In a first for adults in Singapore, scientists are conducting gene editing trials on heart patients to correct defects at ...

The Boston biotech founded by Harvard professor David Sinclair is testing whether cellular reprogramming can turn back the ...

In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians ...

Oncologists' experience with CGT has increased, with more patients treated and earlier lines of therapy being utilized.

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 ...

Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...

A top US regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare ...

In honor of World Blindness Awareness Month, "GMA" is spotlighting a breakthrough gene therapy that's helping some blind ...

With Eli Lilly's recent acquisition of Verve Therapeutics and its PCSK9 candidate, delve into what the target represents.

The trial was small — just 12 children were treated in one or both ears — but it offers important lessons for researchers ...

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.

BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending ...