Sio Gene Therapies Announces First Patient Dosed in High-Dose Cohort of AXO-AAV-GM1 Clinical Trial in Patients with GM1 Gangliosidosis
- On-track to report topline results from low-dose cohort by end of 2020
“AXO-AAV-GM1 is the only gene therapy in the clinic targeting patients with Type I and Type II GM1 gangliosidosis, a devastating and fatal pediatric disease,” said
The Phase 1/2 study (NCT03952637) is designed to evaluate the safety, tolerability, and potential efficacy of AXO-AAV-GM1 delivered intravenously in patients with Type I and Type II GM1 gangliosidosis.
- The low-dose cohort evaluated 1.5x1013 vg/kg AXO-AAV-GM1 gene therapy in a total of five Type II (juvenile) patients. Six-month follow-up data from the low-dose cohort are expected by the end of 2020.
- The high-dose cohort is evaluating a dose of 4.5x1013 vg/kg AXO-AAV-GM1 gene therapy.
AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation and is the only gene therapy in clinical development for both Type I and Type II GM1 gangliosidosis.
GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene that cause impaired production of the β-galactosidase enzyme. Currently, there are no approved treatment options for GM1 gangliosidosis. In 2019, Sio reported clinically meaningful improvements from baseline to six-month follow-up for the first GM1 Type II child dosed with low-dose AXO-AAV-GM1 gene therapy under an expanded access protocol.
AXO-AAV-GM1 delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, with the goal of restoring β-galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1’s ability to improve β-galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.
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Source: Sio Gene Therapies