ESGCT 2021 | LYS-GM101 AAV gene therapy for GM1 gangliosidosis

GM1 gangliosidosis is a rare autosomal lysosomal storage disorder caused by mutations in the GLB1 gene which encodes β-galactosidase (Beta-gal), resulting in toxic accumulation of GM1 ganglioside predominantly in the central nervous system (CNS). Michael Hocquemiller, PhD, Lysogene, Paris, France, describes ongoing research into LYS-GM101, an adeno-associated virus serotype rh.10 (AAVrh.10) vector carrying GLB1 cDNA, administered via a single injection into the cisterna magna. Following promising results obtained in pre-clinical studies in mouse, cat and non-human primate disease models, an open-label two-stage adaptive design study (NCT04273269) is currently underway which will evaluate the safety and efficacy of LYS-GM101 in patients with GM1 gangliosidosis. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.