The first-of-its kind gene therapy uses a patient’s skin cells to create sheets to treat the open wounds caused by the rare, ...

ORLANDO -- A gene therapy that could transform CAR T-cell treatment achieved rapid and potentially durable measurable ...

On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.

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

Miltenyi Biotec Receives ‘Special Recognition Award’ for Pioneering Work in Cell & Gene Therapy at BioSpectrum India ...

A new wave of gene therapy treatments offer a new lease on life for people with inherited diseases such as haemophilia. But ...

Scientists discovered that certain gene changes allow cells to function even when frataxin, the protein lost in Friedreich’s ...

ROME, Italy, Dec. 10, 2025 /PRNewswire/ -- Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex ...

Treating our first patient is a proud moment for Abeona and a testament to the tireless resolve of our team,” said Vish ...

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