After a strong launch for its cancer gene therapy, Ferring Pharmaceuticals is setting the stage for long-term growth.

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 ...

A groundbreaking study titled "An Engineered Intravitreal Injection Retinal-Pigment-Epithelium-Tropic Adeno-Associated Virus ...

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

Net loss per share (GAAP) was $0.62, but remained negative as the company continues to invest heavily in research and development. The pivotal Phase 2 trial for Danon disease was placed on clinical ...

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

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

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 ...

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