WASHINGTON (AP) — Drugmaker Sarepta Therapeutics said late Friday it won't comply with a request from the Food and Drug Administration to halt all shipments of its gene therapy following ...

A single jab of a breakthrough gene therapy could reverse hearing loss in people within weeks, according to new research.

Sarepta Therapeutics said on Monday it will pause all shipments of Elevidys in the United States after two teenage boys with a rare condition called Duchenne muscular dystrophy, who had received ...

Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene therapy.

Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy appeared at risk. Sarepta has reported three patient deaths related to its gene therapies.

The new gene therapy consists of removing the Hepatitis B infected gene along the patient’s genetic code to reduce the surface antigens of the virus and halt the integration of the virus with ...

Rocket Pharmaceuticals, Inc. a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that ...

Shares of drugmaker Sarepta Therapeutics continued to sink Tuesday after the company said it would comply with a Food and Drug Administration request to pause shipments of its gene therapy ...

Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are resolvable FDA clinical reviews acknowledged that the clinical data are ...

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that have attracted attention from regulators ...

Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles.