December 2, 2020 -- Sio Gene Therapies has dosed the first patient in the high dose group of its phase I/II study of its gene therapy, AXO-AAV-GM1, for type 1 and type 2 GM1 gangliosidosis. The high dose study is being conducted at the National Institutes of Health's Clinical Center.
The gene therapy delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, with the goal of restoring β-galactosidase enzyme activity for the treatment of GM1 gangliosidosis. It is delivered intravenously.
AXO-AAV-GM1 has received both orphan drug and rare pediatric drug designations and is the only gene therapy in clinical development for both types of GM1 gangliosidosis, a progressive, fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene.
The company reported clinically meaningful improvements from the baseline with a low dose of the AXO-AAV-GM1 gene therapy for type II gangliosidosis in five juvenile patients. Topline data from the low dose cohort is expected by the end of the year.
Copyright © 2020 scienceboard.net
Member Rewards
Earn points for contributing to market research. Redeem your points for merchandise, travel, or even to help your favorite charity.
Research Topics
Interact with an engaged, global community of your peers who come together to discuss their work and opportunities.
Conferences
Connect
