There's a fresh push to edit the genes of human embryos to prevent diseases and enhance characteristics that parents value.

Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as the company faces intense regulatory scrutiny after two recent patient ...

A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment ...

Over 5 years of treatment in hemophilia B patients, gene therapy research shows sustained factor IX activity and safety.

The CHMP opinion is based on data from the largest and broadest gene therapy clinical programme in DMD to date, including results from the pivotal Phase III EMBARK study that showed treatment with ...

Discover how CRISPR gene editing is creating personalized cancer therapies, enhancing immunotherapy, and offering new hope for hard-to-treat cancers.

FDA weighs new study requirements for Sarepta to confirm safety of gene therapy Elevidys: report By Fraiser Kansteiner Jul 24, 2025 3:12pm Sarepta Sarepta Therapeutics Elevidys U.S. FDA ...

Researchers reduce cardiac arrhythmia after heart attacks using gene therapy by University Hospital of Bonn edited by Lisa Lock, reviewed by Andrew Zinin Editors' notes ...

Rocket Pharmaceuticals will cut 80 jobs and streamline operations, focusing on gene therapies to drive long-term growth.

Roche is following its partner Sarepta and halting shipments of the Duchenne gene therapy Elevidys in some countries, amid safety concerns.

Roche has halted some international shipments of the gene therapy Elevidys, used to treat Duchenne muscular dystrophy, Bloomberg reported July 22. The decision comes in the wake of a similar move ...

Sarepta will halt all shipments of Elevidys, its drug for Duchenne muscular dystrophy patients, by Tuesday evening.