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 ...

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 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment was released ...

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

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

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

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

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...

Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s ...

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

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