Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

In a groundbreaking medical first, doctors have restored vision in children who were born almost completely blind. Four young ...

Solid Biosciences reported recent positive data in developing their gene therapy candidate SGT-003. The phase 1/2 INSPIRE ...

Scientists compared five families and multiple generations to find a gene variant that prevents COPA Syndrome and opens the door to a new gene therapy for the condition.

Pfizer said on Thursday it will stop global development and commercialization of its hemophilia gene therapy, Beqvez, citing ...

Fuse Vectors has raised $5.2 million of funding as it moves closer to commercialising its innovative new approach to ...

Revolutionary gene therapy approaches show promise in treating various retinal conditions, potentially transforming treatment ...

In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants ...

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...

A gene therapy from Regeneron Pharmaceuticals Inc. improved hearing in nearly all deaf children in a clinical trial, results ...

Ten of 11 children born with a rare form of congenital hearing loss experienced improvements after receiving the company’s ...

Ten children who received DB-OTO for profound genetic hearing loss due to variants of the otoferlin gene showed notable ...