A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes ...

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 ...

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

The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended the approval of Vyjuvek for the ...

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

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

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

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

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

Japan's regenerative medicine cold chain logistics market thrives on aging demographics, stringent regulations, and advanced ...

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