Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts ...

The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...

Sarepta Therapeutics is refusing to pull its gene therapy Elevidys (delandistrogene moxeparvovec), despite a request from the ...

As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head, the biotech is standing firm ...

Ocugen has dosed the first subject in its Phase II/III GARDian3 trial of modifier gene therapy candidate, OCU410ST ...

Drugmaker Sarepta Therapeutics said late Friday it won't comply with a request from the Food and Drug Administration to halt ...

Amid a season of regulatory and scientific advances, experts reveal a culture of data hoarding among cell and gene therapy ...

Penn's Kiran Musunuru spoke with The Inquirer about the future of CRISPR technology.

The FDA has placed multiple investigational gene therapy clinical trials on hold, signaling broader platform concerns.

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a ...

IL-1 is considered one of the key mediators involved in the pathogenesis of OA, causing inflammation, joint pain, and ...