 in X-Linked Retinoschisis (XLRS) appeared first on Atsena Therapeutics .
Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-07-09
phase 3
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Celularity & Fountain Life Announce Partnership to Deliver Stem Cell Therapies Under New Florida Law
celularity
2025-07-09
cell therapies
Anixa Biosciences Announces Issuance of Additional U.S. Patent for Ovarian Cancer Vaccine Technology
anixa biosciences
2025-07-09
car-t
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
SAN JOSE, Calif. , July 9, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that the United States Patent and Trademark Office (USPTO) will issue U.S. Patent Number 12,357,593 on July 15, 2025 covering key aspects of its ovarian cancer vaccine technology. The patent includes broad claims related to methods of eliciting an immune response targeting anti-Müllerian hormone receptor, type II (AMHR2), a promising target for ovarian cancer prevention and treatment. Anixa's ovarian cancer vaccine, being developed in a collaboration between Cleveland Clinic and the National Cancer Institute, represents a novel approach to preventing and treating ovarian cancer, particularly among high-risk populations such as those carrying BRCA mutations or with a family history of the disease. The patent includes methods of administering an immunogenic composition comprising a nucleic acid encoding the AMHR2 polypeptide, specifically the extracellular domain of human AMHR2, to elicit an AMHR2-specific immune response. This patent was issued to Cleveland Clinic and Anixa…
FDA Review of Citizen Petition Offers a Fresh Look at NurOwn®'s Evidence of Treatment Effectiveness
BrainStorm Cell Therapeutics
2025-07-08
nurown
GenSight Biologics Reports Cash Position as of June 30, 2025
GenSight Biologics
2025-07-08
gene therapies
PARIS, July 08, 2025--Regulatory News: GenSight Biologics ("GenSight Biologics" or the "Company") (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported its cash position as of June 30, 2025.
Beacon Therapeutics completes enrollment in registrational Phase 2/3 VISTA trial of laru-zova for patients with XLRP
Beacon Therapeutics
2025-07-08
phase 2
phase 2/3
The post Beacon Therapeutics completes enrollment in registrational Phase 2/3 VISTA trial of laru-zova for patients with XLRP appeared first on Beacon Therapeutics .
Lexeo Therapeutics Announces FDA Breakthrough Therapy Designation for LX2006 in Friedreich Ataxia
Lexeo Therapeutics
2025-07-08
breakthrough therapy
Orchard Therapeutics Announces Last Patient Treated in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome
orchard therapeutics
2025-07-07
otl-203
Completion of enrollment achieved nearly one year ahead of schedule Primary analysis anticipated two years post-treatment TOKYO and LONDON and BOSTON, July 07, 2025 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a Kyowa Kirin company, today announced the last patient has been treated in a registrational
Mustang Bio Granted Orphan Drug Designation by U.S. FDA for MB-101 (IL13Ra2-targeted CAR T-cells) to Treat Astrocytomas and Glioblastoma
mustang bio
2025-07-07
car t
orphan drug
glioblastoma
Abeona Therapeutics® Closes Sale of Rare Pediatric Disease Priority Review Voucher for $155 Million
Abeona Therapeutics
2025-07-02
priority review
GenSight Biologics to Partner with Catalent on Gene Therapy Candidate
GenSight Biologics
2025-07-02
gene therapy
GenSight Biologics recently announced the successful transfer of the upstream phase of the manufacturing process for LUMEVOQ, the Company’s gene therapy candidate product to CDMO Catalent.
Nanoscope Therapeutics to Present Groundbreaking Research at ASGCT 2025 Annual Meeting
Nanoscope Therapeutics
2025-07-02
gene therapies
Presentations will highlight durability, efficacy and safety of Nanoscope’s optogenetic therapy platforms DALLAS, May 12, 2025 /PRNewswire/ — Nanoscope Therapeutics Inc., a biotech company committed to restoring vision in blind patients in real-world settings by developing and commercializing novel gene therapies for retinal degenerative diseases, today announced multiple presentations at the 28th annual meeting of the American Society of Gene […] The post Nanoscope Therapeutics to Present Groundbreaking Research at ASGCT 2025 Annual Meeting appeared first on Nanoscope Therapeutics .
Engineered cell cross-talk unlocks CAR-T potential against glioblastoma
Fondazione Telethon
2025-07-02
car t
car-t
cell therapy
gene therapy
glioblastoma
Researchers at San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET, Milan) have developed a new strategy to enhance CAR T cell therapy against glioblastoma by using gene-engineered macrophages to deliver immune-boosting cytokines directly into the tumor. This targeted approach reactivates CAR-T cells, stimulates the broader immune response, and improves tumor control in preclinical models.
Instil Bio Announces U.S. F.D.A. Clearance of Investigational New Drug (IND) Application for AXN-2510, a PD-L1xVEGF Bispecific Antibody, for a Phase 1 Trial in Relapsed/Refractory Solid Tumors
Instil Bio
2025-07-02
ind
phase 1
phase 2
clearance
US phase 1 trial of ‘2510 is expected to be initiated before the end of 2025 Anticipate ImmuneOnco sharing initial safety and efficacy results from phase 2 trial of ‘2510 + chemo in 1L NSCLC in the second half of 2025 DALLAS, July 02, 2025 (GLOBE NEWSWIRE) -- Instil Bio, Inc.
Celularity Hails New Florida Law Opening Patient Access to Stem Cell Therapies
celularity
2025-07-01
cell therapies
Abeona Therapeutics® Announces Option Exercise by Beacon Therapeutics for Novel AAV204 Capsid for Ophthalmology Gene Therapy
Abeona Therapeutics
2025-07-01
gene therapy
abeona therapeutics
beacon therapeutics
CRISPR Therapeutics Named to TIME’s Most Influential Companies List of 2025
CRISPR
2025-06-30
crispr
ZUG , Switzerland and BOSTON, June 30, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, has been named to the fifth annual TIME 100 Most Influential Companies list.
Ferring expands gene therapy manufacturing in Morris County (photos) (updated)
Ferring Pharmaceuticals
2025-06-30
gene therapy
Ferring Pharmaceuticals officially opened doors to its state-of-the-art gene therapy manufacturing facility in Parsippany June 17.
AbbVie to Host Second-Quarter 2025 Earnings Conference Call
Abbvie Investors
2025-06-30
financial results
NORTH CHICAGO, Ill. , June 30, 2025 /PRNewswire/ -- AbbVie (NYSE: ABBV) will announce its second-quarter 2025 financial results on Thursday, July 31, 2025 , before the market opens. AbbVie will host a live webcast of the earnings conference call at 8 a.m. Central time .
Rocket Pharmaceuticals Announces FDA IND Clearance of RP-A701 for the Treatment of BAG3-associated Dilated Cardiomyopathy
Rocket Pharmaceuticals
2025-06-30
ind
clearance
gene therapy
RP-A701 is a first-in-class gene therapy for the treatment of BAG3-associated dilated cardiomyopathy BAG3-associated dilated cardiomyopathy is a rare, inherited heart condition caused by mutations in the BAG3 gene, leading to early-onset, progressive heart failure due to impaired cardiac function,
First DEB patient dosed in trial of KB803 gene therapy eye drops
Epidermolysis Bullosa News
2025-06-27
phase 3
gene therapy
dystrophic epidermolysis bullosa
A first patient has been dosed in a Phase 3 trial testing KB803 gene therapy eye drops for people with dystrophic epidermolysis bullosa.
ProQR Announces CTA Submission for Phase 1 Study of AX-0810 Targeting NTCP
proqr therapeutics
2025-06-27
cta
phase 1
pipeline
first-in-human
First-in-human trial of AX-0810 will evaluate safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers with initial data expected in Q4 2025 Marks the first submitted Clinical Trial Application (CTA) advancing ProQR’s Axiomer™ ADAR-mediated RNA editing platform into clinical development LEIDEN, Netherlands & CAMBRIDGE, Mass., June 26, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the submission of a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) to initiate a Phase 1 clinical trial of its lead pipeline program AX-0810 targeting NTCP, a liver cell protein that transports bile acids into cells.
U.S. Food and Drug Administration Approves Streamlined Patient Monitoring Requirements and Removal of REMS Programs within Bristol Myers Squibb’s Cell Therapy Labels
Bristol Myers Squibb
2025-06-27
cell therapy
U.S. Food and Drug Administration Approves Streamlined Patient Monitoring Requirements and Removal of REMS Programs within Bristol Myers Squibb’s Cell Therapy Labels
bristol myers squibb
2025-06-27
cell therapy
Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome—Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical
2025-06-27
breakthrough therapy
The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts.
Cellectis Reports Results from Shareholders Meeting Held on June 26, 2025
cellectis
2025-06-26
gene therapies
June 26, 2025 – New York (N.Y.) – Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, held today a shareholders general meeting at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4 th floor, 75013 Paris, France. At the meeting, during which approximately 57% of voting rights were exercised, resolutions 1 through 23 and resolutions 25 and 26 were adopted, while resolution 24 was rejected, consistent with the recommendations of the board of directors. The Cellectis shareholders' meeting appointed Mr. André Muller as a member of the Company's Board of Directors, with immediate effect. In addition, at the close of the AGM, the term of Mr Axel-Sven Malkomes expired, and the previously announced resignation of Mr. Pierre Bastid became effective. Mr. Muller serves currently (and this until July 1 st , 2025) as Chief Executive Officer of Idorsia Pharmaceuticals, Ltd. a listed Swiss biotech company. Previously, Mr. Muller served as Chief Financial Officer of Idorsia Pharmaceuticals, Ltd. and Actelion Pharmaceuticals, Ltd. He held various financial positions…
A2 Bio Announces First Patient Dosed in DENALI-1 Phase 1/2 Clinical Study of A2B395, a Novel Allogeneic Precision CAR T Cell Therapy for Patients with Solid Tumors that Express EGFR
A2 Bio
2025-06-26
car t
phase 1
phase 1/2
allogeneic
cell therapy
The post A2 Bio Announces First Patient Dosed in DENALI-1 Phase 1/2 Clinical Study of A2B395, a Novel Allogeneic Precision CAR T Cell Therapy for Patients with Solid Tumors that Express EGFR appeared first on A2 Biotherapeutics .
Pharmacy Practice News: FDA Drops REMS for CAR-T Rx; Will Access Expand?
Prime Therapeutics
2025-06-26
bcma
cd19
car-t
autologous
chimeric antigen receptor
Pharmacy Practice News: As of June 26, 2025, the FDA has removed certain Risk Evaluation and Mitigation Strategy (REMS) requirements from all but one of the currently approved B-cell maturation antigen (BCMA)- and CD19-directed autologous chimeric antigen receptor T-cell (CAR-T) immunotherapies
CRISPR Therapeutics Reports Positive Additional Phase 1 Data for CTX310™ Targeting ANGPTL3 and Provides Update on In Vivo Cardiovascular Pipeline
CRISPR
2025-06-26
crispr
phase 1
New Phase 1 clinical data for CTX310™ continues to demonstrate dose-dependent reductions in triglycerides (TG) and low-density lipoprotein (LDL), with peak reduction of up to 82% in TG and up to 86% in LDL, with a well-tolerated safety profile- -Complete Phase 1 data presentation for CTX310
GenSight Biologics Announces Significant Milestone in New Manufacturing Partnership with Catalent
GenSight Biologics
2025-06-26
gene therapy
gene therapies
leber hereditary optic neuropathy
PARIS, June 26, 2025--Regulatory News: GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced the successful transfer of the upstream phase of the manufacturing process for LUMEVOQ®, the Company’s gene therapy candidate product for the rare mitochondrial disease Leber Hereditary Optic Neuropathy (
Cell therapy weekly: gene therapy manufacturing milestone
GenSight Biologics
2025-06-26
cell therapy
gene therapy
Catch up on top news stories, including a strategic collaboration, expanded manufacturing capabilities, and successful upstream transfer.
FDA Grants RMAT Designation for enGene’s Detalimogene, Enabling Potential for Expedited Review in High-Risk, Non-Muscle Invasive Bladder Cancer
enGene
2025-06-25
rmat
bladder cancer
detalimogene voraplasmid
BOSTON & MONTREAL, June 25, 2025--enGene Holdings Inc. (Nasdaq: ENGN, or "enGene" or the "Company"), a clinical-stage, non-viral gene-based immunotherapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to detalimogene voraplasmid (also known as detalimogene, and previously EG-70), the Company’s lead investigational therapy for the treatment of high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, no
Mailed Self-Collection HPV Tests Improve Cervical Cancer Screening Rates, Study Finds
ASCO Post Journals
2025-06-25
cervical cancer
Mail-in self-collection tests for human papillomavirus (HPV) more than doubled cervical cancer screening participation among never- and under-screened U.S. women, according to a first-of-its-kind stud...
FDA Approves Darolutamide for Metastatic Castration-Sensitive Prostate Cancer
ASCO Post FDA
2025-06-25
prostate cancer
On June 3, 2025, the U.S. Food and Drug Administration approved darolutamide (Nubeqa) as a single agent for the treatment of metastatic castration-sensitive prostate cancer.
Prime Therapeutics’ leading research shows only 1 in 12 remain on a GLP-1 drug for obesity at three years
Prime Therapeutics
2025-06-25
persistence
Findings also show improved persistence with high-potency, obesity-approved GLP-1 products
Sangamo Therapeutics Announces Positive Topline Results From Registrational STAAR Study in Fabry Disease
Sangamo BioSciences
2025-06-24
fabry disease
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Abeona Therapeutics® Announces Publication in The Lancet of Phase 3 VIITAL™ Study Data in Recessive Dystrophic Epidermolysis Bullosa
Abeona Therapeutics
2025-06-24
phase 3
dystrophic epidermolysis bullosa
recessive dystrophic epidermolysis bullosa
Genprex Collaborators Present Positive Preclinical Research on Diabetes Gene Therapy at the 2025 American Diabetes Association 85th Scientific Sessions
Genprex
2025-06-24
gene therapy
Latest Research Demonstrates Promising Improved Glucose Homeostasis by Reprogramming Alpha Cells AUSTIN, Texas — (June 24, 2025) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that its research collaborators presented positive preclinical research from studies of GPX-002, the Company’s […]
Anixa Biosciences Initiates Dosing in Fourth Cohort in its Ovarian Cancer CAR-T Clinical Trial
anixa biosciences
2025-06-23
car-t
phase 1
quarterly
cell therapy
cell therapies
first-in-human
ovarian cancer
chimeric antigen receptor
tumor infiltrating lymphocytes
Fourth cohort dose is three million CAR positive cells; thirty times higher than the first cohort dose SAN JOSE, Calif. , June 23, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that it has dosed its first patient in the fourth dosage cohort in the ongoing Phase 1 clinical trial evaluating its novel chimeric antigen receptor-T cell (CAR-T) therapy for recurrent ovarian cancer. The study is being conducted through a research partnership with Moffitt Cancer Center ("Moffitt") under the direction of Dr. Robert Wenham, Chair of the Gynecologic Oncology Program at Moffitt, the principal investigator. The fourth cohort in the trial will receive a dose of three million CAR-positive cells per kilogram of body weight, representing a thirtyfold increase from the first cohort. No dose-limiting toxicities were observed in the third cohort, enabling advancing to the fourth dosage cohort. This planned escalation marks a key step in assessing the safety and therapeutic effect of CAR-T cell therapy in patients with ovarian cancer. Anixa's FSHR-mediated CAR-T…
Lyell Immunopharma to Participate in the H.C. Wainwright “HCW@Home” Series
lyell immunopharma
2025-06-23
car t
cell therapies
SOUTH SAN FRANCISCO, Calif., June 23, 2025 (GLOBE NEWSWIRE) -- Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical-stage company advancing next-generation CAR T-cell therapies for patients with cancer, announced today that members of its senior management team will participate in the H.C.
Actinium Highlights Expanded Data Set for ATNM-400 in Prostate Cancer Demonstrating Superior Efficacy to Enzalutamide and Ability to Overcome Resistance to ARPI Therapy and PSMA-Ac-225/Lu-177 Labelled Radiotherapy at the Society of Nuclear Medicine & Molecular Imaging Annual Meeting
Actinium Pharma
2025-06-23
aml
cd33
psma
phase 2
pivotal
pipeline
clearance
phase 2/3
quarterly
solid tumor
gene therapies
acute myeloid leukemia
ATNM-400 shows; superior prostate cancer cell killing compared to androgen receptor inhibitor Xtandi ® (enzalutamide), the ability to overcome Xtandi resistance, and activity in combination - ATNM-400 is more efficacious than both Pluvicto ® (177-Lu-PSMA-617) and Ac-225-PSMA-617 targeted radiotherapy in prostate cancer in vitro and in vivo models and overcome resistance in prostate cancer tumors that failed Pluvicto ® therapy - ATNM-400's target is highly differentiated from PSMA as it is implicated in disease biology, contributing to disease progression, faster progression to castration resistance and poorer survival outcomes with target expression reported following androgen receptor inhibitor and PSMA radiotherapy - Data further supports ATNM-400's potential to address critical gaps in prostate cancer treatment as a monotherapy or in sequence with other therapeutic modalities NEW YORK , June 23, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today reported new preclinical data from its first-in-class, non-PSMA targeting radiotherapy prostate cancer candidate ATNM-400,…
OpRegen® (RG6501) 36-Month Visual Acuity Results Featured at Clinical Trials at the Summit 2025
lineage cell therapeutics
2025-06-23
cell therapy
Mean vision gains of +9 letters among patients with extensive coverage of OpRegen cell therapy to the Geographic Atrophy (GA) lesion site Evidence of retinal structural improvement persisted out to 3 years Anatomical and functional improvements occur following a single administration of OpRegen
Imbruvica gets positive EMA Committee opinion for untreated mantle cell lymphoma eligible for Stem Cell Transplant: Janssen-Cilag International
Janssen Global Services
2025-06-22
mantle cell lymphoma
Beerse: Janssen-Cilag International NV, a Johnson & Johnson company, has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a...
Darzalex receives positive EMA Committee opinion for high-risk smouldering multiple myeloma: Janssen-Cilag International
Janssen Global Services
2025-06-21
multiple myeloma
Beerse: Janssen-Cilag International NV, a Johnson & Johnson company, has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has...
Zemcelpro® (UM171 Cell Therapy) receives positive CHMP opinion for treatment of blood cancer patients without access to suitable donor cells
Excellthera
2025-06-20
allogeneic
cell therapy
myelodysplastic
– If approved, Zemcelpro® is expected to: increase access to donor-derived stem cell transplantation, which offers a potentially curative option for haematologic malignancies, including leukemias and myelodysplastic syndromes be the first and only therapy in the European Union with marketing authorization for adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative […]
Capricor Therapeutics Announces Positive 4-Year Data from HOPE-2 Open-Label Extension Study of Deramiocel in Duchenne Muscular Dystrophy
Capricor Therapeutics
2025-06-20
deramiocel
duchenne muscular dystrophy
Anixa Biosciences to Host an Investor Webcast on June 26, 2025
anixa biosciences
2025-06-20
car-t
quarterly
cell therapies
ovarian cancer
tumor infiltrating lymphocytes
SAN JOSE, Calif. , June 20, 2025 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced that it will host an investor webcast presentation on June 26, 2025 at 2:00 PM EDT. During the webcast, Dr. Amit Kumar, Anixa's Chairman and CEO, will conduct an introductory presentation that will cover key aspects of Anixa's business including an overview of the Company's therapeutic portfolio, its business strategy, market opportunity and near-term milestones. After the formal presentation, investors will have an opportunity to ask relevant questions through an interactive Q&A portal. To listen to the webcast or to ask questions during the live event, please pre-register at the following link: https://us02web.zoom.us/webinar/register/WN_g8ClEi_KQzmPJhIvQdD64Q . An archived version of the webcast and presentation will be available on the Company's website, https://ir.anixa.com/events . About Anixa Biosciences, Inc. Anixa is a clinical-stage biotechnology company focused on the treatment and prevention of cancer. Anixa's therapeutic portfolio consists of an ovarian cancer…
Dupilumab treatment eases disease severity, itching in DEB: Study
Epidermolysis Bullosa News
2025-06-20
dystrophic epidermolysis bullosa
Treatment with dupilumab eases itching and disease severity in people with dystrophic epidermolysis bullosa, a small study suggests.
Clinical Milestones, Vector Innovation Key to Gene Therapy, GenoTher Biocluster’s First International Summit, Atamyo Therapeutics’ Progress for LGMDs Highlight Genethon’s Most Recent Newsletter
Genethon
2025-06-18
gene therapy
gene therapies
PARIS, FRANCE (19 June, 2025) – Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), and its spin-off Atamyo Therapeutics made significant advances in their gene therapies for multiple rare diseases as highlighted in Genethon’s most recent Newsletter. Chief among them are key clinical milestones reached, including those … Continued
Lisa Deschamps of AviadoBio and Richard Wilson of Astellas discuss the field of AAV gene therapy, and their partnership on AVB-101, an AAV gene therapy for frontotemporal dementia (FTD)
aviadobio
2025-06-18
aav
gene therapy
The post Lisa Deschamps of AviadoBio and Richard Wilson of Astellas discuss the field of AAV gene therapy, and their partnership on AVB-101, an AAV gene therapy for frontotemporal dementia (FTD) appeared first on AviadoBio .
Lyell Immunopharma Announces Positive New Clinical Data Demonstrating High Rates of Durable Complete Responses from the Phase 1/2 Trial of LYL314 for the Treatment of Aggressive Large B-cell Lymphoma
lyell immunopharma
2025-06-17
phase 1
phase 1/2
b-cell lymphoma
complete response
large b-cell lymphoma
overall response rate
LYL314 demonstrated robust clinical responses, with an 88% overall response rate and a 72% complete response rate in patients treated in the third- or later-line setting (N = 25) 71% of patients with complete response remained in complete response at ≥ 6 months Manageable safety profile appropriate
Bristol Myers Squibb Presents First Data from the Marginal Zone Lymphoma Cohort of the Transcend FL Trial Demonstrating Deep and Durable Responses with Breyanzi (lisocabtagene maraleucel)
bristol myers squibb
2025-06-16
breyanzi
marginal zone lymphoma
BrainStorm Announces New Survival Data from Expanded Access Program Cohort: Remarkably 90% of ALS Patients Survived Over 5 Years with NurOwn®
BrainStorm Cell Therapeutics
2025-06-16
nurown
Bristol Myers Squibb Presents First Data from the Marginal Zone Lymphoma Cohort of the Transcend FL Trial Demonstrating Deep and Durable Responses with Breyanzi (lisocabtagene maraleucel)
Bristol Myers Squibb
2025-06-16
breyanzi
marginal zone lymphoma
Intellia Therapeutics Announces Positive Three-Year Data from Phase 1 Trial of Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema (HAE) at the European Academy of Allergy and Clinical Immunology Congress
Intellia Therapeutics Inc.
2025-06-15
phase 1
hereditary angioedema
With up to three years of follow-up, a single dose of lonvo-z led to a 98% mean reduction in monthly HAE attack rate in all 10 patients All 10 patients were attack-free and treatment-free for a median of 23 months through the latest follow-up, demonstrating the potential of lonvo-z to become the
Study: Long-term Vyjuvek in dystrophic epidermolysis bullosa safe...
Epidermolysis Bullosa News
2025-06-13
vyjuvek
dystrophic epidermolysis bullosa
Vyjuvek is made to deliver healthy COL7A1 gene copies to skin cells, helping them make their own collagen to address blistering and wounds.
Medigene AG Shares Move to General Standard Segment of the Frankfurt Stock Exchange
medigene
2025-06-13
tcr
Planegg/Martinsried (pta021/13.06.2025/12:40 UTC+2) Planegg/Martinsried, June 13, 2025. Medigene AG (Medigene, WKN: A40ESG, symbol: MDG1), an oncology platform company focused on the research and development of T cell receptor (TCR)-guided therapies for the treatment of cancer, announces that, following preliminary insolvency proceedings ordered by the Munich Local Court on April 24, 2024, the management of the ... Read more The post Medigene AG Shares Move to General Standard Segment of the Frankfurt Stock Exchange appeared first on Medigene AG .
Editas Medicine Reports Proprietary Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for Sickle Cell Disease and Beta Thalassemia at the European Hematology Association 2025 Congress in June
Editas
2025-06-13
beta thalassemia
lipid nanoparticle
sickle cell disease
BRCA Genes Increase Risk of Lymphoma in Women with Breast Cancer and Implants
Hematology.org
2025-06-13
breast cancer
BRCA Genes Increase Risk of Lymphoma in Women with Breast Cancer and Implants
Beam Therapeutics Announces New Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 Supporting Differentiated Profile in Sickle Cell Disease (SCD) at European Hematology Association (EHA) 2025 Congress
beam therapeutics
2025-06-13
scd
phase 1
phase 1/2
sickle cell disease
Updated Data from 17 Patients Consistent with Previously Presented Data; All Patients Treated with BEAM-101 Achieved Hemoglobin F (HbF) Induction of >60%, Hemoglobin S (HbS) Reduction to 40%, and Resolution of Anemia Patients Required a Median of One Mobilization Cycle and Experienced Rapid
GenSight Biologics Reaches Agreement with ANSM for Opening of LUMEVOQ® Named Early Access Program (AAC) in France
GenSight Biologics
2025-06-12
gene therapies
PARIS, June 12, 2025--Regulatory News: GenSight Biologics ("GenSight Biologics" or the "Company") (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that it has reached agreement with the French medicines safety agency ANSM to consider expeditiously opening the French named early access program (AAC) for LUMEVOQ
Sumitomo Pharma America Announces that Nuvisertib (TP-3654) Has Received FDA Fast Track Designation for the Treatment of Myelofibrosis
Enzyvant Therapeutics
2025-06-12
fast track
MiNK Therapeutics Announces Virtual Annual Shareholders Meeting
Mink Therapeutics
2025-06-11
allogeneic
cell therapies
NEW YORK , June 10, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics , Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering allogeneic, off-the-shelf invariant natural killer T (iNKT) cell therapies, today announced that its Annual Shareholders Meeting will begin at 9:30 a.m. ET.
Bristol Myers Squibb Presents Late-Breaking Data from Pivotal Phase 3 POETYK PsA-1 Trial Demonstrating Superiority of Sotyktu (deucravacitinib) Compared with Placebo in Adults with Psoriatic Arthritis
Bristol Myers Squibb
2025-06-11
phase 3
pivotal
Bristol Myers Squibb Presents Late-Breaking Data from Pivotal Phase 3 POETYK PsA-1 Trial Demonstrating Superiority of Sotyktu (deucravacitinib) Compared with Placebo in Adults with Psoriatic Arthritis
bristol myers squibb
2025-06-11
phase 3
pivotal
Cabaletta Bio Announces New Rese-cel Safety and Efficacy Data in Patients with Myositis, Lupus and Scleroderma to Be Presented at the EULAR 2025 Congress
cabaletta bio
2025-06-11
lupus
scleroderma
Retifanlimab-dlwr Approved for Anal Cancer in Combination With Carboplatin Plus Paclitaxel and as a Single Agent
ASCO Post FDA
2025-06-10
first-line
The U.S. Food and Drug Administration (FDA) approved retifanlimab-dlwr (Zynyz), a PD-1–blocking monoclonal antibody, with carboplatin and paclitaxel for the first-line treatment of adults with inopera...
FDA Approves Therapy for NSCLC With High c-Met Protein Overexpression
ASCO Post FDA
2025-06-10
c-met
accelerated approval
The FDA granted accelerated approval to telisotuzumab vedotin-tllv (Emrelis), a c-Met–directed antibody and microtubule inhibitor conjugate, for adults with locally advanced or metastatic, nonsquamous...
CRISPR-Cas9–Edited Tumor-Infiltrating Lymphocytes in Metastatic Colorectal Cancer
ASCO Post Journals
2025-06-10
cas9
crispr
autologous
crispr-cas9
first-in-human
tumor-infiltrating lymphocytes
In a first-in-human, single-institution phase I trial reported in The Lancet Oncology,1 Lou et al found that autologous tumor-infiltrating lymphocytes genetically edited with CRISPR-Cas9 to target the...
Use of Bispecific Antibody in Older Patients With Relapsed or Refractory Multiple Myeloma
ASCO Post Journals
2025-06-10
multiple myeloma
Although treatment with the bispecific antibody teclistamab was approved for patients with relapsed or refractory multiple myeloma, based on the MajesTEC-1 study results, only 15% of patients in the p...
SparingVision appoints Dr. Kali Stasi as Chief Medical Officer
Sparing Vision
2025-06-09
gene therapy
SparingVision appoints Dr. Kali Stasi as Chief Medical Officer Experienced global leader in ophthalmology and gene therapy to advance SparingVision’s clinical programs towards registration and commercialization Over 30 years of experience across academic research, biotech, and pharma, including key leadership roles at Novartis, SalioGen, and Adverum, especially in inherited retinal diseases and age-related macular… The post SparingVision appoints Dr. Kali Stasi as Chief Medical Officer appeared first on SparingVision .
Nkarta Appoints Shawn Rose Chief Medical Officer & Head of R&D as Company Resets Senior Leadership Role for Autoimmune Focus
nkarta
2025-06-06
cell therapies
SOUTH SAN FRANCISCO, Calif., June 06, 2025 (GLOBE NEWSWIRE) -- Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat autoimmune disease, today announced the appointment of Shawn Rose, M.D.
REGENXBIO REPORTS NEW POSITIVE FUNCTIONAL DATA FROM PHASE I/II AFFINITY DUCHENNE® TRIAL OF RGX-202
REGENXBIO
2025-06-05
transduction
RGX-202 demonstrating consistent evidence of positively changing disease trajectory for Duchenne All dose level 2 participants exceeded external natural history controls on all functional measures Biomarker data demonstrate consistent, robust microdystrophin expression and transduction levels
Lineage Cell Therapeutics Announces Updates to the 3rd Annual Spinal Cord Injury Investor Symposium
lineage cell therapeutics
2025-06-05
allogeneic
cell therapies
Fully Virtual Event Scheduled for June 27, 2025 CARLSBAD, Calif. --(BUSINESS WIRE)--Jun. 5, 2025-- Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel allogeneic, or “off the shelf”, cell therapies for serious neurological and
MiNK Therapeutics Awarded Prestigious NIAID Grant to Advance Allo-iNKT Cell Therapy for Prevention of GvHD in Stem Cell Transplant Patients
Mink Therapeutics
2025-06-03
allogeneic
cell therapy
Non-dilutive NIH funding supports development of MiNK’s allogeneic iNKT platform for immune regulation in high-risk HSCT settings NEW YORK , June 02, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering allogeneic, off-the-shelf
New insights into long-term dysfunction of edited blood stem cells and how to overcome it
Fondazione Telethon
2025-06-03
aav6
cas9
crispr
crispr-cas9
gene editing
gene therapy
Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET, Milan) have found that gene editing using CRISPR-Cas9 in combination with AAV6 vectors can trigger inflammatory and senescence-like responses in blood stem cells, compromising their long-term ability to regenerate the blood system. The study, published in Cell Reports Medicine, outlines new strategies to overcome this hurdle, improving both the safety and efficacy of gene-editing-based therapies for inherited blood disorders.
Affinia Therapeutics and DCM Foundation Announce Partnership to Increase Awareness About BAG3 Dilated Cardiomyopathy and Critical Need for Genetic Testing to Help Save Lives
Affinia Therapeutics
2025-06-03
cardiomyopathy
Affinia Therapeutics Appoints Hideo Makimura, M.D., Ph.D., as Chief Medical Officer The post Affinia Therapeutics and DCM Foundation Announce Partnership to Increase Awareness About BAG3 Dilated Cardiomyopathy and Critical Need for Genetic Testing to Help Save Lives appeared first on AffiniaTX .
AffyImmune Announces Plenary Oral Presentation on ICAM-1 Directed CAR T Therapy AIC100 at AACR 2025
AffyImmune
2025-06-03
car t
NATICK, Mass., April 28, 2025 /PRNewswire/ -- AffyImmune Therapeutics, Inc., a clinical-stage biopharmaceutical company, today announced an abstract detailing its affinity-tuned CAR T therapy AIC100 will be presented at the American Association for Cancer Research (AACR) Annual Meeting, held April 25-30, 2025 at McCormick Place Convention Center in Chicago, IL. "We look forward to presenting our updated Phase 1 data at
Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-101 for the Treatment of Sickle Cell Disease
beam therapeutics
2025-06-03
orphan drug
base editing
sickle cell disease
CAMBRIDGE, Mass., June 03, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to
Instil Bio Announces Appointment of Jamie Freedman, M.D., Ph.D., as Chief Medical Officer
Instil Bio
2025-06-02
til
pipeline
DALLAS, June 02, 2025 (GLOBE NEWSWIRE) -- Instil Bio, Inc. (“Instil”) (Nasdaq: TIL), a clinical-stage biopharmaceutical company focused on developing a pipeline of novel cancer therapies, today announced the appointment of Jamie Freedman, M.D., Ph.D., as Chief Medical Officer. Dr.
IN8bio Presents Positive Phase 1 Data of INB-200 in Newly Diagnosed GBM Demonstrating Prolonged Progression-Free Survival
In8Bio
2025-06-02
gbm
phase 1
Repeated doses of INB-200 demonstrate extended median progression-free survival (mPFS) of 16.1 months, more than double the expected 6.9 months typically observed with the standard-of-care (SOC) Stupp protocol INB-200 is well-tolerated, showing no serious toxicities beyond those typically observed
REGENXBIO to Host Webcast Discussing Interim Functional Data from the Phase I/II AFFINITY DUCHENNE® Trial of RGX-202
REGENXBIO
2025-06-02
rgx-202
Event will feature Aravindhan Veerapandiyan, M.D., principal investigator of the AFFINITY DUCHENNE ® trial ROCKVILLE, Md. , June 2, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a webcast to discuss new interim functional data from the Phase I/II AFFINITY
BioNTech and Bristol Myers Squibb Announce Global Strategic Partnership to Co-Develop and Co-Commercialize Next-generation Bispecific Antibody Candidate BNT327 Broadly for Multiple Solid Tumor Types
Bristol Myers Squibb
2025-06-02
solid tumor
Atamyo Therapeutics Announces First Patients Dosed with ATA-200 Gene Therapy in LGMD-R5 Clinical Trial
atamyo therapeutics
2025-06-02
lgmd
gene therapy
gene therapies
Evry, France (June 2, 2025) – Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and cardiomyopathies, today announced the dosing with ATA-200 gene therapy of […]
Atamyo Therapeutics annonce le traitement des premiers patients avec sa thérapie génique ATA-200 dans l’essai clinique ciblant la myopathie des ceintures LGMD-R5
atamyo therapeutics
2025-06-02
lgmd
Evry, France (2 juin 2025) – Atamyo Therapeutics, société de biotechnologie au stade clinique spécialisée sur le développement de thérapies géniques de nouvelle génération ciblant les dystrophies musculaires, a annoncé aujourd’hui le traitement des deux […]
BioNTech and Bristol Myers Squibb Announce Global Strategic Partnership to Co-Develop and Co-Commercialize Next-generation Bispecific Antibody Candidate BNT327 Broadly for Multiple Solid Tumor Types
bristol myers squibb
2025-06-02
solid tumor
Novartis Pluvicto™ demonstrates statistically significant and clinically meaningful rPFS benefit in patients with PSMA-positive metastatic hormone-sensitive prostate cancer
Novartis
2025-06-02
psma
Allogene Therapeutics Provides Updated Phase 1 Data Highlighting Durable Responses with ALLO-316 in Heavily Pretreated Advanced Renal Cell Carcinoma at ASCO
Allogene
2025-06-01
car t
phase 1
Data Highlights Transformative Promise of CAR T in Solid Tumors Phase 1 Trial with ALLO-316 Demonstrated Potential to Provide Meaningful Clinical Benefit in Patients with CD70 TPS ≥ 50% Advanced or Metastatic RCC A Single Dose of ALLO-316 Achieved a 31% Confirmed Response Rate Four of Five
Novartis Kisqali® reduces risk of recurrence in younger patients with early breast cancer in NATALEE subgroup analysis
Novartis
2025-06-01
breast cancer
Rheacell, AOP partner on stem cell therapy for epidermolysis bullosa
Epidermolysis Bullosa News
2025-05-30
cell therapy
Two Phase 3 trials are ongoing, and both are testing the stem cell therapy’s safety and effectiveness in epidermolysis bullosa.
Instil Bio to Participate in the 2025 Jefferies Global Healthcare Conference
Instil Bio
2025-05-30
til
DALLAS, May 30, 2025 (GLOBE NEWSWIRE) -- Instil Bio, Inc. (Nasdaq: TIL, "Instil"), a clinical-stage biopharmaceutical company focused on developing a pipeline of novel therapies, today announced that Bronson Crouch, Chief Executive Officer, will present at the Jefferies Global Healthcare Conference
Roivant Reports Financial Results for the Fourth Quarter and Fiscal Year Ended March 31, 2025, and Provides Business Update
roivant
2025-05-29
financial results
BASEL, Switzerland and LONDON and NEW YORK, May 29, 2025 (GLOBE NEWSWIRE) -- Roivant (Nasdaq: ROIV) today reported its financial results for the fourth quarter and fiscal year ended March 31, 2025, and provided a business update. Brepocitinib program progressing well with rapid enrollment in
Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)
beam therapeutics
2025-05-29
orphan drug
base editing
alpha-1 antitrypsin deficiency
CAMBRIDGE, Mass., May 29, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to
Kyverna Therapeutics to Present at the Jefferies Global Healthcare Conference
Kyverna Therapeutics
2025-05-28
cell therapies
EMERYVILLE, Calif. , May 28, 2025 /PRNewswire/ -- Kyverna Therapeutics, Inc. (Kyverna, Nasdaq: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, today announced that Warner Biddle , Chief Executive Officer of Kyverna, will
Nature study reveals novel strategy for in vivo blood stem cell gene therapy
Fondazione Telethon
2025-05-28
lentiviral
gene therapy
gene transfer
Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget, Milan) have discovered a critical early postnatal window that allows for effective in vivo gene transfer to circulating hematopoietic stem and progenitor cells (HSPCs) using lentiviral vectors (LVs), as published in Nature.
AskBio Announces Publication of Complete Results of Phase 1b Trial of AB-1005 Gene Therapy in Participants with Parkinson’s Disease in Movement Disorders
askbio
2025-05-27
rmat
gene therapy
gene transfer
Research Triangle Park, N.C.– MAY 27, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced today the recent publication of the complete results of its Phase 1b trial of AB-1005, a glial cell line-derived neurotrophic factor (GDNF) investigational gene therapy for the treatment of Parkinson’s disease (PD), in Movement Disorders, an official peer-reviewed journal of the International Parkinson and Movement Disorder Society.1 “The exploration of the neurorestorative and neuroprotective potential of GDNF gene transfer is critical to advancing our understanding of AB-1005, which may one day be a groundbreaking treatment to slow the progression of Parkinson’s disease,” said Chad Christine, MD, Professor of Neurology at the University of California, San Francisco, and publication author. “These encouraging results support further evaluation of this therapy in a randomized trial.” The publication, which is available online, states that treatment with AB-1005 was well tolerated with no serious adverse events related to GDNF gene therapy in all 11 participants and is associated with numerical stability in clinical…
FDA Approves Doublet for KRAS -Mutated, Recurrent, Low-Grade Serous Ovarian Cancer
ASCO Post FDA
2025-05-25
ovarian cancer
accelerated approval
Today, the FDA granted accelerated approval to the combination of the dual RAF/MEK inhibitor avutometinib and the FAK inhibitor defactinib (Avmapki Fakzynja Co-pack) for adults with KRAS-mutated, recu...
Strategy for Managing Immunotherapy-Induced Parkinsonism in Patients With Multiple Myeloma
ASCO Post Journals
2025-05-25
multiple myeloma
The JAK (Janus kinase) inhibitor ruxolitinib may prove to be an effective treatment of parkinsonism arising from immunotherapy for patients with multiple myeloma, according to the results of two case ...
Instil Bio and ImmuneOnco to Host Investor and Analyst Breakfast to Discuss the Evolving PD-(L)1xVEGF Bispecific Antibody Landscape and Clinical Trial Updates During the 2025 ASCO Annual Meeting in Chicago
Instil Bio
2025-05-23
til
DALLAS and SHANGHAI, May 23, 2025 (GLOBE NEWSWIRE) -- Instil Bio, Inc. (Nasdaq: TIL, "Instil") and ImmuneOnco Biopharmaceuticals (Shanghai) Inc. (HKEX Code: 1541.HK, “ImmuneOnco”), today announced that they will jointly host an investor and research analyst breakfast in Chicago, Illinois on
Allogene Therapeutics Announces Participation in Upcoming Investor Conferences
Allogene
2025-05-23
car t
allogeneic
SOUTH SAN FRANCISCO, Calif. , May 23, 2025 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™) products for cancer and autoimmune disease, today announced that it will participate in four
Small molecule makes RDEB gene editing more accurate: Study
Epidermolysis Bullosa News
2025-05-23
gene editing
M3814 was used for more accurate editing of genetic mutations causing RDEB, enabling skin cells to restore type VII collagen, a study found.
Blenrep (belantamab mafodotin) combinations receive positive CHMP opinion in relapsed/refractory multiple myeloma
GSK
2025-05-23
multiple myeloma
Positive opinion supported by superior efficacy shown in two head-to-head phase III trials, including overall survival in DREAMM-7.
Adicet Bio to Participate in a Fireside Chat at the 2025 Jefferies Global Healthcare Conference
Adicet Bio
2025-05-22
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--May 21, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced that Chen Schor , President and Chief
Cellectis’ Annual Shareholders General Meeting to be Held on June 26, 2025
cellectis
2025-05-21
gene therapies
New York, NY – May 21, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that it will hold its annual general meeting on June 26, 2025 at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4th floor, 75013 Paris, France. The notice convening the annual general meeting stating the detailed agenda and modalities of participation in the meeting and the report of the board of directors to the shareholders meeting are available on the Cellectis website: https://www.cellectis.com/en/investors/general-meetings/
Janssen-Cilag Guselkumab receives UK nod for Crohn's disease, ulcerative colitis
Janssen Global Services
2025-05-20
crohn's disease
Janssen-Cilag Limited has received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) for guselkumab (Tremfya) to treat Crohn's disease and ulcerative colitis...
REGENXBIO Announces Strategic Royalty Monetization Agreement for Up to $250 Million
REGENXBIO
2025-05-20
priority review
$150 million secured at closing extends cash runway into early 2027 REGENXBIO retains additional potential non-dilutive funding opportunities, including monetization of Priority Review Voucher (PRV) and milestones from AbbVie ROCKVILLE, Md. , May 19, 2025 /PRNewswire/ -- REGENXBIO Inc.
PeproMene Bio, Inc. and the Institute for Follicular Lymphoma Innovation (IFLI) Announce The First Follicular Lymphoma (FL) Patient Treated with BAFF-R Targeting CAR-T Cells (PMB-CT01) Achieves a Complete Response
pepromene bio
2025-05-20
car-t
complete response
follicular lymphoma
To find the article on the web: Click here IRVINE, Calif., May 20, 2025 /PRNewswire/ — PeproMene Bio, Inc. (PMB) a clinical-stage biotech company developing novel therapies to treat cancers and immune disorders, today announced that the first r/r FL patient treated in … Continue reading → The post PeproMene Bio, Inc. and the Institute for Follicular Lymphoma Innovation (IFLI) Announce The First Follicular Lymphoma (FL) Patient Treated with BAFF-R Targeting CAR-T Cells (PMB-CT01) Achieves a Complete Response appeared first on PeproMene Bio Inc .
ASGCT 2025: A Novel Investigational AAV Gene Therapy for Treatment of BAG3 Dilated Cardiomyopathy
Affinia Therapeutics
2025-05-19
aav
gene therapy
cardiomyopathy
The post ASGCT 2025: A Novel Investigational AAV Gene Therapy for Treatment of BAG3 Dilated Cardiomyopathy appeared first on AffiniaTX .
ASGCT 2025: Development of a Flexible High Yielding, High Performing Process for Manufacturing of AFTX-201, a Novel Investigational AAV Gene Therapy for Treatment of BAG3 Dilated Cardiomyopathy
Affinia Therapeutics
2025-05-19
aav
gene therapy
cardiomyopathy
The post ASGCT 2025: Development of a Flexible High Yielding, High Performing Process for Manufacturing of AFTX-201, a Novel Investigational AAV Gene Therapy for Treatment of BAG3 Dilated Cardiomyopathy appeared first on AffiniaTX .
AskBio Presents Complete Results of Phase 1 Trial of AB-1002 Gene Therapy in Participants with Congestive Heart Failure at European Society of Cardiology Heart Failure Meeting
askbio
2025-05-19
aav
gene therapy
heart failure
adeno-associated virus
Research Triangle Park, N.C. – MAY 19, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, presented—during a dedicated late-breaking science session at this year’s European Society of Cardiology Heart Failure meeting—the complete dataset from a Phase 1 trial investigating AB-1002 for the treatment of congestive heart failure (CHF). This year’s meeting is being held in Belgrade, Serbia, from May 17 to 20, 2025. This non-randomized, sequential dose escalation trial includes escalating dose cohorts to evaluate the safety and preliminary efficacy of investigational gene therapy AB-1002 in participants with NYHA Class III non-ischemic heart failure with reduced ejection fraction (HFrEF).1 It is estimated that 64 million people worldwide are living with heart failure, and despite standard of care, mortality and morbidity in heart failure remain very high.2,3 The late-breaking oral presentation highlights that single-dose administration of AB-1002 at 12 months post-dose resulted in the following outcomes:4 Meaningful changes from baseline were considered to be: NYHA FC, ≥1 point; LVEF, ≥5%; MLHFQ, ≥10 points; VO2 max,…
Atsena Therapeutics Announces Positive Clinical Data from Part A of Phase I/II Trial Evaluating ATSN-201 Gene Therapy to Treat X-linked Retinoschisis (XLRS)
atsena therapeutics
2025-05-19
x-linked
gene therapy
x-linked retinoschisis
Atsena Therapeutics Announces Positive Clinical Data from Part A of Phase I/II Trial Evaluating ATSN-201 Gene Therapy to Treat X-linked Retinoschisis (XLRS) First XLRS study ... The post Atsena Therapeutics Announces Positive Clinical Data from Part A of Phase I/II Trial Evaluating ATSN-201 Gene Therapy to Treat X-linked Retinoschisis (XLRS) appeared first on Atsena Therapeutics .
