AXGT - Sio Gene advances early-stage study of nerve cell disorder gene therapy candidate

Sio Gene Therapies (SIOX) has dosed the first patient in the high-dose cohort of Phase 1/2 (Stage 1) study evaluating AXO-AAV-GM1 gene therapy for Type I (infantile) and Type II (late infantile and juvenile onset) GM1 gangliosidosis.High-dose cohort is evaluating a dose of 4.5x1013 vg/kg.Low dose cohort is evaluating 1.5x1013 vg/kg gene therapy in five Type II (juvenile) patients. 6-month follow-up data from the low-dose cohort is expected by year end.AXO-AAV-GM1 has received both Orphan Drug and Rare Pediatric Disease Designations by the FDA for the same indication.GM1 gangliosidosis a rare inherited disorder characterized by the progressive destruction of nerve cells (neurons) in the brain and spinal cord. It is caused by mutations in the GLB1gene leading to impaired production of the beta-galactosidase enzyme.AXO-AAV-GM1 delivers a functional copy of the GLB1 gene, with the goal of restoring ?-galactosidase enzyme activity for the treatment of GM1 gangliosidosis.Recently, the company renamed itself from Axovant Gene Therapies to Sio Gene Therapies.Shares are

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Sio Gene advances early-stage study of nerve cell disorder gene therapy candidate