GM1 gangliosidosis is an autosomal recessive disease that develops due to mutations in the β-galactosidase gene (GLB1) gene. It is characterized by the build-up of GM1 ganglioside in neurons, causing progressive neurodegeneration.
The fatal disease currently lacks approved treatments.
LYS-GM101 is a gene therapy candidate that can potentially re-establish the ability of the brain and spinal cord cells in the central nervous system and the cells in the peripheral nervous system to produce functional beta-galactosidase.
Starting long-term beta-galactosidase expression in GM1 gangliosidosis patients could eliminate gathered GM1-ganglioside lipids and prevent more accumulation.
The gene therapy leverages an adeno-associated viral vector (AAVrh10) to carry the GLB1 gene’s functional copy to the CNS.
During the interventional, multi-centre, single-arm, two-stage adaptive-design Phase I/II trial, the intracisternal transfer of a recombinant adeno-associated virus vector serotype rh.10 delivering the human β-galactosidase gene (GBL1) will be assessed.
The trial, which involves safety and confirmatory efficacy phases, will enroll 16 patients suffering from early or late infantile GM1 gangliosidosis at sites in the US and Europe.
Lysogene expects to complete the trial in 2025.