30 August 2021 - - The first patient has been dosed in the United States with LYS-GM101 investigational gene therapy at CHOC Hospital in a global adaptative-design clinical trial (NCT04273269) for the treatment of GM1 gangliosidosis, French phase 3 gene therapy platform company Lysogene (FR0013233475 LYS) (PAR: LYS) said.

This trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating the intracisternal delivery of a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene.

The clinical trial includes a safety phase and a confirmatory efficacy phase.

The trial will enroll 16 patients with a diagnosis of early or late infantile GM1 gangliosidosis at sites in the US and Europe.

LYS-GM101 ('adeno-associated viral vector serotype rh.10 expressing beta-galactosidase') received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as the Rare Pediatric Disease designation in the US in 2016.

Lysogene is also funding a GM1 gangliosidosis natural history study being conducted by Casimir Trials to collect prospective and/or retrospective videos of children doing certain everyday tasks and behaviors (NCT04310163).

Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system.

The company has built a unique capability to enable a delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS.

A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing.

An adaptive clinical trial in GM1 gangliosidosis is ongoing. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe.

Lysogene has also entered into an exclusive worldwide license agreement with SATT Conectus for a gene therapy candidate for the treatment of the Fragile X syndrome, a genetic disease related to autism.