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

CGT Global, a leading biotechnology company driving the development and delivery of innovative cell and gene therapies, today ...

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

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

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

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

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

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

For nearly three decades, Mayo Clinic researcher Christopher Evans, Ph.D., has pushed to expand gene therapy beyond its ...

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