SIOX - Sio Gene Therapies' AAV9 gene therapy shows beneficial effect in GM1 gangliosidosis in children

Sio Gene Therapies (SIOX) will present new biomarker data from the study of AXO-AAV-GM1, its adeno-associated viral vector ((AAV))9-based gene therapy candidate for the treatment of GM1 gangliosidosis in children.GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene that cause impaired production of the ?-galactosidase enzyme. Key findings from the new biomarker analysis:18-49% reductions from baseline in accumulated substrate, GM1 ganglioside, were observed in cerebrospinal fluid ((CSF)) of 4 out of 5 children in the low-dose cohort at 6 months.3 of 5 children demonstrated CSF GM1 ganglioside levels less than 100 ng/mL at the 6-month follow-up visit.Literature reported mean levels of normal GM1 ganglioside in CSF range from 29.7- 52.3 ng/ml in healthy children 1 month to 18 years of age (Izumi 1993, Kaye 1992, Ginns 1980)These data represent the first direct evidence in humans that intravenously administered AXO-AAV-GM1 gene therapy exerts a measurable

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Sio Gene Therapies' AAV9 gene therapy shows beneficial effect in GM1 gangliosidosis in children