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 ...

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 ...

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 ...

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

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

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, ...

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

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

"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 ...

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 ...