In one of the biggest advances against leukemia and other blood cancers in many years, doctors are reporting unprecedented success by using gene therapy to transform patients' blood cells into ...

An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.

Vertex Pharmaceuticals presents clinical data demonstrating benefits of Casgevy in SCD and TDT patients aged 5+ at ASH ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

Children with hereditary deafness regained their hearing thanks to a type of gene therapy, a new study published on Wednesday found. In a clinical trial, co-led by investigators from Mass Eye and Ear, ...

"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 ...

You’re reading an excerpt – sign up to get the whole newsletter in your inbox.Born in 1949, Chip Hailey is old enough to ...

The FDA has put a clinical hold on a Tenaya Therapeutics gene therapy study while the biotech works to standardize activities related to the immunosuppression regimen across trial sites. Tenaya said ...

People looking to lose weight and lower their blood sugar may someday be able to get a single injection that turns their cells into tiny factories churning out a protein that is essentially the active ...

ROME, Italy, Dec. 10, 2025 /PRNewswire/ -- Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex ...

Hemophilia gene therapies arrived a few years ago with blockbuster expectations. But now, one therapy has been pulled from ...

Miltenyi Biotec has been honoured with the Special Recognition Award for Pioneering Work in Cell & Gene Therapy at the ...