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

From rare diseases to chronic conditions, epigenetic editing unlocks new ways to control gene activity without altering the genetic code.

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 ...

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

A breakthrough therapy is driving aggressive leukaemia into remission. Here’s what the headlines don’t tell you about life ...

A pioneering genetic treatment is saving lives where chemotherapy failed, offering new hope for children and adults with aggressive leukemia. This world-first therapy has kept patients disease-free ...

A Philadelphia-area infant named Baby KJ made international headlines after doctors at Children’s Hospital of Philadelphia and Penn Medicine successfully treated his rare, life-threatening liver ...

Scientists at UCL and GOSH have used groundbreaking base-edited CAR‑T cell therapy — BE‑CAR7 — to treat aggressive T‑cell ...

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, ...

Because of NextCell’s algorithm that allows optimized donor choice for MSC production, its therapy could be completely ...

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

Casgevy safely and effectively prevents vaso-occlusive crises in children ages 5 to 11 with severe sickle cell disease, trial ...