Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase 1/2 Trial of AXO-AAV-GM1 for GM1 Gangliosidosis

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Dec. 15, 2020 (GLOBE NEWSWIRE) — Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically improve the lives of patients with neurodegenerative diseases, today reported positive six-month follow-up data from the low-dose cohort (1.5×1013 vg/kg) of the Company’s dose escalation study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of GM1 gangliosidosis. Initial data from the ongoing Phase 1/2 study in five patients in the low-dose cohort showed that AXO-AAV-GM1 was generally well tolerated with a favorable safety profile and provide early indications of clinical disease stability.

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