The FDA approved labeling changes for elivaldogene autotemcel (eli-cel; Skysona), a one-time gene therapy for patients with ...

Vinay Prasad is returning to his role at the U.S. Food and Drug Administration, a little more than a week after he had left ...

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, ...

US Food and Drug Administration Commissioner Marty Makary said he’s trying to persuade Vinay Prasad, the agency’s former head ...

U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a ...

Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development ...

Sarepta Therapeutics said on Monday it will resume shipping of its muscular disorder gene therapy to patients who can walk, ...

Leveraging Northway Biotech’s 20+ years of CDMO expertise, Diorasis Therapeutics is advancing AAV gene therapy for glaucoma ...

Gene therapy could treat a wide variety of medical problems. It's expensive, though, and not every use is approved. Here's what to know.

Choosing gene therapy Gray was in and out of hospitals seven times during each of the two years before her gene therapy. Then 32, she explained to her doctor that she was too tired to go on.

The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne muscular dystrophy. U.S. distribution has been paused amid safety concerns.

Gene therapy ‒ which has the potential to cure, not just treat disease ‒ has been approved so far only for very rare blood, neuromuscular and eye diseases, though more than 589 clinical trials ...