This is the first time such results have been achieved in both children and adult patients born with a specific type of ...

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 ...

Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...

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

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

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

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

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

RARE faces regulatory setback as FDA issues a CRL for UX111, delaying approval of the MPS IIIA gene therapy until 2026.

A new study shows that gene therapy can significantly improve hearing in both children and adults with congenital deafness ...

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