Sangamo BioSciences, Inc. announced the presentation of new preclinical data in disease models from its In Vivo Protein Replacement Platform™ (IVPRP) programs for MPS I (Hurler syndrome) and MPS II (Hunter syndrome). The data demonstrate that the Company's IVPRP approach enabled stable production of therapeutic levels of replacement enzyme from the liver into the circulation and secondary tissues, including the brain, resulting in significant reduction in biomarkers of the disease and, importantly, statistically significant improvements in cognitive function in treated animals.
The data were presented by Sangamo scientists and their collaborators at the University of Minnesota in two oral presentations at the 2016 Annual WORLDSymposium™ Meeting being held in San Diego from February 29 to March 4, 2016.
Data were presented from mouse models of MPS I and MPS II demonstrating significantly increased levels of human alpha-L-iduronidase (hIDUA) and iduronate 2-sulfatase (hIDS) enzymes, respectively, in the plasma and in secondary tissues, including spleen, lung, muscle and heart. This was achieved after a single intravenous administration of the ZFP Therapeutic®. In both models, the increase in enzyme activity resulted in the significant reduction of glycosaminoglycan (GAG) biomarker levels in all of these tissues, effectively correcting the disease phenotype, as the accumulation of GAGs is associated with MPS I and II disease characteristics. Furthermore, expression of enzymatically active hIDUA and hIDS at therapeutic levels and reduction of tissue GAG levels were stable throughout the four-month studies.
In February, Sangamo received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 1/2 clinical trial for MPS I.
