A rare disease that has cut off children from the outside world now potentially has a cure. A clinical trial to treat severe ...

Fred Hutch bioengineer Dr. Matthias Stephan is working to develop a special foam that could help make gene therapy for blood ...

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy ...

The FDA has added a boxed warning to Sarepta’s Duchenne gene therapy Elevidys after two teens died from liver failure and has ...

Krystal Biotech drives Vyjuvek growth, premium margins, global expansion, a $1B goal, and pipeline data ahead with a $219.50 ...

The U.S. Food and Drug Administration (FDA) is tightening restrictions on a gene therapy used to treat Duchenne muscular ...

Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed ...

Coave Therapeutics SA has nominated a lead gene therapy program, CoTx-101, for the treatment of retinal vascular diseases, ...

The updated label now includes a boxed warning and a new “limitation of use" in addition to the removal of the prior ...

Sarepta Therapeutics’ Elevidys will carry the FDA’s strongest warning label about the risks of liver damage and liver failure ...

Sharonville, Ohio, celebrates the opening of the new Cincinnati Children's Applied Gene and Cell Therapy Center.

Coave Therapeutics unveils lead gene therapy program for retinal vascular diseases, powered by first-in-class vector for ...