Blenrep (belantamab mafodotin) combinations approved in Japan for treatment of relapsed/refractory multiple myeloma
GSK
2025-05-19
multiple myeloma
Second major approval for Blenrep combinations, with more expected in 2025
ASGCT 2025: Engineered AAV Capsids That Target a Novel Human Brain Endothelial Receptor Achieve Robust Transduction in Non-human Primate Central Nervous System After Intravenous Dosing
Affinia Therapeutics
2025-05-19
aav
The post ASGCT 2025: Engineered AAV Capsids That Target a Novel Human Brain Endothelial Receptor Achieve Robust Transduction in Non-human Primate Central Nervous System After Intravenous Dosing appeared first on AffiniaTX .
Janssen launches Tecvayli in Egypt; A treatment for multiple myeloma, a type of blood cancer
Janssen Global Services
2025-05-19
multiple myeloma
Janssen has invested over two decades in research and innovation in the field of multiple myeloma
ASGCT 2025: Novel AAV Capsids That Bind Human Transferrin Receptor (TFRC) Demonstrate Widespread and Preferential CNS Tropism in TFRC-KI Mice After Low-dose Systemic Dosing
Affinia Therapeutics
2025-05-19
aav
The post ASGCT 2025: Novel AAV Capsids That Bind Human Transferrin Receptor (TFRC) Demonstrate Widespread and Preferential CNS Tropism in TFRC-KI Mice After Low-dose Systemic Dosing appeared first on AffiniaTX .
SparingVision Presents PRODYGY data and Research Update in Two Oral Presentations at ASGCT 2025
Sparing Vision
2025-05-19
aav
gene therapy
SparingVision Presents PRODYGY data and Research Update in Two Oral Presentations at ASGCT 2025 Favorable safety profile and preliminary signs of efficacy observed in patients with severe advanced RP in ongoing Phase I/II PRODYGY trial Company also unveils novel triplex dPCR assay to improve detection and quantification of replication-competent AAV genomes in gene therapy… The post SparingVision Presents PRODYGY data and Research Update in Two Oral Presentations at ASGCT 2025 appeared first on SparingVision .
ASGCT 2025: High-Yield, Pan-Serotype Plasmid System for Manufacturing Adeno-Associated Virus Gene Therapies: Cost and Efficiency Benefits for R&D and Commercial Processes
Affinia Therapeutics
2025-05-19
gene therapies
adeno-associated virus
The post ASGCT 2025: High-Yield, Pan-Serotype Plasmid System for Manufacturing Adeno-Associated Virus Gene Therapies: Cost and Efficiency Benefits for R&D and Commercial Processes appeared first on AffiniaTX .
Genethon Presents Two Year Consolidated Results of Its Gene Therapy Trial for Duchenne Muscular Dystrophy: Maintenance of Motor Functions and Significant, Sustained Reduction in CPK Levels in Patients Treated at the Effective Dose at ASGCT 2025
Genethon
2025-05-17
cell therapy
gene therapy
duchenne muscular dystrophy
Paris, France (May 17, 2025) – Genethon unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, May 13 – 17, 2025. Five patients, aged 6 to 10 years, were treated, … Continued
Bladder Cancer Awareness Month 2025: Ferring partners with the community to strengthen its commitment to improve patient outcomes
Ferring Pharmaceuticals
2025-05-16
bladder cancer
[spb_boxed_content type=”coloured” box_link_target=”_self” padding_vertical=”0″ padding_horizontal=”0″ width=”1/1″ el_position=”first last”] Bladder Cancer Awareness Month 2025: Ferring partners with the community to strengthen its commitment to improve patient outcomes [/spb_boxed_content] [spb_blank_spacer height=”30px” width=”1/1″ el_position=”first last”] [spb_blank_spacer height=”30px” width=”1/1″ el_position=”first last”] [spb_boxed_content type=”coloured” box_link_target=”_self” padding_vertical=”0″ padding_horizontal=”0″ width=”1/1″ el_position=”first last”] The forgotten cancer: understanding the unmet need Bladder cancer. These two words deliver a devastating […]
1st US site offering Zevaskyn for RDEB opens in Chicago
Epidermolysis Bullosa News
2025-05-16
zevaskyn
A Chicago hospital became the first qualified treatment center in the U.S. to offer Zevaskyn for recessive dystrophic EB (RDEB).
Century Therapeutics Reports First Quarter 2025 Financial Results and Provides Business Update
Century Therapeutics
2025-05-15
financial results
FDA grants Regenerative Medicine Advanced Therapy designation forBrainChild Bio’s B7-H3 CAR T-cell therapy for incurable pediatric brain tumors Read More
Brainchild Bio
2025-05-15
b7-h3
car t
cell therapy
Orna Therapeutics Presents New Preclinical Data Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases at the American Society of Gene and Cell Therapy Annual Meeting
orna therapeutics
2025-05-15
cell therapy
WATERTOWN, Mass., May 15, 2025 – Orna Therapeutics, a leading biotechnology company developing a proprietary pipeline of in vivo therapies across a broad range of autoimmune and oncology indications, today announced the presentation of new preclinical data supporting its in vivo CAR therapy approach in autoimmune diseases during an oral session at the 28th American… The post Orna Therapeutics Presents New Preclinical Data Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases at the American Society of Gene and Cell Therapy Annual Meeting appeared first on Orna Therapeutics .
Atara Biotherapeutics Announces First Quarter Financial Results and Operational Progress
Atara Biotherapeutics
2025-05-15
financial results
Nkarta Reports First Quarter 2025 Financial Results and Corporate Highlights
nkarta
2025-05-14
open
lupus
nkx019
financial results
myasthenia gravis
Enrollment open in investigator-sponsored trial (IST) of NKX019 in myasthenia gravis; Ntrust-1, Ntrust-2, and IST of NKX019 in systemic lupus erythematosus remain open to enrollment Ntrust-1 expanded to include primary membranous nephropathy cohort Lymphodepletion regimen modified across platform
Exegenesis Bio to Present Data on Next Generation BBB-penetrating AAV Capsids & Intravitreal nAMD Gene Therapy at the American Society of Gene and Cell Therapy Meeting
Exegenesis Bio
2025-05-14
aav
cell therapy
gene therapy
PHILADELPHIA, PA, May 13, 2025 – Exegenesis Bio, a glob […]
Kalaris Reports First Quarter 2025 Financial Results and Provides Business Highlights | Wed, 05/14/2025
Kalaris Therapeutics
2025-05-14
financial results
Actively enrolling nAMD patients in a Phase 1 trial of TH103, a novel, differentiated anti-VEGF agent engineered to potentially provide longer-lasting and increased anti-VEGF activity to treat neovascular and exudative diseases of the retina; initial clinical data is expected in Q4 2025 Cash and
Benitec Biopharma Releases Third Quarter 2025 Financial Results
benitec biopharma
2025-05-14
financial results
Beam Therapeutics to Highlight New Data from BEAM-101 Program in Sickle Cell Disease at European Hematology Association (EHA) 2025 Congress
beam therapeutics
2025-05-14
phase 1/2
sickle cell disease
Presentation to Include Updated Data from 17 Sickle Cell Disease Patients in the Ongoing BEACON Phase 1/2 Clinical Trial Evaluating Safety and Efficacy of BEAM-101 Beam to Host Investor Webcast on Friday, June 13, 2025, at 4:00 p.m. ET CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Beam
IN8bio Presents Preclinical Data Highlighting Potential of INB-619 T Cell Engager (TCE) for Autoimmune Disease at ASGCT 2025
In8Bio
2025-05-14
lupus
Powerful B cell Targeting : INB-619 precisely targets and eliminates B cells from lupus patient samples, including harmful IgG1 and IgM antibodies, demonstrating potential as a treatment for autoimmune diseases Innovative Next Generation TCE Platform: INB-619 gamma-delta (γδ) TCE platform not only
Forge Biologics Announces AAV Development and cGMP Manufacturing Relationship with Fractyl Health to Advance Their Gene Therapy Platform for Patients with Metabolic Diseases
Forge Biologics
2025-05-14
aav
gene therapy
Fractyl Health will leverage Forge’s FUEL™ platform to manufacture AAV for Rejuva, Fractyl's pancreatic gene therapy platform for patients with obesity and type 2 diabetes. The post Forge Biologics Announces AAV Development and cGMP Manufacturing Relationship with Fractyl Health to Advance Their Gene Therapy Platform for Patients with Metabolic Diseases appeared first on Forge Biologics .
MeiraGTx Announces the Presentation of Four Posters at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting
MeiraGTx
2025-05-13
cell therapy
Multiple Poster Presentations Highlight the Depth and Novelty of MeiraGTx’s Technology Platforms for Gene and Cell Therapy LONDON and NEW YORK, May 13, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced
Passage Bio Reports First Quarter 2025 Financial Results and Provides Recent Business Highlights
passage bio
2025-05-13
financial results
Adaptimmune Reports Q1 Financial Results and Provides Business Update
Adaptimmune Therapeutics
2025-05-13
bla
open
pivotal
sarcoma
tecelra
solid tumor
cell therapy
cell therapies
synovial sarcoma
financial results
TECELRA ® launch 2025 YTD metrics: 28 ATCs available; 21 patients aphresed; and 14 doses invoiced $4.0 million Tecelra net sales in Q1 2025 Instituting 2025 Tecelra full year sales guidance of $35-$45 million Lete-cel on track to initiate rolling BLA submission late 2025; approval anticipated in 2026 Adaptimmune had Total Liquidity 1 of $60 million as of March 31, 2025; call at 8 a.m. EDT today Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - May 13, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company redefining the treatment of solid tumor cancers with cell therapy, today reported financial results and provided a business update for the first quarter ended March 31, 2025. Adrian Rawcliffe, Adaptimmune's Chief Executive Officer: "The launch of Tecelra continues to rapidly accelerate, as evidenced by all launch metrics. In Q4 2024 we invoiced 2 patients, in Q1 2025 we invoiced 6 patients, and in Q2 to-date we have invoiced 8 patients. The shape of the pipeline of patients being tested and apheresed continues to support a robust acceleration of sales in Q2 and the second half of the year, and we continue to experience 100% manufacturing success…
Immusoft to Present Data at ASGCT 2025, Demonstrating Positive Results from the World's First Engineered B Cell in a Human Clinical Trial
Immusoft Corporation
2025-05-13
cell therapy
/PRNewswire/ -- Immusoft of CA, a cell therapy company dedicated to improving the lives of patients with diseases modifiable with therapeutic proteins, today...
Orchard Therapeutics Announces New England Journal of Medicine Publication of Long-term Clinical Outcomes from its HSC Gene Therapy for MLD and Multiple Presentations at ASGCT 2025
orchard therapeutics
2025-05-13
lenmeldy
gene therapy
LENMELDY™ is the first and only disease-modifying intravenous infusion proven to extend life expectancy in pre-symptomatic late infantile (PSLI) patients and mitigate the cognitive and/or physical impact of early-onset MLD Six accepted abstracts at ASGCT 2025 detail the therapeutic potential of HSC
Kyverna Therapeutics Provides Business Update and Reports First Quarter 2025 Financial Results
Kyverna Therapeutics
2025-05-13
bla
kyv-101
financial results
myasthenia gravis
stiff person syndrome
Enrollment complete in registrational Phase 2 KYV-101 trial in stiff person syndrome (SPS); on track for topline data in 1H 2026; biologics license application (BLA) filing now anticipated in 1H 2026 Advancing into registrational Phase 3 KYV-101 trial in myasthenia gravis (MG) following successful
Affinia Therapeutics Presents New Data on its Lead AFTX-201 Program in BAG3 Dilated Cardiomyopathy, BBB-Penetrant AAV Capsids, and Proprietary Manufacturing Technology at the 28th American Society of Gene & Cell Therapy 2025 Annual Meeting
Affinia Therapeutics
2025-05-13
aav
cell therapy
cardiomyopathy
Affinia Therapeutics Appoints Hideo Makimura, M.D., Ph.D., as Chief Medical Officer The post Affinia Therapeutics Presents New Data on its Lead AFTX-201 Program in BAG3 Dilated Cardiomyopathy, BBB-Penetrant AAV Capsids, and Proprietary Manufacturing Technology at the 28th American Society of Gene & Cell Therapy 2025 Annual Meeting appeared first on AffiniaTX .
IN8bio Presents Data at ISCT 2025 Demonstrating Proprietary γδ T cell Manufacturing Generates Consistent and Robust Clinical Products
In8Bio
2025-05-12
aml
cell therapies
gamma-delta t cell
Company receives ISCT abstract award for the analytical characterization of its manufactured gamma-delta T cell therapies in the INB-100 trial, which has demonstrated durable, relapse-free survival in AML patients. IN8bio’s proprietary gamma-delta T cell manufacturing platform generates robust and
ProQR Announces Upcoming Scientific Presentations at ASGCT and TIDES Conferences
proqr therapeutics
2025-05-12
cell therapy
LEIDEN, Netherlands & CAMBRIDGE, Mass., May 12, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that it will present at two upcoming scientific conferences: the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, taking place May 13-17, 2025, in New Orleans, Louisiana and the TIDES USA 2025: Oligonucleotide and Peptide Therapeutics Conference, being held May 19-22, 2025, in San Diego, California.
Cellectis Reports Financial Results for the First Quarter 2025
cellectis
2025-05-12
b-all
car t
leukemia
allogeneic
base editing
gene editing
gene therapy
gene therapies
hodgkin lymphoma
financial results
in vivo gene therapy
non-hodgkin lymphoma
hematological malignancies
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
Lasme-cel (UCART22) Phase 1 dataset and late-stage development strategy expected in the third quarter of 2025 Eti-cel (UCART20x22) Phase 1 study in relapsed or refractory B-cell non-Hodgkin lymphoma (r/r NHL) ongoing with readout expected in late 2025 AstraZeneca partnership: R&D activities ongoing on three programs – one allogeneic CAR T for hematological malignancies, one allogeneic CAR T for solid tumors, and one in vivo gene therapy for a genetic disorder Cellectis will present novel non-viral gene editing and base editing research at the 2025 ASGCT annual meeting Cash position of $246 million as of March 31, 2025 [1] provides runway into H2 2027 New York, NY – May 12, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, today provided financial results for the first quarter 2025, ending March 31, 2025, and provided a business update. « We are making progress in our wholly-owned clinical studies and in the three programs under our strategic partnership with AstraZeneca. We will continue to focus our efforts and resources on…
Coave Therapeutics Launches coAAV-CSF-01, a Novel Brain-Targeted Gene Therapy Vector for Neurodegenerative and other CNS Diseases
coave therapeutics
2025-05-09
gene therapy
Celularity Announces Full Year 2024 Operating and Financial Results
celularity
2025-05-09
financial results
Turnstone Biologics Corp. Reports First Quarter 2025 Financial Results and Provides Recent Corporate Highlights
Turnstone Bio
2025-05-08
financial results
SAN DIEGO, May 08, 2025 (GLOBE NEWSWIRE) -- Turnstone Biologics Corp. (“Turnstone” or the “Company”) (Nasdaq-CM: TSBX) , a biotechnology company historically focused on the development of a differentiated approach to treat and cure patients with solid tumors by pioneering selected
ProQR Announces First Quarter 2025 Operating and Financial Results
proqr therapeutics
2025-05-08
financial results
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Strengthened leadership with appointments of Chief Financial Officer and Chief Medical Officer € 132.4 million cash and cash equivalents as of end Q1 providing runway into mid-2027, plus additional potential milestones from Lilly partnership LEIDEN, Netherlands & CAMBRIDGE, Mass., May 08, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform, today reported its financial and operating results for the first quarter ended March 31, 2025, and provided a business update.
AskBio Announces 15 Presentations at American Society of Gene and Cell Therapy 28th Annual Meeting
askbio
2025-05-08
aav
cell therapy
gene editing
gene therapy
heart failure
pompe disease
gene therapies
adeno-associated virus
multiple system atrophy
Research Triangle Park, N.C.– MAY 8, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will deliver 15 presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, which takes place May 13–17, 2025, in New Orleans, Louisiana, USA. The company’s oral and poster presentations will provide updates on key research and development activities in gene therapy and gene editing, as well as advancements in manufacturing capabilities. Featured oral presentations will include “AI Use for Promoter and Transgene Design.” This will be delivered by Mansuo Shannon, Chief Scientific Officer at AskBio, as part of the Novel Approaches to Overcome Limits of Therapeutic Transgene Delivery and Durability scientific session. The presentation will explore how AI/ML (Artificial Intelligence/Machine Learning) may enhance efficacy, safety, and specificity of gene therapies by potentially predicting the best possible genetic sequences for the intended therapeutic outcome. In addition, AskBio’s Srinethe Saravanakumar will present “Vector Assembly Factories in Recombinant Adeno-Associated Virus Type 2 Producing…
Caribou Biosciences Reports First Quarter 2025 Financial Results and Provides Business Update
caribou biosciences
2025-05-08
financial results
Two robust clinical datasets from CB-010 and CB-011 expected to be disclosed in H2 2025 -- -- $212.5 million in cash, cash equivalents, and marketable securities expected to fund the Company’s current operating plan into H2 2027 -- BERKELEY, Calif., May 08, 2025 (GLOBE NEWSWIRE) -- Caribou
Axogen, Inc. Reports 2025 First Quarter Financial Results
axogen
2025-05-08
financial results
Community-Powered Awareness Takes Center Stage: Bionews at WODC 2025
BioNews
2025-05-07
orphan drug
Bionews wrapped up an energizing week at the World Orphan Drug Congress 2025 in Boston, where we had the opportunity to share the evolution of how we engage rare disease communities online. The presentation, led by Marcella Debidda, PhD, and Ethan Ash, senior VP of Business Development, spotlighted Bionews’ new approach to rare community growth: […] The post Community-Powered Awareness Takes Center Stage: Bionews at WODC 2025 appeared first on Bionews .
IN8bio Reports First Quarter 2025 Financial Results and Recent Business Highlights
In8Bio
2025-05-07
aml
leukemia
financial results
acute myeloid leukemia
Phase 1 clinical data of INB-100 continues to demonstrate long-term durable remissions, with 100% of treated Acute Myeloid Leukemia (AML) patients remaining relapse-free with median follow-up of 20.1 months as of January 17, 2025, as presented at the 2025 Transplantation & Cellular Therapy (TCT)
Cryoport Reports First Quarter 2025 Financial Results
Cryoport Inc
2025-05-07
financial results
Adicet Bio Reports First Quarter 2025 Financial Results and Provides Business Updates
Adicet Bio
2025-05-07
open
lupus
financial results
Two programs with clinical data readouts in 2H/2025 Phase 1 clinical trial of ADI-001 in autoimmune diseases ongoing with preliminary clinical data expected in 2H/2025; Trial now open for enrollment to patients with lupus nephritis (LN) and systemic lupus erythematosus (SLE) Preliminary data for
Adaptimmune to Report Q1 2025 Financial and Business Updates on Tuesday, May 13, 2025
Adaptimmune Therapeutics
2025-05-07
open
quarterly
solid tumor
cell therapy
cell therapies
financial results
Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - May 7, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company redefining the treatment of solid tumor cancers with cell therapy, will report financial results and provide business updates for the first quarter of 2025, before the US markets open on Tuesday, May 13, 2025. Following the announcement, the Company will host a live webcast at 8:00 a.m. EDT that same day. The press release will be available in the investor section of Adaptimmune's corporate website . A live webcast and replay can be accessed at https://www.gowebcasting.com/14032 . Call in information is as follows: 1-833-821-0158 (US or Canada) or 1-647-846-2266 (International). Callers should dial in 5-10 minutes prior to the scheduled start time and simply ask to join the Adaptimmune call. About Adaptimmune Adaptimmune is a clinical-stage biopharmaceutical company focused on designing, developing, and delivering cell therapies to transform the lives of people with cancer. The Company's unique engineered T-cell receptor (TCR) platform enables the engineering of T-cells to target and destroy cancers across multiple solid tumor types.…
Forge Biologics Announces Eight Presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting
Forge Biologics
2025-05-06
aav
cell therapy
Oral and poster presentations will highlight data demonstrating the enhanced productivity, recovery, and consistent quality of Forge’s FUEL™ AAV manufacturing platform The post Forge Biologics Announces Eight Presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting appeared first on Forge Biologics .
MiNK Therapeutics to Provide Corporate Update and First Quarter 2025 Financial Report
Mink Therapeutics
2025-05-06
allogeneic
cell therapies
NEW YORK , May 05, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering the discovery, development, and commercialization of allogeneic, off-the-shelf, invariant natural killer T (iNKT) cell therapies to treat cancer and other
Beacon Therapeutics Announces Positive Phase 2 Interim 6-Month Data from DAWN Trial of Laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) at ARVO 2025
Beacon Therapeutics
2025-05-06
x-linked
retinitis pigmentosa
x-linked retinitis pigmentosa
The post Beacon Therapeutics Announces Positive Phase 2 Interim 6-Month Data from DAWN Trial of Laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) at ARVO 2025 appeared first on Beacon Therapeutics .
Cellectis to Report First Quarter Financial Results on May 12, 2025
cellectis
2025-05-06
gene therapies
financial results
New York, NY – May 6, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS- NASD AQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the first quarter 2025 ending March 31, 2025 on Monday, May 12, 2025 after the close of the US market. The press release will be available in the Investors section of Cellectis’ website: https://www.cellectis.com/en/investors/press-releases/ Cellectis will not host a conference call to discuss these results. Our investors relations team remains available for questions at investors@cellectis.com
Actinium Announces Enrollment of First Patient in the Iomab-ACT Commercial CAR-T Trial at the University of Texas Southwestern Medical Center
Actinium Pharma
2025-05-06
cd19
car t
car-t
icans
multiple myeloma
Initial clinical data expected in the second half of 2025 from this first-of-its-kind trial to administer a targeted radiotherapy conditioning agent with a commercial CAR-T therapy - Iomab-ACT supported by results of NIH funded trial with MSK showing effective lymphodepletion of targeted immune cells resulting in negligible rates of CAR-T toxicities ICANS and CRS and CAR T-cell persistence with a novel CD19 CAR-T therapy - Iomab-ACT has the potential to increase the addressable market for CAR-T therapies, which generated $4 billion in sales in 2024, by enabling improved access and better patient outcomes compared to current chemotherapy conditioning agents NEW YORK , May 6, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today announced that the first patient was enrolled on the trial studying Iomab-ACT targeted conditioning with a commercial CAR-T therapy at the University of Texas Southwestern Medical Center (UTSW) ( NCT06768905 ). Initial clinical data from this trial is expected in the second half of 2025. Actinium is developing Iomab-ACT as a targeted radiotherapy…
Caribou Biosciences to Participate in the BofA Securities 2025 Health Care Conference
caribou biosciences
2025-05-06
crispr
BERKELEY, Calif., May 06, 2025 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today announced Rachel Haurwitz, PhD, Caribou’s president and chief executive officer, will participate in a fireside chat at the
Sangamo Therapeutics Announces Important Derisking Milestones in Pathway to Anticipated BLA Submission for ST-920 in Fabry Disease
Sangamo BioSciences
2025-05-06
bla
st-920
fabry disease
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Atara Biotherapeutics Provides Regulatory Updates on EBVALLO™ (tabelecleucel)
Atara Biotherapeutics
2025-05-05
ebvallo
tabelecleucel
As financial prospects for gene therapies dim, an ambitious charity picks up the torch
Fondazione Telethon
2025-05-05
gene therapies
Despite the immense potential of gene therapies, drugmakers have soured on the field. So an ambitious charity is trying to step in.
Orna Therapeutics Announces Presentation at the American Society of Gene and Cell Therapy Annual Meeting Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases
orna therapeutics
2025-05-05
cell therapy
WATERTOWN, Mass., May 5, 2025 – Orna Therapeutics, a biotechnology company dedicated to engineering immune cells in vivo to treat autoimmune and oncology diseases, today announced an upcoming presentation at the 28th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting being held May 13-17, 2025, in New Orleans, Louisiana. The presentation will highlight… The post Orna Therapeutics Announces Presentation at the American Society of Gene and Cell Therapy Annual Meeting Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases appeared first on Orna Therapeutics .
Adicet Bio Reports Inducement Grants under Nasdaq Listing Rule 5635(c)(4)
Adicet Bio
2025-05-01
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Apr. 30, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced it granted inducement awards on April 30,
Adicet Bio to Participate in a Fireside Chat at the Citizens JMP Life Sciences Conference
Adicet Bio
2025-04-30
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Apr. 30, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced that Chen Schor , President and Chief
Medigene AG Has Filed for Insolvency – No Extraordinary Shareholder Meeting According to §92 AktG
medigene
2025-04-29
tcr
Planegg/Martinsried (pta039/29.04.2025/14:30 UTC+2) Planegg/Martinsried. April 29, 2025. Medigene AG (Medigene, WKN: A40ESG, symbol: MDG1), an oncology platform company focused on the research and development of T cell receptor (TCR)-guided therapies for the treatment of cancer, following the ad hoc announcement on April 7, 2025, has filed an application for the opening of insolvency proceedings with ... Read more The post Medigene AG Has Filed for Insolvency – No Extraordinary Shareholder Meeting According to §92 AktG appeared first on Medigene AG .
Atsena Therapeutics to Present at May Scientific Conferences
atsena therapeutics
2025-04-29
gene therapy
Atsena Therapeutics to Present at May Scientific Conferences DURHAM, NC, April 29, 2025 – Atsena Therapeutics, a clinical-stage gene therapy company focused on using the ... The post Atsena Therapeutics to Present at May Scientific Conferences appeared first on Atsena Therapeutics .
Sangamo Therapeutics to Present Neurology Pipeline Advances at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
Sangamo BioSciences
2025-04-29
cell therapy
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
IN8bio to Present Clinical and Preclinical Data from Gamma-Delta T Cell Platform at Upcoming Scientific Conferences
In8Bio
2025-04-29
cell therapies
gamma-delta t cell
NEW YORK, April 29, 2025 (GLOBE NEWSWIRE) -- IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies for cancer and autoimmune diseases, today announced that it will present new clinical and preclinical data from its pipeline of
Be Biopharma to Present at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting
be biopharma
2025-04-28
cell therapy
CAMBRIDGE, Mass. April 28, 2025 – Be Biopharma, Inc. (“Be Bio”), a clinical-stage company pioneering the discovery and development of engineered B Cell Medicines (BCMs), today announced that it will present at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting being held in New Orleans, May 13-17, 2025. Details regarding […] The post Be Biopharma to Present at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting appeared first on Be Biopharma .
Affinia Therapeutics to Present New Data on its AFTX-201 Program in BAG3 Dilated Cardiomyopathy, BBB-Penetrant AAV Capsids, and Proprietary Manufacturing Technology at the 28th Annual American Society of Gene & Cell Therapy 2025 Annual Meeting
Affinia Therapeutics
2025-04-28
aav
cell therapy
cardiomyopathy
Affinia Therapeutics Appoints Hideo Makimura, M.D., Ph.D., as Chief Medical Officer The post Affinia Therapeutics to Present New Data on its AFTX-201 Program in BAG3 Dilated Cardiomyopathy, BBB-Penetrant AAV Capsids, and Proprietary Manufacturing Technology at the 28th Annual American Society of Gene & Cell Therapy 2025 Annual Meeting appeared first on AffiniaTX .
Actinium Presents Data Showing ATNM-400 is More Efficacious than Pluvicto and is Highly Efficacious after Pluvicto Resistance in Prostate Cancer Tumor Models at the American Association for Cancer Research Annual Meeting
Actinium Pharma
2025-04-28
psma
prostate cancer
Results highlight ATNM-400's potential as a transformative therapeutic option for prostate cancer patients with unmet clinical needs as expression of the target receptor for ATNM-400 persists following Pluvicto therapy and ATNM-400 demonstrates sustained tumor control after Pluvicto stops working - ATNM-400 showed greater tumor growth inhibition compared to Pluvicto in prostate cancer models with 99.8% tumor growth inhibition achieved with a single 40 µCi/kg dose of ATNM-400 demonstrating the potential to be offered as an alternative option - ATNM-400 was well tolerated with no apparent toxicities at two different dose levels, with efficient clearance from essential organs NEW YORK , April 28, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today announced preclinical results with ATNM-400 in prostate cancer models presented at the American Association for Cancer Research (AACR) Annual Meeting. ATNM-400 is a novel, non-PSMA targeting, first in class targeted radiotherapy utilizing the Actinium-225 (Ac-225) radioisotope that Actinium is evaluating in prostate cancer models…
Cellectis Presents Non-Viral Gene Editing and Base Editing Innovation at the ASGCT Annual Meeting
cellectis
2025-04-28
aav
talen
lentiviral
base editing
cell therapy
gene editing
gene therapy
gene therapies
New York, NY – April 28, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today unveils research data on TALEN®-mediated non-viral transgene insertion for advancing cellular and gene therapies, and advancements in genetic editing using base editors (TALEB), at the American Society of Gene and Cell Therapy (ASGCT) annual meeting, that will be held on May 13-17, 2025 in New Orleans. The data are presented in two posters: Title: TALEN®- Mediated non-viral Transgene Insertion for the Advancement of Cellular and Gene Therapies. Cell and gene therapy approaches can use gene-editing tools and introduce transgenes to modify disease-associated genes, thus providing a potential therapeutic solution for a wide array of diseases. In this work, Cellectis combines TALEN ®-mediated gene editing with non-viral delivery of transgene for advancing cellular and gene therapies, and explores gene insertion-efficacy and cellular health using single-stranded DNA (ssDNA) for payload delivery in different cell types. This innovative approach has the…
Adicet Bio Announces Oral Presentation Highlighting Preclinical ADI-270 Data at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting
Adicet Bio
2025-04-28
allogeneic
cell therapy
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Apr. 28, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced the acceptance of an abstract for an oral
Actinium Highlights Mutation Agnostic Antileukemic Activity of Actimab-A Against FLT3, NPM1, KMT2A and TP53 Mutations in AML Models Demonstrating Backbone Potential for Acute Myeloid Leukemia Treatment at the American Association for Cancer Research Annual Meeting
Actinium Pharma
2025-04-28
aml
cd33
pivotal
leukemia
phase 2/3
acute myeloid leukemia
Actimab-A significantly potentiated and prolonged efficacy in combination with standard of care targeted therapies including hypomethylating agent azacitidine, FLT3 and menin inhibitors in in-vivo AML models Actimab-A is the only CD33 targeted radiotherapy with Actinium-225 isotope payload in development for AML and other myeloid malignancies Actimab-A is currently being advanced in several combination studies including a Phase 2/3 trial in combination with CLAG-M in relapsed/refractory AML and in combination with Venetoclax and ASTX-727, an oral hypomethylating agent from Taiho Oncology in frontline AML under a CRADA with the NCI NEW YORK , April 28, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today highlighted data presented at the American Association for Cancer Research (AACR) Annual Meeting supporting Actimab-A's mutation agnostic antileukemic effect and backbone therapy potential in preclinical acute myeloid leukemia (AML) models. The preclinical data demonstrate that the combination of Actimab-A with standard of care AML therapies including menin and FLT3…
Combination Immunotherapy Approved for Unresectable or Metastatic Hepatocellular Carcinoma
ASCO Post FDA
2025-04-25
hepatocellular carcinoma
On April 11, 2025, the U.S. Food and Drug Administration (FDA) approved nivolumab (Opdivo) with ipilimumab (Yervoy) for the first-line treatment of adults with unresectable or metastatic hepatocellul...
FDA Approves Nivolumab With Ipilimumab for Unresectable or Metastatic MSI-H/dMMR Colorectal Cancer
ASCO Post FDA
2025-04-25
colorectal cancer
The FDA approved the combination of a PD-1 inhibitor and a CTLA-4 inhibitor for patients with unresectable or metastatic microsatellite instability–high or mismatch repair–deficient colorectal cancer ...
FDA Expands Indication for Lu-177 Vipivotide Tetraxetan in mCRPC
ASCO Post FDA
2025-04-25
psma
prostate cancer
The indication for lutetium Lu-177 vipivotide tetraxetan (Pluvicto) was expanded by the FDA to include adults with PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) who have been t...
AbbVie Reports First-Quarter 2025 Financial Results
Abbvie Investors
2025-04-25
financial results
Reports First-Quarter Diluted EPS of $0.72 on a GAAP Basis, a Decrease of 6.5 Percent; Adjusted Diluted EPS of $2.46 , an Increase of 6.5 Percent; These Results Include an Unfavorable Impact of $0.13 Per Share Related to Acquired IPR&D and Milestones Expense Delivers First-Quarter Net Revenues
Axogen, Inc. to report 2025 first quarter financial results on May 8, 2025
axogen
2025-04-24
financial results
Beacon Therapeutics Announces ARVO 2025 Presentation on Preliminary 6-Month Data from its Phase 2 DAWN Trial of laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP)
Beacon Therapeutics
2025-04-24
x-linked
retinitis pigmentosa
x-linked retinitis pigmentosa
The post Beacon Therapeutics Announces ARVO 2025 Presentation on Preliminary 6-Month Data from its Phase 2 DAWN Trial of laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) appeared first on Beacon Therapeutics .
Ray Therapeutics Awarded $8M CIRM Grant to Advance RTx-015 Gene Therapy for Retinitis Pigmentosa
ray therapeutics
2025-04-24
gene therapy
retinitis pigmentosa
Funding will support ongoing clinical development of optogenetic therapy to restore vision.
U.S. FDA approves second drug product manufacturing facility for Adstiladrin® (nadofaragene firadenovec-vncg)
Ferring Pharmaceuticals
2025-04-24
adstiladrin
[spb_boxed_content type=”coloured” box_link_target=”_self” padding_vertical=”0″ padding_horizontal=”0″ width=”1/1″ el_position=”first last”] U.S. FDA approves second drug product manufacturing facility for Adstiladrin® (nadofaragene firadenovec-vncg) [/spb_boxed_content] [spb_blank_spacer height=”30px” width=”1/1″ el_position=”first last”] [spb_text_block animation=”none” animation_delay=”0″ simplified_controls=”yes” custom_css_percentage=”no” padding_vertical=”0″ padding_horizontal=”0″ margin_vertical=”0″ custom_css=”margin-top: 0px;margin-bottom: 0px;” border_size=”0″ border_styling_global=”default” el_class=”fixme-a” width=”1/1″ el_position=”first last”] Ferring expands production of ADSTILADRIN with new Parsippany, NJ, facility, adding to existing manufacturing sites in Kuopio, […]
Metachromatic Leukodystrophy (MLD) and gene therapy: a game-changing treatment backed by NEJM—Timing Is Everything
Fondazione Telethon
2025-04-24
gene therapy
metachromatic leukodystrophy
If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic origin which leads to the progressive loss of the ability to walk, talk and interact. This was confirmed by a study published in the New England Journal of Medicine which showed that the therapy, if administered early, is able to preserve motor function and cognitive abilities in most patients..The study was conducted at Ospedale San Raffaele in Milan and is the result of more than 20 years of research by the San Raffaele-Telethon Institute for Gene Therapy and the commitment of Fondazione Telethon to transforming research into therapies available for patients.
Aurion Biotech Appoints Edward J. Holland, M.D., as Chief Medical Officer and Eris P. Jordan, O.D., as Chief Development Officer
aurion biotech
2025-04-23
cell therapy
corneal endothelial disease
Key leadership appointments to accelerate global clinical development of AURN001, a transformational cell therapy for corneal endothelial disease Seattle, WA; Cambridge, MA; Tokyo, Japan – 23 April – Aurion Biotech, a clinical-stage regenerative medicine company whose mission is to restore vision to millions of patients, today announced the appointments of Edward J. Holland, M.D., as Chief […]
SparingVision Receives FDA Support to Initiate Phase II trial of SPVN06 in Geographic Atrophy Secondary to Dry-AMD
Sparing Vision
2025-04-22
gene therapy
retinitis pigmentosa
SparingVision Receives FDA Support to Initiate Phase II trial of SPVN06 in Geographic Atrophy Secondary to Dry-AMD Following a positive Type C meeting, the lead gene therapy program will expand beyond retinitis pigmentosa (RP) into geographic atrophy (GA) Advancement of SPVN06 into a second indication is supported by robust clinical safety and preclinical data… The post SparingVision Receives FDA Support to Initiate Phase II trial of SPVN06 in Geographic Atrophy Secondary to Dry-AMD appeared first on SparingVision .
FDA grants Breakthrough Therapy Designation for BrainChild Bio’s B7-H3 CAR T-cell therapy for incurable pediatric brain tumors Read More
Brainchild Bio
2025-04-22
b7-h3
car t
cell therapy
breakthrough therapy
Ferring Announces Initial Data from Phase 3 Trial in Japanese Patients Demonstrating 75% Complete Response Rate at 3 Months with (nadofaragene firadenovec) in BCG-unresponsive NMIBC patients
Ferring Pharmaceuticals
2025-04-21
complete response
[spb_boxed_content type=”coloured” box_link_target=”_self” padding_vertical=”0″ padding_horizontal=”0″ width=”1/1″ el_position=”first last”] Ferring Announces Initial Data from Phase 3 Trial in Japanese Patients Demonstrating 75% Complete Response Rate at 3 Months with (nadofaragene firadenovec) in BCG-unresponsive NMIBC patients1 [/spb_boxed_content] [spb_blank_spacer height=”30px” width=”1/1″ el_position=”first last”] [spb_boxed_content type=”coloured” box_link_target=”_self” padding_vertical=”0″ padding_horizontal=”0″ el_class=”puces-item” width=”1/1″ el_position=”first last”] First results from a Phase 3 trial conducted across 25 centers […]
HuidaGene Selected for Presidential Symposium at ASGCT 2025 Annual Meeting, Showcasing 13 Abstracts Demonstrating Breakthroughs in Gene Therapy
Huidagene Therapeutics Co.
2025-04-19
gene therapy
Blenrep (belantamab mafodotin) combinations approved by UK MHRA in relapsed/refractory multiple myeloma
GSK
2025-04-17
multiple myeloma
Superior efficacy shown in two head-to-head phase III trials, including overall survival in DREAMM-7.
Atsena Therapeutics Granted U.S. FDA Regenerative Medicine Advanced Therapy Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis
atsena therapeutics
2025-04-15
x-linked
gene therapy
x-linked retinoschisis
Atsena Therapeutics Granted U.S. FDA Regenerative Medicine Advanced Therapy Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis Marks fourth FDA designation for ATSN-201, which ... The post Atsena Therapeutics Granted U.S. FDA Regenerative Medicine Advanced Therapy Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis appeared first on Atsena Therapeutics .
HuidaGene at CRISPR MEDiCiNE 2025: A Celebration of Progress, Promise, and Patients
Huidagene Therapeutics Co.
2025-04-12
crispr
HuidaGene Announces Broader Adoption of hfCas12Max Through Sublicensing Agreement to Advance Clinical Cell Therapy Programs
Huidagene Therapeutics Co.
2025-04-10
cell therapy
SparingVision to Highlight Progress of its Gene Therapy Programs at ARVO and ASGCT 2025
Sparing Vision
2025-04-10
gene therapy
SparingVision to Highlight Progress of its Gene Therapy Programs at ARVO and ASGCT 2025 Paris, 10 April 2025 – SparingVision (“the Company”), a clinical-stage genomic medicine company transforming the treatment of retinal disease, announces that it will present a safety update from the ongoing Phase II PRODYGY trial of its lead product candidate, SPVN06, in… The post SparingVision to Highlight Progress of its Gene Therapy Programs at ARVO and ASGCT 2025 appeared first on SparingVision .
Menstrual Cycle May Contribute to SCD Pain Crises
Hematology.org
2025-04-09
scd
Menstrual Cycle May Contribute to SCD Pain Crises
Ferring achieved record sales driven by Reproductive Medicine combined with the emergence of Adstiladrin
Ferring Pharmaceuticals
2025-04-09
adstiladrin
bladder cancer
[spb_boxed_content type=”coloured” box_link_target=”_self” padding_vertical=”0″ padding_horizontal=”0″ width=”1/1″ el_position=”first last”] Ferring achieved record sales driven by Reproductive Medicine combined with the emergence of Adstiladrin [/spb_boxed_content] [spb_blank_spacer height=”30px” width=”1/1″ el_position=”first last”] [spb_boxed_content type=”coloured” box_link_target=”_self” padding_vertical=”0″ padding_horizontal=”0″ width=”1/1″ el_position=”first last”] Continued strong performance in Reproductive Medicine which exceeded sales of €1 billion for the first time Adstiladrin® (nadofaragene firadenovec-vncg), our novel gene-based therapy for bladder cancer, […]
AskBio Elects Dr. Laura Sepp-Lorenzino Independent Member of Board of Directors
askbio
2025-04-08
crispr
gene therapy
gene therapies
Research Triangle Park, N.C. April 8, 2025 – AskBio Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced today that its Board of Directors has elected Independent Director Laura Sepp-Lorenzino, PhD, to the Board, effective April 4, 2025. “We are pleased to welcome Dr. Sepp-Lorenzino to our Board of Directors,” said Gustavo Pesquin, CEO, AskBio. “Laura’s extensive experience as a scientist and adviser will support AskBio’s efforts to stay at the cutting-edge of innovation. She brings to our Board broad expertise across modalities and therapeutic areas, including gene therapy and editing, from discovery through late-stage development. Her contributions will be invaluable as we move our clinical programs through and beyond Phase 2 and advance our next pre-clinical candidates.” Dr. Sepp-Lorenzino brings to AskBio’s Board over 30 years of executive and academic experience and leadership skills. She currently serves as Scientific Advisor, Executive Vice President, at Intellia Therapeutics, where she is helping develop and execute Intellia’s strategic vision and plan, positioning the company as the leader in systemic CRISPR medicines. She is also the…
Atamyo finalise l’étude de choix de dose dans la myopathie des ceintures de type R9 (LGMD-R9) et obtient la désignation de maladie pédiatrique rare de la FDA américaine pour ATA-100
atamyo therapeutics
2025-04-03
lgmd
Evry, France (April 3, 2025) –- Atamyo Therapeutics, société de biotechnologie au stade clinique spécialisée sur le développement de thérapies géniques de nouvelle génération ciblant les dystrophies musculaires, a annoncé aujourd’hui avoir complété la phase […]
Atamyo completes the dose-finding study in Limb-Girdle Muscular Dystrophy Type R9 (LGMD-R9) and obtains US Rare Pediatric Disease Designation for ATA-100
atamyo therapeutics
2025-04-03
lgmd
gene therapies
Evry, France (April 3, 2025) –Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies, announced the completion of the dose-escalation phase of its Phase 1b/2b clinical […]
Novartis receives FDA accelerated approval for Vanrafia® (atrasentan), the first and only selective endothelin A receptor antagonist for proteinuria reduction in primary IgA nephropathy (IgAN)
Novartis
2025-04-03
accelerated approval
Atsena Therapeutics Announces Oversubscribed $150 Million Series C Financing to Further Advance Ocular Gene Therapy Programs
atsena therapeutics
2025-04-02
gene therapy
Atsena Therapeutics Announces Oversubscribed $150 Million Series C Financing to Further Advance Ocular Gene Therapy Programs Financing led by new investor Bain Capital with participation ... The post Atsena Therapeutics Announces Oversubscribed $150 Million Series C Financing to Further Advance Ocular Gene Therapy Programs appeared first on Atsena Therapeutics .
HuidaGene Therapeutics to Present at Multiple Key Conferences Highlighting Advances in CRISPR-based Therapies
Huidagene Therapeutics Co.
2025-04-02
crispr
Nkarta to Participate in an April Investor Conference
nkarta
2025-04-01
cell therapies
SOUTH SAN FRANCISCO, Calif., April 01, 2025 (GLOBE NEWSWIRE) -- Nkarta, Inc. (Nasdaq: NKTX), a biopharmaceutical company developing engineered natural killer (NK) cell therapies, today announced its participation in the following investor conference: 24th Annual Needham Virtual Healthcare
Orsini Selected as the Exclusive Specialty Pharmacy for Cell Therapy ENCELTO™
Neurotech Pharmaceuticals
2025-04-01
encelto
cell therapy
/PRNewswire/ -- Orsini, a leader in rare disease pharmacy solutions, has been selected by Neurotech Pharmaceuticals, Inc., as the exclusive specialty pharmacy...
Adicet Bio Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Adicet Bio
2025-04-01
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Mar. 31, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced it granted inducement awards on March 31,
Vertex Cell Therapy Misses in the Clinic, But It Has Other Shots on the Type 1 Diabetes Goal
CellTrans Inc.
2025-03-31
phase 1/2
cell therapy
Vertex Pharmaceuticals is stopping development of VX-264, an experimental cell therapy/medical device treatment for type 1 diabetes, after an early look at Phase 1/2 data fell short of efficacy goals. This program came from a $950 million acquisition in 2019 that marked Vertex’s entry into type 1 diabetes.
Vertex Ends One Type 1 Diabetes Cell Therapy, Shifts Focus To Another
CellTrans Inc.
2025-03-31
cell therapy
Seeking cell therapy approaches to cure type 1 diabetes, Vertex abandons a candidate encapsulated to avoid immune system detection but hopes to file another candidate for approval in 2026.
Adicet Bio to Participate in 2025 Canaccord Genuity Horizons in Oncology Virtual Conference
Adicet Bio
2025-03-31
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Mar. 31, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced that Chen Schor , President and Chief
AbbVie to Host First-Quarter 2025 Earnings Conference Call
Abbvie Investors
2025-03-31
financial results
NORTH CHICAGO, Ill. , March 31, 2025 /PRNewswire/ -- AbbVie (NYSE: ABBV) will announce its first-quarter 2025 financial results on Friday, April 25, 2025 , before the market opens. AbbVie will host a live webcast of the earnings conference call at 8 a.m. Central time .
