The Food and Drug Administration (FDA) has granted Fast Track designation to LYS-GM101 for the treatment of GM1 gangliosidosis.

GM1 gangliosidosis is a rare inherited lysosomal storage disorder caused by deficient beta-galactosidase activity related to a mutation in the GLB1 gene. This enzyme mutation results in 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. The investigational 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 trial ( Identifier: NCT04273269). The treatment is administered as a single dose intracisternal injection under general anesthesia.

Karen Aiach, Founder, Chairman and CEO of Lysogene commented: “We are pleased that FDA has granted Fast Track designation to LYS-GM101, as it underscores its potential to improve neurocognitive deficits in children with GM1 gangliosidosis, a lethal neurological disease for which there is currently no treatment. 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.”

The FDA previously granted Orphan Drug designation to LYS-GM101 for this indication. The FDA’s Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to therapies that are expected to have an impact on factors such as survival and daily functioning.


Lysogene announces FDA Fast Track designation for LYS-GM101 gene therapy for the treatment of GM1 gangliosidosis. [press release]. Paris, France: Lysogene; July 8, 2021.

This article originally appeared on MPR