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

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

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

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 ...

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

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 ...

Treating our first patient is a proud moment for Abeona and a testament to the tireless resolve of our team,” said Vish ...

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 ...

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 ...

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

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