AAVB-039 advances gene therapy for Stargardt disease, aiming for innovative treatments for inherited retinal disorders.

Sarepta Therapeutics appears to have right-sized itself after laying off over a third of its staff, announcing a significant ...

Want a bespoke baby? Check the beauty, brains, or brawn boxes on the embryo order form. Genetic tinkering is no longer science fiction—it’s a market without legal guidelines or societal buy-in. But ...

Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) surged 15% after receiving a key regulatory boost from the U.S. Food and Drug ...

Sarepta stock surged after the pharma company said it would lay off a third of its workforce and add a black-box warning to ...

Controlling the activity of specific genes in the laboratory through the editing of their epigenetic marks is now a reality ...

The US Food and Drug Administration (FDA) recently granted fast track designation to Sanofi’s SAR446597 for the treatment of geographic atrophy (GA). 1 ...

Eight children in the UK have been spared from devastating genetic diseases thanks to the new three-person in vitro fertilization technique.

Discover the current pipeline candidates advancing through the clinic for the treatment of sickle cell disease.

An expert discusses how recent updates to AUA, EAU and NCCN guidelines have cautiously shifted toward more bladder-sparing approaches for BCG-unresponsive patients, incorporating new FDA-approved ...

The favorable market reaction indicates investor relief at management’s swift measures to rectify the company's worsening ...

It is trying to treat a devastating lifelong disease by fixing it at its source. We are able to fix the actual genetic ...