MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance. LYS-SAF302 is a gene therapy intended to deliver a functional copy of the SGSH gene to the brain. This is a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental state of MPS IIIA patients. The study is interventional, single arm and multicenter. Evolution under treatment will be compared to expected evolution based on natural history studies.

The study is being conducted in France, Germany, Netherlands, U.K. and U.S.A. Patients will be eligible to participate from a variety of additional geographical locations.

Parents interested in learning more about the trial, including eligibility requirements, can contact the site directly ( NCT03612869).


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Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system (CNS). The company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A pivotal clinical trial in MPS IIIA and a phase 1-2 clinical trial in GM1 Gangliosidosis are in preparation, while we are currently collaborating with a major partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism.