This is the first time such results have been achieved in both children and adult patients born with a specific type of ...

Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy.

Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and ...

A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated ...

“We now have emerging data to at least evaluate the efficacy of gene therapy in patients with a risk of or history of stroke,” Sharma said. “Until now, we only had one option that had a long-term ...

Last month, a baby got the world’s first personalized gene-editing treatment. What will this mean for millions of others with ...

The reported dismissal of high-ranking CBER officials Nicole Verdun and Rachael Anatol resurfaced lingering concerns about ...

The biotech has received written agreement from the agency through the START pilot program on key aspects of its trial design for evaluating NGN-401.

Researchers have developed a gene therapy that significantly slowed motor function loss in preclinical models of amyotrophic ...

The Alliance for Regenerative Medicine has voiced deep disappointment about reports that the FDA has put the director of its ...

Three-year-old Gunreet Kaur has a rare inherited condition that affects children’s physical, mental and behavioural ...