Sio Gene Therapies Inc. (NASDAQ: SIOX) stock took an aerial route in the premarket session as it rose 40.85% to $4.00 after it posted positive six-month results from a dose-escalation study of adeno-associated viral vector (AAV)9-based gene therapy candidate for GM1 gangliosidosis, from the low-dose cohort of its Phase III study. The initial results from the Phase 1/2 study of AXO-AAV-GM1 in five patients showed that the therapy was generally safe and well-tolerated, as well as an early indication for disease stabilization.
We are delighted to present promising results on the stability, tolerability, biomarker and partial efficacy of AXO-AAV-GM1, the first gene therapy tested in a GM1 gangliosidosis clinical trial, a life-limiting disorder triggered by GLB1 gene defects that inhibit the function of the beta-galactosidase enzyme. In this first-in-human trial, safety was the main measure, and we are delighted to see a favorable safety profile in the first five children treated with the low-dose,” said Gavin Corcoran, M.D., Sio Gene Therapies Chief R&D Officer.
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