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

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

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 ...

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

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 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 ...

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 ...

Just getting selected to participate in the Bespoke Gene Therapy Consortium was a major hurdle. The eight diseases, including Talia's Charcot-Marie-Tooth disease type 4J or CMT4J, which causes ...