Sarepta Therapeutics will cut 500 jobs and add a serious warning on the label of its muscle-disorder gene therapy Elevidys, the drugmaker said on Wednesday, following the recent deaths of two patients ...

One 7-year-old girl who received a traditional cochlear implant in one ear and the gene therapy in another, recovered almost ...

Up to three in every 1,000 newborns is born with hearing loss in one or both ears. While cochlear implants have long been a ...

Ultragenyx's UX111 gene therapy faces FDA delay due to facility issues; analysts adjust price targets amid uncertain ...

A single shot of gene therapy restored hearing in people born deaf, with most showing improvement within weeks and some regaining full function.

It is trying to treat a devastating lifelong disease by fixing it at its source. We are able to fix the actual genetic ...

A novel gene therapy designed to target a form of inherited deafness restored hearing function in five children who were ...

The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...

The OUTREACH Study – Demonstrating that CAR-T therapies can be effectively administered in community cancer settings: In the ...

BETHESDA, Md. (July 16, 2025) – Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million ...

Indian American chemist receives prestigious CAREER award for research on synthetic DNA ‘programmable’ nanoparticles ...

Just days before South African scientists were set to launch clinical trials of a promising HIV vaccine, their progress was ...