A UC San Diego–led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse model can dramatically improve heart function and extend survival in several inherited forms ...

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

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

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

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.

The FDA has approved Otarmeni, the first-ever gene therapy for genetic hearing loss caused by the OTOF gene, for both pediatric and adult patients after positive results.

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

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

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

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.

Kelly Anne is a deputy editor at Forbes Advisor overseeing the development of various initiatives, including newsletters, ...