Sio Gene's AXO-AAV-GM1 slows disease progression in GM1 gangliosidosis

US clinical-stage drug developer Sio Gene Therapies’ (Nasdaq: SIOX) stock was up as much as 20% and closed up 10% at $2.36 yesterday, after the company presented positive interim data from its ongoing Phase I/II study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of GM1 gangliosidosis, in an oral presentation at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021, held from October 19-22, 2021.

The company also said that the US Food and Drug Administration has granted Fast Track designation to AXO-AAV-GM1.

These follow-up data from five Type II (late-infantile to juvenile) patients in the low-dose cohort and the initial two Type II patients in the high-dose cohort demonstrate an encouraging safety profile and a consistent dose-response in disease biomarkers across the evaluation period.