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 ...

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

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

A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...

For nearly three decades, Mayo Clinic researcher Christopher Evans, Ph.D., has pushed to expand gene therapy beyond its ...

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

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

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.

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 ...

The first gene therapy product approved by the Food and Drug Administration (a treatment for a form of leukemia, approved last summer) costs hundreds of thousands of dollars for each infusion.