Posted by Michael Wonder on 23 Oct 2021
Sio Gene Therapies announces granting of FDA fast track designation for investigational AXO-AAV-GM1 (AAV9-GLB1) gene therapy in patients with GM1 gangliosidosis
21 October 2021 - Sio Gene Therapies today announced that the U.S. FDA has granted fast track designation to AXO-AAV-GM1, its adeno-associated viral vector 9-based gene therapy candidate for the treatment of Type I (early infantile-onset) and Type II (late infantile-onset and juvenile-onset) GM1 gangliosidosis.
AXO-AAV-GM1 delivers a functional copy of the GLB1 gene via an adeno-associated viral vector, with the goal of restoring β-galactosidase enzyme activity for the treatment of GM1 gangliosidosis.
