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.

Ultragenyx Pharmaceuticals RARE announced that the FDA has issued a Complete Response Letter (CRL) for its biologics license ...

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

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

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

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

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

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 ...

AstraZeneca’s rare disease unit Alexion has penned a $825 million deal for adeno-associated virus (AAV) capsids from existing ...

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