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

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

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

The FDA's approval of the first CRISPR-Cas9–based gene therapy marked a major milestone in biomedicine, validating genome ...

Sarepta did not hold an investor call for its second-quarter earnings report or provide an updated full-year revenue outlook.

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, ...

Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...

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

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

Ocugen says it is on track to reshape the market for gene therapies against eye disorders over the next three years, by ...

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