RARE Daily

FDA Grants Lysogene Fast Track Designation for GM1 Gangliosidosis Gene Therapy

July 8, 2021

Rare Daily Staff

The U.S. Food and Drug Administration granted Lysogene Fast Track designation to its LYS-GM101 for the treatment of GM1 gangliosidosis, a rare and fatal, progressive neurodegenerative disease.

Photo: Karen Aiach, founder, chairman and CEO of Lysogene

GM1 gangliosidosis (GM1) is a lysosomal storage disorder caused by mutations in the GLB1 gene, which encodes lysosomal acid beta-galactosidase (β-gal). Reduced β-gal activity results in the accumulation of toxic levels of GM1 ganglioside in neurons throughout the brain, causing rapidly progressing neurodegeneration. GM1 manifests as a continuum of disease and is most severe in the infantile form, which is characterized by onset in the first six months of life by reduced muscle tone, progressive central nervous system dysfunction, and rapid developmental regression.

LYS-GM101 is designed to restore the production of a key enzyme that is missing in patients suffering from GM1 gangliosidosis. There are no approved treatments for the condition. The therapy has the potential to reduce or prevent neuronal damage and thereby improve quality of life and extend the lifespan of young patients with the condition.

Karen Aiach, founder, chairman, and CEO of Lysogene said the designation underscores LYS-GM101’s potential to improve neurocognitive deficits in children with GM1 gangliosidosis. “We have dosed the first patient last month and recruitment is on track, with completion of the first cohort expected by first quarter next year,” she said.

The company has initiated a global adaptative-design clinical trial for the treatment of GM1 gangliosidosis. The trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating an intracisternally delivered recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene. 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 United States and Europe.

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