Posted by Michael Wonder on 15 Mar 2022
Myrtelle receives FDA fast track, rare paediatric disease, and orphan drug designations for its proprietary gene therapy for the treatment of Canavan disease
15 March 2022 - Myrtelle today announced that the U.S. FDA has granted fast track, rare paediatric disease and orphan drug designations for its lead clinical-stage gene therapy, rAAV-Olig001-ASPA, for the treatment of patients with Canavan disease.
Myrtelle’s ongoing Phase 1/2 first in human clinical trial utilises a novel proprietary recombinant adeno-associated virus vector, AAV-Olig, that for the first time directly targets oligodendrocytes in the brain.
