The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas. KJ Muldoon is walking and getting ready to celebrate ...

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male patients Ongoing work in Stargardt disease and cone-rod dystrophies, including ...

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 ...

Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...

Minaris will move its center for viral vector innovation to occupy space within CGT Catapult's collaborator laboratories in London and will work with CGT Catapult on the further development of ...

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

A small group of volunteers will receive multiple injections of the experimental treatments next month, says Unlimited Bio.

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College ...

Editorial: Cutting-edge therapies exist, but the market cannot deliver them cheaply. Britain must build NHS capacity so that cures become collective goods, not expensive products ...

In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a ...

Gene therapy is now being used for certain cancers and sickle cell anemia.A South Florida teen was among the first to get ...

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