Turnstone Biologics Corp. Reports Fourth Quarter and Full Year 2024 Financial Results
Turnstone Bio
2025-03-29
financial results
SAN DIEGO, March 28, 2025 (GLOBE NEWSWIRE) -- Turnstone Biologics Corp. (“Turnstone” or the “Company”) (Nasdaq: TSBX), a biotechnology company historically focused on the development of a differentiated approach to treat and cure patients with solid tumors by pioneering selected tumor-infiltrating
FDA approves Novartis radioligand therapy Pluvicto® for earlier use before chemotherapy in PSMA-positive metastatic castration-resistant prostate cancer
Novartis
2025-03-28
psma
prostate cancer
Actinium Pharmaceuticals Provides Business Update After Presentation at Trump Mar-A-Lago Club Announcing Novel Non-PSMA Prostate Cancer Radiotherapy ATNM-400 and Outlining Revitalized Clinical Pipeline with 2025 Corporate Objectives
Actinium Pharma
2025-03-27
aml
scd
psma
car-t
lung cancer
solid tumor
gene therapy
prostate cancer
sickle cell disease
ATNM-400 is a novel, non-PSMA targeting, first-in-class Actinium-225 radiotherapy for prostate cancer with initial preclinical results to be presented at the AACR Annual Meeting - Actinium is establishing radiopharmaceutical manufacturing infrastructure in 2025 to support expanding clinical trials and to leverage its proprietary Actinium-225 cyclotron manufacturing technology - Data from several clinical trials expected in 2H:2025 across myeloid malignancies, solid tumors and cell and gene therapy conditioning - Actinium's targeted radiotherapy programs expanded to four multi-billion-dollar market opportunities NEW YORK , March 27, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today provided a business update detailing recent achievements and anticipated milestones from its revitalized and expanding clinical pipeline. The update featured the unveiling of ATNM-400, Actinium's new Actinium-225 (Ac-225) solid tumor program, which is a novel, non-PSMA targeting, first-in-class radiotherapy for prostate cancer. Initial preclinical data from ATNM-400 will be presented at AACR…
Kyverna Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2024 Financial Results
Kyverna Therapeutics
2025-03-27
lupus
financial results
myasthenia gravis
stiff person syndrome
Accelerating clinical path to commercialization in stiff person syndrome, myasthenia gravis and lupus nephritis Aligned with the U.S. Food and Drug Administration (FDA) on a registrational Phase 2 trial design for ongoing KYSA-8 trial in stiff person syndrome; 70% of study enrolled with topline
Actinium Pharmaceuticals Announces ATNM-400 a Novel Non-PSMA Targeting First in Class Prostate Cancer Radiotherapy Leveraging Actinium-225
Actinium Pharma
2025-03-27
psma
prostate cancer
99.8% tumor growth inhibition achieved with a single dose of ATNM-400 in preclinical prostate cancer models - ATNM-400 accumulated in tumors for up to 144 hours and showed minimal uptake in healthy tissues in prostate cancer xenograft model - Initial ATNM-400 preclinical data to be presented at AACR including results in Pluvicto resistant prostate cancer models NEW YORK , March 27, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today announced ATNM-400, a novel, non-PSMA targeting, first in class radiotherapy for prostate cancer utilizing the Actinium-225 (Ac-225) radioisotope. Initial preclinical data from ATNM-400 will be presented at the American Association for Cancer Research (AACR) Annual Meeting being held on April 25 - 30, 2025, in Chicago, IL. The ATNM-400 AACR abstract will include the results from in vitro and in vivo studies including biodistribution imaging and efficacy analyses with various dose levels of ATNM-400. Actinium continues to advance ATNM-400 with additional data expected from Pluvicto-resistant prostate cancer models at AACR. Pluvicto…
Actinium Pharmaceuticals to Present Business Update at Trump Mar-A-Lago Club Today
Actinium Pharma
2025-03-26
aml
car-t
leukemia
phase 2/3
allogeneic
lung cancer
solid tumor
gene therapy
gene therapies
myelodysplastic
sickle cell disease
acute myeloid leukemia
Company to highlight recent significant progress made with its Actimab-A and Iomab-ACT clinical programs, leading-edge R&D and radiopharmaceutical manufacturing infrastructure - Revitalized clinical programs focused on 3 separate multi-billion-dollar market opportunities in myeloid malignancies, solid tumors and cell & gene therapy conditioning with clinical data expected in 2025 supporting each addressable market - Presentation follows Investor KOL Event and Company Update on March 25 th NEW YORK , March 26, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, announced it will be presenting a business update today at Trump Mar-a-Lago Club, Florida. This presentation follows an Investor KOL Call and Company Update hosted by Actinium on Tuesday, March 25 th highlighting its revitalized clinical programs and expanded market opportunities. Actinium's Investor KOL Call and Company Update can be accessed for replay here . Sandesh Seth, Actinium's Chairman and CEO, said, "We are honored to have this opportunity to present Actinium Pharmaceuticals and highlight the significant…
Nkarta Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Highlights
nkarta
2025-03-26
nkx019
financial results
Differentiated development program includes two Nkarta clinical trials and two investigator-sponsored trials of NKX019 in rheumatic and neurological diseases Initial data for NKX019 in multiple autoimmune indications expected in second half of 2025 Restructuring and workforce reduction of 34% (53
Actinium Pharmaceuticals to Host KOL Investor Call at 8am ET Today to Highlight Revamped Clinical Programs and Expanded Market Opportunities Including Newly Initiated Actimab-A Solid Tumor Program
Actinium Pharma
2025-03-25
aml
cd33
car-t
pivotal
leukemia
phase 2/3
lung cancer
solid tumor
gene therapy
gene therapies
myelodysplastic
acute myeloid leukemia
Actinium will present 3 separate multi-billion-dollar market opportunities for Actimab-A and Iomab-ACT in myeloid malignancies, solid tumors and cell & gene therapy conditioning - Dr. Ehab Atallah, Professor of Medicine at the Medical College of Wisconsin to highlight Actimab-A clinical results, mutation agnostic mechanism and backbone therapy potential - Clinical proof of concept data from frontline AML trial under NCI CRADA, Actimab-A solid tumor program with PD-1 checkpoint inhibitors and Iomab-ACT commercial CAR-T trial together with other expected 2025 milestones to be outlined - Actinium to highlight manufacturing infrastructure development activity to support revitalized clinical programs NEW YORK , March 25, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, will host an investor call at 8:00 am ET today that will feature KOL Dr. Ehab Atallah, Professor of Medicine at the Medical College of Wisconsin and principal investigator of the Actimab-A + CLAG-M combination trial in patients with relapsed/refractory acute myeloid leukemia (r/r AML). Dr. Atallah will discuss…
Novartis to present new data at AAN, including seven-year disability outcomes and safety analysis of Kesimpta® in people with relapsing multiple sclerosis
Novartis
2025-03-25
multiple sclerosis
Actinium Pharmaceuticals Announces Supply Agreement with Eckert & Ziegler for Ac-225 Radioisotope to Support Comprehensive Development Activities
Actinium Pharma
2025-03-24
aml
cd33
pivotal
leukemia
phase 2/3
lung cancer
solid tumor
acute myeloid leukemia
NEW YORK , March 24, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today announced it has entered into an agreement for the supply of Actinium-225 (Ac-225) with Eckert & Ziegler. Under this agreement, Actinium Pharmaceuticals will have access to Eckert & Ziegler's high-quality Actinium-225 to further develop its lead product Actimab-A as well as additional early and late-stage development candidates for both U.S. and international clinical trials. Targeted radiotherapies using Ac-225 have shown great promise in the treatment of cancer. The radioisotope releases powerful alpha particles with high energy and low penetration depth, enabling precise targeting of tumor cells, including hard-to-reach micrometastases, while minimizing effects on surrounding healthy tissue. Actimab-A is an Ac-225 based radiotherapy agent, directed against CD33, a receptor overexpressed in patients with acute myeloid leukemia (AML) and other myeloid indications. Sandesh Seth, Chairman and CEO at Actium Pharmaceuticals, Inc. commented: "We believe that targeted radiation therapy with Actinium-225…
Engaging the Rare Disease Patient Online: Bionews Presents at World Orphan Drug Congress 2025
BioNews
2025-03-21
orphan drug
Bionews is excited to announce our participation in the World Orphan Drug Congress 2025. This year’s presentation delves into innovative strategies to engage rare disease patients online by exploring practical approaches to reaching these communities, delivering unmatched value, and sharing a taste of the “secret sauce” behind our organic community growth—all supported by data from […] The post Engaging the Rare Disease Patient Online: Bionews Presents at World Orphan Drug Congress 2025 appeared first on Bionews .
Wugen Announces Dosing of First Patients in Pivotal Trial of Off-the-Shelf, Allogeneic, CD7-Targeted CAR-T Cell Therapy WU-CART-007
Wugen Inc.
2025-03-20
cd7
car-t
pivotal
phase 1/2
allogeneic
cell therapy
Previously reported Phase 1/2 data on WU-CART-007 demonstrated clinically manageable safety and evidence of anti-leukemic activity with an overall response rate of 91% and composite complete remission rate of 73% in heavily pretreated patients with R/R T-ALL/LBL The global pivotal Phase 2 T-RRex Study will evaluate WU-CART-007 in pediatric and adult patients ST. LOUIS,... The post Wugen Announces Dosing of First Patients in Pivotal Trial of Off-the-Shelf, Allogeneic, CD7-Targeted CAR-T Cell Therapy WU-CART-007 appeared first on Wugen .
Adaptimmune Provides Q4 and Full Year 2024 Business Update
Adaptimmune Therapeutics
2025-03-20
bla
open
pivotal
sarcoma
tecelra
solid tumor
cell therapy
synovial sarcoma
TECELRA ® launch momentum accelerating – 10 patients apheresed in Q1 2025 and 3 in 2024; 20 ATCs available; all manufacturing and supply goals achieved Lete-cel on track to initiate rolling BLA submission late 2025; approval anticipated 2026 Corporate restructure completed in February 2025 and implementing additional cost reduction for PRAME and CD70 programs At the end of 2024, Adaptimmune had Total Liquidity 1 of $152 million Evaluating all strategic options to maximize shareholder value Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - March 20, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company redefining the treatment of solid tumor cancers with cell therapy, today provided a Q4 and Full Year 2024 business update. Adrian Rawcliffe, Adaptimmune's Chief Executive Officer: "2025 is the year of commercial execution for Tecelra, as we begin to generate value from our promising sarcoma franchise. The launch is going very well and each step in delivering this important medicine: activating our treatment network; identifying biomarker eligible patients; exceptional manufacturing quality and turn-around times, strengthens our sarcoma platform…
Actinium Pharmaceuticals Announces Research Collaboration with Memorial Sloan Kettering to Support Further Clinical Expansion of Actimab-A's Backbone Therapy Strategy
Actinium Pharma
2025-03-20
aml
cd33
pivotal
leukemia
phase 2/3
NEW YORK , March 20, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today announced that is has entered into a sponsored research agreement with Memorial Sloan Kettering Cancer Center (MSKCC) to expand Actimab-A's mutation agnostic mechanism of action. The collaboration has two specific objectives. The first objective is to study Actimab-A in combination with targeted therapies including FLT3 and menin inhibitors to define transcriptional profiles in AML cells following treatment with these combinations. The second objective is to assess the activity of Actimab-A-based combinations on primary AML patient-derived ex vivo samples from patients with and without prior Venetoclax + hypomethylating agent (HMA) treatment. The collaboration will be done with members of MSKCC's leukemia and early drug development specialist teams. Actimab-A has demonstrated a mutation agnostic profile with positive clinical results in high-risk relapsed and refractory (r/r) AML patients including those with a TP53 gene mutation, prior Venetoclax treatment and prior bone marrow transplant (BMT).…
MiNK Therapeutics Reports Fourth Quarter & Full Year 2024 Results and Highlights Business Progress
Mink Therapeutics
2025-03-19
allogeneic
cell therapies
financial results
NEW YORK , March 18, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering the development of allogeneic, off-the-shelf invariant natural killer T (iNKT) cell therapies, today announced its financial results for the fourth quarter and
Actinium Pharmaceuticals to Host Investor KOL Call with Dr. Ehab Atallah of the Medical College of Wisconsin and Provide a Pipeline Update Highlighting Revamped Clinical Programs and Expanded Market Opportunities for Actimab-A and Iomab-ACT with Clinical Data in 2H:2025
Actinium Pharma
2025-03-19
aml
car-t
pivotal
leukemia
phase 2/3
solid tumor
gene therapy
gene therapies
myelodysplastic
sickle cell disease
acute myeloid leukemia
Actinium to highlight 3 separate multi-billion-dollar market opportunities for Actimab-A and Iomab-ACT in myeloid malignancies, solid tumors and cell & gene therapy conditioning - Clinical proof of concept data expected in 2H:2025 from Actimab-A combination with blockbuster immunotherapies KEYTRUDA ® and OPDIVO ® in solid tumor indications - Actimab-A expanded into frontline AML in triplet combination with Venetoclax and ASTX-727, Taiho Oncology's hypomethylating agent, under NCI CRADA with initial clinical data expected by year-end 2025 - Planned pivotal Phase 2/3 trial for Actimab-A + CLAG-M in relapsed/refractory AML patients further supported by recent publication of results in peer-reviewed journal Leukemia reporting 18.4 median overall survival - Iomab-ACT commercial CAR-T trial to initiate patient enrollment with clinical proof of concept data expected in 2H:2025; Iomab-ACT sickle cell disease trial expected to initiate in 1H:2025 - Call Scheduled for 8:00 AM ET on Tuesday, March 25, 2025 NEW YORK , March 19, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today…
Sangamo Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2024 Financial Results
Sangamo BioSciences
2025-03-18
financial results
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Actinium Pharmaceuticals Announces Clinical Trial Program in Solid Tumors Combining Actimab-A with PD-1 Checkpoint Inhibitors KEYTRUDA® and OPDIVO®
Actinium Pharma
2025-03-18
aml
cd33
open
leukemia
solid tumor
acute myeloid leukemia
Trials are designed to demonstrate whether the addition of Actimab-A to either KEYTRUDA ® or OPDIVO ® can result in improved patient outcomes - MDSCs – Myeloid Derived Suppressor Cells in the tumor microenvironment are believed to reduce effectiveness of PD-1 inhibitors like KEYTRUDA ® and OPDIVO ® - Trials supported by preclinical data showing Actimab-A can selectively target and deplete MDSCs which express CD33 - Clinical proof of concept data expected in 2025 could potentially open up a multi-billion-dollar market opportunity for Actimab-A as a combination therapy with PD-1 inhibitors in multiple solid tumors NEW YORK , March 18, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today announced a clinical program comprising of trials studying Actimab-A in combination with either KEYTRUDA ® (pembrolizumab) or OPDIVO ® (nivolumab) which are blockbuster immunotherapies known as PD-1 inhibitors which are approved in multiple solid tumor indications. KEYTRUDA ® developed and commercialized by Merck & Co. and OPDIVO ® developed and commercialized by Bristol Myers Squibb,…
From Research to Reality: Overcoming CMC Challenges in Cell & Gene Therapy
landmark bio
2025-03-18
gene therapy
Translating an R&D asset into a human therapeutic is a risk-filled endeavor that can be complicated by a wide range of development and manufacturing challenges. This webinar reviews the importance of working alongside a partner that has experience developing a CMC strategy, which is critical when shifting from a research mindset to a commercial one. Watch the full webinar The post From Research to Reality: Overcoming CMC Challenges in Cell & Gene Therapy first appeared on Landmark Bio .
ChromaTan and Landmark Bio Awarded National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) Grant to Advance Intensified Recombinant Adeno-Associated Virus (AAV) Production Process
landmark bio
2025-03-17
aav
gene therapy
adeno-associated virus
Grant allows ChromaTan and Landmark Bio to create a scalable, cost-effective, and accessible platform for the manufacturing and characterization of AAV-based gene therapy vectors. Project developed with award from NIIMBL and financial assistance from the National Institute of Standards and Technology. The post ChromaTan and Landmark Bio Awarded National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) Grant to Advance Intensified Recombinant Adeno-Associated Virus (AAV) Production Process first appeared on Landmark Bio .
Tourmaline Bio Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Business Highlights
Talaris Therapeutics
2025-03-13
financial results
– Phase 2 TRANQUILITY trial in patients with elevated high-sensitivity C-reactive protein and chronic kidney disease over-enrolled to 143 total participants, on track to report topline data in the second quarter of 2025 – – Cardiovascular Scientific Advisory Board strengthened with recent
Medigene Files Patent for Unique NK-Specific TCR Construct with European Patent Office
medigene
2025-03-13
tcr
Planegg/Martinsried (pta026/13.03.2025/14:30 UTC+1) Medigene AG (Medigene, FSE: MDG1, Prime Standard), an oncology platform company focused on the research and development of T cell receptor (TCR)-guided therapies for the treatment of cancer, today announced the submission of a patent for a novel natural killer (NK) cell-specific TCR construct to the European Patent Office. With that, Medigene ... Read more The post Medigene Files Patent for Unique NK-Specific TCR Construct with European Patent Office appeared first on Medigene AG .
MeiraGTx Enters into a Strategic Collaboration with Hologen AI to Expedite Phase 3 Development of AAV-GAD for Parkinson’s Disease and Industrialize MeiraGTx’s Proprietary Manufacturing Process
MeiraGTx
2025-03-13
aav
MeiraGTx to receive $200 million in upfront cash consideration - MeiraGTx and Hologen will form a joint venture, Hologen Neuro AI Ltd, with an additional $230 million committed capital from Hologen to fund 100% of the development of AAV-GAD for Parkinson’s disease through to commercialization, as
Cellectis Reports Financial Results for the Fourth Quarter and Full Year 2024 and Provides a Business Update
cellectis
2025-03-13
car t
allogeneic
orphan drug
gene editing
gene therapy
gene therapies
hodgkin lymphoma
financial results
in vivo gene therapy
non-hodgkin lymphoma
hematological malignancies
UCART22 Phase 1 dataset and late-stage development strategy expected in the third quarter of 2025; Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) granted by FDA and ODD granted by the European Commission to UCART22 for the treatment of ALL. UCART20x22 Phase 1 study in relapsed or refractory B-cell non-Hodgkin lymphoma (r/r NHL) ongoing with readout expected in late 2025. AstraZeneca partnership: R&D activities ongoing on three programs – one allogeneic CAR T for hematological malignancies, one allogeneic CAR T for solid tumors, and one in vivo gene therapy for a genetic disorder . Cash position of $264 million as of December 31, 2024 [1] provides runway into mid-2027. Conference call March 14, 2025 at 8:00 am ET / 1:00 pm CET . [1] Cash position includes cash, cash equivalents, restricted cash and fixed-term deposits classified as current -financial assets. Restricted cash was $4.6 million as of December 31, 2024. Fixed-term deposits classified as current-financial assets was $115.8 million as of December 31, 2024. New York, NY – March 13, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company…
Adaptimmune to Report Full Year and Q4 2024 Financial and Business Updates on Thursday, March 20, 2025
Adaptimmune Therapeutics
2025-03-12
open
quarterly
solid tumor
cell therapy
cell therapies
financial results
Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - March 12, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company redefining the treatment of solid tumor cancers with cell therapy, will report financial results and provide business updates for the fourth quarter and full year ended December 31, 2024, before the US markets open on Thursday, March 20, 2025. Following the announcement, the Company will host a live webcast at 8:00 a.m. EDT that same day. The press release will be available in the investor section of Adaptimmune's corporate website . A live webcast and replay can be accessed at https://www.gowebcasting.com/13966 . Call in information is as follows: 1-833-821-0158 (US or Canada) or 1-647-846-2266 (International). Callers should dial in 5-10 minutes prior to the scheduled start time and simply ask to join the Adaptimmune call. About Adaptimmune Adaptimmune is a clinical-stage biopharmaceutical company focused on designing, developing, and delivering cell therapies to transform the lives of people with cancer. The Company's unique engineered T-cell receptor (TCR) platform enables the engineering of T-cells to target and destroy cancers…
Fondazione Telethon submits BLA for etuvetidigene autotemcel
Fondazione Telethon
2025-03-12
bla
gene therapy
wiskott-aldrich
etuvetidigene autotemcel
wiskott-aldrich syndrome
Italian biomedical charity Fondazione Telethon, which is focused on rare genetic diseases, revealed it has submitted a Biologics License Application (BLA) for the gene therapy - etuvetidigene autotemcel – as a treatment of patients with Wiskott-Aldrich syndrome (WAS), a rare genetic disease of the immune system, to the US Food and Drug Administration (FDA).
Atsena Therapeutics Granted U.S. FDA Fast Track Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis
atsena therapeutics
2025-03-12
x-linked
fast track
gene therapy
x-linked retinoschisis
Atsena Therapeutics Granted U.S. FDA Fast Track Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis Marks third FDA designation for ATSN-201, which has also ... The post Atsena Therapeutics Granted U.S. FDA Fast Track Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis appeared first on Atsena Therapeutics .
Neurotech nabs FDA approval to treat rare eye disorder with cell therapy implant Encelto
Neurotech Pharmaceuticals
2025-03-10
encelto
cell therapy
macular telangiectasia type 2
With a U.S. green light for its encapsulated cell therapy technology in the bag, privately held biotech Neurotech Pharmaceuticals is on the verge of making its commercial debut. | The FDA last week approved Neurotech’s cell therapy implant revakinagene taroretcel to treat the vision-robbing neurodegenerative disease macular telangiectasia type 2. The therapy, which will adopt the brand name Encelto, is the first to win the FDA’s blessing in the rare vision disorder.
Luxa Biotechnology Presents Clinical Data from First-in-Human Retinal Pigment Epithelium Cell Therapy Trial for Dry Age-Related Macular Degeneration at the 77th Annual Wills Eye Conference
Luxa Biotechnology, LLC
2025-03-09
cell therapy
dry age-related macular degeneration
AskBio Advances Gene Therapy Clinical Trial for Limb-Girdle Muscular Dystrophy Type 2I/R9 with Dosing of First Participant in Second Cohort
askbio
2025-03-07
aav
lgmd
fast track
gene therapy
Research Triangle Park, N.C. – March 7, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the advancement of the Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) with dosing of the first participant in the second cohort. The recommendation to advance to the second cohort followed the completion of a Data Safety Monitoring Board (DSMB) review of trial recruitment activity, and safety reporting from the first cohort that determined it was safe to proceed to cohort two. DSMBs are independent groups of experts appointed to periodically review information from clinical trials. These reviews typically include risk-benefit assessments and monitoring for serious or unexpected adverse safety events alongside any significant benefit of therapies.1 The LION-CS101 clinical trial is a double-blind, randomized, placebo-controlled, dose-escalation clinical trial to evaluate the safety of AB-1003 gene therapy in adult participants (18–65 years) who have genetic confirmation of LGMD2I/R9. The trial includes…
RI biotech wins FDA approval for MacTel treatment
Neurotech Pharmaceuticals
2025-03-07
cell therapy
The tiny eye insert delivers cell therapy to slow progression of the rare disease
Acepodia Appoints Nobel Laureate Dr. Carolyn Bertozzi to Its Board of Directors-Acepodia® | POWERFUL, ACCESSIBLE CELL THERAPIES FOR PATIENTS WITH CANCER
Acepodia
2025-03-07
cell therapies
ALAMEDA, Calif. & TAIPE, March 7, 2025 — Acepodia (6976: TT) today announced that Dr. Carolyn Bertozzi, Nobel Laureate in Chemistry, has been elected to the company’s Board of Directors through an Extraordinary Shareholders' Meeting. Dr. Bertozzi's appointment will further strengthen Acepodia's leadership in the fields of biochemistry, chemical biology, and innovative drug developmen..
Cellectis to Report Fourth Quarter and Full Year 2024 Financial Results on March 13, 2025
cellectis
2025-03-07
gene therapies
financial results
New York, NY – March 7, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the fourth quarter and full year 2024 ending December 31, 2024 on Thursday, March 13, 2025 after the close of the US market. The publication will be followed by an investor conference call and webcast on Friday, March 14, 2025, at 8:00 AM ET / 1:00 PM CET. The call will include the Company’s fourth quarter and full year 2024 results and an update on business activities. Details for the call are as follows: Dial in information: Domestic: +1-800-225-9448 International: +1-203-518-9708 Conference ID: CLLS24 Webcast Link: https://viavid.webcasts.com/starthere.jsp?ei=1706599&tp_key=4524940f8f
Neurotech’s ENCELTO™ (revakinagene taroretcel-lwey) Approved by the FDA for the Treatment of Adults with Idiopathic Macular Telangiectasia Type 2 (MacTel)
Neurotech Pharmaceuticals
2025-03-07
encelto
macular telangiectasia type 2
Atara Biotherapeutics Announces Fourth Quarter and Full Year 2024 Financial Results and Operational Progress
Atara Biotherapeutics
2025-03-07
financial results
Adicet Bio Reports Fourth Quarter and Full Year 2024 Financial Results and Highlights Recent Company Progress
Adicet Bio
2025-03-07
lupus
financial results
Phase 1 clinical trial of ADI-001 in autoimmune diseases ongoing; on track to report preliminary clinical data from lupus nephritis (LN) patient cohort in 1H25 Additional LN clinical data and preliminary clinical data from other autoimmune patient cohorts anticipated in 2H25 Initiation of ADI-001
Neurotech’s ENCELTO™ (revakinagene taroretcel-lwey) Approved by the FDA for the Treatment of Macular Telangiectasia Type 2 (MacTel)
Neurotech Pharmaceuticals
2025-03-06
encelto
macular telangiectasia type 2
Neurotech Pharmaceuticals, Inc., a private biotech company focused on developing transformative therapies for chronic eye diseases, announced that the U.S. F...
Luxa's stem cell therapy for dry AMD shows early promise
Luxa Biotechnology, LLC
2025-03-06
cell therapy
Neurotech’s ENCELTO™ (revakinagene taroretcel-lwey) Approved by the FDA for the Treatment of Macular Telangiectasia Type 2 (MacTel)
Neurotech Pharmaceuticals
2025-03-06
encelto
macular telangiectasia type 2
CUMBERLAND, R.I., March 06, 2025--Neurotech Pharmaceuticals, Inc., a private biotech company focused on developing transformative therapies for chronic eye diseases, announced that the U.S. Food and Drug Administration (FDA), has approved ENCELTO™ (revakinagene taroretcel-lwey) for the treatment of Macular Telangiectasia type 2 (MacTel).
MiNK Therapeutics to Provide Corporate Update and Fourth Quarter & Full Year 2024 Financial Report
Mink Therapeutics
2025-03-05
allogeneic
cell therapies
NEW YORK , March 04, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics , Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering the discovery, development, and commercialization of allogeneic, off-the-shelf, invariant natural killer T (iNKT) cell therapies to treat cancer and other
Anova Announces First Patient Enrolled to Phase 1/2a Study of DB107 for the Treatment of High-Grade Gliomas
anova evidence
2025-03-05
open
Anova has announced enrollment of the first two patients in the highly anticipated study of DB107 for the treatment of brain tumors. The Phase 1/2a clinical trial is a multicenter, open-label study designed to confirm whether treatment DB107, when added to standard-of-care (SOC), provides clinical benefit to patients with newly diagnosed high-grade gliomas (HGG) when compared to historical performance. The post Anova Announces First Patient Enrolled to Phase 1/2a Study of DB107 for the Treatment of High-Grade Gliomas appeared first on Anova .
Passage Bio Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Recent Business Highlights
passage bio
2025-03-04
financial results
Kyverna Therapeutics to Present at the Leerink Partners Global Biopharma Conference
Kyverna Therapeutics
2025-03-03
cell therapies
EMERYVILLE, Calif. , March 3, 2025 /PRNewswire/ -- Kyverna Therapeutics, Inc. (Kyverna, Nasdaq: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, today announced that Warner Biddle , Chief Executive Officer of Kyverna, will
Adicet Announces Poster Presentations Highlighting ADI-270 Data at the Society for Immunotherapy of Cancer (SITC) 2025 Spring Scientific Meeting
Adicet Bio
2025-03-03
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Mar. 3, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced the acceptance of two abstracts for poster
Be Biopharma Announces Appointment of Susan Abu-Absi, Ph.D. as Chief Operating Officer
be biopharma
2025-03-03
gene therapy
Dr. Abu-Absi Brings 20+ Years of Operational and Manufacturing Experience in Biologics and Cell & Gene Therapy CAMBRIDGE, Mass. March 3, 2025 – Be Biopharma, Inc. (“Be Bio”), a clinical-stage company pioneering the discovery and development of engineered B Cell Medicines (BCMs), today announced the appointment of Susan Abu-Absi, Ph.D., as Chief Operating Officer. With […] The post Be Biopharma Announces Appointment of Susan Abu-Absi, Ph.D. as Chief Operating Officer appeared first on Be Biopharma .
Adicet Bio Receives FDA Fast Track Designation for ADI-001 for the Treatment of Systemic Sclerosis (SSc)
Adicet Bio
2025-02-27
allogeneic
fast track
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Feb. 27, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced the U.S.
Genethon Celebrates Rare Disease Day by Highlighting New Technologies to Advance Gene Therapies and Bring Hope to Millions of Patients Worldwide
Genethon
2025-02-27
gene therapy
gene therapies
PARIS, FRANCE (February 27, 2025) – Genethon, a pioneering gene therapy research organization created by the AFM-Telethon, marks Rare Disease Day February 28, 2025, by highlighting recent technological breakthroughs signaling a next generation of safer, more effective and lower cost gene therapies to expand applications of these life-saving medicines and make them more accessible for … Continued
Medigene and EpimAb Biotherapeutics Enter into a Co-Development Partnership for TCR-Guided T Cell Engagers
medigene
2025-02-27
tcr
Planegg/Martinsried (pta007/27.02.2025/09:00 UTC+1) Planegg/Martinsried and Shanghai, February 27, 2025. Medigene AG (Medigene, FSE: MDG1, Prime Standard) and EpimAb Biotherapeutics, Inc. (EpimAb), announced that the companies have entered a strategic co-development agreement to research and develop off-the-shelf T cell receptor (TCR)-guided T Cell Engagers (TCR-TCEs) for the treatment of immune-related disorders, such as solid tumors. The ... Read more The post Medigene and EpimAb Biotherapeutics Enter into a Co-Development Partnership for TCR-Guided T Cell Engagers appeared first on Medigene AG .
Wugen Announces Appointment of Jim Faulkner, Ph.D., as Chief Technology Officer
Wugen Inc.
2025-02-26
allogeneic
solid tumor
cell therapies
ST. LOUIS, MO and SAN DIEGO, CA, Feb. 26, 2025 – Wugen, Inc., a clinical-stage U.S. biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological and solid tumor malignancies, today announced the appointment of Jim Faulkner, Ph.D., as chief technology officer, initially serving on an interim basis. “Jim brings a specialized skill set... The post Wugen Announces Appointment of Jim Faulkner, Ph.D., as Chief Technology Officer appeared first on Wugen .
Axogen, Inc. Reports 2024 Fourth Quarter and Full-Year Financial Results
axogen
2025-02-25
financial results
FDA Approves First Companion Diagnostic to Identify Patients With Metastatic Breast Cancer Eligible for T-DXd
ASCO Post FDA
2025-02-25
her2
breast cancer
The FDA has approved a label expansion for the PATHWAY anti-HER2/neu (4B5) Rabbit Monoclonal Primary Antibody to identify patients with HR-positive, HER2-ultralow metastatic breast cancer who may be e...
T-DXd Approved by the FDA for Pretreated Patients With HER2-Low or -Ultralow Metastatic Breast Cancer
ASCO Post FDA
2025-02-25
her2
breast cancer
The antibody-drug conjugate fam-trastuzumab deruxtecan-nxki (T-DXd) has been approved for the treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-low or HE...
FDA Approves Preparative Regimen for Allogeneic HSCT in Patients With AML or MDS
ASCO Post FDA
2025-02-25
aml
allogeneic
hematopoietic stem cell
The FDA recently approved treosulfan (Grafapex), an alkylating agent, with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in adult and pediatric pat...
FDA Approves Acalabrutinib With Bendamustine and Rituximab for Previously Untreated Mantle Cell Lymphoma
ASCO Post FDA
2025-02-25
mantle cell lymphoma
On January 16, the U.S. Food and Drug Administration (FDA) granted traditional approval to acalabrutinib (Calquence) with bendamustine and rituximab for adults with previously untreated mantle cell ly...
MiNK Therapeutics Presents First-in-Kind Allo-iNKTs Combination Data in 2L Gastric Cancer at AACR IO Annual Meeting
Mink Therapeutics
2025-02-25
gastric cancer
Addition of AgenT-797 with Checkpoint Inhibitors, BOT/BAL, and Chemotherapy Demonstrates Robust Immune Activation, Offering Potential to Overcome Refractory Gastric Cancers NEW YORK , Feb. 24, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical
Cellectis Presents ‘Smart CAR T’ Strategy to Enhance Efficacy Against Solid Tumors at AACR-IO 2025
cellectis
2025-02-24
fap
car t
car-t
talen
allogeneic
solid tumor
gene editing
cell therapies
gene therapies
hematological malignancies
New York, NY – February 24, 2025 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, showcases an innovative strategy for T cell engineering that leverages the pro-inflammatory properties of interleukin 2 (IL-2) with the objective to enhance CAR T cell efficacy against solid tumors, at the American Association for Cancer Research – Immuno-oncology (AACR-IO), taking place on February 23-26, 2025 in Los Angeles, CA. The data are presented in a poster: CAR induced expression of synthetically engineered FAP-IL2v immunocytokine boosts persistent anti-tumor activity of TALEN-edited allogeneic CAR T-cells without associated IL-2 toxicity Presenter: Shipra Das, Ph.D., Associate Director Immuno-Oncology, at Cellectis. Date/Time: February 25, 2025, 1:45-4:45 p.m. PT Session : Poster Session B CAR T-cell therapies have transformed the treatment landscape for specific hematological malignancies and have shown promising preliminary efficacy in solid tumors. Recent studies suggest a link between the in vivo expansion and persistence of CAR-T cells…
Celularity Enters Strategic Collaboration Agreement with Clinical Stage Cell Therapy Company
celularity
2025-02-24
cell therapy
MeiraGTx Announces The Lancet Publication of Data Demonstrating the Efficacy of rAAV8.hRKp.AIPL1 for the Treatment of Leber Congenital Amaurosis 4 (LCA4) Retinal Dystrophy
MeiraGTx
2025-02-21
leber congenital amaurosis
As disclosed in the paper, 4 out of 4 young children with the AIPL1-related retinal dystrophy, LCA4, benefited substantially from unilateral subretinal administration of rAAV8 .hRKp.AIPL1 with improved visual acuity, functional vision, and protection against progressive retinal degeneration
MiNK Therapeutics Regains Full Compliance with Nasdaq Listing Requirements
Mink Therapeutics
2025-02-21
allogeneic
cell therapies
NEW YORK , Feb. 20, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering the discovery, development, and commercialization of allogeneic, off-the-shelf invariant natural killer T (iNKT) cell therapies for cancer and other
Carisma Therapeutics to Participate in H.C. Wainwright 3rd Annual Cell Therapy Virtual Conference
carisma therapeutics
2025-02-19
cell therapy
PHILADELPHIA , Feb. 19, 2025 /PRNewswire/ -- Carisma Therapeutics Inc. (Nasdaq: CARM) ("Carisma" or the "Company"), a leader in macrophage-focused therapeutics, today announced that Michael Klichinsky , PharmD, PhD, Co-founder and Chief Scientific Officer, will participate in the H.C.
Luxa Biotechnology Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to RPESC-RPE-4W Transplantation for the Treatment of Dry Age-Related Macular Degeneration
Luxa Biotechnology, LLC
2025-02-19
rmat
dry age-related macular degeneration
Landmark Bio’s Mission to Prevent Cell Therapy ‘Do-overs’
landmark bio
2025-02-18
cell therapy
gene therapy
Drug Discovery and Development interviews Landmark Bio CEO Ran Zheng on the realities of large-scale cell and gene therapy manufacturing and the need to incorporate the requirements for GMP and commercial manufacturing early on The post Landmark Bio’s Mission to Prevent Cell Therapy ‘Do-overs’ first appeared on Landmark Bio .
MiNK Therapeutics to Present Updated Data from Phase 2 Study Testing AgenT-797 in Gastric Cancer at AACR IO Annual Meeting
Mink Therapeutics
2025-02-13
allogeneic
cell therapies
gastric cancer
NEW YORK , Feb. 12, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics , Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering the discovery, development, and commercialization of allogeneic, off-the-shelf, invariant natural killer T (iNKT) cell therapies to treat cancer and other
Orchard Therapeutics Appoints Robin Kenselaar to Chief Commercial Officer and Announces Multiple Business Updates Pertaining to its Approved HSC Gene Therapy for MLD
orchard therapeutics
2025-02-12
lenmeldy
gene therapy
First U.S. patients being treated with Lenmeldy™ (atidarsagene autotemcel) in a commercial setting; other launch efforts progressing well following Food and Drug Administration approval last year Reimbursement agreement for Libmeldy ® reached with Spanish National Health System enabling access to
Axogen, Inc. to report 2024 fourth quarter and full year financial results on February 25, 2025
axogen
2025-02-11
financial results
Adicet Bio Receives FDA Fast Track Designation for ADI-001 for the Treatment of Refractory Systemic Lupus Erythematosus (SLE) with Extrarenal Involvement
Adicet Bio
2025-02-05
lupus
allogeneic
fast track
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Feb. 5, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced the U.S.
Orchard Therapeutics Announces Multiple Data Presentations and Receives 2025 New Treatment Award at the 21st Annual WORLDSymposium™
orchard therapeutics
2025-02-03
gene therapy
Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic function in a variety of tissues and organs, including the CNS TOKYO, LONDON and BOSTON, Feb. 03, 2025 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a Kyowa Kirin company, today
A Phase 1/2 Study Evaluating the Safety and Efficacy of Autologous TAC-T Cells in Subjects with Claudin 18.2+ Advanced Solid Tumors
Triumvira Therapeutics
2025-01-31
phase 1/2
autologous
claudin 18.2
cell therapies
INTRODUCTION The T cell antigen coupler (TAC) is a novel, proprietary chimeric receptor that facilitates the re-direction of T cells to tumor cells and activates T cells by co-opting the endogenous T cell receptor complex, with the goal to elicit a safe and durable anti-tumor response. In preclinical models, TAC-engineered T cells effectively eradicate tumor […] The post A Phase 1/2 Study Evaluating the Safety and Efficacy of Autologous TAC-T Cells in Subjects with Claudin 18.2+ Advanced Solid Tumors appeared first on Triumvira Immunologics | Engineered T Cell Therapies .
Adaptimmune to Participate in the Guggenheim Securities SMID Cap Biotech Conference February 6, 2025
Adaptimmune Therapeutics
2025-01-30
quarterly
solid tumor
cell therapy
Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - January 30, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company working to redefine the treatment of solid tumor cancers with cell therapy, today announced that management will participate in the Guggenheim Securities SMID Cap Biotech Conference this February. Guggenheim Securities SMID Cap Biotech Conference, New York, NY Fireside chat with Adrian Rawcliffe, CEO: February 6 at 3:00 p.m. EST Webcast link: https://wsw.com/webcast/guggen2/adap/1969770 This event can also be accessed by visiting 'Events', under the 'News Center' tab, in the 'Investors & Media' section of Adaptimmune's website at www.adaptimmune.com . Following the event, a replay of the presentation will be made available. About Adaptimmune Adaptimmune is a fully integrated cell therapy company working to redefine how cancer is treated. With its unique engineered T cell receptor (TCR) platform, the Company is developing personalized medicines designed to target and destroy difficult-to-treat solid tumor cancers and to radically improve the patient's cancer treatment experience. Forward-Looking Statements This release contains…
Adicet Bio to Participate in a Fireside Chat at the Guggenheim Securities SMID Cap Biotech Conference
Adicet Bio
2025-01-29
allogeneic
cell therapies
gamma delta t cell
REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Jan. 29, 2025-- Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced that Chen Schor , President and Chief
Beacon Therapeutics Granted FDA Regenerative Medicine Advanced Therapy (RMAT)
Beacon Therapeutics
2025-01-28
rmat
The post Beacon Therapeutics Granted FDA Regenerative Medicine Advanced Therapy (RMAT) appeared first on Beacon Therapeutics .
Early detection is a game changer: Novartis makes bold play to prioritize breast health and inspire a new wave of breast cancer screenings
Novartis
2025-01-28
breast cancer
Immusoft to Present Positive Data from the First Engineered B Cell in a Human Clinical Trial at the 21st Annual WorldSymposium™ 2025
Immusoft Corporation
2025-01-27
cell therapy
/PRNewswire/ -- Immusoft Corporation, a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will...
Triumvira Immunologics Presents Updated Clinical Data at the 2025 ASCO Gastrointestinal Cancers Symposium
Triumvira Therapeutics
2025-01-27
allogeneic
autologous
cell therapies
AUSTIN, Texas and HAMILTON, ON and SAN DIEGO — /PRNewswire/ — Triumvira Immunologics, a clinical-stage company developing novel, targeted autologous and allogeneic T cell therapeutics that co-opt the natural biology of T cells to treat patients with solid tumors, recently presented an abstract at the 2025 American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium, which […] The post Triumvira Immunologics Presents Updated Clinical Data at the 2025 ASCO Gastrointestinal Cancers Symposium appeared first on Triumvira Immunologics | Engineered T Cell Therapies .
MiNK Therapeutics Targets Immune Reconstitution to Combat Advanced Gastroesophageal Cancers with Novel Combination of Allo-iNKTs plus BOT/BAL at ASCO GI
Mink Therapeutics
2025-01-24
allogeneic
cell therapies
NEW YORK , Jan. 23, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering the development of allogeneic, off-the-shelf invariant natural killer T (iNKT) cell therapies to treat cancer and other immune-mediated diseases, today
Genethon and Eukarÿs announce a strategic partnership to develop a breakthrough technology to reduce the biomanufacturing cost of gene therapies
Genethon
2025-01-23
gene therapy
gene therapies
Evry, January 23, 2025- Genethon, a pioneer and leader in the research and development of gene therapies for rare diseases, and Eukarÿs, a biotechnology company developing a disruptive technology that significantly increases biomanufacturing yields, today announced the signing of a strategic partnership. The collaboration aims to reduce the production costs of gene therapy medicines by … Continued
Cell Therapy Market Outlook 2034: Rapid Growth Projected with a 20.8% CAGR, Set to Reach US$ 44.6 Billion, Latest Report by TMR
CellTrans Inc.
2025-01-21
cell therapy
Global Cell Therapy market is driven by increasing demand for advanced therapies in oncology, regenerative medicine, and immunology. The market's expansion...
European Commission expands Jemperli (dostarlimab) plus chemotherapy approval to all adult patients with primary advanced or recurrent endometrial cancer
GSK
2025-01-20
endometrial cancer
Expanded approval includes MMRp/MSS tumours, which represent approximately 75% of endometrial cancer cases.
Immusoft Goes For Cell Therapy Plan ‘B’
Immusoft Corporation
2025-01-16
cell therapy
cell therapies
Emerging Company Profile: CEO Sean Ainsworth spoke with Scrip about how the company is developing B-cell therapies to treat a range of diseases, starting with MPS I.
Simnova and Orna Expand Strategic Partnership to Include BCMA-Targeted RNA Therapeutics
orna therapeutics
2025-01-15
bcma
cell therapy
SHANGHAI and CAMBRIDGE, Mass. , Jan. 15, 2025 /PRNewswire/ — Orna Therapeutics, Inc. (“Orna”) and Shanghai Simnova Biotech Co., Ltd. (“Simnova”) are pleased to announce the expansion of their strategic collaboration to include BCMA (B-cell maturation antigen) as a designated biological target for RNA-based therapeutics development. This partnership leverages Orna’s groundbreaking circular RNA (oRNA®) technology and Simnova’s expertise in cell therapy to deliver… The post Simnova and Orna Expand Strategic Partnership to Include BCMA-Targeted RNA Therapeutics appeared first on Orna Therapeutics .
ArsenalBio and Bristol Myers Squibb Achieve Milestone for AB-4000 Series as Part of Ongoing Multi-Program Collaboration to Advance Next-Generation T Cell Therapies for Solid Tumors
arsenal biosciences
2025-01-13
car t
cell therapy
cell therapies
Bristol Myers Squibb exercises an exclusive license option for ArsenalBio’s AB-4000 series programs ArsenalBio is eligible for additional milestone payments and royalties as the programs advance South San Francisco, Calif. – January 13, 2025 – Arsenal Biosciences, Inc. (ArsenalBio), a clinical-stage programmable cell therapy company focused on engineering advanced CAR T cell therapies for solid […]
Kyverna Therapeutics to Highlight Near-Term Strategic Priorities and Key Milestones at the 43rd Annual J.P. Morgan Healthcare Conference
Kyverna Therapeutics
2025-01-13
bla
car t
kyv-101
pivotal
stiff person syndrome
Extending Company's leadership position in autoimmune CAR T with prioritized indication strategy; pivoting to late-stage development and commercialization First-to-market opportunity with KYV-101 in stiff person syndrome; 40% enrolled in pivotal Phase 2 trial with first BLA filing targeted for
Adaptimmune Announces U.S. FDA Breakthrough Therapy Designation Granted to Letetresgene Autoleucel (lete-cel) for Treatment of Myxoid/Round Cell Liposarcoma (MRCLS)
Adaptimmune Therapeutics
2025-01-13
sarcoma
tecelra
ny-eso-1
liposarcoma
solid tumor
cell therapy
synovial sarcoma
breakthrough therapy
letetresgene autoleucel
afamitresgene autoleucel
The Company will present at the Annual J.P. Morgan Healthcare Conference, providing business updates on its sarcoma franchise and other cell therapy pipeline assets Adaptimmune Allo-T program to be featured at the Biotech ShowCase (TM) and the Wuxi Global Forum 2025 Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - January 13, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company working to redefine the treatment of solid tumor cancers with cell therapy, today announced that letetresgene autoleucel (lete-cel), has been granted breakthrough therapy designation by the U.S. FDA for the treatment of patients with unresectable or metastatic myxoid/round cell liposarcoma (MRCLS) who have received prior anthracycline-based chemotherapy, are positive for HLA-A*02:01, HLA-A*02:05, or HLA-A*02:06, and whose tumor expresses the NY-ESO-1 antigen. More details about the Company's sarcoma franchise, including the lete-cel clinical program and launch progress for TECELRA ® (afamitresgene autoleucel), the Company's first commercial product and the first FDA approved engineered cell therapy for a solid tumor, will be provided during the Company's presentation at the…
A2 Biotherapeutics Closes $80 Million Series C Financing to Advance Clinical Development of its Precision Cell Therapies Using its Proprietary TmodTM Technology Platform
A2 Bio
2025-01-09
cell therapies
The post A2 Biotherapeutics Closes $80 Million Series C Financing to Advance Clinical Development of its Precision Cell Therapies Using its Proprietary TmodTM Technology Platform appeared first on A2 Biotherapeutics .
Adicet Bio Provides Corporate Update and Highlights Expected 2025 Milestones
Adicet Bio
2025-01-09
lupus
Adicet made significant progress in 2024 and is well-positioned for success in 2025: Advancing Phase 1 clinical trial evaluating ADI-001 across six autoimmune diseases; preliminary data in lupus nephritis (LN) patients anticipated in 1H25, data from other patient cohorts expected in 2H25 Patient
Orna Therapeutics Appoints Vikas Sinha to its Board of Directors
orna therapeutics
2025-01-09
lnp
lipid nanoparticle
WATERTOWN, Mass., January 9, 2025 – Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of circular RNA medicines and unprecedented lipid nanoparticle (LNP) delivery solutions for oncology and autoimmune diseases, today announced the appointment of Vikas Sinha to its Board of Directors. “We are thrilled to welcome Vikas to our… The post Orna Therapeutics Appoints Vikas Sinha to its Board of Directors appeared first on Orna Therapeutics .
