STAT

In a first, a nonprofit wins FDA approval for rare disease gene therapy

The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...
Fierce Pharma

Italian nonprofit lands FDA approval for 1st gene therapy to treat rare genetic disease

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...
BioSpace

FDA Greenlights First Gene Therapy for Rare Pediatric Disease

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

Vertex's gene therapy shows promise in younger children with blood disorders

Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...