A genetic variant carried by at least one-fifth of the population may do far more than raise the odds of developing Alzheimer ...

With an end-of-week green light from the FDA, Rocket Pharmaceuticals has officially broken into commercial orbit.  | Rocket ...

Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy treatment appeared at risk. The Food and Drug Administration will request that the company ...

Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role ...

Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, "AAV ...

The year 2023 marked a significant milestone for the treatment of sickle cell disease (SCD), with the approval of two gene therapies shown to help reduce the pain experienced by people with this ...

Vinay Prasad, M.D., is out again as the chief medical and scientific officer for the U.S. Food and Drug Administration (FDA).

Scientists have developed a new gene therapy that quiets pain at its source in the brain—without the addictive risks of opioids. Using AI to map how pain is processed, they created a targeted “off ...

When Harith Rajagopalan considers the millions of patients who have taken a GLP-1 drug to treat diabetes or obesity, he sees a revolution that is failing to realize its promise. “We are literally ...

The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring.  | The company is working ...

Rocket Pharmaceuticals received FDA accelerated approval for Kresladi, validating its gene therapy platform. Find out why RCKT stock is a hold.