Atsena Therapeutics Initiates Part B of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis
atsena therapeutics
2025-01-08
x-linked
gene therapy
x-linked retinoschisis
Atsena Therapeutics Initiates Part B of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis Part B of the LIGHTHOUSE study will ... The post Atsena Therapeutics Initiates Part B of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis appeared first on Atsena Therapeutics .
Orna Therapeutics Establishes Strategic Collaboration with Vertex Pharmaceuticals to Develop Next Generation Approaches for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)
orna therapeutics
2025-01-07
lnp
scd
beta thalassemia
lipid nanoparticle
sickle cell disease
— Collaboration to leverage Orna’s differentiated lipid nanoparticle (LNP) delivery solutions for patients with SCD and TDT — — Orna to receive an upfront payment and is eligible to receive potential pre-clinical, clinical, and commercialization milestone payments and royalties — WATERTOWN, Mass., Jan. 7, 2025 /PRNewswire/ — Orna Therapeutics (through its wholly owned subsidiary ReNAgade Therapeutics Inc.)… The post Orna Therapeutics Establishes Strategic Collaboration with Vertex Pharmaceuticals to Develop Next Generation Approaches for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) appeared first on Orna Therapeutics .
Intracerebroventricular B7-H3-targeting CAR T cells for diffuse intrinsic pontine glioma: a phase 1 trial Read More
Brainchild Bio
2025-01-07
b7-h3
car t
glioma
diffuse intrinsic pontine glioma
BrainChild Bio, Inc. to clinically advance BCB-276, an autologous B7-H3 targeting CAR T-cell therapy for incurable pediatric brain tumors Read More
Brainchild Bio
2025-01-07
b7-h3
car t
autologous
cell therapy
GSK’s B7-H3-targeted antibody-drug conjugate, GSK’227, receives US FDA Breakthrough Therapy Designation in late-line relapsed or refractory osteosarcoma
GSK
2025-01-07
b7-h3
osteosarcoma
breakthrough therapy
Regulatory designation based on promising early data in this rare bone cancer.
ArsenalBio to Present at the 43rd Annual J.P. Morgan Healthcare Conference
arsenal biosciences
2025-01-06
car t
cell therapy
cell therapies
South San Francisco, Calif. – January 6, 2025 – Arsenal Biosciences, Inc. (ArsenalBio), a clinical-stage programmable cell therapy company focused on engineering advanced CAR T-cell therapies for solid tumors, today announced that Ken Drazan, M.D., ArsenalBio’s CEO and Chairman, will present at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January […]
Kyverna Therapeutics to Present at the 43rd Annual J. P. Morgan Healthcare Conference
Kyverna Therapeutics
2025-01-06
cell therapies
EMERYVILLE, Calif. , Jan. 6, 2025 /PRNewswire/ -- Kyverna Therapeutics, Inc. (Kyverna, NASDAQ: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimmune diseases, announced that the Company will participate at the 43rd Annual J.P.
Altaris Closes Acquisition of Minaris from Resonac
Altaris
2025-01-05
cell therapy
Altaris finalizes acquisition of Minaris, a pioneering CDMO in cell therapy with 500 plus employees and six global facilities.
Sangamo Therapeutics to Regain Full Rights to Hemophilia A Gene Therapy Program Following Pfizer’s Decision to Cease Development of Giroctocogene Fitelparvovec
Sangamo BioSciences
2024-12-30
gene therapy
hemophilia a
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Altaris to Acquire WuXi AppTec's Cell Therapy Business
Altaris
2024-12-24
cell therapy
Altaris to acquire U.S. and U.K. Advanced Therapies unit, boosting cell therapy manufacturing and process development capabilities.
PeproMene Bio, Inc. and the Institute for Follicular Lymphoma Innovation Announce $11M Investment to Advance PMB-CT01 (BAFF-R CAR T cell therapy) for Relapsed/Refractory Follicular Lymphoma
pepromene bio
2024-12-19
car t
cell therapy
follicular lymphoma
To find the article on the web: PeproMene Bio, Inc. and the Institute for Follicular Lymphoma Innovation Announce $11M Investment to Advance PMB-CT01 (BAFF-R CAR T cell therapy) for Relapsed/Refractory Follicular Lymphoma IRVINE, Calif., Dec. 19, 2024 /PRNewswire/ — PeproMene Bio, Inc. (PMB) … Continue reading → The post PeproMene Bio, Inc. and the Institute for Follicular Lymphoma Innovation Announce $11M Investment to Advance PMB-CT01 (BAFF-R CAR T cell therapy) for Relapsed/Refractory Follicular Lymphoma appeared first on PeproMene Bio Inc .
Aurion Biotech Announces Positive Topline Data for Phase 1/2 Clinical Trial of AURN001, an Allogeneic Cell Therapy Product Candidate for the Treatment of Corneal Edema Secondary to Corneal Endothelial Dysfunction
aurion biotech
2024-12-17
phase 1/2
allogeneic
cell therapy
– Primary endpoint, proportion of responders with >15 letters of improvement in best corrected visual acuity (BCVA), showed statistically significant improvement in high-dose AURN001 arm (p=0.020), compared to Y-27632-only arm– Key secondary endpoints showed statistically significant improvements in change in BCVA and central corneal thickness (CCT) in the high-dose AURN001 arm, compared to Y-27632-only arm– […]
GSK’s B7-H3-targeted antibody-drug conjugate, GSK’227, receives EMA Priority Medicines (PRIME) Designation in relapsed extensive-stage small-cell lung cancer
GSK
2024-12-16
b7-h3
lung cancer
Regulatory designation based on promising preliminary clinical data.
Jemperli (dostarlimab) receives US FDA Breakthrough Therapy Designation for locally advanced dMMR/MSI-H rectal cancer
GSK
2024-12-16
breakthrough therapy
Designation based on data showing no evidence of disease in 100% of all 42 patients who completed treatment with dostarlimab.
Atsena Therapeutics Announces Dosing Completed in Part A of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis
atsena therapeutics
2024-12-16
x-linked
gene therapy
x-linked retinoschisis
Atsena Therapeutics Announces Dosing Completed in Part A of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis DURHAM, NC, December ... The post Atsena Therapeutics Announces Dosing Completed in Part A of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis appeared first on Atsena Therapeutics .
Acepodia Announces 2025 Clinical Development Milestones and Comprehensive Growth Strategies-Acepodia® | POWERFUL, ACCESSIBLE CELL THERAPIES FOR PATIENTS WITH CANCER
Acepodia
2024-12-13
cell therapies
Acepodia (The Company, 6976.TT) announced today at the investor conference the clinical trial progress for its core product candidates ACE1831 and ACE2016, along with a detailed roadmap for 2025 and strategic plans to unlock future value. The Company also showcased groundbreaking developments in its Antibody-Dual-Drugs Conjugation (AD2C) technology platform, further solidifying its leadership in the fields of oncology and autoimmune diseases.
Acepodia Announced CEO Dr. Sonny Hsiao’s Appointment as Chairman and Extraordinary Shareholders’ Meeting Agenda-Acepodia® | POWERFUL, ACCESSIBLE CELL THERAPIES FOR PATIENTS WITH CANCER
Acepodia
2024-12-11
allogeneic
cell therapies
Acepodia (The Company, 6976:TT), a clinical-stage biotechnology company pioneering allogeneic cell therapies, announced today that its current CEO, Dr. Sonny Hsiao, has been unanimously appointed by the Board of Directors to succeed Dr. Patrick Yang as Chairman of the Board, effective January 2025. Dr. Yang, Acepodia’s Co-Founder and current Chairman, will retire at the end of 2024 and transition to the role of Co-Founder and Advisor to..
Accelerated Approval Granted to Zanidatamab-hrii for HER2-Positive Biliary Tract Cancer
ASCO Post FDA
2024-12-10
her2
accelerated approval
The U.S. Food and Drug Administration (FDA) recently granted accelerated approval to zanidatamab-hrii (Ziihera), a bispecific HER2-directed antibody, for previously treated, unresectable or metastatic...
Oral Liquid Imatinib Approved by the FDA in Leukemia
ASCO Post FDA
2024-12-10
leukemia
The U.S. Food and Drug Administration (FDA) recently has approved an oral solution of the tyrosine kinase inhibitor, imatinib mesylate, called Imkeldi, to treat certain types of leukemia and other can...
Undergoing Stem Cell Transplant for Sickle Cell Disease in Childhood
Hematology.org
2024-12-10
sickle cell disease
Undergoing Stem Cell Transplant for Sickle Cell Disease in Childhood
Adding Tafasitamab Cuts Risk of Disease Progression, Relapse, or Death in Patients with Recurrent or Refractory Follicular Lymphoma
Hematology.org
2024-12-10
follicular lymphoma
Adding Tafasitamab Cuts Risk of Disease Progression, Relapse, or Death in Patients with Recurrent or Refractory Follicular Lymphoma
Cellectis announces the drawdown of the third tranche of €5 million under the credit facility agreement entered with the European Investment Bank (EIB)
cellectis
2024-12-10
car t
allogeneic
gene therapies
New York, NY – December 10, 2024 - Cellectis (Euronext Growth: ALCLS – NASDAQ: CLLS) (the “Company”), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it has drawn down the final tranche of €5 million (“Tranche C”) under the credit facility agreement for up to €40 million entered into with the European Investment Bank (the “EIB) on December 28, 2022 (the "Finance Contract"). With the drawdown of Tranche C, the Company has drawn down the full €40 million available under the Finance Contract. Tranche C is expected to be disbursed by the EIB by December 18, 2024. The Company plans to use the proceeds of Tranche C towards the development of its pipeline of allogeneic CAR T-cell product candidates: UCART22 and UCART20x22. As a condition to the disbursement of Tranche C the Company issued 611,426 warrants to the benefit of the EIB, in accordance with the terms of the 14 th resolution of the shareholders’ meeting held on June 28, 2024 and articles L. 228-91 and seq. of the French Commercial Code (the “Tranche C Warrants”). Each Tranche C Warrant allows the EIB to subscribe for one…
Pooled Analysis of Long-Term Outcomes With Nivolumab With or Without Ipilimumab in Advanced Melanoma
ASCO Post Journals
2024-12-10
melanoma
In a pooled analysis of long-term outcomes reported in the Journal of Clinical Oncology, Long et al found that nivolumab plus ipilimumab was associated with better overall survival vs nivolumab monoth...
Orna Therapeutics Presents Data Highlighting Novel panCAR™ Approach to Unlock LNP-Mediated RNA Delivery for B Cell Mediated Diseases at the 66th ASH Annual Meeting
orna therapeutics
2024-12-09
lnp
cd20
cell therapy
–A single dose of Orna’s anti-CD20 panCAR™ resulted in a significant reduction in non-human primate (NHP) B cells, with sustained depletion observed 7 days after dosing– –Approach offers a transient and scalable immune cell therapy without the need for lymphodepletion– WATERTOWN, Mass., December 9, 2024 – Orna Therapeutics, a biotechnology company dedicated to designing and… The post Orna Therapeutics Presents Data Highlighting Novel panCAR™ Approach to Unlock LNP-Mediated RNA Delivery for B Cell Mediated Diseases at the 66th ASH Annual Meeting appeared first on Orna Therapeutics .
Blenrep (belantamab mafodotin) combination accepted for priority review in China in relapsed/refractory multiple myeloma
GSK
2024-12-09
priority review
multiple myeloma
Regulatory submission supported by phase III head-to-head DREAMM-7 trial showing statistically significant efficacy.
Blenrep shows significant overall survival benefit, reducing the risk of death by 42% in multiple myeloma at or after first relapse
GSK
2024-12-09
multiple myeloma
DREAMM-7 trial shows sustained overall survival benefit for Blenrep (belantamab mafodotin) combination versus daratumumab combination
ASH Newborn SCD Screening Program in Sub-Saharan Africa Found Feasible
Hematology.org
2024-12-08
scd
ASH Newborn SCD Screening Program in Sub-Saharan Africa Found Feasible
Disease Modifying Therapies Remain Underused in SCD Despite Safety and Efficacy
Hematology.org
2024-12-07
scd
Disease Modifying Therapies Remain Underused in SCD Despite Safety and Efficacy
AffyImmune Appoints Deyaa Adib, MD as Chief Medical Officer
AffyImmune
2024-11-25
cell therapy
chimeric antigen receptor
NATICK, Mass., Nov. 25, 2024 /PRNewswire/ -- AffyImmune Therapeutics, a clinical-stage biotechnology company committed to developing its novel, first-in-class ICAM-1 targeting chimeric antigen receptor (CAR) T cell therapy for solid tumors, today announced the appointment of Deyaa Adib, M.D., as Chief Medical Officer (CMO). Dr. Adib brings over 28 years of experience in pharmaceutical research and development in oncology,
Blenrep combinations accepted for review by the US FDA for the treatment of relapsed/refractory multiple myeloma
GSK
2024-11-25
multiple myeloma
Regulatory submission supported by phase III head-to-head DREAMM-7 and DREAMM-8 trials showing statistically significant efficacy.
BioLineRx and Ayrmid Ltd. Enter into Exclusive License Agreement to Commercialize APHEXDA® (motixafortide) through Gamida Cell Ltd.
Gamida Cell
2024-11-21
ayrmid ltd.
gamida cell
– BioLineRx to receive $10 million upfront payment from Ayrmid Ltd. (parent company of Gamida Cell) plus up to $87 million in commercial milestones, as well as royalties on net sales ranging from 18% to 23% – – BioLineRx retains rights to develop and commercialize motixafortide in solid tumors...
Pioneering Advances in Cell Therapy: Kristin Yarema, President & CEO of Poseida Therapeutics
poseida therapeutics
2024-11-20
cell therapy
Linerixibat shows positive Phase III results in cholestatic pruritus (relentless itch) in primary biliary cholangitis (PBC)
GSK
2024-11-19
primary endpoint met
Primary endpoint met with a statistically significant improvement in itch over 24 weeks compared with placebo
Sangamo Therapeutics Announces U.S. FDA Clearance of IND Application for ST-503 for the Treatment of Idiopathic Small Fiber Neuropathy, a Type of Chronic Neuropathic Pain
Sangamo BioSciences
2024-11-19
ind
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Adicet Bio Announces First Lupus Nephritis Patient Dosed in Phase 1 Clinical Trial of ADI-001 in Autoimmune Diseases
Adicet Bio
2024-11-18
lupus
stiff person syndrome
Enrollment underway for lupus nephritis (LN) patients Preliminary clinical data in LN anticipated in 1H25 Initiation of patient enrollment in systemic lupus erythematosus (SLE), systemic sclerosis (SSc), idiopathic inflammatory myopathy (IIM) and stiff person syndrome (SPS) expected in 1Q25;
Adicet Opens Enrollment for ADI-270 Phase 1 Clinical Trial in Metastatic/Advanced Clear Cell Renal Cell Carcinoma
Adicet Bio
2024-11-18
car t
cell therapy
First gamma delta CAR T cell therapy with the potential to address solid tumors entering clinical trials Preliminary Phase 1 clinical data expected in the first half of 2025 REDWOOD CITY, Calif. & BOSTON --(BUSINESS WIRE)--Nov. 18, 2024-- Adicet Bio, Inc.
Acepodia Announces FDA Clearance of Investigational New Drug Application for ACE1831 in IgG4-Related Disease
Acepodia
2024-11-15
cell therapies
/PRNewswire/ -- Acepodia (6976:TT), a clinical-stage biotechnology company developing first-in-class cell therapies with its unique Antibody-Cell Conjugation...
Blenrep shows overall survival benefit in head-to-head DREAMM-7 phase III trial for relapsed/refractory multiple myeloma
GSK
2024-11-14
multiple myeloma
Full data to be presented at 2024 American Society of Hematology Annual Meeting in December.
Affinia Therapeutics and Forge Biologics Announce AAV cGMP Manufacturing Partnership
Forge Biologics
2024-11-13
aav
gene therapy
cardiomyopathy
"We’re proud to support Affinia’s vision of bringing hope to patients affected by cardiomyopathy through their innovative gene therapy.” The post Affinia Therapeutics and Forge Biologics Announce AAV cGMP Manufacturing Partnership appeared first on Forge Biologics .
Atamyo Therapeutics obtient l’autorisation réglementaire aux Etats-Unis pour l’essai clinique de ATA-200, sa thérapie génique pour traiter la myopathie des ceintures LGMD-2C/R5
atamyo therapeutics
2024-11-12
lgmd
Evry, France (le 12 novembre 2024) – Atamyo Therapeutics, société de biotechnologie au stade clinique spécialisée sur le développement de thérapies géniques de nouvelle génération ciblant les dystrophies musculaires et les cardiomyopathies, a annoncé aujourd’hui […]
IND for ATA-200, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD2C/R5), cleared to proceed by FDA
atamyo therapeutics
2024-11-12
ind
gene therapy
gene therapies
Evry, France (November 12, 2024) – Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and cardiomyopathies, today announced that the U.S. Food & Drug Administration […]
Sangamo Therapeutics Reports Recent Business Highlights and Third Quarter 2024 Financial Results
Sangamo BioSciences
2024-11-12
financial results
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
A2 Bio Highlights Progress of Tmod TM CAR T-Cell Clinical Programs during 2024 Annual Meeting of the Society for Immunotherapy of Cancer
A2 Bio
2024-11-09
car t
The post A2 Bio Highlights Progress of Tmod TM CAR T-Cell Clinical Programs during 2024 Annual Meeting of the Society for Immunotherapy of Cancer appeared first on A2 Biotherapeutics .
Neurotech Provides Update on BLA for NT-501 as a Treatment for Macular Telangiectasia Type 2 (MacTel)
Neurotech Pharmaceuticals
2024-11-08
bla
macular telangiectasia type 2
Neurotech Pharmaceuticals, Inc., an innovator in sustained drug delivery for chronic retinal diseases, today announced that the U.S. Food and Drug Administra...
Carisma Unveils Promising Pre-Clinical Data on Anti-GPC3 In Vivo CAR-M Therapy for Hepatocellular Carcinoma
carisma therapeutics
2024-11-08
hcc
gpc3
hepatocellular carcinoma
New findings showcase the potential of in vivo CAR-M technology as an effective, off-the-shelf treatment for hepatocellular carcinoma (HCC) PHILADELPHIA , Nov. 8, 2024 /PRNewswire/ -- Carisma Therapeutics Inc. (Nasdaq: CARM ) ("Carisma" or the "Company"), a clinical-stage biopharmaceutical company
Axogen, Inc Reports Third Quarter 2024 Financial Results and Provides BLA Update
axogen
2024-11-07
bla
financial results
Carisma Therapeutics Reports Third Quarter 2024 Financial Results and Recent Business Highlights
carisma therapeutics
2024-11-07
gpc3
financial results
Initial results from the Phase 1 study of CT-0525, lead product candidate, expected in the first quarter of 2025 Nomination of a development candidate for liver fibrosis program expected in the first quarter of 2025 New preclinical efficacy data from the anti-GPC3 in vivo CAR-M therapy to be
Mustang Bio Granted Orphan Drug Designation by U.S. FDA for MB-108 (HSV-1 oncolytic virus) to Treat Malignant Glioma
mustang bio
2024-11-07
glioma
orphan drug
Carisma Therapeutics to Present New Data on Anti-GPC3 In Vivo CAR-M Therapy for Hepatocellular Carcinoma at SITC 2024
carisma therapeutics
2024-11-05
gpc3
hepatocellular carcinoma
Pre-clinical data demonstrate robust anti-tumor activity and a novel off-the-shelf approach for GPC3+ solid tumors PHILADELPHIA , Nov. 5, 2024 /PRNewswire/ -- Carisma Therapeutics Inc. (Nasdaq: CARM ) ("Carisma" or the "Company"), a clinical-stage biopharmaceutical company focused on discovering
Orna Therapeutics Announces Data Presentation at the 66th ASH Annual Meeting
orna therapeutics
2024-11-05
lnp
lipid nanoparticle
WATERTOWN, Mass., November 5, 2024 – Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of circular RNA medicines and unprecedented lipid nanoparticle (LNP) delivery solutions for oncology and autoimmune diseases, today announced an upcoming poster presentation at the 66th American Society of Hematology (ASH) Annual Meeting in San Diego from December 7-10, 2024. The presentation will outline data supporting the exploration of its in vivo CAR therapy approach in oncology and autoimmune diseases. Presentation details are as follows: The post Orna Therapeutics Announces Data Presentation at the 66th ASH Annual Meeting appeared first on Orna Therapeutics .
Cellectis Presents Multiple Strategies to Enhance CAR T-cell Efficacy in Solid Tumors at the SITC Annual Meeting
cellectis
2024-11-05
fap
car t
talen
allogeneic
gene editing
cell therapies
gene therapies
New York, NY – November 5, 2024 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that pre-clinical data to enhance CAR T cell activity against solid tumors while preventing potential toxicity, will be presented at the Society for Immunotherapy of Cancer’s 39 th Annual Meeting (SITC), that will take place on November 6-10, 2024 in Houston, Texas. The data will be presented in a poster: Title: Breaking barriers in solid tumors with SMART allogeneic CAR T-cells Date / Time: November 9 th , 2024 from 9:00am to 8:30pm ET Presenter: Beatriz Aranda-Orgilles, Associate Director, Immuno Oncology at Cellectis Poster number: 254 Despite the success of CAR T-cell therapies treating blood cancers, these cutting-edge technologies continue to face obstacles in solid tumors. A main barrier is the hostile tumor microenvironment (TME), which forms an immunosuppressive barrier and restricts T-cell infiltration into the tumor. Other contributing causes such as tumor antigen diversity or low expression of CAR-targeted tumor-associated…
Cellectis Provides Business Updates and Financial Results for Third Quarter 2024
cellectis
2024-11-04
car t
pivotal
allogeneic
cell therapy
gene editing
gene therapy
gene therapies
financial results
in vivo gene therapy
hematological malignancies
UCART22 and UCART20x22: enrollment ongoing, Phase 1 dataset and late-stage development strategy to be presented in 2025 AstraZeneca partnership: R&D activities are ongoing on three programs – one allogeneic CAR T for hematological malignancies, one allogeneic CAR T for solid tumors, and one in vivo gene therapy for a genetic disorder Appointed Adrian Kilcoyne, M.D., MPH, MBA, an industry leader in the advancement of cell therapy treatment, as Chief Medical Officer Cash position of $2 64 million as of September 30, 2024 [1] ; cash runway projection into 2027 Conference call scheduled for 8:00 am ET / 2:00 pm CET on November 5, 2024 New York, NY – November 4, 2024 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, today provided business updates and reported financial results for the nine-month period ending September 30, 2024. “This quarter, we were thrilled to welcome Dr. Kilcoyne to Cellectis as Chief Medical Officer. Dr. Kilcoyne joins us at a pivotal time for the Company, bringing extensive experience in drug development as we are…
Cellectis to Report Third Quarter 2024 Financial Results on November 4, 2024
cellectis
2024-10-30
gene therapies
financial results
New York, NY – October 30, 2024 - Cellectis (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the third quarter 2024 ending September 30, 2024 on Monday, November 4, 2024 after the close of the US market. The publication will be followed by an investor conference call and webcast on Tuesday, November 5, 2024 at 8:00 AM ET / 2:00 PM CET. The call will include the Company’s third quarter results and an update on business activities. Details for the call are as follows: Dial in information: Domestic: +1-800-225-9448 International: +1-203-518-9708 Conference ID: CLLSQ3 Webcast Link: https://viavid.webcasts.com/starthere.jsp?ei=1688144&tp_key=affca25222
GSK enters agreement to acquire CMG1A46 from Chimagen Biosciences to expand immunology pipeline
GSK
2024-10-29
lupus
Agreement reinforces GSK’s portfolio in the treatment of lupus and underlying drivers of autoimmune disease
Cellectis to Present Data on TALE-Base Editors and Non-Viral Gene Therapy at the ESGCT 31st Annual Congress
cellectis
2024-10-22
talen
gene editing
gene therapy
gene therapies
New York, NY – October 22, 2024 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that they will be showcasing pre-clinical data that permit the design of an efficient and specific TALE base editors (TALEB) as well as a process to enhance the efficacy of non-viral gene insertion in hematopoietic stem and progenitor cells (HSPCs) at the European Society of Cell and Gene Therapy 31 st annual congress, that will take place on October 22-25, 2024, in Roma, Italy. The data will be presented in two posters: Controlling C-to-T editing with TALE base editors Presenter: Alexandre Juillerat, Ph.D., Vice-President Gene Editing & NY Lab Head at Cellectis Date/Time: Thursday, October 24 from 2:00pm to 3:30pm CET Poster number: P0666 TALE base editors (TALEB) are fusions of a transcription activator-like effector domain (TALE), split-DddA deaminase halves, and an uracil glycosylase inhibitor (UGI). The C-to-T class of TALEB edits double strand DNA by converting a cytosine (C) to a thymine (T) via the formation of an uracil…
Be Bio Announces $82 Million Financing and Transition to Clinical Stage Company
be biopharma
2024-10-22
hemophilia b
October 22, 2024 12:00 PM Eastern Daylight Time CAMBRIDGE, Mass.–(BUSINESS WIRE)–Be Biopharma, Inc. (“Be Bio” or “the Company”), a leader in the discovery and development of engineered B Cell Medicines (BCMs), today announced key milestones alongside a new round of funding as its lead program, BE-101 for Hemophilia B, enters the clinic, and its second […] The post Be Bio Announces $82 Million Financing and Transition to Clinical Stage Company appeared first on Be Biopharma .
Sangamo Therapeutics Announces Alignment With FDA on Accelerated Approval Pathway for ST-920 in Fabry Disease With BLA Submission Expected in 2025
Sangamo BioSciences
2024-10-22
bla
st-920
fabry disease
accelerated approval
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Coave Therapeutics Showcases Breakthrough in Peptide-Based Conjugated AAV Vectors Using ALIGATER™ Platform in a Late-Breaking Abstract at ESGCT 2024
coave therapeutics
2024-10-21
aav
Novartis receives positive CHMP opinion for Kisqali® to help reduce risk of recurrence in people with HR+/HER2- early breast cancer
Novartis
2024-10-18
her2
breast cancer
Gepotidacin accepted for priority review by US FDA for treatment of uncomplicated urinary tract infections in female adults and adolescents
GSK
2024-10-16
priority review
Gepotidacin could be the first in a new class of oral antibiotic treatment for uUTIs in over 20 years.
Isatuximab Plus VRd Approved by FDA for Newly Diagnosed, Transplant-Ineligible Multiple Myeloma
ASCO Post FDA
2024-10-10
multiple myeloma
On September 20, the U.S. Food and Drug Administration (FDA) approved the monoclonal antibody isatuximab-irfc (Sarclisa) in combination with bortezomib, lenalidomide, and dexamethasone for adults with...
Food Deserts, Limited Access to Transportation Linked to More Complications Among Preschool Children with SCD
Hematology.org
2024-10-07
scd
Food Deserts, Limited Access to Transportation Linked to More Complications Among Preschool Children with SCD
Orchard Therapeutics and Er-Kim Announce Partnership to Broaden Access to Libmeldy to Eligible Patients in Turkey and Certain Eurasian Countries
orchard therapeutics
2024-10-07
gene therapies
ISTANBUL and LONDON, and BOSTON, Oct. 07, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics , recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe and Er-Kim , an international pharmaceutical company specializing in the
Forge Biologics Announces the FUEL™ AAV Manufacturing Platform
Forge Biologics
2024-10-02
aav
FUEL™ platform can achieve a 2-6x increase in productivity compared to industry standard, and includes pEMBR 2.0™ Ad helper and modified rep/cap plasmids. The post Forge Biologics Announces the FUEL™ AAV Manufacturing Platform appeared first on Forge Biologics .
Atamyo Therapeutics Observes LGMD Awareness Day with Updates on Key Milestones in its Clinical Development of Gene Therapies for Patients Suffering from Limb-Girdle Muscular Dystrophies
atamyo therapeutics
2024-09-30
lgmd
gene therapies
Evry, France (September 30, 2024) – In recognition of LGMD Awareness Day today, Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and cardiomyopathies, announced the […]
Adjuvant Dabrafenib Plus Trametinib for Stage III BRAF V600–Mutated Melanoma: Final Results of the COMBI-AD Trial
ASCO Post Journals
2024-09-25
melanoma
The final follow-up analysis of the phase III COMBI-AD trial was presented at the 2024 ASCO Annual Meeting as well as published in The New England Journal of Medicine.
Combination Targeted Therapy Produces Durable Remissions in Some Patients With Relapsed Diffuse Large B-Cell Lymphoma
ASCO Post Journals
2024-09-25
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
The results from a phase Ib/II study of a five-drug targeted therapy regimen—venetoclax, ibrutinib, prednisone, obinutuzumab, and lenalidomide (ViPOR)—in the treatment of relapsed or refractory diffus...
Altaris to Acquire Minaris, Global Cell Therapy Leader
Altaris
2024-09-24
cell therapy
Altaris to acquire Minaris Regenerative Medicine, a leading CDMO in cell therapy, expanding global manufacturing capabilities.
AffyImmune Appoints Drs. Rick Rutter and Jorge Nieva to Board of Directors
AffyImmune
2024-09-23
cell therapy
chimeric antigen receptor
September 23, 2024 08:00 AM Eastern Daylight Time NATICK, Mass.--(BUSINESS WIRE)--AffyImmune Therapeutics, a clinical-stage biotechnology company committed to developing its novel, first-in-class ICAM-1 targeting chimeric antigen receptor (CAR) T cell therapy for solid tumors, today announced the appointment of Rick Rutter, Ph.D., and Jorge Nieva, M.D., to its Board of Directors. The addition of these
News link: A lymphoma survivor talks about his treatments with CAR T-cell therapy to fight cancer | Lymphoma Awareness Month – CBS Los Angeles
pepromene bio
2024-09-20
car t
cell therapy
Irvine, CA, September 20, 2024 – A lymphoma survivor talks about his treatments with CAR T-cell therapy to fight cancer | Lymphoma Awareness Month – CBS Los Angeles, originally aired on September 19, 2024. To find the media clip … Continue reading → The post News link: A lymphoma survivor talks about his treatments with CAR T-cell therapy to fight cancer | Lymphoma Awareness Month – CBS Los Angeles appeared first on PeproMene Bio Inc .
An article from City of Hope: Relapsed Lymphoma Patient Thrives After CAR T Cell Trial
pepromene bio
2024-09-20
car t
car-t
Irvine, CA, September 20, 2024 – Relapsed Lymphoma Patient Thrives After CAR T Cell Trial – originally posted by City of Hope, on September 17, 2024. To find the article on the web: https://www.cityofhope.org/relapsed-lymphoma-patient-thrives-after-car-t-cell-trial About PMB-CT01 PMB-CT01 is … Continue reading → The post An article from City of Hope: Relapsed Lymphoma Patient Thrives After CAR T Cell Trial appeared first on PeproMene Bio Inc .
FDA approves Novartis Kisqali® to reduce risk of recurrence in people with HR+/HER2- early breast cancer
Novartis
2024-09-17
her2
breast cancer
Blenrep (belantamab mafodotin) combinations in relapsed/refractory multiple myeloma accepted for regulatory review in Japan
GSK
2024-09-17
multiple myeloma
Regulatory submission supported by phase III head-to-head DREAMM-7 and DREAMM-8 trials.
Atamyo Therapeutics to Provide Corporate Overviews and Updates on Its Pipeline Progress at 6 Upcoming Conferences in the US and Europe
atamyo therapeutics
2024-09-16
gene therapies
Evry, France (September 16, 2024) – Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular disease, today announced its participation in six upcoming conferences in the US and Europe […]
Nate Hardy to Join Arsenal Biosciences, Inc. as Chief Financial Officer
arsenal biosciences
2024-09-16
car t
cell therapy
cell therapies
South San Francisco, Calif. – September 16, 2024 – Arsenal Biosciences, Inc. (ArsenalBio), a clinical stage programmable cell therapy company focused on engineering advanced CAR T-cell therapies for solid tumors, today announced that Nate Hardy will join its management team as Chief Financial Officer, effective October 7, 2024. Mr. Hardy will lead financial strategy, manage […]
Aurion Biotech Launches Vyznova® in Japan for the Treatment of Bullous Keratopathy of the Cornea
aurion biotech
2024-09-15
allogeneic
cell therapy
World’s first approved allogeneic cell therapy for corneal endothelial diseases Prof. Shigeru Kinoshita, M.D., cell therapy inventor, successfully treats first patients Seattle, WA; Cambridge, MA; Tokyo, Japan – September 24, 2024 – Aurion Biotech, whose mission is to restore vision to millions of patients with life-changing regenerative therapies, today announced the commercial launch of Vyznova® […]
Blenrep (belantamab mafodotin) in combination receives Breakthrough Therapy Designation in China for treatment of relapsed/refractory multiple myeloma
GSK
2024-09-13
multiple myeloma
breakthrough therapy
Granted based on results from phase III head-to-head DREAMM-7 trial.
Professor Isabelle Richard has received the Fifth Annual LGMD2i Pioneer Award from Cure LGMD2i Foundation and the Speak Foundation
atamyo therapeutics
2024-09-12
lgmd
This recognition is for Isabelle Richard’s groundbreaking research on LGMD. Isabelle Richard, Ph.D., is Research Director at National Center of Scientific Research (CNRS) in France and head of the Progressive Muscular Dystrophies Laboratory at Genethon. We are […]
New Studies Explore Relationships Between Heart Failure and Lymphoma, Arterial Thromboembolism and Cancer
ASCO Post Journals
2024-09-10
heart failure
In two studies recently reported in JACC: CardioOncology, researchers examined the impact of preexisting heart failure on mortality in older patients in the United States with newly diagnosed Hodgkin ...
Muscular Dystrophy Association and Forge Biologics Announce AAV Development and Manufacturing Partnership
Forge Biologics
2024-09-09
aav
gene therapy
"Forge has proven to be a trusted partner to gene therapy innovators, advancing many critical programs for rare diseases" Sharon Hesterlee, Ph.D., CRO at the MDA. The post Muscular Dystrophy Association and Forge Biologics Announce AAV Development and Manufacturing Partnership appeared first on Forge Biologics .
ArsenalBio Announces $325 Million Financing to Advance Programmable Cell Therapy Programs through Clinical Development
arsenal biosciences
2024-09-04
car t
cell therapy
cell therapies
Oversubscribed Series C welcomes multiple new investors South San Francisco, Calif. – September 4, 2024 – Arsenal Biosciences, Inc. (ArsenalBio), a clinical stage programmable cell therapy company focused on engineering advanced CAR T-cell therapies for solid tumors, today announced the close of an oversubscribed $325 million Series C financing round. The funding round included new […]
Cellectis Presents Pre-Clinical Evidence of MUC1 CAR T-cells Reducing Triple-Negative Breast Cancer While Preserving Safety
cellectis
2024-09-03
car t
talen
breast cancer
cell therapies
gene therapies
chimeric antigen receptor
September 3 rd , 2024 – New York (NY) – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today published a scientific article in Science Advances suggesting that TALEN ® -edited MUC1 CAR T-cells could be a potential treatment option for advance-stage triple negative breast cancer (TNBC) patients with limited therapeutic options. Globally, breast cancer continues to be the most prevalent malignancy in women. Among all subtypes, triple-negative breast cancer (TNBC) stands out as the most aggressive form with high metastatic potential and poor survival rates. Despite a few emerging targeted therapies under investigation, surgery, chemotherapy and radiation therapy continue to be the standard of care, and their success remains limited. As an alternative, Chimeric Antigen Receptor (CAR) T-cell therapies could hold promise for advance-stage TNBC patients as tumor-associated MUC1 antigen is overexpressed in a large number of patients thus offering a distinct target for treatment. In this article, Cellectis described its multi-layered CAR…
Acepodia Enters Strategic Clinical Collaboration with Pfizer to Accelerate Development of Antibody-Cell Conjugation-Based Cell Therapies in Autoimmune Diseases
Acepodia
2024-09-03
cell therapies
/PRNewswire/ -- Acepodia (6976:TT), a clinical-stage biotechnology company developing first-in-class cell therapies with its unique Antibody-Cell Conjugation...
Aurion Biotech to Present at the 42nd Congress of the European Society of Cataract and Refractive Surgeons (ESCRS) and Ophthalmology European Futures Forum
aurion biotech
2024-09-03
allogeneic
cell therapy
Presentations at conferences will feature clinical data and business updates on Aurion Biotech’s allogeneic cell therapy AURN001 for the treatment of corneal endothelial diseases Seattle, WA; Cambridge, MA; Tokyo, Japan – September 3, 2024 – Aurion Biotech, whose mission is to restore vision to millions of patients with life-changing regenerative therapies, today announced that it […]
Cellectis Publishes a Molecular Therapy Article on a SMART DUAL CAR T-cell Approach for Treating Recalcitrant Solid Tumors
cellectis
2024-08-26
fap
car t
talen
allogeneic
mesothelin
solid tumor
cell therapy
gene editing
gene therapies
adoptive cell therapy
August 26, 2024 - New York, NY – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today published an article in Molecular Therapy demonstrating TALEN ® -mediated gene editing capabilities for design of SMART DUAL CAR T-cells, which efficiently target immunotherapy recalcitrant solid tumors while mitigating potential safety risks. Adoptive cell therapy based on CAR T cells has proven to be lifesaving for many cancer patients. However, its therapeutic efficacy has so far been restricted to only a few malignancies, with solid tumors proving to be especially recalcitrant to efficient therapy. One key factor for this are cancer-associated fibroblasts (CAFs), that modulate the tumor microenvironment (TME) to inhibit T cell infiltration and induce T cell dysfunction. Additionally, the sparsity of tumor-specific antigens (TSA) and expression of CAR-directed tumor-associated antigens (TAA) on normal tissues often results in on-target off-tumor cytotoxicity, raising safety concerns. Using TALEN® gene editing technology, Cellectis presents an…
FDA Approves Immunotherapy for Relapsed or Refractory CTCL
ASCO Post FDA
2024-08-25
t-cell lymphoma
Today, the FDA approved denileukin diftitox-cxdl (Lymphir), a novel immunotherapy for adult patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL) who have received at least one prior s...
GSK receives US FDA Breakthrough Therapy Designation for its B7-H3-targeted antibody-drug conjugate in relapsed or refractory extensive-stage small-cell lung cancer
GSK
2024-08-20
b7-h3
lung cancer
breakthrough therapy
Regulatory designation based on promising early clinical evidence observed with GSK5764227 in this tumour type
AffyImmune Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for AIC100 in Recurrent Anaplastic Thyroid Cancer
AffyImmune
2024-08-14
rmat
thyroid cancer
RMAT designation follows FDA’s review of available preliminary clinical evidence that AIC100 has the potential to address unmet medical need in recurrent anaplastic thyroid cancer (ATC) RMAT designation enables increased dialogue with the FDA to support the potential expedited development and review of AIC100 in clinical evaluation for ATC NATICK, Mass.--(BUSINESS WIRE)--AffyImmune, a clinical-stage biotechnology
Novartis receives FDA accelerated approval for Fabhalta® (iptacopan), the first and only complement inhibitor for the reduction of proteinuria in primary IgA nephropathy (IgAN)
Novartis
2024-08-08
accelerated approval
PeproMene Bio announced study demonstrating strong polyfunctionality of novel chimeric antigen receptor (CAR) T cells
pepromene bio
2024-08-07
cd19
car t
chimeric antigen receptor
IRVINE, Calif., Aug. 7, 2024 /PRNewswire/ — PeproMene Bio, Inc., a clinical-stage biotech company developing novel therapies for cancers and immune disorders, today announced that City of Hope-developed BAFF-R CAR T, known as PMB-CT01, demonstrated better polyfunctionality than CD19 CAR T cells, which are … Continue reading → The post PeproMene Bio announced study demonstrating strong polyfunctionality of novel chimeric antigen receptor (CAR) T cells appeared first on PeproMene Bio Inc .
Cellectis Appoints Dr. Adrian Kilcoyne as Chief Medical Officer
cellectis
2024-08-07
car t
car-t
pivotal
allogeneic
cell therapy
gene editing
cell therapies
gene therapies
nk cell therapy
New York, NY – August 7, 2024 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the appointment of Dr. Adrian Kilcoyne, M.D., MPH, MBA as its Chief Medical Officer, effective immediately. “We’re thrilled to welcome Dr. Kilcoyne to Cellectis. He is a strategic, forward-thinking drug developer who is passionate about delivering life-saving therapies to patients. His clinical vision and proven leadership, as well as his extensive experience, will strengthen our clinical development efforts as we advance our product pipeline of next-generation CAR T-cell therapies” said André Choulika, Ph.D., Chief Executive Officer at Cellectis. Before joining Cellectis, Dr. Kilcoyne was Chief Medical Officer and Head of Research and Development at Celularity, advancing their oncology allogeneic CAR-T and NK Cell therapy programs. Prior to this, he was Chief Medical Officer at Humanigen. He has held numerous Oncology leadership roles across Research and Development, Medical Affairs, Commercial, Health Economic Outcome Research and…
Sangamo Therapeutics Reports Recent Business Highlights and Second Quarter 2024 Financial Results
Sangamo BioSciences
2024-08-06
financial results
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Cellectis Provides Financial Results for the Second Quarter 2024
cellectis
2024-08-06
bla
leukemia
orphan drug
gene editing
gene therapies
priority review
financial results
acute lymphoblastic leukemia
ODD and RPDD granted by the FDA and ODD granted by the European Commission to UCART22 for the treatment of ALL ODD granted by the FDA to CLLS52 (alemtuzumab) for ALL treatment Cash position of $273 million as of June 30, 2024 [1] ; cash runway projection into 2026 New York, NY – August 6, 2024 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, today provided business updates and reported financial results for the six-month period ending June 30, 2024. "Over the past months, we have achieved a significant milestone with the granting of ODD designations by the Food and Drug Administration and the European Commission, complemented by the FDA’S Rare Pediatric Disease Designation. We have overcome major challenges, which reflects our ongoing commitment to innovation. Driven by an unwavering belief in our ability to revolutionize the healthcare field, we continue our pursuit of advancement with the confidence that our work will lead to the launch of a life-saving drug product. Our determination is the engine of our future success” said André…
FDA Grants Orphan Drug Designation to Cellectis’ CLLS52 (alemtuzumab) For ALL Treatment
cellectis
2024-08-01
talen
leukemia
orphan drug
gene editing
gene therapies
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
New York, NY – August 1, 2024 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Cellectis’ CLLS52 (alemtuzumab), an Investigational Medicinal Product (IMP) used as part of the lymphodepletion regimen associated with UCART22, evaluated in the BALLI-01 clinical trial in relapsed/refractory B-cell acute lymphoblastic leukemia (ALL). “We are excited that the FDA granted CLLS52 (alemtuzumab) ODD designation status. The importance of adding alemtuzumab to the lymphodepletion regimen has been demonstrated in Cellectis’ BALLI-01 study, where the addition of this lymphodepletion agent to the fludarabine and cyclophosphamide regimen was associated with sustained lymphodepletion and significantly higher UCART22 cell expansion allowing for greater clinical activity ”, said Mark Frattini, M.D., Ph.D. Chief Medical Officer at Cellectis . Cellectis is the inventor of the combination of CD52 knockout UCART cells with a lymphodepleting regimen…
Novartis Scemblix® granted FDA Priority Review for the treatment of adults with newly diagnosed CML
Novartis
2024-07-29
priority review
Addition of Ibrutinib to Immunochemotherapy and ASCT Improves Survival in Mantle Cell Lymphoma
ASCO Post Journals
2024-07-25
autologous
mantle cell lymphoma
Results in the European Mantle Cell Lymphoma Network phase III TRIANGLE trial indicate that the addition of ibrutinib to immunochemotherapy and autologous stem cell transplantation (ASCT) significantl...
FDA Grants Orphan Drug and Rare Pediatric Disease Designation Status to Cellectis’ UCART22 product candidate for Acute Lymphoblastic Leukemia (ALL) Treatment
cellectis
2024-07-25
cd19
cd22
open
car t
leukemia
phase 1/2
allogeneic
orphan drug
cell therapies
gene therapies
hematopoietic stem cell
acute lymphoblastic leukemia
ALL represents 10% of all leukemia cases in the United States, progresses rapidly, and is typically fatal within weeks or months if left untreated [1] There is an urgent need to develop new therapies for ALL for patients who are not candidates for hematopoietic stem cell transplantation (HSCT) or relapse after FDA ODD and RPDD designations for UCART22 marks an important step towards developing allogeneic CAR T products that would be readily available for all patients New York, NY – July 25, 2024 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug (ODD) and Rare Pediatric Disease Designation (RPDD) Status to UCART22 product candidate for the treatment of Acute Lymphoblastic Leukemia (ALL). ALL represents about 10% of all leukemia cases in the United States, progresses rapidly, and is typically fatal within weeks or months if left untreated. It is estimated that 6,660 new cases of ALL and 1,560 deaths related to the disease occurred in the US in 2022…
Sangamo Therapeutics Reports on Pfizer’s Announcement of Positive Topline Results From Phase 3 Trial of Hemophilia A Gene Therapy Candidate
Sangamo BioSciences
2024-07-24
gene therapy
hemophilia a
The Investor Relations website contains information about Sangamo Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
ViiV Healthcare announces positive new data for Apretude use during pregnancy at AIDS 2024
GSK
2024-07-23
open
Open label extension show maternal and pregnancy outcomes with Apretude exposure were comparable to those with no cabotegravir exposure.
