LYS-GM101

Lysogene is scheduled to start an adaptive Phase I/II trial for the long-term treatment of GM1 gangliosidosis, or Landing disease, with our gene therapy product candidate LYS-GM101.

LYS-GM101 is a gene therapy candidate designed to restore the ability of brain and spinal cord cells within the central nervous system (CNS), as well as the cells within the peripheral nervous system, to manufacture functional beta-galactosidase. In patients with GM1 gangliosidosis, initiating long-term expression of beta-galactosidase has the dual effect of removing accumulated GM1-ganglioside lipids and preventing further buildup.


LYS-GM101 uses an adeno-associated viral vector (AAVrh10) to deliver a functional copy of the GLB1 gene to the CNS. 

Treatment is administered via a one-time injection into a fluid-filled space in the back of the head called the cisterna magna. This ensures that the therapy reaches the brain and spinal cord. The injection itself takes 5 to 15 minutes and is delivered under general anesthesia. 

LYS-GM101 gene therapy is expected to reduce existing neuronal dysfunction and prevent further damage, potentially preventing or reversing cognitive and motor dysfunction and improving outcomes for patients.

In 2017, LYS-GM101 received Orphan Drug Designation for the treatment of GM1 gangliosidosis in the European Union and in the US, as well as Rare Pediatric Disease Designation in the US.

GM1 Gangliosidosis

GM1 gangliosidosis is a rare inherited neurodegenerative lysosomal storage disorder caused by mutations in the GLB1 gene—the gene that instructs cells to produce an enzyme called beta-galactosidase.

This enzyme is responsible for the metabolism of a substance called GM1-ganglioside in neurons. When the GLB1 gene is faulty and activity of beta-galactosidase is disrupted, GM1-ganglioside accumulates at toxic levels, causing neurodegeneration associated with severe neurological symptoms. This damage to the nervous system results in delayed development, behavioral disturbances, seizures and a range of other cognitive and physical symptoms. There is currently no approved treatment for GM1 gangliosidosis.

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