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GlobeNewswire·

Sio Gene Therapies Announces Positive Interim Safety and Biomarker Data from Ongoing Phase 1/2 Clinical Study of AXO-AAV-GM1 Gene Therapy in GM1 Gangliosidosis

Consistent dose-dependent improvements across biomarker measures Normalization of serum beta-galactosidase activity and GM1 ganglioside in CSF in the high-dose cohort No overt disease progression in six out of seven patients treated across low- and h