In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

Hemophilia usually is an inherited bleeding disorder in which the blood does not clot properly. Patients with severe hemophilia A may have to manage their condition with regular at-home injections or ...

Five-year clinical study results show this revolutionary one-time treatment allowed 94% of patients to stop routine factor IX prophylaxis DETROIT, March 23, 2026 /PRNewswire/ -- The Barbara Ann ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Biogen has discontinued all gene therapy programs using adeno-associated virus (AAV) capsids, instead shifting resources to modalities that have the highest likelihood of achieving better treatment ...

Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...

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 ...

The rare pediatric disease affects approximately one in one million children globally. Mutations in the ITGB2 gene disrupt ...

With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...

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