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

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 ...

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

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

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

JABSOM Cell and Molecular Biology researcher Dr. Jesse Owens has spent the better part of two decades chasing a vision that ...

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 ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

Aradigm Health, a new benefits platform aimed at easing the financial sting of coverage for cell and gene therapies, has ...

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

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

Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, ...