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MPS IIIA

Lysogene’s most advanced product is rAAV vector serotype rh.10 carrying the human N-sulfoglucosamine sulfohydrolase (hSGSH) for the treatment of the mucopolysaccharidosis type IIIA (MPS IIIA) or Sanfilippo syndrome type A. Lysogene’s gene therapy is delivered directly to the CNS in one neurosurgical procedure. It is hoped that the delivery of the missing SGSH gene provides a permanent source of functional enzyme in the brain that reverses phenotypic abnormalities of CNS neural cells.

The recently completed Phase I/II study in four MPS IIIA children (three patients around 6 years of age and one patient nearly 3 at the time of treatment) demonstrated that the gene therapy and neurosurgical procedure is safe and well tolerated, and exploratory efficacy profiles are encouraging (Tardieu et al. 2014).

Lysogene is currently preparing to initiate a Phase II/III pivotal clinical trial with its next generation gene therapy formulation.

 

GM1 gangliosidosis

Lysogene’s GM1 program includes the development of an AAV based treatment for GM1 gangliosidosis.

In February 2015, Lysogene entered into a strategic collaboration with University of Massachusetts Medical School (UMMS) and Auburn University (AU). Through the collaboration, Lysogene, UMMS and AU will develop IND-supporting preclinical studies in GM1-gangliosidosis using AAV gene therapy technology. The collaboration will combine Lysogene’s outstanding translational and clinical expertise in gene therapy for CNS disorders with the unique preclinical expertise and infrastructure of UMMS and AU to design and test innovative AAV-based gene therapy approaches to treat GM1-gangliosidosis.

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