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.

A small group of patients with an otherwise incurable form of T‑cell leukemia have seen their cancer driven into remission by ...

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 ...

For men with hemophilia B, receipt of gene therapy comprising an infusion of etranacogene dezaparvovec results in sustained endogenous factor IX expression and low annualized bleeding rates over five ...

Fondazione Telethon announced that FDA has approved its Biologics License Application (BLA) for Waskyra (etuvetidigene ...

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

The Food and Drug Administration this week granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...

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

Sarepta Therapeutics (NASDAQ:SRPT) leads in genetic therapies, contributing to ongoing discussions surrounding the nasdaq ...

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 ...