Vinay Prasad is returning to his role at the U.S. Food and Drug Administration to resume overseeing vaccine, gene therapy and ...

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

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona ...

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 ...

The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

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

The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...

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

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

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.