While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...

The firm said the agency cited chemistry, manufacturing, and controls issues in its complete response letter, but had no concerns about product quality.

In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy ...

CRISPR Therapeutics is revolutionizing medicine with gene editing. Learn more about CRSP's breakthroughs, stock rally, and ...

Scientific organizations recently called for a 10-year ban on human germline editing. But will we ever be ready? And how would we even know?

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further innovations in safety, efficacy, and treatment strategies are needed.

Biotechnology New Gene Therapy Reverses Three Diseases With Shots to the Bloodstream The treatments rescued damaged blood and immune cells in newborn mice with just one shot.

After a patient died following a high dose of Neurogene’s Rett syndrome gene therapy candidate, the biotech is sharing details about the event and the new safety protocols being incorporated ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.