If successful, the access model for sickle cell disease gene therapies could serve as a blueprint for other such treatments ...

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...

Leveraging Northway Biotech’s 20+ years of CDMO expertise, Diorasis Therapeutics is advancing AAV gene therapy for glaucoma ...

Ixo-vec reduced anti-VEGF injection frequency and was well tolerated in patients with wet AMD. A phase 3 pivotal trial ...

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...

Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development ...

Shares of beleaguered drugmaker Sarepta Therapeutics jumped in afterhours trading Monday after the company said it would ...

Sarepta Therapeutics said on Monday it will resume shipping of its gene therapy Elevidys to patients with a rare muscular ...

But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...

Sarepta Therapeutics shares snapped a three-day losing streak on Tuesday, as analysts said resumption of U.S. shipments for ...

A new study in Nature shows that delivering a single injection of gene therapy at birth may offer years-long protection ...