Sangamo Reports Presentation of New Data from Proprietary ZFP Therapeutic Programs for Lysosomal Storage Disorders at ASGCT

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Sangamo BioSciences, Inc.
SGMO
announced that new data from the company's proprietary ZFP Therapeutic® programs in disease models of lysosomal storage disorders (LSDs) were presented last week at the 18th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). "These new data significantly 'de-risk' Sangamo's approach for Hunter and Hurler syndromes and potentially many other monogenic diseases, such as the hemophilias, that are currently treated using lifelong enzyme replacement therapy," stated Edward Lanphier, Sangamo's president and chief executive officer. The data demonstrated durable expression of levels of functional enzyme sufficient to correct biomarkers of disease in mouse models of Hunter and Hurler syndromes, enabled by Sangamo's zinc finger nuclease (ZFN)-mediated genome editing technology. The LSD programs are part of Sangamo's In Vivo Protein Replacement Platform (IVPRP) portfolio of ZFP Therapeutics. The IVPRP uses ZFN-mediated genome editing to precisely insert disease-corrective genes into the albumin locus in the genome of liver cells. The correct version of the enzyme, which is defective in a genetic disease, is then made by the powerful machinery that normally drives albumin expression, leading to the production and secretion of the missing enzyme by the liver. A significant and sustained reduction in glycosaminoglycan (GAG) levels (a biomarker of lysosomal storage disorder progression) was observed in the urine and secondary tissue (liver, spleen, kidneys and lungs) in Hurler and Hunter mice 21 days post treatment with ZFNs. The accumulation of GAG oligosaccharides in the lysosomes of cells leads to dysfunction in several tissues throughout the body and is associated with symptoms of these LSDs. Production of the corrective enzyme was also shown to be durable through the twelve week study, consistent with earlier studies of the IVPRP for clotting factor, Factor IX, which demonstrated sustained production for over a year. The data also demonstrated that in models of Hurler and Hunter syndrome, high levels of human IDUA (hIDUA) and human IDS (hIDS) enzyme activity respectively, could be detected in the liver (site of expression) and plasma of treated animals. In addition, functional enzymes could be detected in secondary tissues such as the spleen, kidneys, and lungs, demonstrating that they had been taken up into these tissues from the circulation. "Our study demonstrates that the levels of enzyme present in various tissues were sufficient for the phenotypic correction of Hurler and Hunter syndromes mice, as measured by the reduction in GAG biomarkers, which suggests that the IVPRP can be successfully applied to these and potentially other LSDs," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "We are committed to advancing these programs through IND-enabling studies and into clinical trials as rapidly as possible to provide patients with potentially life-long protein replacement from a one-time therapy." Additionally, at the meeting's Presidential Symposium on Friday, May 15, 2015, in one of four studies selected from all abstracts submitted to the conference, data were presented that demonstrated the success of a proprietary method for precise and highly efficient targeted gene addition in CD34+ hematopoietic stem and progenitor cells (HSPCs). Finally, Dr. Philip Gregory, D.Phil., Sangamo's senior vice president of research and chief scientific officer, was one of three speakers invited to participate in the ASGCT Education Session, "Emerging Field Review: Gene Editing" on the final day of the meeting. His presentation covered recent technical developments made by Sangamo's scientists and collaborators in the field of genome editing using the research supporting Sangamo's HIV stem cell program and Sangamo and Shire's Huntington's disease program as examples.
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