Blenrep (belantamab mafodotin) combinations in multiple myeloma application accepted for review by the European Medicines Agency
GSK
2024-07-19
multiple myeloma
Regulatory submission supported by phase III head-to-head DREAMM-7 and DREAMM-8 trials
AffyImmune Appoints Dr. Daniel Janse as Chief Executive Officer
AffyImmune
2024-07-15
car t
cell therapies
NATICK, Mass.--(BUSINESS WIRE)--AffyImmune, a clinical-stage biotechnology company committed to developing novel, first-in-class CAR T cell therapies, today announced the appointment of Daniel Janse, Ph.D. as President and Chief Executive Officer. Dr. Janse brings twenty years of expertise and an extensive background in biopharmaceutical research and development, operations, and leadership to drive the company forward. “We
Cellectis Reports Results from Shareholders Meeting Held on June 28, 2024
cellectis
2024-06-28
gene therapies
June 28, 2024 – New York (N.Y.) – Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, held today a shareholders general meeting at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4 th floor, 75013 Paris, France. At the meeting, during which approximately 40 % of shares were exercised, resolutions 1 through 28 were adopted and resolution 29 was rejected, consistent with the recommendations of the management. The detailed results of the vote and the resolutions are available on Cellectis’ website: https://www.cellectis.com/en/investors/general-meetings/
Why BTD / RMAT Designations Matter
aurion biotech
2024-06-25
rmat
by Greg Kunst, CEO Aurion Biotech Biotech is a numbers game, but often the outcomes are not binary. Drugs work..sometimes, or they achieve partial effect, or they work only in certain populations. All too often, clear answers are not available because human biology is exceptionally complex. Sometimes, the numbers are more straightforward. Since FDA’s Center […]
BlueSphere Bio To Partner with NMDP BioTherapies℠ for the Company’s First-In-Human TCR-based Product Candidate in Combination with Allogeneic Hematopoietic Cell Transplantation Trial
bluesphere bio
2024-06-25
allogeneic
gene therapies
PITTSBURGH, June 25, 2024 (GLOBE NEWSWIRE) — BlueSphere Bio, a drug development company focused on novel T-cell receptor (TCR) based therapies for oncology and non-oncology indications, today announced a collaboration with NMDP BioTherapies℠ (formerly Be The Match BioTherapies), an organization offering solutions for companies developing and commercializing cell and gene therapies, to advance BlueSphere’s first clinical trial evaluating […]
FDA Grants Accelerated Approval to Repotrectinib for NTRK Fusion–Positive Solid Tumors
ASCO Post FDA
2024-06-25
accelerated approval
Today, the FDA granted accelerated approval to repotrectinib (Augtyro) for adult and pediatric patients aged 12 years and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTR...
Aurion Biotech Receives Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation for Its Drug Candidate AURN001
aurion biotech
2024-06-25
allogeneic
cell therapy
breakthrough therapy
corneal endothelial disease
First allogeneic cell therapy to receive both FDA designations for the treatment of corneal edema secondary to corneal endothelial disease Seattle, WA; Cambridge, MA; Tokyo, Japan — 18 June 2024—Aurion Biotech, whose mission is to restore vision to millions of patients with life-changing regenerative therapies, today announced that the U.S. Food and Drug Administration (FDA) […]
Power Of Attorney For Cell Therapy Outsourcing
bluesphere bio
2024-06-24
cell therapy
Cellectis Publishes a Scientific Article Unveiling Three Key Factors for Efficient TALE Base Editing
cellectis
2024-06-20
cas9
crispr
base editing
gene editing
gene therapies
New York, NY – June 20, 2024 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the publication of a manuscript in Scientific Reports, demonstrating how three key factors can be determinant for efficient TALE base editing. TALE base editors are a recent and important addition to the gene editing landscape. By design, TALE base editors do not create break within DNA strands as does CRISPR/Cas9, or other engineered nucleases, and is a promising therapeutic strategy for genetic diseases. A key aspect to broaden the scope of possible applications is our comprehension of design rules. TALE base editors rely on the deamination of cytidines within double strand DNA, leading to the formation of an uracil (U) intermediate. These molecular tools are fusions of transcription activator-like effector domains (TALE) for specific DNA sequence binding, split-DddA deaminase halves that will, upon catalytic domain reconstitution, initiate the conversion of a cytosine (C) to a thymine (T), and an uracil glycosylase inhibitor (UGI).…
Cellectis Unveils a Non-Viral Gene Therapy Approach for Sickle Cell Disease in Nature Communications
cellectis
2024-06-12
scd
talen
autologous
gene editing
gene therapy
gene therapies
sickle cell disease
New York, NY – June 12, 2024 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the publication of a scientific article in Nature Communications, unveiling a non-viral gene therapy approach for sickle cell disease. Sickle cell disease (SCD) is one of the most common inherited diseases worldwide. SCD is caused by a single point mutation in the HBB gene, which encodes the β subunit of hemoglobin (Hb). Normally, red blood cells adopt a disc-like shape that allows them to move easily through the blood vessels and deliver oxygen throughout the body. In sickle cell disease, red blood cells become crescent or “sickle”-shaped, a dysfunctional state that impairs blood flow, oxygen delivery and triggers multiple debilitating symptoms including intense pain crisis. Cellectis leverages TALEN® technology and a non-viral gene repair template delivery to develop a clinically relevant gene editing process in hematopoietic stem and progenitor cells (HSPCs). This process enables efficient HBB gene correction with high precision,…
FDA Grants Accelerated Approval to Lisocabtagene Maraleucel for Relapsed or Refractory Follicular Lymphoma
ASCO Post FDA
2024-06-10
breyanzi
follicular lymphoma
accelerated approval
On May 15, the FDA granted accelerated approval to lisocabtagene maraleucel (Breyanzi) for adult patients with relapsed or refractory follicular lymphoma who have received two or more prior lines of s...
FDA Approves Lisocabtagene Maraleucel for Relapsed or Refractory Mantle Cell Lymphoma
ASCO Post FDA
2024-06-10
breyanzi
mantle cell lymphoma
On May 30, the FDA approved lisocabtagene maraleucel (Breyanzi) for adult patients with relapsed or refractory mantle cell lymphoma who have received at least two prior lines of systemic therapy, incl...
BlueSphere Bio Establishes Strategic Collaboration with the National Cancer Institute to Advance Treatments for Rare Respiratory Disease
bluesphere bio
2024-06-04
cell therapy
PITTSBURGH, June 04, 2024 (GLOBE NEWSWIRE) — BlueSphere Bio, a T-cell receptor (TCR) T-cell therapy company utilizing a powerful TCR discovery platform to identify novel therapeutic candidates for patients with hematologic malignancies and solid tumors, today announced a strategic collaboration with the National Cancer Institute (NCI), part of the National Institutes of Health. The collaboration will […]
Cellectis Receives Orphan Drug Designation for UCART22, its Allogeneic CAR T Product for Patients with Acute Lymphoblastic Leukemia
cellectis
2024-06-04
cd19
cd22
open
car t
leukemia
phase 1/2
allogeneic
orphan drug
cell therapy
gene therapies
acute lymphoblastic leukemia
New York, NY – June 4, 2024 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that the European Commission (EC) has granted an Orphan Drug Designation (ODD) to its product candidate UCART22, for the treatment of Acute Lymphoblastic Leukemia (ALL). UCART22 is an allogeneic CAR T-cell product candidate targeting CD22 and evaluated in BALLI-01, a Phase 1/2 open-label dose-escalation and dose-expansion study, designed to evaluate the safety, expansion, persistence and clinical activity of UCART22 in patients with relapse/refractory ALL. ALL represents 12% of all leukemia cases, progresses rapidly, and is typically fatal within weeks or months if left untreated [1] . In 2024, the 10-year prevalence is estimated at 1.9 in 100,000 persons in the European Union (EU). Based on the preliminary clinical data generated with UCART22 in heavily pretreated patients who were relapsed or refractory to approved medicinal products, the European Medicines Agency (EMA) considered that the significant benefit of UCART22 has been…
BlueSphere Bio Announces IND Clearance of its First in Human Candidate and New Cell Therapy Portfolio for High-Risk Leukemia Patients
bluesphere bio
2024-06-03
ind
tcr t
leukemia
allogeneic
autologous
cell therapy
TCX-101 Clinical Program: TCR T-Cell therapy product candidate (BSB-1001) to be delivered simultaneously with allogeneic stem cell transplant in patients with active disease or high risk of relapse First patient to be enrolled in 4Q2024 Nomination of Three additional novel TCR candidates expand HLA population coverage and addressable market size TCX 102 Development Program:Autologous TCR-based cell […]
Blenrep combination reduced the risk of disease progression or death by nearly 50% versus standard of care combination in relapsed/refractory multiple myeloma
GSK
2024-06-02
multiple myeloma
DREAMM-8 phase III trial showed statistically significant and clinically meaningful improvement.
AffyImmune Announces Confirmed Complete Response in Patient with Anaplastic Thyroid Cancer
AffyImmune
2024-05-30
car t
autologous
thyroid cancer
complete response
The first complete response from a single intravenous (IV) dose of autologous CAR T in a patient with solid tumors - Unprecedented safety and early efficacy data to be presented with complete response in poster session at ASCO 2024 Annual Meeting NATICK, Mass., May 23, 2024--(BUSINESS WIRE)--AffyImmune Therapeutics, a clinical-stage biotechnology company committed to developing novel,
A2 Bio Highlights Progress of CAR T-Cell Clinical Programs in Three Poster Presentations during the American Society of Clinical Oncology (ASCO) 2024 Annual Meeting
A2 Bio
2024-05-29
car t
The post A2 Bio Highlights Progress of CAR T-Cell Clinical Programs in Three Poster Presentations during the American Society of Clinical Oncology (ASCO) 2024 Annual Meeting appeared first on A2 Biotherapeutics .
Cellectis’ Annual Shareholders General Meeting to be Held on June 28, 2024
cellectis
2024-05-29
gene therapies
New York, NY – May 29, 2024 - Cellectis (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that it will hold its annual general meeting on June 28, 2024 at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4th floor, 75013 Paris, France. The notice convening the annual general meeting stating the detailed agenda and modalities of participation in the meeting is available on the Cellectis website: https://www.cellectis.com/en/investors/general-meetings/
Cellectis Reports Financial Results for First Quarter 2024
cellectis
2024-05-28
gene editing
gene therapy
gene therapies
financial results
Cellectis announced completion of the additional equity investment of $140M by AstraZeneca Cash position of $143 million as of March 31, 2024 [1] ; cash runway projection into 2026 [2] Conference call and webcast scheduled for tomorrow, May 29, 2024 at 8:00AM ET / 2:00PM CET New York, NY – May 28, 2024 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, today provided business updates and reported financial results for the three-month period ending March 31, 2024. “We are thrilled to have announced the closing of the additional equity investment of $140 million by AstraZeneca. This followed AstraZeneca’s initial payment of $105 million, composed of a $80 million equity investment and a $25 million upfront payment under our research collaboration. Following AstraZeneca’s additional investment, we expect our cash runway to fund operations into 2026. We will continue to focus our efforts and expenses on advancing its core clinical trials BALLI-01, NATHALI-01 and AMELI-01, which remain wholly owned assets, while building, within our owned…
Cellectis to Report First Quarter 2024 Financial Results on May 28, 2024
cellectis
2024-05-25
gene therapies
financial results
New York, NY – May 27, 2024 - Cellectis (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the first quarter 2024 ending March 31, 2024 on Tuesday, May 28, 2024 after the close of the US market. The publication will be followed by an investor conference call and webcast on Wednesday, May 29, 2024 at 8:00 AM ET / 2:00 PM CET. The call will include the Company’s first quarter results and an update on business activities. Details for the call are as follows: Dial in information: Domestic: 1-877-451-6152 International: 1-201-389-0879 Conference ID: 13746795 Webcast Link: https://viavid.webcasts.com/starthere.jsp?ei=1672725&tp_key=f83e7ab481
New data at ASCO showcases the transformational potential of GSK's oncology portfolio
GSK
2024-05-23
pivotal
Pivotal data will be shared from the DREAMM-8 and DREAMM-7 phase III trials.
A2 Bio Announces First Patient Dosed in EVEREST-2 Phase 1 Clinical Trial of Novel Mesothelin Logic-Gated CAR T
A2 Bio
2024-05-16
car t
mesothelin
The post A2 Bio Announces First Patient Dosed in EVEREST-2 Phase 1 Clinical Trial of Novel Mesothelin Logic-Gated CAR T appeared first on A2 Biotherapeutics .
Excision BioTherapeutics Announces Data from the Phase 1/2 Trial of EBT-101 in HIV And In Vivo Efficacy Data in Herpes Virus and Hepatitis B
Excision Biotherapeutics
2024-05-13
phase 1/2
FDA Grants Accelerated Approval to Lifileucel for Unresectable or Metastatic Melanoma
ASCO Post FDA
2024-05-10
amtagvi
melanoma
autologous
accelerated approval
On February 16, the FDA granted accelerated approval to lifileucel (Amtagvi), a tumor-derived autologous T-cell immunotherapy, for previously treated adult patients with unresectable or metastatic mel...
Acepodia Announces Preliminary Clinical Data From Phase 1 Clinical Trial of ACE1831, First-Ever Anti-CD20 Antibody Conjugated Allogenic Gamma Delta T Cell Therapy for Non-Hodgkin's Lymphoma
Acepodia
2024-05-08
cd20
cell therapy
cell therapies
gamma delta t cell
/PRNewswire/ -- Acepodia (6976:TT), a clinical stage biotechnology company developing first-in-class cell therapies with its unique Antibody-Cell Conjugation...
William Blair Fireside chat with Acepodia-Acepodia® | POWERFUL, ACCESSIBLE CELL THERAPIES FOR PATIENTS WITH CANCER
Acepodia
2024-05-08
cell therapies
Acepodia’s Clinical Update: Harnessing the Versatility of Bioorthogonal Chemistry Host:Andy Hsieh, Ph.D., Biotech Research Analyst at William Blair Guests:Sonny Hsiao, Ph.D., Acepodia CEO/PresidentCarolyn Bertozzi, Ph.D., Acepodia Chief Scientific Advisor; Nobel Laureate in Chemistry in 2022; Anne T. and Robert M. Bass Professor of Chem..
ArsenalBio Announces Presentation of Four Abstracts at ASGCT Annual Meeting Highlighting New Mechanisms for Leveraging CAR T Cells to Address Solid Tumor Cancers
arsenal biosciences
2024-05-06
car t
solid tumor
cell therapy
cell therapies
Company to share research on new approaches to overcome the immunosuppressive tumor microenvironment and combat T cell exhaustion through integrated circuit T cell technology South San Francisco, Calif. – May 6, 2024 – Arsenal Biosciences, Inc. (ArsenalBio), a clinical stage programmable cell therapy company focused on engineering advanced CAR T-cell therapies for solid tumors, […]
Cellectis Announces Completion of the Additional Equity Investment by AstraZeneca
cellectis
2024-05-06
gene therapies
Completion of the additional equity investment of $140M by AstraZeneca, as previously announced on November 1 and 15, 2023 New York, NY – May 6, 2024 - Cellectis (Euronext Growth: ALCLS – NASDAQ: CLLS) a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that, following clearance from the French Ministry of Economy and satisfaction of all other closing conditions, AstraZeneca (LSE/STO/Nasdaq: AZN) completed the additional equity investment of $140M in Cellectis, as previously announced by Cellectis on November 1 and 15, 2023 (the “ Additional Investment ”). As part of the Additional Investment, AstraZeneca subscribed today for 10,000,000 “class A” convertible preferred shares and 18,000,000 “class B” convertible preferred shares, in each case at a price of $5.00 per convertible preferred share, issued by the board of directors of Cellectis pursuant to the authorizations granted by the extraordinary general meeting of the shareholders of Cellectis held on December 22, 2023. Prior to their conversion into ordinary shares, the “class A" convertible preferred shares have single voting rights…
Cellectis Appoints Arthur Stril as Interim Chief Financial Officer
cellectis
2024-05-02
pivotal
gene therapy
gene therapies
New York, NY - May 2, 2024 – Cellectis (the “Company”) (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the resignation of Mr. Bing Wang from his position of the Company with immediate effect, and the concomitant appointment of Mr. Arthur Stril as interim Chief Financial Officer, replacing Mr. Bing Wang. Mr. Stril joined Cellectis in 2018 as Vice President, Corporate Development, and was appointed Chief Business Officer in 2020. He has been managing Cellectis’ business development and portfolio management teams and most recently led the execution of the Company’s strategic collaboration and investment agreements with AstraZeneca. As interim Chief Financial Officer, Mr. Stril will oversee the finance and investor relations functions, and continue to oversee the business development functions. He will remain based in Cellectis’ New York office. “On behalf of Cellectis’ Board and team, I would like to warmly thank Bing for his contribution to the Company during his tenure, he was a key member of our leadership team during important corporate…
Arsenal Biosciences Announces First Patient Dosed in Phase 1/2 Clinical Trial of AB-2100 in Development as a Treatment for Clear-cell Renal Cell Carcinoma
arsenal biosciences
2024-04-30
open
car t
phase 1/2
cell therapy
cell therapies
South San Francisco, Calif. – April 30, 2024 – Arsenal Biosciences, Inc. (ArsenalBio), a clinical stage programmable cell therapy company focused on engineering advanced CAR T-cell therapies for solid tumors, today announced that the first patient has been dosed with AB-2100 in a multi-center, open-label Phase 1/2 clinical trial for patients with clear-cell renal cell […]
Forge Biologics Announces Nine Presentations at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting
Forge Biologics
2024-04-30
aav
cell therapy
gene therapies
“As we continue to advance how genetic medicines are manufactured, we are thrilled to present data showcasing our novel molecular findings, platform process improvements, and regulatory knowledge all centered around efficient, safe, and scalable manufacturing of AAV gene therapies" The post Forge Biologics Announces Nine Presentations at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting appeared first on Forge Biologics .
Anova to Manage Study of DB107 Following Newly Awarded $11.8m CIRM Grant
anova evidence
2024-04-30
glioma
Anova, a technology enabled CRO dedicated to accelerating promising treatments, has collaborated with University of California at San Francisco (UCSF), University of California at San Diego (UCSD), the University of Southern California (USC) and Denovo Biopharma to manage a newly awarded $11.8M California Institute for Regenerative Medicine (CIRM) grant to support the development of DB107 in patients with newly diagnosed high-grade glioma. The post Anova to Manage Study of DB107 Following Newly Awarded $11.8m CIRM Grant appeared first on Anova .
Cellectis Reports Financial Results for the Fourth Quarter and Full Year 2023
cellectis
2024-04-29
cd19
car t
autologous
gene editing
cell therapies
gene therapies
financial results
Preliminary results of NATHALI-01 and updated results of BALLI-01 Phase I clinical studies presented at the American Society of Hematology (ASH) 65th Annual Meeting Execution of strategic collaboration and investment agreements with AstraZeneca Cécile Chartier, Ph.D., appointed as a director of the Cellectis’ Board of Directors Drawdown of the second tranche of €15 million under the credit facility agreement entered into with the European Investment Bank (EIB) Cash position of $156 million as of December 31, 2023 [1] [1] Cash position includes cash, cash equivalents, restricted cash and fixed-term deposits classified as current -financial assets . Restricted cash was $5 million as of December 31, 2023. Fixed-term deposits classified as current-financial assets was $15 million as of December 31, 2023. New York, NY – April 29, 2024 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, today provided business updates and reports preliminary financial results for the fourth quarter and full year 2023, ending December 31, 2023. “Cellectis…
A2 Bio to Highlight Progress of CAR T Clinical Programs in Three Poster Presentations at 2024 American Society of Clinical Oncology (ASCO) Annual Meeting
A2 Bio
2024-04-24
car t
The post A2 Bio to Highlight Progress of CAR T Clinical Programs in Three Poster Presentations at 2024 American Society of Clinical Oncology (ASCO) Annual Meeting appeared first on A2 Biotherapeutics .
US FDA accepts for priority review GSK’s application for an expanded indication of Jemperli (dostarlimab) plus chemotherapy to include all adult patients with primary advanced or recurrent endometrial cancer
GSK
2024-04-24
priority review
endometrial cancer
Application supported by statistically significant and clinically meaningful progression-free and overall survival data from RUBY trial
Cellectis Presents Novel TALEN® Editing Processes Enabling Highly Efficient Gene Correction and Gene Insertion in HSPCs
cellectis
2024-04-22
talen
cell therapy
gene editing
gene therapy
gene therapies
This novel editing approach might unlock new strategies for the treatment of metabolic and neurological diseases Non-viral circular ssDNA delivery associated to TALEN® gene editing allows high levels of gene insertion in long-term repopulating HSPCs New York, NY – April 22, 2024 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, will present first data exploring novel TALEN® editing processes in hematopoietic stem and progenitor cells (HSPCs) at the American Society of Gene and Cell Therapy (ASGCT) being held on May 7-11, 2024. “These two posters showcase the potential and versatility of the TALEN® technology to promote efficient gene insertion in HSPCs. We show that circular single strand DNA templates can be efficiently delivered to HSPCs and enable unprecedented efficiency of gene insertion without compromising the viability, fitness and differentiation capacity of edited cells” commented Julien Valton, Ph.D., Vice President of Gene Therapy at Cellectis. “We also illustrate a novel TALEN® mediated-DNA template insertion approach…
EAGLE-1 phase III data show potential for gepotidacin as a new oral treatment option for uncomplicated urogenital gonorrhoea (GC) amid growing resistance to existing treatments
GSK
2024-04-17
pivotal
GSK has announced positive results from the pivotal EAGLE-1 phase III trial for gepotidacin.
Novartis Kesimpta® six-year efficacy data show substantial benefits in recently diagnosed treatment-naïve people with relapsing multiple sclerosis
Novartis
2024-04-17
multiple sclerosis
Cellectis Publishes a Novel Intronic Gene Editing Approach For the Treatment of Inborn Metabolic Diseases by Edited HSPCs
cellectis
2024-04-10
talen
gene editing
gene therapy
gene therapies
mucopolysaccharidosis type i
New York, NY – April 10, 2024 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the publication of a new research paper in Molecular Therapy , demonstrating that TALEN-mediated intron editing of hematopoietic stem and progenitor cells (HSPCs) enables transgene expression restricted to the myeloid lineage. This approach could unlock new therapeutic avenues for the treatment of inborn metabolic diseases as well as neurological diseases that require delivery of therapeutics to the brain. About HSPCs Gene editing in hematopoietic stem and progenitor cells (HSPCs) has enabled the treatment of multiple previously uncurable genetic diseases. Edited therapeutic HSPCs can engraft in the patient’s bone marrow, self-replicate, differentiate and populate other organs, propagating the therapeutic effects systemically and indefinitely after a single intervention. In this paper, Cellectis developed an intron-specific gene insertion strategy for HSPC, that restricts the expression of a therapeutic protein named IDUA to the myeloid…
Cellectis Announces Two Poster Presentations on Novel TALEN® Editing Process for Gene Correction and Gene Insertion in HSPCs at the ASGCT Annual Meeting
cellectis
2024-04-08
talen
cell therapy
gene editing
gene therapy
gene therapies
New York, NY – April 8, 2024 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that preliminary data exploring novel TALEN ® editing process in hematopoietic stem and progenitor cells (HSPCs) will be presented at the American Society of Gene and Cell Therapy (ASGCT) 27 th Annual Meeting, to be held in Baltimore, Maryland, on May 7-11, 2024. Poster presentations: Title: Circularization of Non-Viral Single-Strand DNA Template for Gene Correction and Gene Insertion Improves Editing Outcomes in HSPCs Presenter : Alex Boyne, Gene Editing Platform Manager at Cellectis Session Date/Time: May 9, 2024 at 12PM ET Session Title: Nonviral Therapeutic Gene Delivery and Synthetic/Molecular Conjugates Presentation Room: Exhibit Hall Final Abstract Number: 1235 Cellectis presents the development of a novel gene editing process, leveraging the TALEN ® technology and non-viral DNA template delivery, enabling highly efficient gene correction and gene insertion in hematopoietic stem and progenitor cells (HSPCs). Title: Intron Editing of…
Arsenal Biosciences Announces Presentation of Four Abstracts at AACR Annual Meeting Highlighting New CAR T-Focused Research
arsenal biosciences
2024-04-05
car t
solid tumor
cell therapy
cell therapies
Company to share preclinical data on the utility of engineered T cells intended for the treatment of ovarian, kidney, and other solid tumor cancers South San Francisco, Calif. – April 5, 2024 – Arsenal Biosciences, Inc. (ArsenalBio), a clinical-stage programmable cell therapy company focused on engineering advanced CAR T-cell therapies for solid tumors, today […]
Nobel laureate Carolyn Bertozzi and Acepodia Co-Founder Sonny Hsiao on the science of antibody-cell conjugates
Acepodia
2024-04-03
allogeneic
Dr. Bertozzi and Dr. Hsiao describe how Acepodia is leveraging biorthogonal chemistry to conjugate antibodies to allogeneic γδ2 T cells.
Eureka Therapeutics Announces UCSF Benioff Children’s Hospitals as First California Site for ARYA-2 Clinical Trial Targeting Pediatric Liver Cancers
Eureka Therapeutics
2024-04-02
cell therapy
cell therapies
Emeryville, California, April 2nd, 2024 – Eureka Therapeutics, Inc., a clinical-stage biotechnology company developing novel T-cell therapies to treat cancer, today announced the activation of UCSF Benioff Children’s Hospitals as the first California site to join the ARYA-2 clinical trial (NCT04634357). The ARYA-2 trial is evaluating Eureka’s investigational ARTEMIS® ET140203 T-cell therapy which targets the The post Eureka Therapeutics Announces UCSF Benioff Children’s Hospitals as First California Site for ARYA-2 Clinical Trial Targeting Pediatric Liver Cancers appeared first on Eureka .
FDA Grants Accelerated Approval to Zanubrutinib Plus Obinutuzumab for Relapsed or Refractory Follicular Lymphoma
ASCO Post FDA
2024-03-25
cd20
follicular lymphoma
accelerated approval
Today, the FDA granted accelerated approval to the small-molecule BTK inhibitor zanubrutinib (Brukinsa) in combination with the anti-CD20 monoclonal antibody obinutuzumab for patients with relapsed or...
Unlocking Clinical Trial Recruitment Success: Bionews Presents at World Orphan Drug Congress 2024
BioNews
2024-03-20
orphan drug
The session will highlight how Bionews’ large reach and access among rare disease patients, combined with strong community activation expertise and a patient-centric approach, accelerates trial recruitment programs. During the presentation, Bionews also will share key insights from our recent groundbreaking study, the 2024 Rare Disease Survey. Bionews will be participating in the World Orphan […] The post Unlocking Clinical Trial Recruitment Success: Bionews Presents at World Orphan Drug Congress 2024 appeared first on Bionews .
CRX100 Elicits Activity, Is Well Tolerated in Recurrent, Platinum-Resistant Ovarian Cancer
bioeclipse therapeutics
2024-03-19
cell therapy
ovarian cancer
Combination treatment with the novel adoptive Natural Killer-Like T (NK-T) cell therapy CRX100 and a tumor-specific oncolytic vaccinia virus generated responses and was well tolerated in a small cohort of patients with recurrent, platinum-resistant ovarian cancer, according to data from a phase 1 trial (NCT04282044) presented at the 2024 SGO Annual Meeting on Women’s Cancer.
Forge Biologics’ Novel AAV Gene Therapy FBX-101 for Patients with Krabbe Disease is Granted UK’s Innovation Passport Designation
Forge Biologics
2024-03-19
aav
gene therapy
krabbe disease
Designation grants Forge's FBX-101 therapy access to enter the Innovative Licensing and Access Pathway (ILAP). The post Forge Biologics’ Novel AAV Gene Therapy FBX-101 for Patients with Krabbe Disease is Granted UK’s Innovation Passport Designation appeared first on Forge Biologics .
Eureka Therapeutics Advances ARYA-3 Clinical Trial to Phase II for Treatment of Liver Cancer Using GPC3-Targeting ARTEMIS® T-cell Therapy
Eureka Therapeutics
2024-03-18
gpc3
cell therapy
cell therapies
Emeryville, California, March 18th, 2024 – Eureka Therapeutics, Inc., a clinical-stage biotechnology company developing novel T-cell therapies to treat cancer, today announced the advancement of its ARYA-3 clinical trial to Phase II (NCT04864054). This milestone represents one of the first engineered T-cell programs targeting solid tumors to reach Phase II. The ARYA-3 trial is evaluating The post Eureka Therapeutics Advances ARYA-3 Clinical Trial to Phase II for Treatment of Liver Cancer Using GPC3-Targeting ARTEMIS® T-cell Therapy appeared first on Eureka .
GSK announces positive results from DREAMM-8 phase III trial for Blenrep versus standard of care combination in relapsed/refractory multiple myeloma
GSK
2024-03-07
multiple myeloma
Trial unblinded early at an interim analysis based on Independent Data Monitoring Committee recommendation
A dual-receptor T-cell platform with Ab-TCR and costimulatory receptor achieves specificity and potency against AML
Eureka Therapeutics
2024-03-04
aml
Blood. 8 February 2024: Tao Dao, Guangyan Xiong, Sung Soo Mun, Jeremy Meyerberg, Tatyana Korontsvit, Jingyi Xiang, Ziyou Cui , Aaron Y Chang, Casey Jarvis, Winson Cai, Hanzhi Luo, Aspen Pierson, Anthony Daniyan, Sarah Yoo, Sumiko Takao, Michael Kharas, Alex Kentsis, Cheng Liu, David A Scheinberg The post A dual-receptor T-cell platform with Ab-TCR and costimulatory receptor achieves specificity and potency against AML appeared first on Eureka .
Novel CD19-specific γ/δ TCR-T cells in relapsed or refractory diffuse large B-cell lymphoma
Eureka Therapeutics
2024-02-15
cd19
tcr-t
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
Journal of Hematology & Oncology. 21 January 2023: Chenggong Li, Fen Zhou, Jing Wang, Qi Chang, Mengyi Du, Wenjing Luo, Yinqiang Zhang, Jia Xu, Lu Tang, Huiwen Jiang, Lin Liu, Haiming Kou, Cong Lu, Danying Liao, Jianghua Wu, Qiuzhe Wei, Sha Ke, Jun Deng, Cheng Liu, Heng Mei & Yu Hu The post Novel CD19-specific γ/δ TCR-T cells in relapsed or refractory diffuse large B-cell lymphoma appeared first on Eureka .
GSK receives US FDA Fast Track designation for bepirovirsen in chronic hepatitis B
GSK
2024-02-12
fast track
Designation underscores the unmet need for medicines that can achieve functional cure in patients with chronic hepatitis B (CHB)
GSK’s RSV vaccine, Arexvy, accepted under Priority Review in US for the prevention of RSV disease in adults aged 50-59 at increased risk
GSK
2024-02-06
priority review
GSK is the first company to file for regulatory approval to extend RSV vaccination to adults aged 50-59 at increased risk.
DREAMM-7 phase III trial shows Blenrep combination nearly tripled median progression-free survival versus standard of care combination in patients with relapsed/refractory multiple myeloma
GSK
2024-02-05
multiple myeloma
Results from an interim analysis of the DREAMM-7 phase III head-to-head trial.
Acepodia Announces FDA Clearance of Investigational New Drug Application for ACE2016, a First-in-Class Allogeneic Anti-EGFR Cell Therapy
Acepodia
2024-02-05
allogeneic
cell therapy
cell therapies
/PRNewswire/ -- Acepodia (6976:TT), a clinical stage biotechnology company developing first-in-class cell therapies with its unique Antibody-Cell Conjugation...
Forge Biologics Announces Positive FBX-101 Clinical Trial Update in Patients with Krabbe Disease Identified by Newborn Screening Ahead of RUSP Vote
Forge Biologics
2024-01-29
krabbe disease
Five patients have received FBX-101, all of whom were identified by newborn screening. The post Forge Biologics Announces Positive FBX-101 Clinical Trial Update in Patients with Krabbe Disease Identified by Newborn Screening Ahead of RUSP Vote appeared first on Forge Biologics .
PeproMene Bio, Inc. Announces Complete Remission of the Cohort 1 First Patient Treated for Relapsed and Refractory B-cell Acute Lymphoblastic Leukemia (B-ALL) in the Phase 1 Clinical Trial of PMB-CT01 (BAFFR-CAR T Cells) at City of Hope
pepromene bio
2024-01-23
b-all
car t
leukemia
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
IRVINE, Calif., Jan. 18, 2024 /PRNewswire/ — PeproMene Bio, Inc., a clinical-stage biotech company developing novel therapies to treat cancers and immune disorders, today announced that the cohort 1 first patient treated in its Phase 1 relapsed or refractory B-cell acute lymphoblastic … Continue reading → The post PeproMene Bio, Inc. Announces Complete Remission of the Cohort 1 First Patient Treated for Relapsed and Refractory B-cell Acute Lymphoblastic Leukemia (B-ALL) in the Phase 1 Clinical Trial of PMB-CT01 (BAFFR-CAR T Cells) at City of Hope appeared first on PeproMene Bio Inc .
Cellectis announces the drawdown of the second tranche of €15 million under the credit facility agreement entered with the European Investment Bank (EIB)
cellectis
2024-01-16
car t
allogeneic
gene therapies
New York, NY – January 16, 2024 - Cellectis (Euronext Growth: ALCLS – NASDAQ: CLLS) (the “Company”), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it has drawn down the second tranche of €15 million (“Tranche B”) under the credit facility agreement for up to €40 million entered into with the European Investment Bank (the “EIB) on December 28, 2022 (the "Finance Contract"). Tranche B is expected to be disbursed by the EIB by January 25, 2024. The Company plans to use the proceeds of Tranche B towards the development of its pipeline of allogeneic CAR T-cell product candidates: UCART22, UCART20x22, and UCART123. As a condition to the disbursement of Tranche B the Company issued 1,460,053 warrants to the benefit of the EIB, in accordance with the terms of the 14 th resolution of the shareholders’ meeting held on June 27, 2023 and articles L. 228-91 and seq. of the French Commercial Code (the “Tranche B Warrants”). Each Tranche B Warrant allows the EIB to subscribe for one ordinary share of the Company, at a price of €2.53, corresponding to 99% of the volume-weighted average price…
US FDA Open To ‘Compromise’ In Regulating Pancreatic Islet Cell Transplantation
CellTrans Inc.
2024-01-12
open
FDA’s Peter Marks says there may be a way to reduce the regulatory burden for pancreatic islet cell treatment for diabetes. Researchers in Canada and Europe note access to therapy is limited in the US since FDA regulates islet cells as a drug while other countries regulate them as a tissue or organ.
Arsenal Biosciences to Present at the 42nd Annual J.P. Morgan Healthcare Conference
arsenal biosciences
2024-01-02
car t
cell therapy
cell therapies
South San Francisco, Calif. – January 2, 2024 – Arsenal Biosciences, Inc. (ArsenalBio), a clinical stage programmable cell therapy company engineering advanced CAR T-cell therapies for solid tumors, today announced that Ken Drazan, MD, the company’s Chairman and Chief Executive Officer, will present a corporate update at the 42nd Annual J.P. Morgan Healthcare Conference on […]
Cellectis Reports Results from Shareholders Meeting Held on December 22, 2023
cellectis
2023-12-22
gene therapies
December 22, 2023 – New York (N.Y.) – Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, held today a shareholders general meeting at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4 th floor, 75013 Paris, France. At the meeting, resolutions 1 through 5 as well as resolutions 7 and 8 were adopted and resolution 6 was rejected, consistent with the recommendations of the board of directors. Cellectis’ shareholders supported the contemplated $140M additional investment by AstraZeneca in the Company, to be effected by way of subscription of 10,000,000 newly created “class A” convertible preferred shares and 18,000,000 newly created “class B” convertible preferred shares, in each case at a price of $5.00 per share (the “Additional Investment”). The completion of the Additional Investment remains subject to the clearance of such investment from the French Ministry of Economy pursuant to applicable foreign direct investment regulations, and other customary closing conditions. The Company’s shareholders also approved the appointments of Mr. Marc Dunoyer…
Scientific publication update: ASH meeting abstract 2023 (Promising Safety and Anti-Lymphoma Efficacy of Autologous Pmb-CT01 (BAFFRCAR T Cell) Therapy in a First-in-Human Phase 1 Study)
pepromene bio
2023-12-15
autologous
Irvine, CA, December 15, 2023 – An oral and poster abstract, “Promising Safety and Anti-Lymphoma Efficacy of Autologous Pmb-CT01 (BAFFRCAR T Cell) Therapy in a First-in-Human Phase 1 Study”, has been published at American Society of Hematology Meeting 2023. … Continue reading → The post Scientific publication update: ASH meeting abstract 2023 (Promising Safety and Anti-Lymphoma Efficacy of Autologous Pmb-CT01 (BAFFRCAR T Cell) Therapy in a First-in-Human Phase 1 Study) appeared first on PeproMene Bio Inc .
Bionews empowers Friedreich’s Ataxia community with inaugural live webinar
BioNews
2023-12-05
friedreich's ataxia
Panel of experts and patients provided tips on management, treatment, and financial planning for Friedreich's Ataxia patients and caregivers. The post Bionews empowers Friedreich’s Ataxia community with inaugural live webinar appeared first on Bionews .
GSK announces positive results from DREAMM-7 head-to-head phase III trial for Blenrep in relapsed/refractory multiple myeloma
GSK
2023-11-27
multiple myeloma
Blenrep plus BorDex showed statistically significant progression-free survival (PFS) benefit versus daratumumab plus BorDex.
First-Line Dabrafenib Plus Trametinib vs Standard Chemotherapy for Pediatric Low-Grade Glioma With BRAF V600 Mutations
ASCO Post Journals
2023-11-25
glioma
Dabrafenib plus trametinib shows significant improvement in response rate, progression-free survival, and safety in pediatric patients with BRAF V600–mutant low-grade glioma. The study was funded by Novartis.
Cellectis’ Shareholders Meeting to be Held on December 22, 2023
cellectis
2023-11-17
gene therapies
November 17, 2023 – New York (N.Y.) – Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that it will hold a general meeting on December 22, 2023 at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4 th floor, 75013 Paris, France. The notice of meeting as published today in the BALO (Bulletin des Annonces Legales Obligatoires), including the agenda, the text of the resolutions and the terms of participation, is online on the Cellectis website at the following address: https://www.cellectis.com/en/investors/general-meetings/
Athersys Reports Third Quarter 2023 Financial Results and Business Highlights
athersys
2023-11-16
financial results
Alaunos Therapeutics Announces Third Quarter 2023 Financial Results, Phase 1 Clinical Data and Continued Exploration of Strategic Alternatives
Alaunos
2023-11-14
tcr-t
phase 1/2
cell therapy
financial results
TCR-T Library Phase 1/2 trial achieved an 87% disease control rate of eight evaluable patients with metastatic, refractory solid tumors; TCR-T cell therapy was well tolerated in all treated patients hunTR® discovered new HLA class I and class II restricted TCRs that recognize driver mutations,
Cellectis Reports Financial Results for Third Quarter and First Nine Months 2023
cellectis
2023-11-06
aml
car t
allogeneic
gene editing
gene therapy
gene therapies
financial results
Strategic Collaboration and Investment Agreements signed with AstraZeneca Updated results of the Phase I BALLI-01 Trial evaluating UCART22 in r/r B-cell ALL and preliminary results of the Phase I NATHALI-01 Trial evaluating UCART20x22 in r/r B-cell NHL to be presented at ASH 65 th Annual meeting. Clinical trials ongoing: BALLI-01 (evaluating UCART22), NATHALI-01 (evaluating UCART20x22) and AMELI-01 (evaluating UCART123) studies for patients with r/r B-cell ALL, r/r B-cell NHL and r/r AML, respectively. Preclinical data on HSPC gene therapy and a comprehensive analysis of TALE-BE editing determinants presented at ESGCT 2023 30 th annual congress. Preclinical data on Multi-armored Allogeneic MUC1-CAR T-cells targeting Triple-Negative Breast at SITC 2023 annual event. Cash position of $72 [1] million as of September 30, 2023. Cash runway into 2026 including Initial AstraZeneca Investment and Potential Additional AstraZeneca Investment. Conference call scheduled for 8.00am ET / 2.00 pm CET on November 7, 2023 New York, NY – November 6, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing…
Cellectis to Present Preliminary Results of NATHALI_01 and Updated Results of the BALLI_01 Phases I Trials at the American Society of Hematology (ASH) 65th Annual Meeting
cellectis
2023-11-02
cd20
cd22
b-all
car-t
icans
leukemia
allogeneic
gene therapies
hodgkin lymphoma
non-hodgkin lymphoma
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
New York, NY – November 2, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that preliminary results of the Phase I NATHALI-01 clinical trial evaluating UCART20x22 in patients with relapsed or refractory non-Hodgkin lymphoma (r/r NHL) and updated results of the Phase I BALLI-01 clinical trial evaluating UCART22 in patients with relapsed or refractory CD22+ B-cell acute lymphoblastic leukemia, (r/r B-ALL) will be presented at the American Society of Hematology 65 th Annual Meeting (ASH 2023), that will take place on December 9-12, 2023 in San Diego (CA) and online. These data will be presented in two poster sessions: Poster Presentation (P2110) Title: Preliminary Results of Nathali-01: A First-in-Human Phase I/IIa Study of UCART20x22, a Dual Allogeneic CAR-T Cell Product Targeting CD20 and CD22, in Relapsed or Refractory (R/R) Non-Hodgkin Lymphoma (NHL) Session Name: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: Poster I Presenter: Dr. Jeremy Abramson (Massachusetts General Hospital…
Cellectis Announces Strategic Collaboration and Investment Agreements with AstraZeneca
cellectis
2023-11-01
ind
gene editing
gene therapy
Collaboration leverages Cellectis’ gene editing technologies and manufacturing capabilities to develop up to 10 novel cell & gene therapy candidate products Cellectis to receive up to $245M in cash (up to $220M equity investment and $25M upfront payment), with potential for additional milestones, plus tiered royalties New York, NY – November 1, 2023 - Cellectis (Euronext Growth: ALCLS – NASDAQ: CLLS) today announced it has entered into (i) a Joint Research Collaboration Agreement (the “Collaboration Agreement”, (ii) an investment agreement relating to an initial equity investment of $80M, and (iii) a memorandum of understanding (the “MOU”) relating to an additional equity investment of $140M, with AstraZeneca (LSE/STO/Nasdaq: AZN). The Collaboration Agreement aims to accelerate the development of next generation therapeutics in areas of high unmet need, including oncology, immunology and rare diseases. Under the terms of the Collaboration Agreement, AstraZeneca will leverage Cellectis’ proprietary gene editing technologies and manufacturing capabilities to design novel cell and gene therapy candidate products. As part of the Collaboration Agreement, 25 genetic targets have been…
Cellectis to Report Third Quarter 2023 Financial Results on November 6, 2023
cellectis
2023-11-01
gene therapies
financial results
New York, NY – November 1, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the third quarter ended September 30, 2023 on Monday, November 6, 2023 after the close of the US market. The announcement will be followed by an investor conference call and webcast on Tuesday, November 7, 2023 at 8:00 AM ET / 2:00 PM CET. The call will include the Company’s third quarter results and an update on business activities. Details for the call are as follows: Dial in information: Domestic: 1-877-451-6152 International: 1-201-389-0879 Conference ID: 13741035 Webcast Link: https://viavid.webcasts.com/starthere.jsp?ei=1632799&tp_key=bff99741ab
Cellectis Presents Pre-Clinical Data on Multi-armored Allogeneic MUC1-CAR T-cells Targeting Triple-Negative Breast Cancer at the Society for Immunotherapy of Cancer (SITC) 38th Annual Meeting
cellectis
2023-10-31
car t
car-t
talen
allogeneic
cell therapy
gene editing
breast cancer
gene therapies
New York, NY – October 31, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that pre-clinical data on MUC1-CAR T-cells to overcome key challenges of targeting solid tumors, will be presented at the Society for Immunotherapy of Cancer’s 38 th Annual Meeting (SITC 2023), that will take place on November 1-5, 2023 at San Diego Convention Center in San Diego (CA). The data will be presented by Laurent Poirot, Ph.D., SVP Immunology of Cellectis, in a poster session that will be held November 4 th 2023 from 9:00 a.m. to 8:30 p.m. PDT, at San Diego Convention Center, Hall A. The poster is number 254 and it is entitled “ TGF-Beta Blockade Combined with Activation-Induced IL12 Secretion Synergize to Optimize Potency of MUC1-CAR T-cells in Preclinical Targeting of Triple-Negative Breast Cancer”. While during SITC 2022 Cellectis presented how TALEN®-mediated gene editing allows programming of various functions addressing both safety and potency aspects of allogenic CAR T-cell therapy, this year the company’s presentation…
Cellectis to Present Pre-Clinical Data on HSPC Gene Therapy Program and Comprehensive Analysis of TALE-BE at the ESGCT 30th Annual Congress
cellectis
2023-10-24
talen
cell therapy
gene editing
gene therapy
gene therapies
New York, NY – October 24, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that they will be showcasing pre-clinical data on its program of gene therapy for HSPC as well as comprehensive analysis of TALE-BE editing determinants at the European Society of Gene and Cell Therapy (ESGCT) 30 th annual congress that will take place on October 24-27, 2023 in Brussels, Belgium. The data will be presented in three posters: Intronic editing enables lineage specific expression of therapeutics relevant for HSPC gene therapy (Poster N°646) Presenter: Eduardo Seclen, Senior Scientist & Team Leader, Gene Editing Date/Time: Wednesday October 25 th from 18:15 to 19:30 and Thursday October 26 th from 19:30 to 20:30 Intronic editing enables lineage specific expression of therapeutics relevant for HSPC gene therapy. TALEN®-mediated intron editing of the CD11b locus results in the lineage-specific expression of a reporter transgene in myeloid cells, with negligible expression in HSPC or other cellular subsets in vitro and in vivo .…
Cellectis and Imagine Institute Publish A Proof-Of-Concept Study of a Gene Surgery Candidate to Treat Activated Phosphoinositide 3-Kinase Δ Syndrome Type 1 (APDS1)
cellectis
2023-10-12
aav
talen
allogeneic
gene editing
gene therapies
October 12, 2023 - New York, NY – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the publication of a new research paper in Molecular Therapy – Methods & Clinical Development , demonstrating the efficacy of its TALEN-mediated gene correction of mutated PIK3CD gene in APDS1 T-cells. The research work described in this article was jointly conducted by Imagine Institute and Cellectis teams. About APDS1: Activated phosphoinositide 3-kinase δ syndrome (also known as APDS type 1 or APDS1) is a rare but devastating disease caused by gain-of-function mutations in the PIK3CD gene and resulting in a combined immunodeficiency. Approved treatments for APDS1 consist in prophylactic measures including long term antibiotics and Ig (immunoglobulin) replacement therapy. Allogeneic hematopoietic stem/progenitor cell (HSPC) transplantation has been proposed as a definitive treatment for APDS1. However, the lack of compatible donor as well as graft failure, graft instability, and poor graft function are still major challenges that must…
Athersys Reports Interim Analysis Results of MASTERS-2 Clinical Study with MultiStem in Ischemic Stroke, Signs Memorandum of Understanding (MOU) for Global ARDS License with Healios
athersys
2023-10-10
ischemic stroke
Cellectis to Present Pre-Clinical Data on Multi-armored Allogeneic MUC1-CAR T-cells Targeting Triple-Negative Breast Cancer at the Society for Immunotherapy of Cancer (SITC) 38th Annual Meeting
cellectis
2023-09-27
car t
talen
allogeneic
breast cancer
gene therapies
New York, NY – September 27, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that pre-clinical data on MUC1-CAR T-cells to overcome key challenges of targeting solid tumors, will be presented at the Society for Immunotherapy of Cancer’s 38 th Annual Meeting (SITC 2023), that will take place on November 1-5, 2023 at San Diego Convention Center in San Diego (CA). Poster Presentation: Laurent Poirot, SVP Immunology of Cellectis, will present a poster on the benefits of combining potency attributes for TALEN ® -edited smart CAR T-cells targeting MUC1 in preclinical models of triple-negative breast cancer. Title : TGF-Beta Blockade Combined with Activation-Induced IL12 Secretion Synergize to Optimize Potency of MUC1-CAR T-cells in Preclinical Targeting of Triple-Negative Breast Cancer. Poster Number : 254 Presenter: Laurent Poirot, Ph.D., SVP Immunology, Cellectis. Date/Time : Poster will be on display Saturday, November 4 th , 2023 from 9:00 a.m. to 8:30 p.m. PDT, at San Diego Convention Center, Hall A. Full text of…
FDA Approves Melphalan Hepatic Delivery System for Adult Patients With Unresectable Hepatic-Dominant Metastatic Uveal Melanoma
ASCO Post FDA
2023-09-25
melanoma
On August 14, the U.S. Food and Drug Administration (FDA) approved the HEPZATO KIT, a melphalan hepatic delivery system, as a liver-directed treatment for adult patients with metastatic uveal melanoma...
