Regeneron Pharmaceuticals said on Monday its experimental gene therapy showed improved hearing in 10 of 11 children between ...

ElevateBio is seeking out partnerships for its lead gene therapy after the construct reduced the levels of a toxic protein in ...

As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...

UMass Chan researchers demonstrated the promise of a gene therapy to correct a gene mutation causing maple syrup urine disease.

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

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

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

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

A single injection of a healthy gene allowed blind children to recognize objects, shapes, and even read — offering hope for ...

Regeneron’s gene therapy, dubbed DB-OTO, is a cell-selective AAV gene therapy for children with hearing loss stemming from a ...

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