LYS-GM101 is a gene therapy candidate being developed to treat GM1 gangliosidosis, a fatal autosomal recessive disorder. Credit: PublicDomainPictures from Pixabay.

Lysogene has started dosing participants in the US with experimental gene therapy, LYS-GM101, to treat GM1 gangliosidosis in the international adaptative-design Phase I/II clinical trial.

GM1 gangliosidosis is an autosomal recessive disease that develops due to mutations in the β-galactosidase gene (GLB1) gene. It is characterised by the build-up of GM1 ganglioside in neurons, causing progressive neurodegeneration.

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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 (AAVrh.10) delivering the human β-galactosidase gene (GBL1) will be assessed.

The trial, which involves safety and confirmatory efficacy phases, will enrol 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.

Lysogene founder, chairman and CEO Karen Aiach said: “We are pleased with the rapid progress we are making with our teams in our effort to bring a new therapeutic solution to patients and families affected by GM1 gangliosidosis.

“By promptly opening a new site in the US and dosing the second patient we demonstrate our quality and timely execution despite a challenging and persistent Covid-19 environment.”

In 2016, LYS-GM101 secured the Rare Pediatric Disease designation in the US, followed by orphan drug designation in 2017 to treat GM1 gangliosidosis in the US and the European Union.

Currently, the company is also funding a GM1 gangliosidosis natural history study to obtain prospective and/or retrospective videos of children doing specific daily tasks and behaviours. The study is being performed by Casimir Trials.