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 ...

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

Gene therapy has been headline news in recent years, in part due to the rapid development of biotechnology that enables doctors to administer such treatments. Broadly, gene therapies are ...

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

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

Since receiving gene therapy, Will Ungerer, shown in a recent picture, can do everyday things, including climb stairs easily and get dressed on his own. He’s also on a swim team. S. UNGERER ...

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

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

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 ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root ...