Subjects ” Neurodegenerative Diseases
The Food and Drug Administration (FDA) has granted LYS-GM101 fast-track status for the treatment of GM1 gangliosidosis.
GM1 gangliosidosis is a rare hereditary lysosomal storage disorder caused by deficient beta-galactosidase activity associated with a mutation in the GLB1 gene. This enzyme mutation leads to an accumulation of GM1 ganglioside, which leads to progressive neurodegeneration.
LYS-GM101 is a gene therapy designed to replace the gene mutation and restore the production of the beta-galactosidase enzyme. Investigative therapy uses an adeno-associated viral vector to deliver a functional copy of the GLB1 gene to the CNS.
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The company is currently investigating the efficacy and safety of LYS-GM101 in 16 patients with infantile GM1 gangliosidosis in a Phase 1/2 study (ClinicalTrials.gov Identifier: NCT04273269). Treatment is given as a single dose intracisternal injection under general anesthesia.
Karen Aiach, Founder, Chairman and CEO of Lysogene, commented, “We are pleased that the FDA has awarded LYS-GM101 Fast Track status, which underscores its potential to improve neurocognitive deficits in children with GM1 gangliosidosis, one deadly neurological disorder for which there is currently no treatment. We dosed the first patient last month and recruitment is on track, with the first cohort expected to complete in the first quarter of next year. ”
The FDA previously granted orphan drug status to LYS-GM101 for this indication. The FDA’s fast-track status enables accelerated review of therapies designed to treat serious or life-threatening conditions. In general, the designation is given to therapies that are expected to influence factors such as survival and daily functioning.
reference
Lysogene Announces FDA Fast Track Status for LYS-GM101 Gene Therapy for the Treatment of GM1 Gangliosidosis. [press release]. Paris, France: Lysogen; July 8, 2021.
This article originally appeared on MPR
Subjects:
Genetic Disorders Treatments for Neurodegenerative Diseases
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