Currently, GenAns Biotech is conducting investigator-initiated trials for several gene therapies, including GA001 for ...

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

Sarepta Therapeutics (NASDAQ:SRPT) has hired a lobbying firm with close ties to the Trump administration as regulatory ...

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

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...

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

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

Shares of drugmaker Sarepta Therapeutics surged in afterhours trading Monday after the company said it would resume shipping ...

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

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

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.

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 ...