Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

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 ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...

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

Henry Ford Health has treated the first Michigan patient outside clinical trials with Roctavian, the newly approved gene therapy for severe hemophilia A.

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

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.

"This was the first proof that 'gene therapy in a box' could work." Gene therapies or cell therapies that involve genetically modified cells today are available at only a limited number of research ...

Professor Paul Gissen told delegates at ELRIG 2026 that gene therapy has begun to change the outlook for some childhood ...

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 ...