Lysogene announced dosing of the first patient in the United States with LYS-GM101 investigational gene therapy at CHOC Hospital (CHOC) in a global adaptative-design clinical trial (NCT04273269) for the treatment of GM1 gangliosidosis. This trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating the intracisternal delivery of a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human ß-galactosidase gene (GBL1). The clinical trial includes a safety phase and a confirmatory efficacy phase. The trial will enroll 16 patients with a diagnosis of early or late infantile GM1 gangliosidosis at sites in the US and Europe. More information can be found on www.clinicaltrials.gov/ct2/show/NCT04273269. GM1 gangliosidosis is a fatal autosomal recessive disease caused by mutations in the GLB1 gene leading to accumulation of GM1 ganglioside in neurons resulting in progressive neurodegeneration. No treatment has been approved so far for this disease. LYS-GM101 (‘adeno-associated viral vector serotype rh.10 expressing beta-galactosidase’) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as the Rare Pediatric Disease designation in the US in 2016. Lysogene is also funding a GM1 gangliosidosis natural history study being conducted by Casimir Trials to collect prospective and/or retrospective videos of children doing certain everyday tasks and behaviors (NCT04310163).