Pfizer announced in February 2025 that it was taking Beqvez (fidanacogene elaparvovec), its gene therapy for hemophilia B, ...

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 ...

Up to three in every 1,000 newborns is born with hearing loss in one or both ears. While cochlear implants have long been a life-changing option, they involve surgery and can't fully replicate the ...

The idea behind intra-articular gene therapy for treating osteoarthritis (OA) is to deliver the gene-altering vector or cells directly to the precise site of the disease with a single injection — so ...

Gene therapies for rare diseases are frequently developed then discarded by drug companies because they can’t afford to produce the treatment for more patients. Gene therapies for rare diseases are ...

UF scientist develops safer gene therapy restoring vision in children with rare inherited eye diseases; pivotal trials begin ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

Sarepta Therapeutics is still assessing how best to prevent liver injury from its commercialized Duchenne muscular dystrophy gene therapy. But Sarepta partner Hansa Biopharma has encouraging ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

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 ...