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Gene Therapy

Huntington's Disease Duchenne Muscular Dystrophy Clinical Trials Hearing Loss Hemophilia A CAR-T Therapy Sickle Cell Disease Patient Safety Mucopolysaccharidosis II OTOF-related Hearing Loss Sarepta Therapeutics Rett Syndrome Cardiovascular Disease Adeno-Associated Virus (AAV) Genetic Agency Otoferlin Deafness Regulatory Affairs Safety Concerns Hematopoietic Stem Cell Therapy Cholesterol Management Hypertrophic Cardiomyopathy siRNA Therapy RNA Interference Dystrophic Epidermolysis Bullosa FDA Elevidys Chronic Diseases Market Trends Ophthalmology Hearing Loss Treatments Bluebird Bio Kriya Therapeutics CRISPR Technology AAV-SLB101 Cancer Treatment NGLY1 Deficiency

FDA Agrees Three‑Year Phase I/II Data Can Support uniQure’s AMT‑130 Accelerated BLA

It opens a route for accelerated approval with negotiations underway over the design of a required post‑approval confirmatory trial.

FDA Approves First Gene Therapy for Inherited Hearing Loss

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

Sarepta Reports Early siRNA Data Showing Muscle Delivery as Shares Surge

Plaintiff Firms Intensify Biotech Securities Suits as Key Deadlines Near for Nektar, uniQure and REGENXBIO

Taysha Advances Rett Gene Therapy Program With Pivotal Dosing, FDA Alignment and Q2 Data Timeline

uniQure Investors Face April 13 Deadline in Securities Class Action Over FDA Setback

uniQure Soars 36% After FDA Biologics Chief Vinay Prasad Announces April Exit

FDA Rejects Regenxbio’s Hunter Syndrome Gene Therapy Application

Lilly Strikes Up to $1.12 Billion Pact With Seamless Therapeutics for Hearing‑Loss Gene Editing

Lilly Licenses MeiraGTx Eye Gene Therapy in Deal Worth Up to $475 Million

Sarepta Shares Plunge After ESSENCE Phase 3 Miss in Duchenne Trial

FDA Setback Puts uniQure’s Huntington’s Gene Therapy Filing in Doubt

Kriya Nets $320 Million Series D as Odyssey Raises $213 Million and Amgen Retools Research Leadership

Regulators Halt Elevidys Gene Therapy Worldwide After Safety Failures

Sarepta Defies FDA, Keeps Duchenne Gene Therapy on the Market

Sarepta Shares Plummet on FDA Review of Elevidys Following Third Patient Death

Eli Lilly to Acquire Verve Therapeutics as Gene-Editing Trial Begins

Sarepta Halts Elevidys Shipments for Non-Ambulatory Patients After Second Fatal Liver Failure