Ipilimumab Plus Nivolumab May Improve Outcomes in Refractory Metastatic Melanoma, Study Suggests
ASCO Post Journals
2023-09-25
melanoma
Researchers have discovered that the combination of the CTLA-4 inhibitor ipilimumab and the PD-1 inhibitor nivolumab may be an effective second-line therapy in patients with PD-1 blockade–refractory ...
AIVITA Biomedical Announces Case Report Suggesting Positive Collateral Benefits in Systemic Lupus Erythematosus Achieved Through the Use of Its Dendritic Cell Vaccine Kit
Aivita BioMedical
2023-09-21
lupus
The post AIVITA Biomedical Announces Case Report Suggesting Positive Collateral Benefits in Systemic Lupus Erythematosus Achieved Through the Use of Its Dendritic Cell Vaccine Kit appeared first on AIVITA Biomedical .
Lantidra Chronology: 19 Years From IND Submission To Approval For The Cellular Therapy
CellTrans Inc.
2023-09-19
ind
lantidra
Pink Sheet’s Drug Review Profile looks at the timeline behind the development and FDA review of CellTrans’ treatment for type 1 diabetes.
Lantidra’s First-Cycle Complete Response Letter Centered On Inspection And Product Quality Issues
CellTrans Inc.
2023-09-16
lantidra
cell therapy
complete response
Although the CRL delayed the cell therapy’s approval by almost two years, additional CMC-related testing requested by the FDA ultimately came to be seen as a good thing by sponsor CellTrans because it provided assurance of product consistency.
PeproMene Bio, Inc. Announced Completion of the First Dose Cohort and Opening of the Second Dose Cohort in Its B-cell Non-Hodgkin Lymphoma (B-NHL) Phase 1 Clinical Trial of PMB-CT01 (BAFFR-CAR T Cells)
pepromene bio
2023-09-13
car t
hodgkin lymphoma
non-hodgkin lymphoma
IRVINE, Calif., Sept. 11, 2023 /PRNewswire/ — PeproMene Bio, Inc., a clinical-stage biotech company developing novel therapies to treat cancers and immune disorders, today announced that the first dose cohort of its phase 1 relapsed or refractory B-cell Non-Hodgkin Lymphoma (r/r B-NHL) … Continue reading → The post PeproMene Bio, Inc. Announced Completion of the First Dose Cohort and Opening of the Second Dose Cohort in Its B-cell Non-Hodgkin Lymphoma (B-NHL) Phase 1 Clinical Trial of PMB-CT01 (BAFFR-CAR T Cells) appeared first on PeproMene Bio Inc .
CellTrans’ Lantidra: Type 1 Diabetes Cell Therapy Overcame The Odds On Nontraditional Path To Approval
CellTrans Inc.
2023-09-12
bla
lantidra
cell therapy
Despite a host of challenges with donislecel’s development and BLA, the US FDA approved based on clinical data from only 30 patients, using a clinically meaningful insulin independence endpoint and in a more carefully tailored population. Pink Sheet’s Drug Review Profile takes a deep dive into the FDA review of the first approved allogenic pancreas islet cell product.
Accelerated Approval for Elranatamab-bcmm in Patients With Relapsed or Refractory Multiple Myeloma
ASCO Post FDA
2023-09-10
bcma
multiple myeloma
accelerated approval
On August 14, the U.S. Food and Drug Administration (FDA) granted accelerated approval to elranatamab-bcmm (Elrexfio), a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager, for ad...
Anova Supports the Design and Authorization of Phase 2 Study of DB107 In Promising Turn-Around of Targeted Treatment to Treat Newly Diagnosed High Grade Glioma
anova evidence
2023-08-31
glioma
Anova Enterprises, Inc. (Anova), an organization dedicated to accelerating the development of promising treatments to market announced the FDA authorization of a Phase II clinical trial to re-assess a targeted treatment in light of newly discovered pharmacogenomic predictor data in patients with newly diagnosed high-grade glioma (HGG) The post Anova Supports the Design and Authorization of Phase 2 Study of DB107 In Promising Turn-Around of Targeted Treatment to Treat Newly Diagnosed High Grade Glioma appeared first on Anova .
Athersys Reports Second Quarter 2023 Financial Results and Business Highlights
athersys
2023-08-16
financial results
Alaunos Therapeutics Announces Second Quarter 2023 Financial Results, Interim Clinical Data and Exploration of Strategic Alternatives
Alaunos
2023-08-14
tcr-t
phase 1/2
cell therapy
financial results
TCR-T Library Phase 1/2 trial achieved an 83% disease control rate of six evaluable patients with metastatic, refractory solid tumors; TCR-T cell therapy was well tolerated in all evaluable patients Company to wind down TCR-T Library Phase 1/2 trial and concurrently explore potential partnering
Intricon will open first facility dedicated to production of sensor-driven medical devices in Costa Rica
Altaris
2023-08-10
open
Cellectis Provides Full Report for Second Quarter 2023 Financial Results
cellectis
2023-08-07
gene therapies
financial results
financial statements
New York, NY – August 7, 2023 - Cellectis S.A. (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the release of the full report of the financial results for the second quarter 2023 (ended June 30, 2023) and filing of the corresponding 6-K with the SEC. Financial Results The interim condensed consolidated financial statements of Cellectis, have been prepared in accordance with International Financial Reporting Standards, as issued by the International Accounting Standards Board (“IFRS”). We present certain financial metrics broken out between our two reportable segments – Therapeutics and Plants – in the appendices of this Q2 2023 financial results press release. On January 13, 2023, Calyxt, Cibus Global LLC (Cibus) and certain other parties named therein, entered into an Agreement and Plan of Merger (the “Merger Agreement”), pursuant to which, subject to the terms and conditions thereof, Calyxt and Cibus will merge in an all-stock transaction (the “Calyxt Merger”). As a consequence of the foregoing, Calyxt met the “held-for-sale”…
Cellectis Provides Business Updates and Preliminary Financial Results for Second Quarter 2023
cellectis
2023-08-03
aml
cd19
b-all
car t
allogeneic
cell therapy
gene therapies
financial results
financial statements
Updated clinical & translational data on BALLI-01 trial (evaluating UCART22 in r/r B-cell ALL) presented at the EHA annual meeting * Clinical trials ongoing: BALLI-01 (evaluating UCART22), NATHALI-01 (evaluating UCART20x22) and AMELI-01 (evaluating UCART123) studies for patients with r/r B-cell ALL, r/r B-cell NHL and r/r AML, respectively * Preclinical data presented on HBB gene correction of sickle cell mutation & TALE Base Editors (TALE-BE) at ISCT 2023 annual event * Appointment of Cécile Chartier, Ph.D., as Director to Cellectis’ Board of Directors * Cash position [1] of $89 million as of June 30, 2023. Cash runway into Q3 2024 * Full financial statements for the second quarter of 2023 will be released in the coming days * Conference call scheduled for 8AM ET/2PM CET on August 4, 2023 New York, NY – August 3, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today provided business updates and preliminary financial results for the six-month period ending June 30, 2023. Full report of the financial results for the second…
Fondazione Telethon announces commitment to make gene therapy for Wiskott-Aldrich syndrome available to patients
Fondazione Telethon
2023-08-02
gene therapy
wiskott-aldrich
wiskott-aldrich syndrome
This is the second gene therapy abandoned by industry that Fondazione Telethon is taking over, so that it reaches the patients who need it.
Cellectis to Report Second Quarter 2023 Financial Results on August 3, 2023
cellectis
2023-07-26
gene therapies
financial results
New York, NY – July 27, 2023 - Cellectis S.A. (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the second quarter ended June 30, 2023 on Thursday, August 3, 2023 after the close of the US market. The announcement will be followed by an investor conference call and webcast on Friday, August 4, 2023 at 8:00 am ET / 2:00 pm CET. The call will include the Company’s second quarter results and an update on business activities. Details for the call are as follows: Dial in information: Domestic: 1-877-451-6152 International: 1-201-389-0879 Conference ID: 13739252 Webcast Link: https://viavid.webcasts.com/starthere.jsp?ei=1619596&tp_key=40e9fac8fc
Cellectis Appoints Dr. Cécile Chartier, Ph.D. to its Board of Directors
cellectis
2023-07-12
ind
til
car t
allogeneic
gene editing
gene therapy
gene therapies
tumor-infiltrating lymphocytes
New York, NY – July 12, 2023 - Cellectis S.A. (the “Company”) (Euronext Growth: ALCLS- NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that during the annual shareholders meeting held on June 27, 2023, Cécile Chartier, Ph.D., has been appointed as a Director of the Company’s Board of Directors, with immediate effect. “We are very excited to welcome Dr. Chartier as a director of Cellectis’ Board of Directors. Cécile’s extensive experience in the development of next generation cell and gene therapies coupled with her deep knowledge of the U.S. biotechnology industry will be a huge asset to the Company” said André Choulika, Ph.D., Chief Executive Officer at Cellectis. “ We look forward to her contribution and insights as we continue advancing the development of our product candidates.” “I am honored to have the opportunity to contribute to the development of truly breakthrough gene editing therapies. I am keen to lend my insights to the Cellectis’ Board of Directors to serve a patient population with severe unmet medical needs, where a successful allogeneic CAR T-cell…
After First Approval, Experts Point to Barriers in Cell Therapy for Type 1 Diabetes
CellTrans Inc.
2023-07-12
cell therapy
The FDA recently approved the first cellular therapy for Type 1 diabetes and others may not be far behind. But experts say challenges still exist to the widespread application of these treatments.
Type 1 diabetes: New cell therapy reduces need for insulin
CellTrans Inc.
2023-07-11
lantidra
cell therapy
The FDA has recently approved a cellular therapy for type 1 diabetes for the first 1. The therapy, called Lantidra, can reduce the need for insulin injections.
FDA Approves First Cellular Therapy to Treat Patients with Type 1 Diabetes
CellTrans Inc.
2023-07-07
lantidra
Today, the U.S. Food and Drug Administration approved Lantidra, the
A Startup Wins the First FDA Approval of a Cell Therapy for Type 1 Diabetes
CellTrans Inc.
2023-07-02
lantidra
cell therapy
CellTrans’s Lantidra is now the first FDA-approved cell therapy for type 1 diabetes. The therapy is made from islet cells sourced from deceased donors.
Lantidra: first cell therapy approved for Type 1 diabetes
CellTrans Inc.
2023-06-30
lantidra
allogeneic
cell therapy
FDA-approved allogeneic pancreatic islet cell therapy Lantidra could provide another treatment option for Type 1 diabetes.
FDA Approves Lantidra, the First Ever Cell Therapy for Type 1 Diabetes
CellTrans Inc.
2023-06-29
lantidra
cell therapy
FDA OKs Pancreatic Islet Cell Therapy for Type 1 Diabetes
CellTrans Inc.
2023-06-29
lantidra
cell therapy
Approval of Lantidra, a beta cell therapy derived from donor pancreatic islets, came despite transplant surgeons' concerns about FDA regulating these cells as biologics rather than transplanted organs.
First FDA-Approved Islet Cell Transplant Performed
CellTrans Inc.
2023-06-29
lantidra
Key takeaways:A man with type 1 in Illinois has received the first FDA-approved islet-cell replacement treatment, Lantidra, and he is now producing his own insulin.The treatment works by restoring the body’s beta cells, potentially eliminating the need for insulin injections.The FDA approved Lantidra (donislecel) in 2023.
FDA approves novel pancreatic islet cell therapy for adults with type 1 diabetes
CellTrans Inc.
2023-06-29
lantidra
cell therapy
The FDA approved an allogenic pancreatic islet cellular therapy for adults with type 1 diabetes who struggled to achieve their target HbA1c due to repeated episodes of severe hypoglycemia, according to an agency press release. The novel cell therapy (Lantidra, CellTrans Inc.) is made from deceased donor pancreatic cells and is administered as a single infusion into the patient’s hepatic
Cellectis S.A. Reports Results from Annual Shareholders’ General Meeting Held on June 27, 2023
cellectis
2023-06-28
gene therapies
June 28, 2023 – New York (N.Y.) – Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, hold its annual general meeting on June 27, 2023 at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4 th floor, 75013 Paris, France. At the meeting, during which more than 72% of shares were exercised, Resolutions 1 through 28 were adopted and resolution 29 was rejected, according to the management recommendations. The detailed results of the vote and the resolutions are available on the company’s website: https://www.cellectis.com/en/investors/general-meetings/
NeOnc Technologies Holdings Appoints Anova to Deliver its Phase 1/2 Trial of NEO212 for Primary Brain Tumors and for Brain Metastasis in Combination with Standard of Care Therapy
anova evidence
2023-06-27
phase 1/2
NeOnc Technologies Holdings, Inc., a clinical stage biopharmaceutical company, has received authorization by the U.S. Food and Drug Administration (FDA) for the Phase 1/2 clinical study of NEO212, the Company’s drug conjugate therapeutic under development. NEO212 is designed to more efficiently deliver a DNA alkylating agent to the brain for treatment of primary brain tumors and for brain metastases that arise from other cancers. The post NeOnc Technologies Holdings Appoints Anova to Deliver its Phase 1/2 Trial of NEO212 for Primary Brain Tumors and for Brain Metastasis in Combination with Standard of Care Therapy appeared first on Anova .
GSK receives US FDA Fast Track designation for investigational vaccine against gonorrhoea
GSK
2023-06-27
fast track
Fast Track designation accelerates the vaccine candidate’s path to US FDA submission for the prevention of Neisseria gonorrhoeae infection
Cellectis Presents Updated Clinical and Translational Data on BALLI-01 at the European Hematology Association (EHA) 2023
cellectis
2023-06-08
cd19
open
b-all
car t
icans
leukemia
allogeneic
autologous
cell therapy
gene therapies
complete response
hematopoietic stem cell
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
New York, NY – June 9, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, presented today updated clinical and translational data on its clinical trial BALLI-01 (evaluating UCART22) at the European Hematology Association (EHA) 2023. The data presented support the preliminary safety and efficacy of UCART22 in a heavily pretreated relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) population. “These clinical data are very positive for patients with r/r B-ALL who have failed multiple lines of treatment options including chemotherapy, CD19 directed CAR T-cell therapy and allogeneic stem cell transplant and encourage further enrollment into the BALLI-01 clinical study” said Nicolas Boissel, M.D., Ph.D., Head of Hematology Adolescent and Young Adult Unit at Hôpital Saint-Louis, Paris, France. The poster presentation at EHA highlights the following data: BALLI-01 is a Phase 1/2a open-label study, evaluating the safety and clinical activity of UCART22 in patients with relapsed/refractory B-cell acute lymphoblastic…
GSK receives US FDA file acceptance for Jemperli (dostarlimab) plus chemotherapy for the treatment of dMMR/MSI-H primary advanced or recurrent endometrial cancer
GSK
2023-06-06
priority review
endometrial cancer
Submission accepted for Priority Review
Eureka Therapeutics Awarded $10.6 Million CIRM Grant to Support Clinical Trial of ARTEMIS® T-Cell Therapy in Pediatric Liver Cancer
Eureka Therapeutics
2023-06-06
cell therapy
cell therapies
Emeryville, California, June 6, 2023 – Eureka Therapeutics, Inc., a clinical-stage biotechnology company developing novel T-cell therapies to treat cancer, today announced that the California Institute for Regenerative Medicine (CIRM) awarded the Company a $10.6 million grant to support its ongoing ARYA-2 Phase I study of ET140203 for the treatment of pediatric patients with refractory/relapsed The post Eureka Therapeutics Awarded $10.6 Million CIRM Grant to Support Clinical Trial of ARTEMIS® T-Cell Therapy in Pediatric Liver Cancer appeared first on Eureka .
Cellectis Showcased Preclinical Data at an Oral Presentation and Two Poster Presentations at the 29th International Society for Cell & Gene Therapy (ISCT 2023) Annual Event
cellectis
2023-06-05
cd19
cd20
cd22
car t
talen
allogeneic
autologous
cell therapy
gene editing
gene therapy
cell therapies
gene therapies
Preclinical data on UCART20x22, Cellectis’ first allogeneic dual CAR T-cell product candidate to treat B-NHL, presented at an oral session Encouraging data presented on gene editing process using Cellectis TALEN® technology to develop highly efficient HBB gene correction of sickle cell mutation Comprehensive analysis to better design efficient TALE Base Editors (TALE-BE) using Cellectis’ TALEN® technology presented in a poster June 5, 2023 – New York (NY) – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, showcased preclinical data at an oral presentation and two poster presentations at the International Society for Cell & Gene Therapy (ISCT) 29th annual event that took place at the Paris Convention Center in Paris, France on May 31 - June 3, 2023. “Our breadth of scientific presence at the 29th ISCT annual event reflects the type of cutting-edge research our teams undertake, which we believe is essential to address patients’ unmet medical need. We are proud of the pre-clinical results presented and remain deeply focused on the…
Cellectis Publishes an Article in Cancer Immunology Research Demonstrating Preclinical Evidence of UCART20x22 Product Candidate to Target a Broad Spectrum of Patients with B-cell Malignancies
cellectis
2023-05-31
cd19
cd20
cd22
car t
allogeneic
autologous
cell therapy
cell therapies
gene therapies
hodgkin lymphoma
non-hodgkin lymphoma
May 31, 2023 - New York, NY – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today publishes an article in Cancer Immunology Research demonstrating pre-clinical proof-of-concept data of UCART20x22 product candidate, Cellectis’ first allogeneic dual CAR T-cell targeting the CD20 and CD22 antigens, to overcome current mechanisms of resistance to CAR T-cell therapies in B-cell Non-Hodgkin lymphoma (B-NHL), while providing a potential alternative to CD19 directed therapy. B-cell Non-Hodgkin lymphoma (B-NHL) remains one of the most common cancers worldwide, with reports an estimate of 544,000 new cases and 260,000 deaths worldwide in 2020. Despite the groundbreaking efficacy of current CAR T-cell therapies, studies on patients treated with autologous CAR T-cells are revealing several causes for relapses that includes: antigen loss, low antigen expression or insufficient CAR T-cell potency and persistence, among others. While several suitable targets to treat B-cells malignancies have been identified, CD19 has been the focus of attention…
Alaunos Therapeutics to Present Early Data from TCR-T Library Phase 1/2 Trial at 2023 American Society of Clinical Oncology Annual Meeting
Alaunos
2023-05-25
tcr-t
phase 1/2
solid tumor
cell therapy
TCR-T cell therapy shows first-in-human response for non-viral TCR-T therapy; effectively targets high frequency TP53 and KRAS driver mutations across multiple solid tumor indications Treatment was well tolerated with a manageable safety profile and persistence of TCR-T cells in peripheral blood
Cellectis’ Annual Shareholders General Meeting to be Held on June 27, 2023
cellectis
2023-05-24
gene therapies
May 24, 2023 – New York (N.Y.) – Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that it will hold its annual general meeting on June 27, 2023 at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4th floor, 75013 Paris, France. The notice convening the annual general meeting stating the detailed agenda and modalities of participation in the meeting is available on the Cellectis website: https://www.cellectis.com/en/investors/general-meetings/
Cellectis Presents Clinical Data on AMELI-01 and Preclinical Data on Multiplex Engineering for Superior Generation of CAR T-cells at ASGCT 2023
cellectis
2023-05-17
aml
open
car t
icans
leukemia
allogeneic
cell therapy
gene therapies
complete response
acute myeloid leukemia
May 17, 2023 – New York (NY) – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today presents clinical data on its Phase 1 AMELI-01 clinical trial (evaluating UCART123) that were unveiled in an oral presentation at the 64th American Society of Hematology (ASH) annual meeting, as well as preclinical data on multiplex engineering for superior generation of CAR T-cells, at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting. Oral presentation: AMELI-01, a study evaluating UCART123, an allogeneic CAR T-cell product candidate, in relapsed/refractory acute myeloid leukemia (r/r AML) The oral presentation highlights the following clinical data: Preliminary Clinical Data from the AMELI-01 Study Presented at ASH 2022 AMELI-01 is a Phase 1 open-label dose-escalation trial evaluating the safety, tolerability, expansion and preliminary activity of UCART123 given at escalating dose levels after lymphodepletion (LD) with either fludarabine and cyclophosphamide (FC) or FC with alemtuzumab (FCA) in patients with relapsed or…
ArsenalBio Announces Presentations Highlighting Preclinical Data on AB-1015 and AB-2100 at ASGCT Annual Meeting
arsenal biosciences
2023-05-16
car t
cell therapy
cell therapies
ovarian cancer
Company to share preclinical data on the development of integrated circuit T cell therapies for the potential treatment of ovarian cancer and kidney cancer SOUTH SAN FRANCISCO, Calif.—May 16, 2023—Arsenal Biosciences, Inc. (ArsenalBio), a clinical stage programmable cell therapy company engineering advanced CAR T-cell therapies for solid tumors, today announced that it will present preclinical […]
Ray Therapeutics Closes Oversubscribed $100M Series A Financing to Advance Optogenetic Gene Therapies for Blinding Diseases
ray therapeutics
2023-05-16
gene therapy
gene therapies
Top-tier syndicate led by Novo Holdings A/S and joined by Deerfield Management, Norwest Venture Partners, Platanus, MRL Ventures Fund, the therapeutics-focused corporate venture fund of Merck & Co., Inc., and existing investor, 4BIO Capital. Funds will advance Ray Therapeutics’ optogenetics gene therapy programs for blinding retinal diseases.
Cellectis Publishes Article in Frontiers in Immunology Unveiling Pre-Clinical Data on a Novel Treatment Paradigm for Successful CAR T Immunotherapy Against Stroma-rich Solid Tumors
cellectis
2023-05-12
fap
car t
car-t
talen
mesothelin
cell therapy
gene editing
mesothelioma
breast cancer
gene therapies
adoptive cell therapy
chimeric antigen receptor
May 12, 2023 - New York, NY – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today published an article in Frontiers Bioenginnering, demonstrating the efficacy of its TALEN® engineered FAP UCART-cells in cancer-associated fibroblast (CAF) depletion, reduction of desmoplasia and tumor infiltration. Adoptive cell therapy based on chimeric antigen receptor-engineered T (CAR-T) cells has proven to be lifesaving for many cancer patients. However, its therapeutic efficacy has so far been restricted to only a few malignancies, with solid tumors proving to be especially recalcitrant to efficient therapy. Poor intra-tumor infiltration by T cells and T cell dysfunction due to a desmoplastic, immunosuppressive microenvironment are key barriers for CAR T-cell success against solid tumors. - Cancer-associated fibroblasts (CAFs) are critical components of the tumor stroma, evolving specifically within the tumor microenvironment (TME). The CAF secretome is a significant contributor to the extracellular matrix and a plethora of cytokines and growth…
GSK presents pivotal data at ESPID confirming effectiveness of its 5-in-1 meningococcal ABCWY vaccine candidate, with demonstrated coverage against a panel of 110 MenB strains
GSK
2023-05-12
pivotal
GSK today presented preliminary positive results from the phase III trial
Cellectis Announces Poster Presentation on BALLI-01 at the European Hematology Association (EHA) 2023
cellectis
2023-05-11
cd19
cd22
open
b-all
car t
talen
leukemia
allogeneic
lentiviral
cell therapy
gene therapies
chimeric antigen receptor
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
BALLI-01 (evaluating UCART22) abstract demonstrates safety and preliminary efficacy of UCART22 in heavily pretreated r/r B-ALL population New York, NY – May 11, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced the release of an abstract, which was accepted for presentation at the European Hematology Association (EHA) Hybrid Congress, taking place on June 8-15, 2023 in Frankfurt, Germany. Cellectis will present, in a poster session, updated clinical and translational data on its BALLI-01 clinical trial (evaluating UCART22) in patients with relapsed/refractory B-cell acute lymphoblastic Leukemia (r/r B-ALL). “Cellectis is excited to present updated clinical and translational data from its BALLI-01 clinical trial (evaluating UCART22) in patients with relapsed/refractory B-cell acute lymphoblastic leukemia. These data are very encouraging for patients who have limited, if any, treatment options, especially for those who have failed prior CD19 directed CAR T-cell therapy and allogeneic stem cell transplant” said Mark…
Alaunos Therapeutics Reports First Quarter 2023 Financial Results
Alaunos
2023-05-10
tcr-t
precigen
phase 1/2
precigen
inc.
financial results
Actively enrolling patients and manufacturing cryopreserved products for TCR-T Library Phase 1/2 trial; expect to report interim data in the third quarter of 2023 Fully prepaid loan with Silicon Valley Bank; now a debt-free company Amended exclusive licensing agreement with Precigen, Inc.,
FDA Approves Polatuzumab Vedotin-piiq Plus R-CHP for Previously Untreated DLBCL–Not Otherwise Specified and High-Grade B-Cell Lymphoma
ASCO Post FDA
2023-05-10
dlbcl
b-cell lymphoma
On April 19, the U.S. Food and Drug Administration (FDA) approved the antibody-drug conjugate polatuzumab vedotin-piiq (Polivy) with a rituximab product, cyclophosphamide, doxorubicin, and prednisone ...
FDA Approves Omidubicel-onlv to Reduce Time to Neutrophil Recovery and Infection in Patients With Hematologic Malignancies
ASCO Post FDA
2023-05-10
omisirge
On April 17, the U.S. Food and Drug Administration (FDA) approved omidubicel-onlv (Omisirge) for use in adult and pediatric patients aged 12 years and older with hematologic malignancies who are plann...
Cellectis Provides Business Update and Reports Financial Results for First Quarter 2023
cellectis
2023-05-04
car t
talen
leukemia
allogeneic
gene therapies
financial results
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
First r/r ALL patient dosed in Europe with Cellectis’ UCART22 product candidate manufactured in-house Cellectis implements CLLS52 for the first time in the clinic with Sanofi’s alemtuzumab Cellectis stops enrollment and treatment of patients in the MELANI-01 clinical trial evaluating UCARTCS1 product candidate in r/r MM Encouraging preclinical data on TALEN ® -edited MUC1 CAR T-cells presented at the AACR 2023 annual meeting Two abstracts accepted for oral and poster presentations at the upcoming ASGCT annual meeting Cash position [1] of 88$ million as of March 31, 2023 Conference call scheduled for 8AM ET/2PM CET on May 5, 2023 New York, NY – May 4, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today provided a business update and announced its results for the three-month period ending March 31, 2023. “ Cellectis took a notable step forward this quarter with the first patient being dosed in France with our in-house manufactured product candidate UCART22 in the BALLI-01 clinical study. UCART22 is currently the most advanced…
Cellectis Announces Oral Presentation on AMELI-01 and Poster Presentation on Multiplex Engineering for Superior Generation of CAR T-cells at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting
cellectis
2023-05-02
aml
open
car t
car-t
cd123
talen
leukemia
allogeneic
lentiviral
solid tumor
cell therapy
gene therapies
acute myeloid leukemia
chimeric antigen receptor
New York, NY – May 2, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will present clinical data on its Phase 1 AMELI-01 clinical trial (evaluating UCART123) that were presented in an oral presentation at the 64th American Society of Hematology (ASH) annual meeting, as well as preclinical data on multiplex engineering for superior generation of CAR T-cells, at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place May 16-20, 2023 in Los Angeles, CA. Oral presentation: AMELI-01, a study evaluating UCART123, an allogeneic CAR T-cell product candidate, in relapsed/refractory acute myeloid leukemia (r/r AML) The presentation includes preliminary clinical data from the Phase 1, open-label, dose-escalation trial, AMELI-01, in patients with r/r AML administered UCART123 following lymphodepletion (LD) with either fludarabine and cyclophosphamide (FC) or FC with alemtuzumab (FCA). The data show that adding alemtuzumab to the FC regimen was associated with improved LD and significantly…
Cellectis to Report First Quarter 2023 Financial Results on May 4, 2023
cellectis
2023-04-27
gene therapies
financial results
New York, NY — April 27, 2023 — Cellectis (Euronext Growth: ALCLS - Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies , today announced that it will report financial results for the first quarter ended March 31, 2023, on Thursday, May 4, 2023 after the close of the US market. The announcement will be followed by an investor conference call and webcast on Friday, May 5, 2023 at 8:00 am ET / 2:00 pm CET. The call will include the Company’s first quarter results and an update on business activities. Details for the call are as follows: Dial-in Information: Domestic: 1-877-451-6152 International: 1-201-389-0879 Conference ID: 13737755 Webcast Link: https://viavid.webcasts.com/starthere.jsp?ei=1608110&tp_key=92e9a57eb8
Alaunos Therapeutics Announces Poster Presentation at the American Society of Clinical Oncology (ASCO) 2023 Annual Meeting
Alaunos
2023-04-26
cell therapy
HOUSTON, April 26, 2023 (GLOBE NEWSWIRE) -- Alaunos Therapeutics, Inc. (“Alaunos” or the “Company”) (Nasdaq: TCRT), a leading T-cell receptor (TCR) cell therapy company advancing a clinical-stage pipeline of therapeutics for solid tumors, today announced a poster presentation for its lead non-viral
European Medicines Agency validates marketing authorisation application for Jemperli (dostarlimab) plus chemotherapy for the treatment of dMMR/MSI-H primary advanced or recurrent endometrial cancer
GSK
2023-04-25
pivotal
endometrial cancer
Regulatory submission based on pivotal data from Part 1 of the RUBY phase III trial
Cellectis Implements CLLS52 for the First Time in the Clinic with Sanofi’s Alemtuzumab
cellectis
2023-04-24
aml
car t
talen
allogeneic
gene editing
gene therapies
Cellectis demonstrated that the addition of alemtuzumab to the lymphodepletion regimen was associated with prolonged lymphodepletion and significantly higher cell expansion and clinical activity New York (NY) – April 24, 2023 – Cellectis (the “Company”) (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced it has implemented the use of alemtuzumab as a Cellectis Investigational Medicinal Product (IMP), coded as CLLS52, as part of the lymphodepletion regimen for UCART22 in the BALLI-01 clinical trial in relapsed/refractory B-cell ALL, for UCART123 in the AMELI-01 clinical trial in relapsed/refractory AML, and for UCART20x22 in the NatHaLi-01 clinical trial in relapsed/refractory B-cell NHL. In May 2021, Cellectis entered into partnership and supply agreements with Sanofi regarding alemtuzumab. Under the agreements, Sanofi is supplying alemtuzumab to support Cellectis’ clinical trials and the parties agreed to enter into discussions to execute a commercial supply of alemtuzumab under pre-agreed financial conditions. “As previously reported, the importance of…
Cellectis Presents Preclinical Data on TALEN®-edited MUC1 CAR T-cells to Enhance Efficacy in Targeting Triple Negative Breast Cancer at the American Association for Cancer Research (AACR) Annual Meeting
cellectis
2023-04-17
car t
talen
allogeneic
solid tumor
cell therapy
breast cancer
gene therapies
April 17, 2023 – New York (NY) – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today released preclinical data on TALEN ® -edited MUC1 CAR T-cells at the American Association for Cancer Research (AACR) Annual Meeting 2023 . The data showed the capability of armored allogeneic MUC1 CAR T-cells to excel in the immune suppressive tumor micro-environment suggesting that they could be an effective option in treating relapsed and refractory triple negative breast cancer (TNBC) patients with limited therapeutic options. Tumor-associated MUC1 antigen is overexpressed in a large number of TNBC patients offering an effective discriminatory target for CAR T-cell therapy. Cellectis’ MUC1 CAR T-cells are allogeneic and target Mucin 1 for TNBC and a variety of epithelial cancers. As other solid tumor targets can be plagued by safety concerns due to off-tumor expression, MUC1 is of high interest as its expression in normal epithelium is restricted to apical membranes. Additionally, MUC1 heavy glycosylation in normal tissue contrasts with…
Ray Therapeutics to Present at Upcoming Scientific Conferences
ray therapeutics
2023-04-11
gene therapies
April 11, 2023 08:00 AM Eastern Daylight Time SAN FRANCISCO–(BUSINESS WIRE)–Ray Therapeutics, a biotech company developing optogenetic gene therapies for patients with blinding diseases, announced today that Peter Francis, M.D., Ph.D., Chief Scientific and Medical Officer, will present at two upcoming scientific conferences: the 2023 Cell & Gene Meeting on the Med and the Eighth …
Cellectis Announces Dosing of the First Patient in Europe with its In-house Manufactured Product Candidate UCART22
cellectis
2023-04-11
cd19
cd22
open
car t
leukemia
allogeneic
gene therapies
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
UCART22 is currently the most advanced allogeneic CAR T-cell product in development for relapsed or refractory B-cell acute lymphoblastic leukemia The BALLI-01 study (evaluating UCART22) is actively enrolling patients with relapsed or refractory B-cell ALL after FCA lymphodepletion New York, NY – April 11, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that the first patient in Europe has been dosed in France with its in-house manufactured product candidate UCART22 and completed the 28-day Dose Limiting Toxicity period. “Our team has worked tirelessly to expand our BALLI-01 clinical study (evaluating UCART22) to Europe . Dosing our first patient in France with our UCART22 product candidate manufactured in-house is an important advancement for Cellectis” said Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis. “By targeting the CD22 antigen, we aim at offering a novel therapeutic alternative to patients living with relapsed/refractory B-cell ALL, including those patients who have relapsed or did not…
Cellectis enters into warrant agreement with the European Investment Bank related to credit facility agreement and announces the drawdown of the first tranche of €20 million
cellectis
2023-04-04
car t
allogeneic
gene therapies
New York, NY – April 4, 2023 – Cellectis (Euronext Growth: ALCLS – NASDAQ: CLLS) (the “Company”), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies today announced that the Company entered into the warrant agreement (the “Warrant Agreement”) and finalized the related ancillary documents required under the credit facility with the European Investment Bank (“EIB”) (the "Finance Contract") for up to €40 million previously announced on December 28, 2022. The Company also announced the drawdown of the first tranche of €20 million (“Tranche A”) under the Finance Contract, expected to be disbursed by the EIB in early April 2023 . The Company plans to use the proceeds of Tranche A towards the development of its pipeline of allogeneic CAR T-cell product candidates: UCART22, UCART20x22, UCART123 and UCARTCS1. The disbursement of Tranche A is subject to, among other conditions, (i) the issuance of a specified number of warrants to the benefit of EIB (the “Tranche A Warrants”) and (ii) the completion of certain clinical development milestone by a Cellectis’ licensee, and, as of the date of this press release, each…
Alaunos Therapeutics Appoints Dr. Robert J. Hofmeister to Board of Directors
Alaunos
2023-03-30
cell therapy
Industry leader brings more than 25 years of scientific leadership and expertise in T-cell therapy development HOUSTON, March 30, 2023 (GLOBE NEWSWIRE) -- Alaunos Therapeutics, Inc. (“Alaunos” or the “Company”) (Nasdaq: TCRT), a leading T-cell receptor (TCR) cell therapy company advancing a
Cellectis Announces Poster Presentation on TALEN®-edited MUC1 CAR T-cells Targeting Triple Negative Breast Cancer at the American Association of Cancer Research (AACR) Annual Meeting
cellectis
2023-03-14
car t
talen
allogeneic
cell therapy
gene editing
breast cancer
gene therapies
adoptive cell therapy
New York, NY – March 14, 2023 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that preclinical data exploring purposeful armoring of CAR T-cells to enhance efficacy of MUC1 CAR T-cells in targeting triple-negative breast cancer, will be presented at the American Association of Cancer Research (AACR) Annual Meeting, to be held in Orlando, Florida on April 14-19, 2023. “We are proud to present updated preclinical data on our product candidate UCARTMUC1 for solid tumors at AACR 2023” said Laurent Poirot, Ph.D., Senior Vice President Immunology at Cellectis. “As immune-therapies continue to develop for solid tumors, the tumor microenvironment (TME) poses many challenges that CAR T-cells need to overcome for efficient tumor cell clearance. Our preclinical data make us confident that UCART MUC1 could be a valuable product candidate for patients with highly unmet medical needs.” Presentation includes: Poster presentation: Therapeutic options for triple negative breast cancer (TNBC) remain limited to date despite it being…
GSK announces positive pivotal phase III data for 5-in-1 Meningococcal ABCWY vaccine candidate
GSK
2023-03-14
pivotal
MenABCWY combination vaccine candidate met all its primary endpoints of the pivotal phase III clinical trial
Cellectis Provides Business Update and Reports Fourth Quarter and Full Year 2022 Financial Results
cellectis
2023-03-08
aml
gene therapies
financial results
Entered into a € 40 million credit facility with the European Investment Bank and closed an approximatively $25 million follow-on equity offering to support Cellectis’ research, development and innovation activities Positive preliminary clinical data from Phase 1 BALLI-01 study (evaluating UCART22) for patients with r/r B-cell ALL presented in a Live Webcast; First patient dosed with UCART22 product candidate fully manufactured in-house Encouraging preliminary results from Phase 1 AMELI-01 study (evaluating UCART123) for patients with AML presented at an oral session at ASH 2022; AMELI-01 currently enrolling in a two-dose regimen arm at DL2 Partner Allogene Therapeutics initiated Phase 2 of ALPHA2 trial in relapsed/refractory Large B Cell Lymphoma (r/r LBCL) Cellectis partnered with Primera Therapeutics to edit mutations in the mitochondrial DNA (mtDNA) in vivo Cash position of $95 million as of December 31, 2022 [1] Conference call scheduled for 8AM ET/2PM CET on March 9, 2023 New York (N.Y) - March 8, 2023 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving…
Calyxt, Inc. Reports its Fourth Quarter 2022 Financial Results and Provides Corporate Update
cellectis
2023-03-06
gene editing
gene therapies
financial results
New York, NY – March 6, 2023 – Cellectis S.A. (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that Calyxt, Inc. (Nasdaq: CLXT), a plant-based synthetic biology company for which Cellectis owns 49.1% (as of December 31, 2022) of its issued and outstanding common stock, today announced operating and financial results for its fourth quarter ended December 31, 2022. The contents of Calyxt’s announcement are included below: Announced proposed merger with Cibus Global and filed initial registration statement on Form S-4 — —Current customer projects under development are on track— —Signed agreement with Evologic Technologies to further develop and scale production of its Plant Cell Matrix™ and BioFactory TM technologies— Merger Agreement with Cibus On January 17, 2023, Calyxt announced it had entered into a definitive merger agreement with Cibus Global, LLC (Cibus), a leader in precision gene editing in agriculture, under which Calyxt and Cibus will merge in an all-stock transaction. The merger will create a new industry-leading company that combines…
Cellectis to Report Fourth Quarter and Year-End 2022 Financial Results on March 8, 2023
cellectis
2023-03-01
gene therapies
financial results
March 1 st , 2023 - New York, NY - Cellectis (Euronext Growth: ALCLS - Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the fourth quarter and year-end 2022 ending December 31 st , 2022, on Wednesday, March 8, 2023, after the close of the US market. The announcement will be followed by a conference call and live audio webcast on Thursday, March 9, 2023, at 8:00 AM ET / 2:00 PM CET. The call will include the Company’s fourth quarter results, year-end results, and an update on business activities. Details for the call are as follows: Dial-in information: Domestic: 1-877-451-6152 International : 1-201-389-0879 Call Me Link: https://callme.viavid.com/viavid/?callme=true&passcode=13727032&h=true&info=company-email&r=true&B=6 Webcast Link: https://viavid.webcasts.com/starthere.jsp?ei=1598389&tp_key=25a6baeb0f
US FDA Advisory Committee votes in support of trials designed to evaluate Jemperli (dostarlimab-gxly) as a potential treatment for mismatch repair-deficient/microsatellite instability-high locally advanced rectal cancer
GSK
2023-02-09
fast track
In January 2023, the US FDA granted dostarlimab-gxly Fast Track designation for the treatment of dMMR/MSI-H locally advanced rectal cancer.
Cellectis Announces Closing of Global Offering and Exercise of Underwriters’ Option to Purchase Additional Shares
cellectis
2023-02-07
gene therapies
NEW YORK (NY) - February 7, 2023 - Cellectis S.A. (“Cellectis” or the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today: the initial closing on February 7, 2023 of the previously announced underwritten global offering by way of a capital increase of 8,800,000 American Depositary Shares (the “ADSs”), each representing the right to receive one ordinary share of Cellectis, nominal value €0.05 per share (the “Global Offering”), which launched on February 2, 2023; and the exercise by the underwriters, Jefferies LLC and Barclays Capital Inc., of their option (the “Option”) to purchase an additional 1,107,800 ordinary shares (the “Additional Ordinary Shares”) of the Company to be delivered in the form of an aggregate of 1,107,800 ADSs (the “Additional ADSs”). The Additional ADSs will be listed on the Nasdaq Global Market under the symbol “CLLS”, and the Company’s Additional Ordinary Shares will be listed on Euronext Growth in Paris under the symbol “ALCLS”. The closing of the Option is expected to occur on February 8, 2023, subject to…
Cellectis Supplements the Announcement of the Pricing of Follow-On Offering with Allocation of Share Capital Information
cellectis
2023-02-03
open
gene therapies
NEW YORK—February 3, 2023 — Cellectis S.A. (“Cellectis” or the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, in accordance with French regulatory requirements, is supplementing its previous announcement of the pricing of its underwritten global offering by way of a capital increase of 8,800,000 American Depositary Shares (“ADS”), each representing the right to receive one ordinary share of Cellectis, nominal value €0.05 per share (the “Global Offering”), which launched and priced on February 2, 2023, at a price to the public of $2.50 per ADS as announced on February 2, 2023. The closing of the Global Offering is expected to occur on or about February 7, 2023, subject to customary closing conditions. Jefferies LLC and Barclays Capital Inc. are acting as joint book-running managers for the Global Offering. Allocation of the Share Capital The following table presents the expected allocation of Cellectis’ share capital following the settlement and delivery of the ADSs sold in the Global Offering: Bpifrance Participations, Baillie Gifford & Co. and…
Cellectis Announces Launch of Follow-On Offering
cellectis
2023-02-02
gene therapies
NEW YORK, February 2, 2023 - Cellectis S.A. (“Cellectis” or the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced the launch of a global offering of USD 22 million of its American Depositary Shares (“ADS”), each representing one ordinary share of Cellectis, nominal value €0.05 per share (the “Global Offering”) pursuant to an underwriting agreement, to be entered into by Cellectis, Jefferies LLC and Barclays Capital Inc. The Global Offering is comprised of a public offering of ADS in the United States of America only and a private placement in other countries, including in European Union member States, exclusively to “qualified investors” within the meaning of Article 2(e) of Regulation (EU) 2017/1129, as amended (the “Prospectus Regulation”). Jefferies LLC and Barclays Capital Inc. (the “Underwriters”) are acting as joint book-running managers for the Global Offering. The Company plans to use the net proceeds of the Global Offering to fund the continued clinical development of UCART123, UCART22, UCART20x22, and UCARTCS1, and for…
Cellectis Announces Pricing of Follow-On Offering
cellectis
2023-02-02
gene therapies
NEW YORK—February 2, 2023 -- Cellectis S.A. (“Cellectis” or the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announces the pricing of its previously announced underwritten global offering by way of a capital increase of 8,800,000 American Depositary Shares (“ADS”), each representing the right to receive one ordinary share of Cellectis, nominal value €0.05 per share (the “Global Offering”), comprised of a public offering of ADS in the United States of America only and a private placement in other countries including in European Union member States exclusively to “qualified investors” within the meaning of Article 2(e) of Regulation (EU) 2017/1129, as amended (the “Prospectus Regulation”), which launched on February 2, 2023, at a price to the public of $2.50 per ADS. The aggregate net proceeds to be received by Cellectis from the sale of ADSs in the Global Offering are expected to be approximately $20.2 million, equivalent to approximately €18.5 million, after deducting the estimated expenses related to the Global Offering and the underwriting…
Benlysta granted Orphan Drug Designation by US FDA for the potential treatment of systemic sclerosis
GSK
2023-02-01
orphan drug
GSK plans to initiate a phase II/III trial for systemic sclerosis associated interstitial lung disease (SSc-ILD) in the first half of 2023.
Cellectis Amends $20 Million Convertible Note Under Collaboration Agreement with its Partner Cytovia Therapeutics
cellectis
2023-01-20
gene therapies
New York (NY) – January 20, 2023 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that it has amended certain financial terms of the $20 million convertible note issued by its partner, Cytovia Therapeutics, LLC (“Cytovia”) in payment of the upfront collaboration consideration provided for pursuant to the research collaboration and non-exclusive license agreement between Cellectis and Cytovia. The amended and restated note provides for automatic conversion into common stock of Cytovia in the case of certain fundamental transactions pursuant to which Cytovia becomes a public reporting company and for conversion at Cellectis’ option in connection with certain financing transactions, upon a company sale and at final maturity. In each case such conversion is subject to a 9.9% ownership cap, with the balance issuable in the form of pre-funded warrants . Among other changes, the amended and restated note increased the applicable interest rate of the note to 10% per annum, subject to a 10% step up upon the occurrence and…
Exegenesis Bio Announces FDA Clearance of Investigational New Drug (IND) Application for EXG102-031: A Novel Gene Therapy for the Treatment of neovascular Age-Related Macular Degeneration (nAMD)
Exegenesis Bio
2023-01-18
ind
gene therapy
– Phase 1 clinical trial to be initiated in Q1 20 […]
Calyxt and Cibus Announce the Signing of a Definitive Merger Agreement
cellectis
2023-01-17
gene editing
gene therapies
New York (N.Y) – January 17, 2023 - Cellectis (Euronext Growth: ALCLS - Nasdaq: CLLS), a clinical-stage biotechnology company using its gene editing platform to deliver life-saving cell and gene therapies, announced today that Cibus Global LLC, a Delaware limited liability company (“ Cibus ”), and Calyxt, Inc. (NASDAQ: CLXT) (“Calyxt”), announced that Calyxt and Calypso Merger Subsidiary, LLC, a Delaware limited liability company and wholly-owned subsidiary of Calyxt, entered into an Agreement and Plan of Merger with Cibus and certain other parties named therein (the “Merger Agreement”), pursuant to which, subject to the terms and conditions thereof, Calyxt and Cibus will merge in an all-stock transaction. The transaction combines two companies in agriculture-based gene editing, with facilities for trait development and next-generation plant breeding. Cellectis currently holds a 49.1 % equity interest in Calyxt. Following the closing of the merger, Cellectis is expected to own approximately 2.5% of the equity interests of the combined company. In connection with the Merger Agreement, Cellectis executed a voting agreement with Cibus to vote in favor of and approve all the…
Cellectis Establishes an At-The-Market (ATM) Program on Nasdaq
cellectis
2023-01-04
gene therapies
Maximum Potential Dilution of Approximately 23.04%, Based on Share Capital of Cellectis as of September 30, 2022 New York, NY, January 4, 2023 – Cellectis S.A. (“Cellectis” or the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it has filed a prospectus supplement with the Securities and Exchange Commission (“SEC”), pursuant to which it may offer and sell to eligible investors a maximum gross amount of up to $60.0 million of American Depositary Shares (“ADS”), each representing one ordinary share of Cellectis, nominal value €0.05 per share, from time to time in sales deemed to be an “at the market offering” pursuant to the terms of a sales agreement with Jefferies LLC (“Jefferies”), acting as sales agent. The timing of any sales will depend on a variety of factors. The at-the-market (“ATM”) program is presently intended to be effective through the expiration of the existing registration statement, i.e. July 6, 2025, unless terminated prior to such date in accordance with the sales agreement or the maximum amount of the program…
Mayflower Bioventures Launches its First Spin-Out, Primera Therapeutics, in Strategic Collaboration with Cellectis to Develop a Gene Editing Platform to Treat Mitochondrial Diseases
cellectis
2022-12-29
gene editing
gene therapy
gene therapies
New York (N.Y) - December 29, 2022: Following the formation of Mayflower Bioventures, a cell and gene therapy accelerator stood up from Hibiscus BioVentures and Mayo Clinic, comes the launch of their first start-up, Primera Therapeutics, Inc. (Primera). Cellectis (Euronext Growth: ALCLS - Nasdaq: CLLS), a clinical-stage biotechnology company using its gene editing platform to deliver life-saving cell and gene therapies, and Primera announced today the execution of a Collaboration Agreement under which the Parties will work collaboratively to edit mutations in the mitochondrial DNA (mtDNA) in vivo to treat the root cause of associated diseases. Inherited mitochondrial diseases caused by genetic changes in the mtDNA have very limited to no treatment options, and often result in early childhood death. An estimated 1 in 5,000 people worldwide suffer from mtDNA – associated diseases. Primera is advancing pioneering mtDNA gene editing therapies developed in the Mayo Clinic lab of Drs. Steven Ekker and Karl Clark to precisely fix a patient’s mutated mtDNA and potentially achieve a cure for the disease. Primera’s initial clinical focus is on rare mitochondrial diseases for which there are…
Cellectis secures a €40 million credit facility from the European Investment Bank to support its Research, Development and Innovation activities
cellectis
2022-12-28
car t
allogeneic
gene therapies
The credit facility will enable Cellectis to support the development of its UCART product candidates pipeline The credit facility consists of three tranches of €20 million, €15 million, and €5 million respectively, each redeemable in fine in 6 years The credit facility is part of the European Investment Bank’s strategy to support biotech companies developing a high-level of expertise in various therapeutic areas with significant unmet medical needs Paris, France - December 28, 2022 – Cellectis (Euronext Growth: ALCLS – NASDAQ: CLLS) (the “Company“), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it has entered into a €40 million credit facility agreement with the European Investment Bank (“EIB”) (the “Finance Contract”). The Company plans to use the facility toward the development of its pipeline in the field of allogeneic CAR T-cell product candidates, UCART22, UCART20x22, UCART123 and UCARTCS1. The €40 million facility is divided into three tranches: €20 million for the first tranche (“ Tranche A ”), €15 million for the second tranche (“ Tranche B ”) and €5 million for the…
Cellectis Announces First Dosing of a Patient with its In-house Manufactured Product Candidate UCART22 for the treatment of r/r B-cell ALL
cellectis
2022-12-22
cd19
cd22
open
b-all
car t
leukemia
allogeneic
cell therapy
gene editing
gene therapy
gene therapies
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
o The first patient completed the 28-day Dose Limiting Toxicity (DLT) observation period without complication New York, NY – December 22, 2022 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that for the first time, a patient was dosed in the United States with its in-house manufactured product candidate UCART22, and completed the 28 day DLT period on December 14th, 2022, without complication. “First dosing of a patient with a product candidate manufactured in-house is a major milestone for Cellectis. UCART22 has been developed to potentially offer a therapeutic alternative for patients with r/r B-ALL, including patients that have relapsed from or unable to receive CD19-directed therapy. The ability to have our manufacturing completely in-house maximizes the chances that eligible patients can be treated without delay.” said Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis. “This is a transformational step forward for Cellectis: our in-house manufacturing capabilities would allow us to move product…
PeproMene Bio, Inc. Announced Complete Response of The First Patient Treated in Its B-cell Non-Hodgkin Lymphoma (B-NHL) Phase 1 Clinical Trial of PMB-CT01 (BAFFR-CAR T Cells) at City of Hope
pepromene bio
2022-12-20
car t
hodgkin lymphoma
complete response
non-hodgkin lymphoma
Irvine, Calif., 20 Dec, 2022 — PeproMene Bio, Inc., a clinical-stage biotech company developing novel therapies to treat cancers and immune disorders, today announced that the first patient treated in its phase 1 relapsed or refractory B-cell Non-Hodgkin Lymphoma (r/r … Continue reading → The post PeproMene Bio, Inc. Announced Complete Response of The First Patient Treated in Its B-cell Non-Hodgkin Lymphoma (B-NHL) Phase 1 Clinical Trial of PMB-CT01 (BAFFR-CAR T Cells) at City of Hope appeared first on PeproMene Bio Inc .
Cellectis Announces Positive Preliminary Clinical Data for UCART22 in ALL and UCART123 in AML
cellectis
2022-12-13
aml
cd19
cd22
open
car t
icans
leukemia
allogeneic
autologous
cell therapy
gene therapies
complete response
acute lymphoblastic leukemia
UCART22: anti-tumor activity observed in 60% (n=3) of patients at DL3 using FCA lymphodepletion UCART123: 25% (n=2) of patients at DL2 in the FCA arm achieved meaningful response; one patient experienced a durable minimal residual disease (MRD)-negative complete response that continues beyond 12 months BALLI-01 study (evaluating UCART22) now enrolling patients with product candidate manufactured in-house at DL2 AMELI-01 study (evaluating UCART123) now enrolling patients in a two-dose regimen arm at DL2 New York, NY – December 13, 2022 - Cellectis S.A. (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today will host a live webcast reviewing updated clinical data on its Phase 1/2a BALLI-01 clinical trial (evaluating UCART22) and on its Phase 1 AMELI-01 clinical trial (evaluating UCART123) that were presented in an oral session on December 12, 2022 at the 64th Annual Meeting of the American Society of Hematology (ASH). Preliminary Data from the BALLI-01 Clinical Study BALLI-01 is a Phase 1/2a open-label dose-escalation trial evaluating the safety…
European Medicines Agency accepts marketing authorisation application for momelotinib for the treatment of myelofibrosis
GSK
2022-12-02
pivotal
Application includes data from key phase III trials, including the pivotal MOMENTUM trial.
Cellectis to Host a Live Webcast and Provide a Company Update on December 13, 2022
cellectis
2022-12-01
gene therapies
New York, NY –December 1, 2022 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, will host a live webcast to review the Company’s clinical data on the AMELI-01 study (evaluating UCART123) presented at the ASH Annual Meeting, and provide a Company update, on December 13, 2022. The event will feature presentations by the management team and will be followed by a Q&A. Cellectis Live Webcast Tuesday, December 13, 2022 7:30am ET/1:30pm CET To register and access the live webcast: https://lifescievents.com/event/cellectis-webcast/ Following the live webcast, a replay will be available under the “Events and Webcasts” section on the Investor page of the Company’s website: https://cellectis.com/en/investors/events-and-webcasts/
New global survey reveals opportunities to address organ damage risk with people living with lupus earlier in the course of their disease
GSK
2022-11-29
lupus
Survey examined healthcare professional approaches to preventing organ damage
GSK provides an update on Zejula (niraparib) US prescribing information
GSK
2022-11-11
pivotal
Zejula is an oral, once-daily PARP inhibitor currently being evaluated in multiple pivotal trials.
Cellectis Publishes Manuscript in Frontiers Bioengineering and Biotechnology Unveiling Efficient Multitool/Multiplex Gene Engineering combining TALEN® and TALE Base Editors (TALE-BE)
cellectis
2022-11-10
cas9
car t
talen
crispr
allogeneic
gene editing
gene therapies
November 10, 2022 - New York, NY – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today published a manuscript in Frontiers Bioengineering and Biotechnology demonstrating the feasibility of efficient multiplex gene engineering using a combination of two different molecular tools: Cellectis’ TALEN® gene editing technology (TALE nuclease) and a TALE-BE (TALE Base editor). TALE base editors are a recent and very important addition to the gene editing landscape. This approach does not create DNA double strand breaks as does CRISPR/Cas9, or other engineered nucleases, and is a promising therapeutic strategy for genetic diseases. A key aspect to broaden the scope of possible applications is our comprehension of design rules. Cellectis used a screening approach to gain in-depth insights into the editing rules driving TALE-BE activity and applied this knowledge to design highly efficient TALE-BE compatible with potential therapeutic application. Moreover, TALE-BE show insignificant levels of by-products such as indels. Cellectis scientists…
Cellectis Presents Pre-Clinical Data on TALEN®-edited Smart CAR T-cells Overcoming Key Challenges of Targeting Solid Tumors at SITC 2022
cellectis
2022-11-10
car t
talen
allogeneic
solid tumor
cell therapy
gene editing
breast cancer
gene therapies
New York, NY – November 10, 2022 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that preclinical data will be presented on TALEN®-edited smart CAR T-cells overcoming key challenges of targeting solid tumors at the Society for Immunotherapy of Cancer’s (SITC) 37th Annual Meeting. The data will be presented today in two poster sessions titled: “Multi-armored allogeneic MUC-1 CAR T-cells efficiently control triple negative breast cancer tumor growth” (Poster Number: 217) and “TALEN®-edited smart CAR T-cells leverage solid tumor microenvironment for specific and effective immunotherapy” (Poster Number: 325). The poster presentations highlight the following preclinical data: Multi-armored allogeneic MUC-1 CAR T-cells efficiently control triple negative breast cancer tumor growth · This poster highlights TALEN®-edited smart CAR T-cells targeting MUC1- expressing solid tumors. · MUC1 is a tumor-associated antigen that is overexpressed in triple-negative breast cancer (TNBC) and other solid tumor malignancies. · MUC-1 CAR…
GSK provides update on DREAMM-3 phase III trial for Blenrep in relapsed/refractory multiple myeloma
GSK
2022-11-07
multiple myeloma
DREAMM-3 did not meet its primary endpoint of progression-free survival (PFS)
Cellectis to Showcase Clinical Data from AMELI-01 and Preclinical Data from UCARTCS1 at ASH 2022
cellectis
2022-11-03
aml
open
car-t
cd123
talen
leukemia
allogeneic
lentiviral
gene editing
gene therapies
multiple myeloma
acute myeloid leukemia
chimeric antigen receptor
· AMELI-01 (evaluating UCART123) abstract, selected for oral presentation, highlights that adding alemtuzumab was associated with improved activity · UCARTCS1 abstract, selected for poster presentation, demonstrated anti-tumor activity in vivo and in vitro supporting the potential benefit of UCARTCS1 first in-human study for patients with r/r MM New York, NY – November 3, 2022 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced the release of two abstracts, which were accepted for presentation at the 64th American Society of Hematology (ASH) Annual Meeting taking place from December 10 to 13, 2022. The Company will present, in an oral session on December 12, preliminary clinical data from its AMELI-01 clinical trial (evaluating UCART123) in patients with relapsed/refractory acute myeloid leukemia (r/r AML). Amsterdam University Medical Center (location VUmc), in collaboration with Cellectis, will also present, in a poster session on December 10, preclinical data supporting anti-tumor activity for Cellectis’ UCARTCS1…
Cellectis Provides Business Update and Reports Financial Results for Third Quarter and First Nine Months 2022
cellectis
2022-11-03
gm1
til
car t
talen
pivotal
leukemia
melanoma
allogeneic
cell therapy
gene therapy
gene therapies
b-cell lymphoma
financial results
large b-cell lymphoma
acute myeloid leukemia
ASH 2022 sponsored programs – UCART123 abstract selected for oral presentation & preclinical data on UCARTCS1 selected for poster presentation, in collaboration with the Amsterdam University Medical Center -Preclinical data on TALEN®-edited smart CAR T-cells to be presented at SITC 2022 -Partner Allogene Therapeutics announced having initiated industry’s first allogeneic CAR T Phase 2 trial -Iovance Biotherapeutics, Inc. announced having dosed the first patient and completed safety observation period in its IOV-GM1-201 clinical trial -Mark Frattini, M.D., Ph.D., appointed Chief Medical Officer -Cash position[1] of $103 million as of September 30, 2022 -Conference call scheduled for 8AM ET/1PM CET on November 4, 2022 [1] Cash position includes cash, cash equivalent, current financial assets and restricted cash. Restricted cash was $5million as of September 30, 2022. November 3, 2022 - New York (N.Y.) - Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop potentially life-saving cell and gene therapies, announced its results for the three-month and nine-month periods ending September 30, 2022.…
GSK’s respiratory syncytial virus older adult vaccine candidate granted Priority Review by US FDA
GSK
2022-11-02
priority review
The US FDA has accepted a Biologics License Application and granted Priority Review for its RSV older adult vaccine candidate.
Cellectis to Report Third Quarter 2022 Financial Results
cellectis
2022-10-27
gene therapies
financial results
October 27, 2022 - New York, NY –– Cellectis (Euronext Growth: ALCLS - Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the third quarter ended September 30th, 2022, on Thursday, November 3rd, 2022, after the close of the US market. The announcement will be followed by a conference call and live audio webcast on Friday, November 4th, 2022, at 8:00 AM EDT / 2:00 PM CET. The call will include the Company’s third quarter results and an update on business activities. Details for the call are as follows: Friday, November 4th at 8:00 a.m. Eastern Time: Domestic: 1-877-413-2411 International :1-201-389-0882 Conference ID :13732988 Webcast: https://viavid.webcasts.com/starthere.jsp?ei=1571941&tp_key=ebcf814947
GSK’s older adult respiratory syncytial virus (RSV) vaccine candidate shows 94.1% reduction in severe RSV disease and overall vaccine efficacy of 82.6% in pivotal trial
GSK
2022-10-13
pivotal
Data showed overall vaccine efficacy against RSV-lower respiratory tract disease in adults aged 60 years and above
Cellectis Presents Data on Two TALEN®-based Gene Therapy Preclinical Programs for Patients with Sickle Cell Disease and Mucopolysaccharidosis type I at ESGCT 2022
cellectis
2022-10-11
talen
cell therapy
gene editing
gene therapy
gene therapies
sickle cell disease
mucopolysaccharidosis type i
New York, NY – October 11, 2022 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that the Company will present both an oral and poster at the European Society of Gene and Cell Therapy’s (ESGCT) 29th Congress, to be held in Edinburgh from October 11-14, 2022. Arianna Moiani, Ph.D., Senior Scientist & Team Leader Innovation Gene Therapy, will give an oral presentation on encouraging pre-clinical data that leverages TALEN® gene editing technology to develop a hematopoietic stem and progenitor cell (HSPCs)-based gene therapy to treat sickle cell disease. Eduardo Seclen, Ph.D., Senior Scientist & Team Leader, Gene Editing, will present a poster illustrating a TALEN®-based gene editing approach that reprograms HSPCs to secrete alpha-L-iduronidase (IDUA), a therapeutic enzyme missing in Mucopolysaccharidosis type I (MPS-I). “The pre-clinical data presented at ESGCT further demonstrate our ability to leverage TALEN® gene editing technology to potentially address genetic diseases, namely, sickle cell disease and lysosomal…
Ray Therapeutics and Forge Biologics Expand Their Viral Vector cGMP Partnership to Encompass Plasmid DNA Manufacturing
ray therapeutics
2022-10-10
aav
plasmid dna
gene therapy
viral vector
retinitis pigmentosa
Building on AAV manufacturing partnership with new plasmid production, Forge’s end-to-end manufacturing services will help to advance Ray Therapeutics’ gene therapy for patients living with retinitis pigmentosa and other blinding diseases.
Cellectis to Present Pre-Clinical Data on TALEN®-edited Smart CAR T-cells Supporting Improved Solid Tumor Targeting at the Society for Immunotherapy of Cancer’s (SITC) 37th Annual Meeting
cellectis
2022-10-05
car t
talen
allogeneic
solid tumor
breast cancer
gene therapies
New York, NY – October 5, 2022 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that pre-clinical data on TALEN®-edited smart CAR T-cells to overcome key challenges of targeting solid tumors, will be presented at the Society for Immunotherapy of Cancer’s 37th Annual Meeting (SITC 2022), to be held in Boston, M.A. and virtually on November 8-12, 2022. Presentations include: Poster presentation: Cellectis will present a poster on TALEN®-edited smart CAR T-cells targeting MUC1- expressing solid tumors. MUC1 is a tumor-associated antigen that is overexpressed in triple-negative breast cancer (TNBC) and other solid tumor malignancies. Title: Multi-armored allogeneic MUC-1 CAR T-cells efficiently control triple negative breast cancer tumor growth Poster Number: 217 Presenter: Piril, Erler Ph.D., Scientist II, Immuno-Oncology, Cellectis Date/Time: Thursday, November 10th, posters will be on display from 9:00 AM- 9:00 PM, Hall C Poster presentation: Cellectis will present a poster on innovative T-cell engineering strategies…
Cellectis Appoints Mark Frattini, M.D., Ph.D. as Chief Medical Officer
cellectis
2022-09-28
car t
leukemia
cell therapies
gene therapies
hematological malignancies
New York, NY – September 28, 2022 - Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the appointment of Mark Frattini, M.D., Ph.D., as Chief Medical Officer, effective immediately. Dr. Frattini has over 20 years of experience in the field of hematological malignancies and joined Cellectis in August 2020 as Senior Vice President of Clinical Sciences. Mark has been responsible for Cellectis’ clinical leadership including the clinical development strategy of the Company’s current immune-oncology UCART product candidates. He has also been serving as a core member of the senior clinical team and has been managing a team of physicians and clinical scientists. As Chief Medical Officer, Dr. Frattini will oversee clinical research and development for Cellectis’ UCART clinical trial programs. He will remain based in Cellectis’ New York office and is joining the Company’s executive committee. “Mark already had an impressive track record before joining Cellectis. He has continued to lead our clinical teams successfully over the…
Cellectis Announces Participation in Four Upcoming Investor Conferences in September
cellectis
2022-09-01
gene therapies
New York, NY – September 1, 2022 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that management plans to participate in four upcoming investor conferences. Details for the events are below: Citi's 17th Annual Biopharma Conference Format: Panel Date: September 7th, 2022 Time: 9:40AM- 10:25AM ET Location: Four Seasons Hotel, Boston, MA Webcast Link: http://www.veracast.com/webcasts/citigroup/biopharma2022/3DniPm.cfm Wells Fargo 2022 Healthcare Conference Format: Investor meetings Date: September 7-9, 2022 Location: Encore Boston Harbor, Everett, MA Baird Global Healthcare Conference Format: Presentation Date: September 13, 2022 Time: 10:15AM-10:45AM ET Location: Intercontinental Barclay Hotel, New York City, NY Jefferies Cell and Genetic Medicine Summit Format: Presentation Date: September 29, 2022 Time: Presentation time to be posted on Cellectis.com Location: Palace Hotel, New York City, NY Live webcasts and replays of these presentations will be available under the “Events and Webcasts” section on the Investor page…
US FDA accepts new drug application for GSK’s momelotinib for the treatment of myelofibrosis
GSK
2022-08-17
pivotal
Regulatory submission included data from the pivotal MOMENTUM phase III clinical trial
Cellectis Provides Business Update and Reports Financial Results for Second Quarter 2022
cellectis
2022-08-04
aml
ind
cd19
cd20
cd22
b-all
car t
cd123
leukemia
allogeneic
cell therapy
gene therapy
gene therapies
hodgkin lymphoma
multiple myeloma
financial results
non-hodgkin lymphoma
acute myeloid leukemia
acute lymphoblastic leukemia
b-cell acute lymphoblastic leukemia
IND clearance received from FDA for UCART20x22 for the treatment of Non-Hodgkin Lymphoma -Cellectis unveiled novel immune-evasive universal allogeneic CAR T-cell in Nature Communications; awarded oral presentation at ASGCT in May -Mr. Axel-Sven Malkomes & Dr. Donald A Bergstrom, M.D., Ph.D. appointed as Directors of Cellectis’ Board of Directors -Cash position [1] of $135 million as of June 30, 2022 -Conference call scheduled for 8AM ET/2PM CET on August 5, 2022 [1] Cash position includes cash, cash equivalents and current financial assets and restricted cash. Restricted cash was $5 million as of June 30, 2022. New York, NY – August 4, 2022 – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today provided business update and announced its results for the six-month period ending June 30, 2022. “This is a very exciting time at Cellectis: earlier this week, we were proud to announce the FDA clearance of our Investigational New Drug application for UCART20x22, our product candidate being developed for patients with relapsed and refractory…
Cellectis Receives IND clearance for UCART20x22, its First in-house Manufactured Product Candidate for the Treatment of B-cell Malignancies
cellectis
2022-08-01
ind
cd19
cd20
cd22
car t
talen
allogeneic
gene therapy
gene therapies
hodgkin lymphoma
non-hodgkin lymphoma
· UCART20x22 is Cellectis’ first allogeneic dual CAR T, targeting CD20 and CD22 simultaneously · UCART20x22 is Cellectis’ first product candidate fully designed, developed and manufactured in-house · Phase 1/2a clinical trial NatHaLi-01 expected to begin in second half of the year August 1st, 2022 - New York, NY – Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has cleared Cellectis’ Investigational New Drug (IND) application to initiate a Phase 1/2a clinical trial of UCART20x22 for patients with relapsed or refractory Non-Hodgkin Lymphoma (r/r NHL). The Company plans to begin enrolling patients in the NatHaLi-01 study in the second half of the year. “We are delighted that the FDA has cleared our IND application for UCART20x22”, said André Choulika, PhD, Chief Executive Officer of Cellectis. “This is a very exciting product candidate, for two reasons: UCART20x22 will be our first dual allogeneic CAR T product candidate to enter clinical development, and dual targeting of…
Cellectis to Report Second Quarter 2022 Financial Results
cellectis
2022-07-28
gene therapies
financial results
July 28, 2022 - New York, NY –– Cellectis (Euronext Growth: ALCLS - Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that it will report financial results for the second quarter ended June 30th, 2022, on Thursday, August 4th, 2022, after the close of the US market. The announcement will be followed by a conference call and webcast on Friday, August 5th, 2022, at 8:00 AM EDT / 2:00 PM CET. The call will include the Company’s second quarter results and an update on business activities. Details for the call are as follows: Friday, August 5th at 8:00 am Eastern Time: Domestic: 1-877-451-6152 International:1-201-389-0879 Conference ID: 13731385 Webcast: https://viavid.webcasts.com/starthere.jsp?ei=1558912&tp_key=9b34665e01
Luminary Therapeutics selected as a 2022/2023 Showcase Company in NIH/NCI’s investor initiative program
luminary therapeutics
2022-07-19
allogeneic
Minneapolis, MN USA Jul 19 2021 /EINPresswire.com/ – Luminary Therapeutics was selected as a 2022/2023 Showcase Company in NIH/NCI’s investor initiative program. Luminary Therapeutics is a clinical stage allogeneic cellular therapy company. Its gamma delta expansion platform coupled with its non-viral cell engineering process is unique in the industry. Luminary Therapeutics’ novel ligand-based CAR designed […] The post Luminary Therapeutics selected as a 2022/2023 Showcase Company in NIH/NCI’s investor initiative program appeared first on Luminary Therapeutics .
GSK selects Target the Future grant recipient in innovation challenge supporting multiple myeloma community
GSK
2022-06-29
multiple myeloma
GSK today announced the first recipient of the Target the Future Think Tank Challenge.
GSK announces positive pivotal phase III data for its respiratory syncytial virus (RSV) vaccine candidate for older adults
GSK
2022-06-10
pivotal
GSK today announced positive headline results from a pre-specified efficacy interim analysis of the AReSVi 006 phase III trial.
Ray Therapeutics Receives $4M in Funding From the California Institute for Regenerative Medicine (CIRM)
ray therapeutics
2022-04-27
retinitis pigmentosa
Funding will Advance Development of Ray Therapeutics’ Optogenetics Technology Platform - Company will Advance Ray-001 for the Treatment of Retinitis Pigmentosa -
BioEclipse Therapeutics™ Awarded Almost $8 Million Grant by California Institute for Regenerative Medicine (CIRM) to Fund Ongoing Phase 1 CRX100 Cancer Immunotherapy Clinical Trial
bioeclipse therapeutics
2022-04-26
open
BioEclipse Therapeutics announced today it has been awarded a $7,999,689.00 grant by the California Institute for Regenerative Medicine (CIRM) to support its ongoing clinical study: A Phase 1, open-label, dose-escalation study of CRX100 in patients with advanced solid tumors.
Ray Therapeutics and Forge Biologics Announce Viral Vector Contract Development and cGMP Manufacturing Partnership
ray therapeutics
2022-03-01
gene therapy
viral vector
gene therapies
Posted on March 1, 2022 (March 1, 2022) by Forge Biologics SAN DIEGO, CALIFORNIA AND COLUMBUS, OHIO March 1, 2022 – Ray Therapeutics, a biotechnology company developing optogenetic gene therapies for patients with retinal degenerative conditions, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced a manufacturing partnership that will advance Ray Therapeutics’ lead optogenetics gene therapy …
China’s National Medical Products Administration approves Benlysta (belimumab) for adult patients with active lupus nephritis
GSK
2022-02-10
lupus
First and only biologic approved in China for both systemic lupus erythematosus and lupus nephritis
Ray Therapeutics closes $6M seed financing to advance novel optogenetics platform to treat blinding diseases
ray therapeutics
2022-01-04
gene therapy
retinitis pigmentosa
Top-tier advanced therapies investor 4BIO Capital led the financing round. The financing will be used to advance its lead optogenetics gene therapy Ray-001 into clinical trials for retinitis pigmentosa.
New data presented at ASH 2021 highlight potential of Blenrep (belantamab mafodotin-blmf) in combination with standard of care therapies in earlier lines of multiple myeloma treatment
GSK
2021-12-13
multiple myeloma
GSK announced new data from the DREAMM-9 phase I trial and two GSK collaborative studies investigating the potential use of Blenrep.
GSK unveils Target the Future, a global, multi-year programme to help address key challenges affecting the multiple myeloma community
GSK
2021-12-01
multiple myeloma
$100,000 grant to be awarded to best idea submitted to Target the Future Think Tank Challenge
GSK showcases progress from the DREAMM clinical trial programme in multiple myeloma at the 2021 ASH Annual Meeting
GSK
2021-11-18
multiple myeloma
11 abstracts on Blenrep will be presented at the upcoming American Society of Hematology (ASH) Annual Meeting and Exposition.
Primary endpoint met in COMET-TAIL Phase III trial evaluating intramuscular administration of sotrovimab for early treatment of COVID-19
GSK
2021-11-12
primary endpoint met
Phase III IM administration data for sotrovimab.
GSK announces positive Phase III efficacy and safety data for daprodustat in patients with anaemia due to chronic kidney disease
GSK
2021-11-05
pivotal
Pivotal trials in non-dialysis and dialysis populations published simultaneously in the New England Journal of Medicine
FDA grants Priority Review to ViiV Healthcare’s New Drug Application for cabotegravir long-acting for prevention of HIV
GSK
2021-09-28
priority review
Final FDA decision anticipated by 23 January 2022; if approved, cabotegravir would be the first long-acting therapy for HIV PrEP
GSK to highlight continued progress in oncology pipeline and portfolio with data presented at ESMO
GSK
2021-09-09
cell therapy
The data reflects our commitment to strengthening our oncology pipeline across immuno-oncology, synthetic lethality & oncology cell therapy
SK bioscience and GSK start Phase 3 trial of adjuvanted COVID-19 vaccine candidate
GSK
2021-08-31
phase 1/2
Advance to Phase 3 follows positive interim Phase 1/2 immunogenicity and safety data
GSK receives FDA accelerated approval for JEMPERLI (dostarlimab-gxly) for adult patients with mismatch repair-deficient (dMMR) recurrent or advanced solid tumours
GSK
2021-08-17
accelerated approval
GARNET study demonstrated objective response rate of 41.6% across dMMR solid tumours
European Commission approves Benlysta for adult patients with active lupus nephritis
GSK
2021-05-05
lupus
First and only biologic approved for both systemic lupus erythematosus and lupus nephritis
FDA grants accelerated approval for GSK’s JEMPERLI (dostarlimab-gxly) for women with recurrent or advanced dMMR endometrial cancer
GSK
2021-04-22
endometrial cancer
accelerated approval
FDA grants accelerated approval for GSK’s JEMPERLI (dostarlimab-gxly) for women with recurrent or advanced dMMR endometrial cancer
GSK receives CHMP positive opinion recommending approval of Benlysta for adult patients with active lupus nephritis
GSK
2021-03-26
lupus
This CHMP opinion follows the recent label expansion by the US FDA to include lupus nephritis
Bio-Techne and Luminary Therapeutics sign license agreement for use of Bio-Techne’s TcBuster™ for the development of Luminary’s CAR-T cell therapies
luminary therapeutics
2021-02-23
car-t
cell therapy
cell therapies
Minneapolis, MN USA Feb 23 2021 – Bio-Techne Corporate (NASDAQ: TECH) today announced the signing of a license agreement with Luminary Therapeutics for the use of TcBuster™ for the manufacturing of gene-modified cell therapies. TcBuster™ is Bio-Techne’s proprietary non-viral transposon-based gene delivery system used to advance cell therapy manufacturing. Under the terms of this […] The post Bio-Techne and Luminary Therapeutics sign license agreement for use of Bio-Techne’s TcBuster™ for the development of Luminary’s CAR-T cell therapies appeared first on Luminary Therapeutics .
ViiV Healthcare announces FDA approval of Cabenuva (cabotegravir, rilpivirine), the first and only complete long-acting regimen for HIV treatment
GSK
2021-01-21
pivotal
The approval is based on the pivotal phase III ATLAS and FLAIR studies that included more than 1,100 patients from 16 countries.
FDA approves GSK’s BENLYSTA as the first medicine for adult patients with active lupus nephritis in the US
GSK
2020-12-17
lupus
Approval builds on nearly 10 years of experience in lupus
Sanofi and GSK announce a delay in their adjuvanted recombinant protein-based COVID-19 vaccine programme to improve immune response in the elderly
GSK
2020-12-11
phase 1/2
Phase 1/2 interim results showed an immune response comparable to patients who recovered from COVID-19 in adults aged 18 to 49 years
ViiV Healthcare receives FDA Breakthrough Therapy Designation for investigational, long-acting cabotegravir for HIV prevention
GSK
2020-11-17
breakthrough therapy
This is intended to facilitate the development and expedite the review of drugs that address serious or life-threatening medical conditions.
Hope & Gene Therapy. A Map for the Future of Diabetic Retinopathy.
Adverum
2020-11-16
gene therapy
diabetic retinopathy
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam, quis nostrud exercitation ullamco laboris nisi ut aliquip ex ea commodo consequat. Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. The post Hope & Gene Therapy. A Map for the Future of Diabetic Retinopathy. appeared first on Adverum Biotechnologies .
Medicago and GSK announce start of Phase 2/3 clinical trials of adjuvanted COVID-19 vaccine candidate
GSK
2020-11-12
phase 2/3
Based on the positive Phase 1 results, Medicago has decided to launch the Phase 2/3 clinical trial with GSK’s pandemic adjuvant.
GSK highlights progress from the BLENREP (belantamab mafodotin-blmf) development programme in multiple myeloma at ASH Annual Meeting
GSK
2020-11-11
multiple myeloma
Studies presented will demonstrate belantamab mafodotin's potential in combination with standard therapies in earlier lines of treatment.
Sanofi and GSK initiate Phase 1/2 clinical trial of COVID-19 adjuvanted recombinant protein-based vaccine candidate
GSK
2020-09-03
phase 1/2
Over 400 participants being enrolled in Phase 1/2 study.
Vir Biotechnology and GSK start phase 2/3 study of COVID-19 antibody treatment
GSK
2020-08-31
phase 2/3
Phase 2/3 study will investigate the safety and efficacy of antibody treatment in preventing hospitalisation due to COVID-19.
European Commission approves BLENREP (belantamab mafodotin) for the treatment of patients with relapsed and refractory multiple myeloma
GSK
2020-08-26
bcma
multiple myeloma
BLENREP is a first-in-class humanised anti-BCMA treatment for patients whose disease has progressed despite the current standard of care.
FDA approves GSK’s BLENREP (belantamab mafodotin-blmf) for the treatment of patients with relapsed or refractory multiple myeloma
GSK
2020-08-06
multiple myeloma
BLENREP is the fifth major medicine approval for GSK in 2020
GSK receives positive CHMP opinion recommending approval of belantamab mafodotin for the treatment of relapsed and refractory multiple myeloma
GSK
2020-07-24
multiple myeloma
If approved, it will be the second major regulatory milestone for GSK’s oncology portfolio this year.
GSK announces FDA advisory committee votes in favour of positive benefit/risk profile for belantamab mafodotin for patients with relapsed/refractory multiple myeloma
GSK
2020-07-14
multiple myeloma
FDA ODAC voted 12-0 in favour of the demonstrated benefit of monotherapy treatment with belantamab mafodotin
Luminary Therapeutics and Case Western Reserve University Enter Formal Collaboration
luminary therapeutics
2020-07-07
car-t
chimeric antigen receptor
Minneapolis, July 7, 2020 —Luminary Therapeutics (Luminary Tx) and Case Western Reserve University have entered into a formal collaboration agreement that includes an option for Luminary to exclusively license a novel BAFF target for use in CAR-T (chimeric antigen receptor T cells) constructs. The BAFF target was discovered by Reshmi Parameswaran, PhD, an assistant professor […] The post Luminary Therapeutics and Case Western Reserve University Enter Formal Collaboration appeared first on Luminary Therapeutics .
GSK announces FDA Advisory Committee meeting to review belantamab mafodotin for the treatment of patients with relapsed/refractory multiple myeloma
GSK
2020-06-19
multiple myeloma
GSK announces FDA Advisory Committee meeting to review belantamab mafodotin
GSK announces new data presentations from the DREAMM programme exploring investigational belantamab mafodotin in patients with relapsed/refractory multiple myeloma
GSK
2020-06-04
pivotal
multiple myeloma
16 presentations, including new analyses from the pivotal DREAMM-2 study and initial results from the DREAMM-4 study
DREAMM-2 and DREAMM-6 data at ASCO reinforce the potential of GSK’s investigational belantamab mafodotin in patients with relapsed/refractory multiple myeloma
GSK
2020-05-27
multiple myeloma
13-month update on the DREAMM-2 study shows median overall survival of 13.7 months and median duration of response of 11 months
FDA grants priority review of Nucala for patients with Hypereosinophilic Syndrome (HES)
GSK
2020-05-27
priority review
An approval would give Nucala a third indication in an eosinophil-driven disease
Nucala (mepolizumab) is the first anti-IL5 biologic to report positive phase 3 results in patients with nasal polyps
GSK
2020-04-03
pivotal
Pivotal data support regulatory filing for additional eosinophil-driven disease
GSK announces European Medicines Agency (EMA) accepted marketing authorisation application for belantamab mafodotin for the treatment of relapsed or refractory multiple myeloma
GSK
2020-02-03
multiple myeloma
Belantamab mafodotin accepted for accelerated assessment by the EMA’s Committee for Human Medicinal Products (CHMP)
US Food and Drug Administration (FDA) grants priority review of belantamab mafodotin for patients with relapsed or refractory multiple myeloma
GSK
2020-01-21
bcma
priority review
multiple myeloma
Belantamab mafodotin has potential to be the first anti-BCMA treatment available to patients
ViiV Healthcare receives complete response letter from US FDA for use of investigational cabotegravir and rilpivirine long-acting regimen in the treatment of HIV
GSK
2019-12-21
complete response
ViiV Healthcare received a CRL from the FDA regarding its application for cabotegravir and rilpivirine.
GSK announces positive headline results in phase 3 study of Benlysta in patients with lupus nephritis
GSK
2019-12-18
lupus
BLISS-LN achieves primary endpoint and all major secondary endpoints. On-track for regulatory submission during the first half of 2020.
Pivotal DREAMM-2 study demonstrated a clinically meaningful overall response rate with belantamab mafodotin (GSK2857916) for patients with relapsed/refractory multiple myeloma
GSK
2019-12-16
pivotal
multiple myeloma
Today announced treatment with the investigational single-agent belantamab mafodotin.
Nucala (mepolizumab) is the first treatment to show a significant reduction in flares for patients with Hypereosinophilic Syndrome (HES)
GSK
2019-11-13
pivotal
Positive data from a pivotal study supports new regulatory filing in HES
Our R&D
Gamida Cell
2019-10-30
omisirge
cell therapies
PIPELINE We’re advancing potentially curative cell therapies for cancer. Our proprietary nicotinamide (NAM) technology enhances and expands innate cells, offering potentially curative options for patients living with blood cancers. Explore our pipeline below for information on our clinical development candidates and ongoing clinical trials. The safety and efficacy of GDA-201 and Omisirge® (omidubicel-onlv) for aplastic […]
Intravenous Benlysta is the first biologic treatment to be approved for children with lupus in Europe
GSK
2019-10-29
lupus
European Commission has made a decision to extend to children five years and older, the existing adult indication for intravenous Benlysta
GSK and Lyell Immunopharma join forces to develop the next generation of cancer cell therapies
GSK
2019-10-08
cell therapies
Collaboration will combine Lyell’s technologies with GSK’s pipeline of cell therapies and manufacturing capability
GSK receives positive CHMP opinion for intravenous Benlysta in children with lupus aged five years and above
GSK
2019-09-20
lupus
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion
GSK announces positive headline results from the pivotal DREAMM-2 study for multiple myeloma
GSK
2019-08-23
pivotal
multiple myeloma
Belantamab mafodotin (GSK2857916) on track for regulatory submission by the end of 2019
ViiV Healthcare announces start of first-ever study to identify and evaluate approaches to implementing its once-monthly injectable HIV treatment in clinical practice
GSK
2019-07-08
priority review
The long-acting injectable regimen has been granted Priority Review status by the FDA, with a target approval date set for December 29, 2019
U.S. Food and Drug Administration accepts GSK’s application for ZEJULA (niraparib) in late stage ovarian cancer with priority review
GSK
2019-06-24
ovarian cancer
priority review
TESARO, submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for ZEJULA (niraparib).
GSK joins forces with the University of California to advance genomic research and improve drug discovery
GSK
2019-06-13
crispr
New ‘Laboratory for Genomics Research’ unites CRISPR pioneers with industry expertise to help unravel mysteries of the human genome
GSK announces data on anti-BCMA antibody-drug conjugate in patients with relapsed/refractory multiple myeloma to be presented at European Hematology Association (EHA) Congress
GSK
2019-06-12
bcma
multiple myeloma
GSK today announced new data on belantamab mafodotin will be presented at the 24th Congress of the European Hematology Association.
Healthcare Professionals
Gamida Cell
2019-05-22
allogeneic
cell therapy
Gamida Cell is developing allogeneic cell therapy products and candidates that are potentially curative for patients with hematologic malignancies. We are committed to supporting healthcare professionals who care for patients and share our goal of improving clinical outcomes. Clinical Trials Gamida Cell’s clinical development candidates are designed to address areas of significant medical need. Read […]
GSK receives US approval of Benlysta for intravenous use in children with lupus aged five years and above
GSK
2019-04-26
lupus
FDA approval marks the first medicine in the US approved for children with systemic lupus erythematosus (SLE)
GSK announces further positive data from DREAMM-1 study of anti-BCMA antibody-drug conjugate in patients with relapsed/refractory multiple myeloma
GSK
2019-03-21
bcma
multiple myeloma
Median progression-free survival extends to twelve months.
Data from GARNET study indicates robust activity of dostarlimab in patients with advanced or recurrent endometrial cancer
GSK
2019-03-19
phase 1/2
endometrial cancer
TESARO announced the presentation of data from the Phase 1/2 GARNET study
ViiV Healthcare presents positive, 48-week data from two pivotal phase III studies showing long-acting, injectable two-drug regimen of cabotegravir and rilpivirine has similar efficacy to daily, three-drug oral treatment in adults living with HIV-1 infection
GSK
2019-03-07
pivotal
Comprehensive data from ATLAS and FLAIR studies presented today at the 2019 Conference on Retroviruses and Opportunistic Infections
GSK announces positive phase 3 results for daprodustat in patients with anaemia associated with chronic kidney disease
GSK
2018-10-29
pivotal
Second of three pivotal studies intended to support regulatory filing in Japan in 2019
ViiV Healthcare submits New Drug Application to US FDA for single-tablet, two-drug regimen of dolutegravir and lamivudine for treatment of HIV
GSK
2018-10-18
priority review
Priority review voucher used with NDA submission with anticipated target action date of six months.
GSK and Tres Cantos Open Lab Foundation announce additional £5m funding for open innovation research in diseases impacting the developing world
GSK
2018-09-21
open
Additional £5m in GSK funding to support the Foundation’s research and development programmes in diseases of the developing world.
GSK receives complete response letter from US FDA for use of mepolizumab in COPD patients
GSK
2018-09-07
complete response
GlaxoSmithKline plc(LSE/NYSE: GSK) today received a complete response letter (CRL) from the US FDA regarding its application for mepolizumab
ViiV Healthcare reports positive 48-week results for first pivotal, phase III study for novel, long-acting, injectable HIV-treatment regimen
GSK
2018-08-15
pivotal
ATLAS study meets primary endpoint, showing similar efficacy of a once-a-month, investigational, injectable two-drug regimen
GSK and Adaptimmune complete transition of NY-ESO SPEAR T-cell therapy programme to GSK
GSK
2018-07-24
cell therapy
GlaxoSmithKline plc and Adaptimmune Therapeutics plc, announced the transition of the development programme for GSK3377794 to GSK.
GSK presents new results showing low rates of organ damage with long term use of Benlysta
GSK
2018-06-13
lupus
Results showing low rates of organ damage progression in patients with active systemic lupus erythematosus treated with Benlysta
GSK signs strategic agreement to transfer rare disease gene therapy portfolio to Orchard Therapeutics
GSK
2018-04-12
gene therapy
Agreement strengthens Orchard’s position as a global leader in gene therapy for rare diseases.
GSK starts phase III study of Benlysta and rituximab combination in systemic lupus erythematosus
GSK
2018-03-20
lupus
GSK today announced the start of a phase III study investigating Benlysta (belimumab)
GSK’s meningitis B vaccine Bexsero receives Breakthrough Therapy Designation from US FDA for prevention of Invasive Meningococcal Disease in children 2-10 years of age
GSK
2018-02-07
breakthrough therapy
GlaxoSmithKline plc announced that it has received Breakthrough Therapy Designation from the U.S. FDA for its meningitis B vaccine Bexsero
GSK presents promising new data for priority BCMA asset from its emerging Oncology pipeline at 59th ASH meeting
GSK
2017-12-11
bcma
Data from dose expansion phase of DREAMM-1 study
New data supports the safety and efficacy of GSK’s Shingrix in preventing shingles in autologous haematopoietic stem cell transplant patients
GSK
2017-12-06
autologous
GSK announced new data from a Phase III clinical study
GSK receives European marketing authorisation for self-injectable formulation of Benlysta for the treatment of systemic lupus erythematosus
GSK
2017-11-13
lupus
The approval is for a single-dose prefilled syringe and a single-dose prefilled pen presentation, administered as a once weekly
First long-term efficacy analysis on the effect of GSK’s Benlysta on rate of organ damage progression in SLE versus standard therapy alone
GSK
2017-11-08
lupus
GSK announced results of the first study assessing levels of organ damage in patients with active systemic lupus erythematosus (SLE)
GSK’s investigational BCMA antibody-drug conjugate receives Breakthrough Therapy Designation from US FDA for relapsed and refractory multiple myeloma
GSK
2017-11-02
bcma
multiple myeloma
breakthrough therapy
GlaxoSmithKline plc (LSE/NYSE: GSK) announced it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration
GSK receives approval for Benlysta in Japan for the treatment of systemic lupus erythematosus
GSK
2017-09-27
lupus
GSK announced today that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved Benlysta (belimumab)
GSK receives CHMP positive opinion for self-injectable formulation of Benlysta for systemic lupus erythematosus
GSK
2017-09-15
lupus
GSK today announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion
GSK exercises option on phase I/II NY-ESO T-cell therapy (GSK3377794)
GSK
2017-09-07
cell therapy
Today GSK announced that it has exercised the option to obtain an exclusive global license from Adaptimmune
GSK receives FDA approval for a new self-injectable formulation of Benlysta (belimumab) for systemic lupus erythematosus
GSK
2017-07-21
lupus
GSK announced today that the US Food and Drug Administration (FDA) has approved a new subcutaneous formulation of Benlysta
GSK announces headline phase III results of mepolizumab in patients with severe chronic obstructive pulmonary disease
GSK
2017-05-10
pivotal
GSK plc (LSE/NYSE:GSK) announced preliminary results of two pivotal phase III studies evaluating the efficacy and safety of mepolizumab
GSK submits regulatory application in Japan for belimumab in systemic lupus erythematosus
GSK
2016-12-13
lupus
Belimumab is a human monoclonal antibody that selectively targets B-lymphocyte stimulator an important factor in the survival of B cells.
