The Food and Drug Administration is adding a new warning to a gene therapy linked to two patient deaths earlier this year.

EU regulator says treatment could fill major gap for patients who lack a suitable stem cell donor and leads to sharp drops in ...

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

MeiraGTx Holdings has signed a deal with Eli Lilly , potentially worth over $475 million, granting the pharma giant rights to ...

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...

The FDA added a boxed warning to delandistrogene moxeparvovec (Elevidys) gene therapy for Duchenne muscular dystrophy and ...

The U.S. Food and Drug Administration said on Friday it approved new labeling for Sarepta Therapeutics' gene therapy Elevidys ...

Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, ...

The alliance extends a deal streak in genetic medicine for Lilly and hands the company rights to an experimental treatment ...

Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis.

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn ...

MeiraGTx is cashing in on a gene therapy that restored vision to 11 children who were born legally blind, licensing the ...