gene therapy
The latest news about gene therapy: headlines, AI summaries and the full story from every source.
- Ocugen Outlicenses Gene Therapy Candidate OCU400 to Celairis Therapeutics for Asia-Pacific Region
- Novartis Shares Decline Following Safety Concerns Regarding Zolgensma Gene Therapy
- Ocugen Outlines Clinical Milestones for OCU400 and Targets Three BLA Submissions by 2028
- uniQure Reports Q3 Results with EPS Beat and Revenue Growth Amid Cash Runway Extension
- FDA Grants Priority Review for uniQure's Huntington's Disease Gene Therapy
- FDA Issues Draft Guidance to Accelerate Personalized Gene Therapy Development
- Troy Short Joins Nanoscope Therapeutics as Senior Vice President of Manufacturing and Supply Chain
- Cystic Fibrosis: The Remaining 10% Still Lack Effective Treatments
- J.P. Morgan Initiates Overweight Coverage on Neurogene
- Rocket Pharmaceuticals Advances Phase 2 Trial for RP-A501 in Danon Disease
- Ultragenyx to Sell FDA Priority Review Voucher for $210 Million
- Nanoscope Therapeutics Announces Participation in Upcoming Ophthalmology Conferences
- Abeona Therapeutics Reports Progress on ZEVASKYN Launch, Revenue Growth
- BofA Upgrades Candel Therapeutics Following Positive Outlook for Prostate Cancer Gene Therapy
- Intellia Prepares for Potential Launch of Lonvo-z as FDA Decision Nears
- A Gene Therapy Could Help a Child with Batten Disease — But Access Remains Elusive
- Opus Genetics Shares Rise Ahead of Upcoming Trial Data Release
- Bayer's AskBio Completes Enrollment in Phase 2 Heart Failure Gene Therapy Trial
- Eli Lilly and Sanofi Backed Scribe Therapeutics Surges Nearly 50% in Upsized IPO
- Ocugen Signs Licensing Term Sheet for OCU400 with Roots Pharmaceutical
- Taysha's R&D Chief Sells $1.2 Million in Stock Amid Strong Share Performance
- Vertex Pharmaceuticals Hits 52-Week High Amid Regulatory and Developmental Catalysts
- MeiraGTx Secures $400M Investment from Oberland Capital
- Barclays Upgrades UniQure N.V. (QURE) to Overweight Amid Advancements in Huntington's Disease Program
- Remix Therapeutics to Go Public via Reverse Merger with Passage Bio
- Precigen Secures FDA Orphan Drug Exclusivity for PAPZIMEOS
- FDA Issues Draft Guidance to Accelerate Gene Therapy Development
- AvenCell Therapeutics Announces Phase 1a Results of CD123 CAR-T Therapy for AML at EHA 2026
- Regeneron Announces EMA Accepted Review for Otarmeni Gene Therapy Application
- 4D Molecular Therapeutics Reports Q1 Results, Advances Clinical Programs
- Uniqure NV (QURE) Announces Plans to Seek UK Approval for AMT-130 Gene Therapy
- Sarepta Therapeutics' Q1 Performance Surpasses Expectations Amid Elevidys Challenges
- Regeneron Receives FDA Approval for Gene Therapy Treating Genetic Hearing Loss
- Vertex Pharmaceuticals Exceeds Profit Estimates as Sales of New Cystic Fibrosis Drug Surge
- Opus Genetics Secures $155 Million Financing Agreement to Support Clinical Development
- Citizens Initiates Coverage on Opus Genetics with Outperform Rating
- TD Cowen Increases Price Target for Regeneron Pharmaceuticals Ahead of Fiscal Q1 Results
- Regeneron Offers Free Gene Therapy for Rare Hearing Loss in the U.S., Undecided on Overseas Pricing
- Regeneron Agrees to Lower Drug Prices and Offer Free Hearing-Loss Therapy Under Deal with Trump
- FDA Accepts BLA for Ultragenyx's Gene Therapy for Sanfilippo Syndrome
- Replimune Announces Staff Cuts, MeiraGTx Reacquires Eye Gene Therapy