GSK announces phase lll study of mepolizumab meets co-primary endpoints and all secondary endpoints in patients with eosinophilic granulomatosis with polyangiitis
GSK
2016-11-23
pivotal
Both co-primary endpoints and all secondary endpoints were met in a pivotal phase III study.
GSK announces positive results in fourth consecutive pivotal trial of Benlysta® (belimumab) in SLE
GSK
2016-11-13
pivotal
Data in Japan, China and South Korea will form basis of new regulatory submissions for Benlysta
GSK announces regulatory submissions for subcutaneous formulation of Benlysta® (belimumab) for patients with systemic lupus disease
GSK
2016-09-23
lupus
It has filed regulatory submissions in the US and Europe for Benlysta® (belimumab) for approval.
ARIA study shows superior efficacy of Triumeq® for treatment-naïve women living with HIV
GSK
2016-07-18
open
ViiV Healthcare today presented 48-week data from the phase IIIb, open-label, international, multi-centre ARIA study
StrimvelisTM receives European marketing authorisation to treat very rare disease, ADA-SCID
GSK
2016-05-27
ada-scid
gene therapy
GSK, Fondazione Telethon and Ospedale San Raffaele gain approval to provide life-saving gene therapy to patients.
GSK, Fondazione Telethon and Ospedale San Raffaele announce publication of pivotal safety and efficacy of gene therapy for children with ADA-SCID
GSK
2016-05-25
pivotal
ada-scid
gene therapy
GSK today announced the publication in BLOOD of the long-term safety and efficacy data from an analysis of 18 children with ADA-SCID
GSK receives positive CHMP opinion in Europe for Strimvelis™, the first gene therapy to treat very rare disease, ADA-SCID
GSK
2016-04-01
ada-scid
gene therapy
GSK announces a positive opinion recommending marketing authorisation for Strimvelis
GSK and Miltenyi Biotec establish cell and gene therapy collaboration
GSK
2016-03-16
car-t
gene therapy
Collaboration includes discovery programme for CAR-T cell-based oncology therapies
GSK names winners of 2015 Discovery Fast Track Challenge
GSK
2016-03-04
fast track
Six winning proposals were selected from 378 entries submitted from 21 countries across Europe and North America
GSK receives positive top-line results from sirukumab phase III programme supporting regulatory filings for rheumatoid arthritis in 2016
GSK
2015-12-16
top-line results
GSK today announced it has received positive top-line results from the phase III programme investigating sirukumab
GSK announces positive results from phase III BLISS-SC study of Benlysta® (belimumab) administered subcutaneously in patients with systemic lupus erythematosus
GSK
2015-11-07
lupus
GSK announces positive results from phase III BLISS-SC study of Benlysta® (belimumab) in patients with systemic lupus erythematosus
GSK’s candidate shingles vaccine demonstrates 90% efficacy against shingles in people 70 years of age and over
GSK
2015-10-27
pivotal
GSK today announced that the second pivotal phase III study of its candidate vaccine Shingrix™ successfully met its primary objective
GSK to divest ofatumumab for auto-immune indications to Novartis for up to $1 billion plus royalties
GSK
2015-08-21
multiple sclerosis
GSK announced an agreement with Novartis Pharma to divest its rights in ofatumumab for auto-immune indications, incl. multiple sclerosis.
GSK announces outcome of US FDA Advisory Committee recommending approval of mepolizumab for the treatment of adults with severe asthma
GSK
2015-06-11
bla
GSK announced outcome regarding the BLA for mepolizumab as an add-on maintenance treatment for severe asthma with eosinophilic inflammation.
GSK, Fondazione Telethon and Ospedale San Raffaele announce EU regulatory submission for gene therapy to treat rare disease ADA-SCID
GSK
2015-05-05
ada-scid
gene therapy
Today announced the submission of a marketing application to the EMA for a gene therapy (GSK2696273) to treat patients with a rare disease
GSK’s 2015 Discovery Fast Track Challenge seeks early drug discovery partnerships with academics in Europe, Canada and US
GSK
2015-03-24
fast track
GSK is encouraging scientists to submit their novel early drug discovery research proposals into its Discovery Fast Track Challenge
Pivotal phase III study of GSK shingles candidate vaccine meets its primary endpoint
GSK
2014-12-18
pivotal
GSK announced a phase III study to assess the efficacy of HZ/su, investigational vaccine for shingles, has met its primary endpoint.
GSK names winners of 2014 Discovery Fast Track Challenge
GSK
2014-12-01
fast track
GSK announced the winners of its second Discovery Fast Track Challenge for academic researchers & drug discovery scientists at GSK
GSK launches first call for proposals for research in to non-communicable diseases in sub-Saharan Africa
GSK
2014-11-14
open
GSK launched first call for proposals for its Africa NCD Open Lab, to support much-needed scientific research into NCDs in Africa.
Stiefel, a GSK company, announces start of phase III study of subcutaneous ofatumumab for pemphigus vulgaris
GSK
2014-10-07
pemphigus vulgaris
Stiefel, announced phase III study to evaluate the efficacy and safety of subcutaneous ofatumumab in patients with pemphigus vulgaris
GSK announces overall survival results from phase III BREAK-3 study of Tafinlar® (dabrafenib) in patients with BRAF V600E-mutant metastatic melanoma
GSK
2014-09-29
melanoma
GSK updated results for Tafinlar® from a planned analysis of the phase III BREAK-3 study in 250 patients with BRAFV600E metastatic melanoma.
GSK joins with research councils in UK and South Africa to tackle non-communicable diseases in Africa
GSK
2014-09-09
open
GSK announced a £5m collaboration with UK and SAMRC, to support much-needed research into NCDs in Africa, part of GSK Open Lab initiative.
Margaux discovering the world thanks to gene therapy
Fondazione Telethon
2014-09-05
ada-scid
gene therapy
She was the first Belgian girl with the rare immunodeficiency ADA-SCID treated with the gene therapy approach developed by Telethon researchers. From the risk of a life of seclusion, she has conquered the possibility of doing whatever she likes.
GSK and Genmab announce positive interim result for phase III study of ofatumumab as maintenance therapy for relapsed CLL
GSK
2014-07-31
cll
GSK & GEN announced that an Independent Data Monitoring Committee (IDMC) interim analysis of a phase III study, PROLONG (OMB 112517)
Trametinib (Mekinist™) and dabrafenib (Tafinlar™) combination demonstrated overall survival benefit compared to vemurafenib; phase III BRAF V600-mutant metastatic melanoma study stopped early
GSK
2014-07-17
melanoma
GSK announced that the IDMC recommended COMBI-v phase III study of Mekinist™, in combination with Tafinlar™ be stopped early.
GSK receives EU marketing authorisation for Mekinist™ (trametinib) for patients with unresectable or metastatic melanoma with a BRAF V600 mutation
GSK
2014-07-04
melanoma
GSK announced, the EC has granted marketing authorisation for trametinib as treatment of patients with unresectable/metastatic melanoma
GSK and Genmab announce top-line results from a Phase III study of ofatumumab versus physicians’ choice for bulky fludarabine-refractory CLL
GSK
2014-06-27
cll
top-line results
GSK & GEN announced today that the Phase III study of ofatumumab (Arzerra™) did not meet its primary endpoint of PFS.
GSK announces the start of a phase III study with eltrombopag in patients with myelodysplastic syndromes
GSK
2014-06-25
myelodysplastic
GSK announced the start of a Phase III study to evaluate effects of eltrombopag with azacitidine versus placebo in patients with MDS.
GSK announces start of phase III cardiovascular outcomes study with losmapimod in patients with acute coronary syndrome
GSK
2014-06-05
pivotal
GSK today announced the start of a pivotal phase III study to evaluate the effects of losmapimod in patients presenting with ACS.
GSK announces Phase III ALTTO results for anti-HER2 therapy combination in the adjuvant breast cancer treatment setting
GSK
2014-06-01
her2
breast cancer
GSK today announced that the Phase III study of two anti-HER2 agents, lapatinib (Tykerb™/Tyverb™) and trastuzumab.
motherapy for the treatment of relapsed or refractory diffuse large b-cell lymphoma
GSK
2014-05-19
dlbcl
b-cell lymphoma
large b-cell lymphoma
diffuse large b-cell lymphoma
GSK & GEN announced that the Phase III study ORCHARRD of Arzerra™ or rituximab plus chemotherapy for DLBCL did not meet its primary endpoint
GSK receives positive CHMP opinion for Mekinist™ (trametinib) in metastatic melanoma with a BRAF V600 mutation
GSK
2014-04-25
melanoma
(LSE/NYSE: GSK) today announced that the Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion on Mekinist™
GSK and Genmab receive FDA approval for Arzerra
GSK
2014-04-17
cll
GSK & GEN announced today that the FDA has approved a sBLA for Arzerra® (ofatumumab) for CLL
GSK presents data from Phase III STABILITY study of darapladib in patients with chronic coronary heart disease
GSK
2014-03-30
pivotal
GSK presented data from the pivotal Phase III STABILITY study of darapladib at the American College of Cardiology 63rd Annual Session
GSK publishes historical quarterly restated financial information
GSK
2014-03-21
quarterly
GSK will adopt a revised presentation for the analysis of its Pharmaceuticals and Vaccines turnover by segment, product and therapeutic area
GSK announces positive results from phase III studies for mepolizumab in severe eosinophilic asthma
GSK
2014-03-12
pivotal
GSK announced that, pivotal phase III study of mepolizumab, an investigational IL-5 antagonist monoclonal antibody, met its primary endpoint
GSK increases stake in Indian Pharmaceuticals subsidiary to 75 per cent after Open Offer
GSK
2014-03-10
open
GSK announced that, it has successfully increased its stake in its publicly-listed pharmaceuticals subsidiary in India.
GSK kicks off 2014 Discovery Fast Track Challenge for academic drug hunters in Europe, Canada and USA
GSK
2014-02-25
fast track
GSK is inviting academic scientists to enter their most innovative drug research proposals into its 2014 Discovery Fast Track Challenge.
GSK gains FDA Breakthrough Therapy designation for Promacta®/Revolade® (eltrombopag) for severe aplastic anaemia
GSK
2014-02-03
breakthrough therapy
GSK announced that the U.S. FDA has granted Breakthrough Therapy designation for Promacta®/Revolade® (eltrombopag).
GSK announces headline results for Phase III study of the combination of Tafinlar® (dabrafenib) and Mekinist® (trametinib) in metastatic melanoma
GSK
2014-01-24
melanoma
GSK announced that a Phase III study of the combination of Tafinlar® (dabrafenib) and Mekinist® (trametinib) met its primary endpoint of PFS
Tafinlar® receives FDA Breakthrough Therapy designation for non-small cell lung cancer with BRAF mutation
GSK
2014-01-13
lung cancer
breakthrough therapy
GSK announced today that the U.S. FDA has granted Breakthrough Therapy designation for Tafinlar® (dabrafenib).
Prosensa regains rights to drisapersen from GSK and retains rights to all other programmes for the treatment of Duchenne muscular dystrophy (DMD)
GSK
2014-01-13
dmd
duchenne muscular dystrophy
Prosensa Holding N.V. (NASDAQ: RNA) and GlaxoSmithKline (GSK) today announced that Prosensa has regained all rights from GSK to drisapersen.
GSK gains accelerated FDA approval for combination use of Mekinist® (trametinib) and Tafinlar® (dabrafenib)
GSK
2014-01-09
melanoma
GSK announced today that the FDA has approved Mekinist® (trametinib) for use with Tafinlar® (dabrafenib) for the treatment melanoma
GSK and MMV announce FDA Breakthrough Therapy designation for tafenoquine for Plasmodium vivax malaria
GSK
2013-12-20
breakthrough therapy
GSK and Medicines for Malaria Venture (MMV) announced today that the FDA has granted Breakthrough Therapy designation for tafenoquine.
GSK and Genmab Receive Priority Review from FDA for Arzerra® (ofatumumab) as 1st Line Treatment for Chronic Lymphocytic Leukaemia (CLL)
GSK
2013-12-17
cll
priority review
GSK and Genmab Receive Priority Review from FDA for Arzerra® (ofatumumab) as 1st Line Treatment for Chronic Lymphocytic Leukaemia (CLL)
GSK initiates voluntary open offer to increase stake in its pharmaceuticals subsidiary in India
GSK
2013-12-16
open
GSK today announced a Voluntary Open Offer to increase its stake in its publicly-listed pharmaceuticals subsidiary in India.
GSK and Theravance announce positive results from pivotal phase III study for fluticasone furoate/vilanterol in asthma
GSK
2013-12-06
pivotal
GSK & THRX announced positive results from a phase III efficacy and safety study of fluticasone furoate “FF”/vilanterol “VI”.
GSK and Amicus Therapeutics announce revised Fabry agreement
GSK
2013-11-20
fabry disease
GSK & Amicus announced that Amicus has obtained global rights to develop chaperone migalastat HCl as monotherapy for Fabry disease.
GSK announces top-line results from pivotal Phase III study of darapladib in chronic coronary heart disease
GSK
2013-11-12
pivotal
top-line results
Study did not meet the primary endpoint measure.
GSK names inaugural winners in unique competition for academic drug hunters
GSK
2013-11-06
fast track
GSK announced selection of eight winners in its first Discovery Fast Track competition.
Regulatory update – GSK and Genmab announce submission to US regulatory authorities for Arzerra® (ofatumumab) as 1st line treatment of Chronic Lymphocytic Leukaemia (CLL)
GSK
2013-10-18
cll
GSK and Genmab A/S announced today the submission of a supplemental Biologics License Application to the US Food and Drug Administration
Regulatory update – GSK and Genmab announce European submission to regulatory authorities for Arzerra® (ofatumumab) as 1st line treatment of Chronic Lymphocytic Leukaemia (CLL)
GSK
2013-10-04
cll
GSK & Genmab A/S announced submission of Marketing Authorisation for Arzerra in combination with alkylator-based therapy, for CLL patients
GSK and Prosensa announce primary endpoint not met in Phase III study of drisapersen in patients with Duchenne Muscular Dystrophy
GSK
2013-09-20
dmd
duchenne muscular dystrophy
GSK & Prosensa announced that Phase III clinical study of drisapersen, treatment for DMD did not meet the primary endpoint.
GSK receives Priority Review from FDA for dabrafenib/trametinib combination in metastatic melanoma
GSK
2013-09-16
melanoma
priority review
GSK announced that the FDA has granted Priority Review designation to the use of Tafinlar® & Mekinist® for the treatment of melanoma
FDA grants GSK and Genmab’s Arzerra® (ofatumumab) Breakthrough Therapy designation for previously untreated chronic lymphocytic leukaemia
GSK
2013-09-13
cll
breakthrough therapy
GSK & Genmab A/S announced today that FDA has granted Breakthrough Therapy designation for Arzerra® (ofatumumab) with chlorambucil for CLL
The investigational MAGE-A3 antigen-specific cancer immunotherapeutic does not meet first co-primary endpoint in Phase III melanoma clinical trial
GSK
2013-09-05
melanoma
In line with IDMC unanimous recommendation, GSK will continue the DERMA trial until the second co-primary endpoint is assessed.
GSK announces phase III study of vercirnon in patients with moderate-to-severe Crohn’s disease did not meet its primary endpoint
GSK
2013-08-23
crohn's disease
GSK announced Phase III studies, investigating vercirnon in patients with Crohn's disease did not achieve the primary endpoint
Regulatory update: albiglutide US PDUFA date extended by three months
GSK
2013-08-02
pdufa
GSK announced that PDUFA goal date for albiglutide, treatment for adult patients with type 2 diabetes, has been extended by three months.
Regulatory update – GSK announces US submission for dabrafenib/trametinib combination in metastatic melanoma
GSK
2013-07-09
melanoma
GSK today announced submission of NDAs to the FDA for use of dabrafenib, a BRAF inhibitor, in combination with trametinib, a MEK inhibitor.
GlaxoSmithKline’s drisapersen (previously GSK2402968/PRO051) to receive Food and Drug Administration Breakthrough Therapy designation
GSK
2013-06-27
breakthrough therapy
Breakthrough Therapy designation is one of several programmes created by the FDA to expedite the development and review of drugs.
GSK announces Phase III data for TYKERB/TYVERB® (lapatinib) in combination with chemotherapy for advanced HER2-positive gastric cancer
GSK
2013-06-03
her2
gastric cancer
GSK announced study of lapatinib with chemotherapy in patients with HER2-positive advanced gastric cancer did not meet the primary endpoint
GSK and Genmab announce positive top-line results from pivotal trial of ARZERRA® (ofatumumab) combined with chlorambucil in previously untreated chronic lymphocytic leukaemia
GSK
2013-05-29
cll
pivotal
top-line results
GSK & Genmab announce phase III study of ARZERRA® with chlorambucil versus chlorambucil in patients with CLL met its primary endpoint.
Funding boost for GSK’s open innovation research into diseases affecting the developing world
GSK
2013-05-03
open
GSK announced a funding of up to £5m from the Wellcome Trust to support discovering and developing new treatments for diseases
Regulatory update: Trametinib US PDUFA date extended to 3 September 2013
GSK
2013-05-01
pdufa
GSK today announced that the US Prescription Drug User Fee Act (PDUFA) goal date for trametinib.
Regulatory update: GlaxoSmithKline receives complete response from FDA for candidate pandemic H5N1 adjuvanted influenza vaccine
GSK
2013-03-25
complete response
GSK announced today that it has received a Complete Response letter from the FDA .
ViiV Healthcare announces FDA priority review designation for dolutegravir as a potential treatment for HIV infection
GSK
2013-02-15
priority review
ViiV announced that the FDA has granted a priority review designation to dolutegravir submitted for the treatment of HIV infection.
Regulatory update – GSK announces European submission for MEK monotherapy and BRAF/MEK combination therapy in metastatic melanoma
GSK
2013-02-07
melanoma
GSK announced submission of a Marketing Authorisation Application to EMA for MEK with BRAF for the treatment of adult patients with melanoma
GlaxoSmithKline starts Phase III study to test combined BRAF/MEK inhibition in patients with BRAF positive melanoma following surgery
GSK
2013-02-01
melanoma
GSK today announced the start of COMBI-AD, a Phase III study evaluating the combination of dabrafenib, its BRAF inhibitor and trametinib.
Regulatory update – GSK announces submission of albiglutide BLA to the US FDA for the treatment of type 2 diabetes
GSK
2013-01-14
bla
GSK announced a regulatory submission to the FDA for albiglutide, an investigational treatment for adult patients with type 2 diabetes.
GSK announces further initiatives to advance openness and collaboration to help tackle global health challenges
GSK
2012-10-11
open
GSK CEO Sir Andrew Witty will outline new steps to build on progress resulting from open innovation approach to R&D.
European Commission grants marketing authorisation for pazopanib (Votrient®) in the treatment of certain advanced soft tissue sarcoma subtypes
GSK
2012-08-07
sarcoma
soft tissue sarcoma
GSK announced today the EC has granted pazopanib marketing authorisation for the treatment of aSTS who have received prior chemotherapy.
Regulatory update – GSK announces submissions in the EU and US for dabrafenib and trametinib
GSK
2012-08-03
melanoma
GSK announced submissions in EU & US related to single-agent use of dabrafenib & trametinib to treat BRAF V600 mutation metastatic melanoma.
GlaxoSmithKline and Amicus Therapeutics expand Fabry disease collaboration
GSK
2012-07-17
fabry disease
GSK & FOLD announced an expansion of their collaboration to develop the investigational pharmacological migalastat HCl for Fabry disease.
US Regulatory Update – Tykerb® (lapatinib)
GSK
2012-07-12
her2
breast cancer
GSK announced it has withdrawn a sNDA to the FDA for lapatinib with trastuzumab for HER2 (ErbB2)-positive metastatic breast cancer
GSK announces start of Phase III programme for combination of dabrafenib (GSK2118436) and trametinib (GSK1120212) in BRAF V600 mutation-positive metastatic cutaneous melanoma
GSK
2012-05-29
melanoma
The start of a Phase III programme evaluating the combination of dabrafenib, its BRAF inhibitor, and trametinib, its MEK inhibitor,
FDA approves Votrient® for treatment of patients with certain types of advanced soft tissue sarcoma
GSK
2012-04-26
sarcoma
soft tissue sarcoma
GSK announced the U.S. FDA has approved Votrient for the treatment of soft tissue sarcoma who have received prior chemotherapy.
Shionogi-ViiV Healthcare announces initial data from pivotal phase III study of dolutegravir in HIV
GSK
2012-04-02
pivotal
SPRING-2 study meets primary endpoint of non-inferiority of dolutegravir compared to raltegravir over 48 weeks in treatment of HIV patients.
GSK announces positive vote from FDA panel for pazopanib in certain advanced soft tissue sarcomas
GSK
2012-03-20
sarcoma
soft tissue sarcoma
GSK announced the ODAC voted 11:2 in support a favourable benefit; risk assessment for use of Votrient in patients with soft tissue sarcoma.
GSK and Theravance announce initial outcomes from pivotal Phase III studies for once-daily Relovair™* in COPD and asthma
GSK
2012-01-09
pivotal
GSK & THRX announced completion of phase III registration programme for Relovair in patients with COPD and in patients with asthma.
GlaxoSmithKline and Human Genome Sciences initiate phase III trial of belimumab (Benlysta®) administered subcutaneously in subjects with systemic lupus erythematosus
GSK
2011-12-15
lupus
GSK & HGS announced dosing has been initiated in BLISS-SC, a new Phase III study of belimumab administered SC to adults with SLE.
GSK presents phase lll data for lapatinib at San Antonio breast cancer symposium
GSK
2011-12-08
her2
breast cancer
Improvement in disease-free survival does not reach statistical significance in study of Tykerb monotherapy in HER2 positive breast cancer.
Regulatory Update: GlaxoSmithKline submits additional information for candidate meningococcal and Hib combination vaccine
GSK
2011-12-01
bla
complete response
GSK submitted information to the FDA supporting BLA for combination vaccine MenHibrix. submitted in response to Complete Response letter.
GSK joins WIPO Re:Search open innovation platform as part of its commitment to tackling diseases of the developing world
GSK
2011-10-26
open
GSK joined WIPO Re:Search in innovation strategy to help accelerate the development of treatments of NTDs affecting over a billion each year
Regulatory Update: GlaxoSmithKline receives Complete Response letter from FDA for candidate meningococcal and Hib combination vaccine
GSK
2011-09-23
complete response
GSK announced it's received a Complete Response letter from FDA for MenHibrix ,active immunization of infants 6 weeks through 15 months
GlaxoSmithKline and Amicus Therapeutics commence second phase III study of Amigal™ for Fabry disease
GSK
2011-09-12
fabry disease
GlaxoSmithKline and Amicus Therapeutics commence second phase III study of Amigal™ for Fabry disease
Lapatinib clinical trial update
GSK
2011-09-09
her2
breast cancer
Review - Phase III study of lapatinib & trastuzumab in patients with HER2+ early stage breast cancer to continue trials without modification
GSK welcomes external scientists to diseases of the developing world research campus
GSK
2011-06-28
open
GSK will provide support, expertise and facilities as part of our open innovation strategy to spur global collaboration in research
GSK and Theravance announce results of two pivotal Phase III studies for Relovair in COPD
GSK
2011-06-02
pivotal
GSK and THRX announced the results of two pivotal 6-month efficacy and safety phase III studies of Relovair for patients with COPD.
GlaxoSmithKline and Human Genome Sciences receive positive opinion in Europe from the CHMP for Benlysta® belimumab
GSK
2011-05-20
lupus
GSK & HGS announced that EMA's CHMP issued a positive opinion, recommending marketing authorisation for Benlysta, a lupus therapy.
Regulatory update – GSK and Valeant respond to FDA on ezogabine
GSK
2011-04-18
complete response
GSK & VRX submitted on 15/04/11 the response to the FDA Complete Response letter received on 30 November 2010 for the NDA for ezogabine.
Regulatory Update - Tyverb® lapatinib
GSK
2011-04-15
her2
breast cancer
GSK submitted a MAA to EMA for lapatinib in with paclitaxel for the treatment of metastatic breast cancer with over-express HER2 (ErbB2).
GlaxoSmithKline and Human Genome Sciences announce FDA approval of Benlysta® (belimumab) for the treatment of systemic lupus erythematosus
GSK
2011-03-09
lupus
GSK & HGS announced that the FDA has approved Benlysta for the treatment of active, autoantibody-positive SLE who are receiving therapy.
GlaxoSmithKline and Human Genome Sciences announce publication of BLISS-52 phase lll study results for Benlysta® in The Lancet
GSK
2011-02-07
lupus
GSK & HGS announced publication Benlysta in autoantibody-positive patients with active systemic lupus erythematosus (SLE) in The Lancet.
GSK receives FDA Complete Response letter for Avodart for prostate cancer risk reduction supplemental indication
GSK
2011-01-26
prostate cancer
complete response
GSK received a Complete Response letter from the FDA for the sNDA for Avodart for reducing the risk of prostate cancer in men.
GlaxoSmithKline announces start of two phase III studies in advanced/metastatic melanoma
GSK
2011-01-24
melanoma
GSK announced the start of two global Phase III studies in advanced or metastatic melanoma patients with a BRAF V600 mutation.
GSK and Prosensa announce start of Phase III study of investigational Duchenne Muscular Dystrophy medication
GSK
2011-01-19
dmd
duchenne muscular dystrophy
GSK and Prosensa announced - first patient has commenced treatment in the Phase III study investigating GSK2402968 in ambulant boys with DMD
GlaxoSmithKline and ChemoCentryx announce initiation of Phase III study of GSK’786, formerly Traficet-EN™, for the treatment of Crohn’s disease
GSK
2011-01-12
crohn's disease
GSK & ChemoCentryx announced, first patient with Crohn's disease has initiated treatment in study comparing Traficet-EN, to placebo.
Phase III study in HER2-positive advanced breast cancer shows overall survival benefit when Tykerb is combined with paclitaxel
GSK
2010-12-09
her2
breast cancer
Phase III study, women with untreated HER2+ metastatic breast cancer improved when treated with a combination of lapatinib & paclitaxel
Data from Tykerb investigational phase III studies in neo-adjuvant HER2-positive breast cancer presented at breast cancer symposium
GSK
2010-12-09
her2
breast cancer
Results from 2 Phase III studies examining Tykerb with trastuzumab in the neo-adjuvant setting of HER2+ breast cancer with chemotherapy
GlaxoSmithKline and Human Genome Sciences announce FDA extension of Benlysta® PDUFA target date to 10th March 2011
GSK
2010-12-02
bla
pdufa
priority review
GSK & HGS announced, FDA has extended the PDUFA target date for its priority review of BLA for Benlysta as a potential treatment for SLE.
GSK and Valeant announce receipt of U.S. FDA Complete Response letter for ezogabine
GSK
2010-11-30
complete response
GSK & VRX announced receipt of CRL from FDA for NDA for ezogabine*, a drug studied for the treatment of adults with partial-onset seizures.
Human Genome Sciences and GlaxoSmithKline announce vote of FDA advisory committee to recommend approval of Benlysta® for systemic lupus erythematosus
GSK
2010-11-15
lupus
GSK & HGS announced, FDA has voted 13 to 2 to recommend BENLYSTA for the treatment of autoantibody-positive patients with active SLE.
New study findings show no benefit of prescription omega-3 fatty acids in prevention of recurrent symptomatic atrial fibrillation in affected patients
GSK
2010-11-14
atrial fibrillation
Findings from study show treatment with high-dose prescription omega-3 fatty acids did not reduce the recurrence of symptomatic AF.
GSK and Amicus Therapeutics enter exclusive worldwide agreement to develop and commercialise Amigal™ for Fabry disease
GSK
2010-10-28
fabry disease
GSK and Amicus today announced a definitive agreement to develop and commercialise AmigalTM (migalastat HCl),
GSK, Fondazione Telethon and Fondazione San Raffaele to collaborate on gene therapy for rare diseases
GSK
2010-10-17
gene therapy
GSK, Fondazione Telethon and Fondazione San Raffaele today announced a new strategic alliance to research and develop novel treatments.
GSK and Valeant announce new U.S. FDA PDUFA goal date for ezogabine
GSK
2010-08-29
pdufa
GSK and VRX announced today FDA has extended the PDUFA goal date for ezogabine* to 30 Nov 2010, the original goal date was 30 August 2010.
GlaxoSmithKline and Human Genome Sciences announce FDA priority review designation for Benlysta® (belimumab) as a potential treatment for systemic lupus erythematosus
GSK
2010-08-18
lupus
priority review
GSK and Human Genome Sciences, Inc. today announced that the FDA has granted a priority review designation to Benlysta.
GSK and Genmab announce topline results from the concluded pivotal trial of Arzerra (ofatumumab) in fludarabine and alemtuzumab refractory chronic lymphocytic leukemia
GSK
2010-08-08
pivotal
leukemia
top line results
chronic lymphocytic leukemia
GSK and GEN announced today top line results from the concluded pivotal trial.
GlaxoSmithKline Receives Complete Response from FDA for Candidate Meningococcal and Hib Combination Vaccine
GSK
2010-06-10
complete response
Meningococcal and Hib diseases are caused by potentially deadly bacteria that can lead to meningitis and other very serious complications.
Regulatory update – BLA filed for Benlysta® (belimumab)
GSK
2010-06-09
bla
GSK announced today that Human Genome Sciences, Inc. (HGS) has submitted a Biologics License Application (BLA) to the U.S.
GlaxoSmithKline and Human Genome Sciences announce topline 76-week results of phase 3 trial of Benlysta™ in systemic lupus erythematosus
GSK
2010-04-19
lupus
GlaxoSmithKline (GSK) and Human Genome Sciences, Inc. (Nasdaq: HGSI) and today announced topline secondary endpoints from BLISS-76.
GlaxoSmithKline receives conditional marketing authorisation in the EU for Arzerra® (ofatumumab)
GSK
2010-04-18
cll
GSK and Genmab AS confirmed that EC has granted a conditional marketing authorisation for Arzerra for the treatment of refractory CLL.
GlaxoSmithKline and XenoPort receive FDA Complete Response letter for Horizant™ (GSK1838262/XP13512) for RLS
GSK
2010-02-16
complete response
GSK and XNPT received a Complete Response letter from FDA regarding NDA for gabapentin enacarbil) Extended-Release Tablets.
Results announcement for the fourth quarter 2009
GSK
2010-02-03
quarterly
See the quarterly results page for more information
GSK’s Tykerb® receives accelerated approval for first-line combination treatment of hormone receptor positive, HER2+/ErbB2+ metastatic breast cancer
GSK
2010-01-28
her2
breast cancer
accelerated approval
GlaxoSmithKline announced today that the U.S. Food and Drug Administration (FDA) has granted accelerated approval.
GSK announces ‘open innovation’ strategy to help deliver new and better medicines for people living in the world’s poorest countries
GSK
2010-01-19
open
'Open Lab' established with $8m seed funding for new research
GSK initiates second pivotal Phase III trial for investigational cardiovascular medication Darapladib
GSK
2009-12-09
pivotal
GSK today announced initiation of the second large-scale Phase III clinical trial to evaluate long-term treatment.
GlaxoSmithKline and XenoPort announce extension of GSK1838262 (XP13512) FDA review date to 9 February 2010
GSK
2009-11-05
pdufa
GSK and XNPT today announced that FDA has extended the original PDUFA goal date for its review of NDA for GSK to February 9, 2010.
World’s largest malaria vaccine trial now underway in seven African countries
GSK
2009-11-02
pivotal
Pivotal testing of RTS,S is on track for target enrollment of 16,000 children
GlaxoSmithKline and Human Genome Sciences announce positive results in second of two phase 3 trials of Benlysta in systemic lupus erythematosus
GSK
2009-11-01
lupus
Benlysta 10 mg/kg plus standard of care met its primary efficacy endpoint by achieving a statistically significant improvement.
Results announcement for the third quarter 2009
GSK
2009-10-28
quarterly
See the quarterly results page for more information.
GSK and Genmab receive accelerated approval for Arzerra™
GSK
2009-10-26
accelerated approval
Today, GlaxoSmithKline (GSK) and Genmab A/S (OMX: GEN) announced the accelerated approval of Arzerra TM (ofatumumab).
GlaxoSmithKline and Human Genome Sciences announce full presentation at ACR of positive phase 3 study results for Benlysta in systemic lupus erythematosus
GSK
2009-10-19
lupus
Benlysta (belimumab) significantly reduced SLE disease activity, disease flare rates and fatigue; delayed time-to-first SLE disease flare.
GlaxoSmithKline and Prosensa form alliance to fight Duchenne Muscular Dystrophy
GSK
2009-10-13
duchenne muscular dystrophy
GlaxoSmithKline plc (GSK) announced today that it has entered into a unique partnership with the UK Government.
GlaxoSmithKline update on FDA review of Cervarix
GSK
2009-09-29
bla
GSK announced the FDA has not yet completed the review of BLA for CERVARIX vaccine, review will continue beyond September 29, 2009.
GlaxoSmithKline and Genmab announce results from a study of Arzerra in rituximab refractory follicular NHL
GSK
2009-08-16
top-line results
GlaxoSmithKline (GSK) and Genmab A/S (OMX: GEN) announced today top-line results from an international multi-center study of Arzerra.
GlaxoSmithKline and Genmab announce top-line results for ofatumumab in rheumatoid arthritis
GSK
2009-07-28
top-line results
GSK & GEN announced results from a Phase III study of ofatumumab, treatment of RA in patients with no response to methotrexate.
Results announcement for the second quarter 2009
GSK
2009-07-21
quarterly
See the quarterly results page for more information
Human Genome Sciences and GlaxoSmithKline announce positive Phase 3 study results for Benlysta™ in systemic lupus erythematosus
GSK
2009-07-20
lupus
GSK & HGS announced that Benlysta met the primary endpoint in BLISS-52, Phase 3 trials in patients with serologically active SLE.
Regulatory update for REZONIC™ (casopitant mesylate)
GSK
2009-06-22
complete response
GSK confirmed today that it has received a complete response letterfrom the FDA related to the application for casopitant
FDA extends review of Arzerra™ (ofatumumab)
GSK
2009-06-16
bla
GSK & Genmab announced, FDA informed the companies they have extended the action date for the ofatumumab BLA application by three months.
FDA advisory panel makes favorable recommendation for ARZERRA (ofatumumab)
GSK
2009-05-28
cll
GSK & GEN announced that FDA voted 10:3 that ofatumumab, reasonably likely to predict clinical benefit for patients with chronic CLL.
GlaxoSmithKline and Genmab seek European marketing authorisation of Arzerra™ (ofatumumab) in advanced stage blood cancer
GSK
2009-02-05
cll
cd20
GSK & Genmab announced submission of MAA to EMEA for Arzerra, treatment of CLL, it targets a binding site on the CD20 molecule of B-cells.
Results announcement for the fourth quarter 2008
GSK
2009-02-04
quarterly
See the quarterly results page for more information.
GlaxoSmithKline and Genmab submit Arzerra (Ofatumumab) application to FDA for the treatment of advanced stage blood cancer
GSK
2009-01-29
bla
cll
GSK & GEN announced submission of BLA to the FD) for Arzerra to treat patients whose CLL is resistant (refractory) to previous therapies.
How Newborn Liver Cells Shape Growth and Future Treatments
Fondazione Telethon
1970-01-01
gene therapy
A discovery at SR-Tiget reveals the key liver cells driving organ growth and advancing pediatric gene therapy
FDA blesses CellTrans' Lantidra, the first cell therapy for Type 1 diabetes
CellTrans Inc.
1970-01-01
lantidra
cell therapy
gene therapy
Much attention has been paid to Vertex’s efforts to develop a stem cell therapy for Type 1 diabetes. | Much attention has been paid to Vertex’s efforts to develop a stem-cell therapy for Type 1 diabetes. But flying under the radar with an allogenic (donor) gene therapy for the disorder has been Chicago startup CellTrans. Thursday, the FDA signed off on CellTrans’ Lantidra (donislecel), the first cell therapy for type 1 diabetes.
Kyverna Therapeutics Highlights Potential of KYV-101 in Multiple Sclerosis with Data from Phase 1 Investigator-Initiated Trials to be Presented at ECTRIMS, Read More
Kyverna Therapeutics
1970-01-01
kyv-101
multiple sclerosis
‘Poor Quality’ Submission: CellTrans’ Lantidra BLA Marked By Missing And Incongruent Data
CellTrans Inc.
1970-01-01
bla
lantidra
Kyverna Therapeutics to Host Virtual KOL Event to Highlight Neuroimmunology CAR T Franchise, Read More
Kyverna Therapeutics
1970-01-01
car t
REGENXBIO Announces Completion of Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program
REGENXBIO
1970-01-01
pivotal
gene therapy
Kyverna Therapeutics to Host Conference Call on Interim Phase 2 Data from KYSA-6 Study of KYV-101 in Generalized Myasthenia Gravis, Read More
Kyverna Therapeutics
1970-01-01
kyv-101
myasthenia gravis
A Tripartite Alliance to Cure Seven Genetic Diseases
Fondazione Telethon
1970-01-01
gene therapies
Fondazione Telethon, Ospedale San Raffaele, and GlaxoSmithKline have entered into a strategic alliance to develop and make available innovative gene therapies for seven severe rare genetic diseases.
REGENXBIO Announces Completion of Enrollment in Pivotal Trials of Subretinal Surabgene Lomparvovec for Wet AMD
REGENXBIO
1970-01-01
pivotal
gene therapy
Over 1,200 participants enrolled in ATMOSPHERE ® and ASCENT ® pivotal trials, representing largest global gene therapy program ever conducted Subretinal surabgene lomparvovec on track to be first gene therapy for wet AMD Topline pivotal data expected in Q4 2026 ROCKVILLE, Md. , Oct.
The Armenise-Harvard Foundation invests one million dollars in brain research
Fondazione Telethon
1970-01-01
gene therapy
Thanks to the Career Development Award, neuroscientist Gabriele Ciceri will establish a new laboratory at the San Raffaele-Telethon Institute for Gene Therapy.
CellTrans’ Donislecel: Insulin Independence Data May Offer Path To Market In Type 1 Diabetes
CellTrans Inc.
1970-01-01
allogeneic
Proportion of subjects with freedom from exogenous insulin ‘might support the efficacy’ of the allogeneic islet cellular therapy, US FDA says after concluding that issues in two studies limit interpretability of primary analyses based on severe hypoglycemic events and HbA1c levels. An advisory committee will weigh clinical efficacy, safety and product characterization issues.
CellTrans’s Lantidra: The first cell therapy to personalise Type 1 diabetes treatment
CellTrans Inc.
1970-01-01
lantidra
cell therapy
Gene editing
Fondazione Telethon
1970-01-01
gene editing
gene therapy
A particular case of gene therapy is based on gene editing, a recent technology that intervenes in a precise manner to find and correct genetic errors in DNA, even at a single-letter level. Genetic editing is viewed as very promising for the gene therapy of the future, because it could enable faulty genes to be …
Kyverna Therapeutics Provides Business Update and Reports Second Quarter 2025 Financial Results, Read More
Kyverna Therapeutics
1970-01-01
financial results
Kyverna Therapeutics to Highlight Interim Phase 2 Data from KYV-101 KYSA-6 Study in Myasthenia Gravis at AANEM 2025, Read More
Kyverna Therapeutics
1970-01-01
kyv-101
myasthenia gravis
Beta Thalassemia: clinical trial provides encouraging evidence for efficacy of gene therapy
Fondazione Telethon
1970-01-01
gene therapy
beta thalassemia
Gene therapy, especially if administered early, could be an effective treatment strategy for beta thalassemia, a genetic disease that is quite common in Mediterranean countries, affecting over 7000 patients in Italy only.
ERC Consolidator Grants Awarded to Two SR-Tiget Researchers
Fondazione Telethon
1970-01-01
gene therapy
Daniela Cesana and Alessio Cantore presented two projects, respectively to investigate the impact, safety and efficacy of gene therapy through the 'signatures' of circulating DNA.
‘Poor Quality’ Submission: CellTrans’ Lantidra BLA Marked By Missing And Incongruent Data
CellTrans Inc.
1970-01-01
bla
lantidra
FDA's review of documents and source data files to assess product efficacy and subject safety was complicated by missing and inconsistent data, the clinical reviewer said, concluding there was insufficient data monitoring during clinical studies or in preparing the documents for regulatory submission.
Kyverna Therapeutics Provides Business Update and Reports Third Quarter 2025 Financial Results, Read More
Kyverna Therapeutics
1970-01-01
financial results
Aicardi-Goutières syndrome: when the immune system detects viruses where there are none
Fondazione Telethon
1970-01-01
gene therapy
An interview with Anna Kajaste-Rudnitski from the San Raffaele Telethon Institute for Gene Therapy in Milan, author of a study that gives new insights into this rare syndrome and provides also useful information to investigate the interaction between the immune system and viruses, including Sars-CoV-2.
Kyverna Therapeutics Highlights Neuroimmunology CAR T Franchise and Registrational Phase 3 Trial Design in Myasthenia Gravis at Virtual KOL Event, Read More
Kyverna Therapeutics
1970-01-01
car t
myasthenia gravis
REGENXBIO Reports Third Quarter 2025 Financial Results and Operational Highlights
REGENXBIO
1970-01-01
financial results
Gene therapy targeted to the brain: a complex but not impossible challenge
Fondazione Telethon
1970-01-01
gene therapy
Angela Gritti from the San Raffaele Telethon Institute of Milan talks about the efforts of applying gene therapy to one of the most difficult body organs to access.
Cell and Gene Therapies: A Review of Current and Future Treatments
CellTrans Inc.
1970-01-01
gene therapies
REGENXBIO to Host Conference Call on November 6 to Discuss Third Quarter 2025 Financial Results and Operational Highlights
REGENXBIO
1970-01-01
financial results
ROCKVILLE, Md. , Oct. 29, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Thursday, November 6 , at 8:00 a.m. ET to discuss its financial results for the third quarter ended September 30, 2025 , and operational highlights.
REGENXBIO Announces Completion of Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program
REGENXBIO
1970-01-01
pivotal
gene therapy
accelerated approval
Patients treated with RGX-202 demonstrate consistent, robust microdystrophin expression and functional improvement compared to natural history in Phase I/II portion of AFFINITY DUCHENNE ® trial supporting potential approval via the accelerated approval pathway REGENXBIO continues to enroll patients
Attya Omer Named Runner-Up in the Nature Inspiring Women in Science Awards
Fondazione Telethon
1970-01-01
gene therapy
The scientist of SR-TIGET and Università Vita-Salute San Raffaele recognized internationally for her vision in advancing gene therapy and inspiring women in science.
REGENXBIO to Host Conference Call on November 6 to Discuss Third Quarter 2025 Financial Results and Operational Highlights
REGENXBIO
1970-01-01
financial results
Gene Therapy Demonstrates Efficacy in Treating Wiskott-Aldrich Syndrome
Fondazione Telethon
1970-01-01
gene therapy
wiskott-aldrich
wiskott–aldrich
wiskott-aldrich syndrome
wiskott–aldrich syndrome
A clinical study confirms the efficacy of gene therapy as a treatment for Wiskott–Aldrich Syndrome (WAS), a serious rare genetic disease affecting blood cells.
Kyverna Therapeutics Announces Positive Interim Phase 2 Data from KYSA-6 Study of KYV-101 in Generalized Myasthenia Gravis at AANEM 2025, Read More
Kyverna Therapeutics
1970-01-01
kyv-101
myasthenia gravis
CellTrans’ Donislecel: Insulin Independence Data May Offer Path To Market In Type 1 Diabetes
CellTrans Inc.
1970-01-01
allogeneic
Proportion of subjects with freedom from exogenous insulin ‘might support the efficacy’ of the allogeneic islet cellular therapy, US FDA says after concluding that issues in two studies limit interpretability of primary analyses based on severe hypoglycemic events and HbA1c levels. An advisory committee will weigh clinical efficacy, safety and product characterization issues.
Kyverna Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2024 Financial Results, Read More
Kyverna Therapeutics
1970-01-01
financial results
Kyverna Therapeutics Provides Business Update and Reports First Quarter 2025 Financial Results, Read More
Kyverna Therapeutics
1970-01-01
financial results
FDA blesses CellTrans' Lantidra, the first cell therapy for Type 1 diabetes
CellTrans Inc.
1970-01-01
lantidra
cell therapy
CellTrans’s Lantidra: The first cell therapy to personalise Type 1 diabetes treatment
CellTrans Inc.
1970-01-01
lantidra
cell therapy
Precision and personalised medicine has a significant impact on how pharma companies and other stakeholders engage with patients, providing treatments that better fit specific patient populations, rather than generating inconsistent results in a broader patient population.
Cell and Gene Therapies: A Review of Current and Future Treatments
CellTrans Inc.
1970-01-01
gene therapies
Gene therapy: target liver
Fondazione Telethon
1970-01-01
gene therapy
gene transfer
TIGET: a liver-directed gene transfer platform applicable to various metabolic diseases was developed thanks to a group of reserchers led by Cantore.
TIGEM secures ERC grant for programmable gene‑circuit therapy
Fondazione Telethon
1970-01-01
gene therapy
di Bernardo’s DIMERCIRCUITS integrates AI and synthetic biology to advance precision gene therapy.
Kyverna Therapeutics Highlights Potential of KYV-101 in Rheumatoid Arthritis with Phase 1 Data from Investigator-Initiated Trial Presented at ACR Convergence 2025, Read More
Kyverna Therapeutics
1970-01-01
kyv-101
REGENXBIO Announces Completion of Enrollment in Pivotal Trials of Subretinal Surabgene Lomparvovec for Wet AMD
REGENXBIO
1970-01-01
pivotal
U.S. FDA Approves World’s First Cell Therapy (Lantidra) for Type 1 Diabetes
CellTrans Inc.
1970-01-01
lantidra
cell therapy
Janssen's bladder cancer drug hits snag in Korea's orphan drug review
Janssen Global Services
1970-01-01
orphan drug
bladder cancer
REGENXBIO Reports Third Quarter 2025 Financial Results and Operational Highlights
REGENXBIO
1970-01-01
bla
pivotal
financial results
duchenne muscular dystrophy
RGX-202 program for Duchenne muscular dystrophy advancing rapidly, topline results expected early Q2 2026 and BLA submission mid-2026 Pivotal trial enrollment completed in October; confirmatory trial open and enrolling New 12-month analysis shows all participants demonstrate improved functional