The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying the agency was investigating the three deaths due to acute liver failure following treatment with Sarepta's AAVrh74 gene therapies.

BioMarin's Roctavian gene therapy shows long-term effectiveness in treating severe hemophilia A, reducing bleeding and improving quality of life for patients.

Using gene therapy to treat hereditary deafness is safe and effective in both children and adults, according to new findings from a multicenter clinical trial.

Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene therapy recipients showed stable hearing recovery and faster improvements in auditory and speech performance than cochlear implantation recipients.

RDH12, a gene therapy for RDH12-associated Leber congenital amaurosis (RDH12-LCA) RESEARCH TRIANGLE PARK, N.C., July 23, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders,

The UC Davis Department of Ophthalmology has received a five-year, $3.6 million grant from the National Eye Institute to explore a new way to treat vision loss using gene therapy.

The Vision Center at Children’s Hospital Los Angeles is developing a protocol for a phase 1 clinical trial for the first gene therapy for boys with blue cone monochromacy, which impairs color vision and severely affects visual function.

Sarepta Therapeutics said it will temporarily pause all shipments of delandistrogene moxeparvovec (Elevidys) for Duchenne muscular dystrophy in the U.S. The company previously refused the FDA's request to halt distribution to all Duchenne patients.