16 February 2021 - FBX-101 is a first-in-human gene therapy utilising an adeno-associated virus to deliver a functioning copy of the GALC gene intraveniously to cells in the central nervous system and peripheral organs.
Forge Biologics today announced that the U.S. FDA has granted fast track, orphan drug, and rare paediatric disease designations to FBX-101 for the treatment of patients with Krabbe disease.