In 2019, a non-randomized, phase 1/2 clinical trial started aiming at evaluating the safety and efficacy of a single dose AAV9-GLB1 (AXO-AAV-GM-1) after IV infusion to subjects, aged 2–12 years old, with Type II GM1 gangliosidosis (NCT03952637). The gene discussed is GLB1; the disease is GM1 gangliosidosis.