Three 2026 Breakthrough Prize winners reflect on developing Luxturna, a gene therapy that treats blindness caused by rare ...

A breakthrough FDA-approved treatment is changing how genetic deafness is treated.

The FDA has granted accelerated approval to Regeneron Pharmaceuticals’ Otarmeni™ (lunsotogene parvec-cwha) as the first gene ...

Researchers at National Taiwan University have developed a liver-directed IL-10 gene therapy that strengthens cancer-fighting ...

Beacon is investigating the gene therapy in other pivotal trials, including the VISTA trial that is expected to readout in H2 ...

Two deaf siblings from Elbridge, NY have been able to hear for the first time after undergoing a groundbreaking gene therapy approved by the FDA.

Dr. Marty Makary noted it had "record speed approval," the fastest approval of any gene therapy device combination in U.S.

Regeneron’s drug restored hearing in 11 of 12 children in a rare, inherited condition that causes deafness.

The FDA has approved Otarmeni, the first-ever gene therapy for genetic hearing loss caused by the OTOF gene, for both ...

The therapy is part of a plan to develop gene therapies that can be efficiently delivered at lower doses, enabling them to be used against more than just “ultra-rare” conditions.

CRISPR gene therapy increases haemoglobin and fetal haemoglobin in sickle cell disease, reducing vaso occlusive events in ...

Even though Elevidys beat Wall Street expectations, analysts expect investor focus to shift toward Sarepta's early-stage RNA ...