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.
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