A pioneering genetic treatment is saving lives where chemotherapy failed, offering new hope for children and adults with ...

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

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

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, ...

Zevaskyn, the first gene therapy for recessive dystrophic epidermolysis bullosa, showed significant wound healing in phase 3 ...

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...

"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 ...

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 ...

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 ...

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 ...