GM1 gangliosidosis is a fatal autosomal recessive disease caused by mutations in the GLB1 gene leading to accumulation of GM1 ganglioside in neurons resulting in progressive neurodegeneration.
No treatment has been approved so far for this disease.
The Fast Track program facilitates the development and accelerates the review of new drugs for serious conditions, which have the potential to address unmet medical needs.
The purpose is to expedite the availability of new treatment options for patients.
A product that receives Fast Track designation is eligible for more frequent interactions with FDA, potential eligibility for accelerated approval, priority review, and rolling Biologics License Application review.
The trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating an intracisternally delivered recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene (NCT04273269).
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.
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.
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