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

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

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 ...

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

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

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

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

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 ...

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

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

The CMS said on Wednesday that 33 states, plus the District of Columbia and Puerto Rico, will participate in the “Cell and Gene Therapy Access Model” to centrally coordinate insurance coverage ...