LYS-GM101 builds on Lysogene's extensive experience in direct to CNS adeno-associated viral vector -based gene therapy clinical development.
The IND clearance follows the recent clinical trial authorization granted by the MHRA in the United Kingdom. Lysogene intends to initiate its global, multi-center, single-arm, two-stage, adaptive-design clinical trial of LYS-GM101 in patients with a diagnosis of early or late infantile GM1 gangliosidosis.
The clinical trial will include a safety phase and a confirmatory efficacy phase.
The company intends to dose a total of 16 patients, with dosage of the first patient expected in the first half of 2021.
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 Rare Pediatric Disease designation in the US in 2016.
Leading international gene therapy and Lysosomal Storage Disease centers plan to participate in the clinical trial (NCT04273269).
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 in infantile and juvenile GM1 gangliosidosis (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 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 adaptative clinical trial in GM1 gangliosidosis is in preparation. 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 is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism.
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