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

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 ...

Connecticut is now participating in a new federal program aimed at expanding treatment access for patients with sickle cell ...

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.

Uppsala University Hospital-led investigators report that gene-edited donor islet cells survived 12 weeks inside a man with ...

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

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

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

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 ...

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 ...