Three 2026 Breakthrough Prize winners reflect on developing Luxturna, a gene therapy that treats blindness caused by rare ...

Stuart Orkin and Swee Lay Thein shared a Breakthrough Prize in Life Sciences for their research on genetic causes of sickle ...

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 ...

The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time treatment that proved to be life-changing for a small number of children in a ...

In LAMA2-related muscular dystrophy, weakened extracellular matrix (yellow) leads to muscle fiber degeneration and the infiltration of immune cells (red) into the tissue. Researchers at the University ...

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 ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

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.

Cell and gene therapies are currently niche products, even within specialty pharmacy, drugs that, by most definitions, are ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...