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

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

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 ...

D Molecular Therapeutics has posted 60-week data on a gene therapy, providing evidence of durability in diabetic macular edema and sending its stock up more than 40% in premarket trading.

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

For nearly three decades, Mayo Clinic researcher Christopher Evans, Ph.D., has pushed to expand gene therapy beyond its ...

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

In a major win for Sarepta Therapeutics Inc., US regulators are recommending that patients who can walk be allowed to take ...

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

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

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

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 ...