The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and ...

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

Researchers hoped they had finally found a way to get gene therapies past the blood-brain barrier. Then the first patient ...

An experimental gene therapy seems to slow the progression of Huntington’s disease by about 75 per cent, and researchers are ...

An Illinois family pushed for both boys to receive the potentially life changing medical treatment after one was approved and ...

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

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 ...

A baby born with a rare genetic disorder has taken his first steps after receiving a groundbreaking, personalized gene ...

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...

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

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read ...

Later-stage financing deals for cell and gene therapy companies reflects a broader derisking trend in the pharma industry.