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Richmond, California May 31 , 2019
Sangamo Therapeutics has announced that the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has issued a positive opinion on the application for orphan medicinal product designation (OMPD) for SB-318 and SB-913, Sangamo's genome editing product candidates for the treatment of rare lysosomal storage disorders Mucopolysaccharidosis Type I (MPS I) and MPS II, respectively.

The EMA's OMPD is granted to medicines intended for the treatment, prevention or diagnosis of life-threatening or chronically debilitating conditions that are rare and affect less than five in 10,000 persons in the European Union (EU). The designation provides incentives to advance the development and commercialization of orphan medicines, which include access to the EU centralized authorization procedure and potential for market exclusivity for a period of up to ten years.

MPS I and MPS II are caused by mutations in the genes encoding alpha-L-iduronidase (IDUA) and iduronate 2-sulfatase (IDS) enzymes, respectively. Using Sangamo's zinc finger nuclease (ZFN) genome editing technology, SB-318 (for MPS I) and SB-913 (for MPS II) are designed as a single treatment strategy intended to provide stable, continuous production of the IDUA or IDS enzyme for the lifetime of the patient.

SB-318 and SB-913 have already received Orphan Drug, Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). The phase 1/2 clinical trials for these programs, evaluating SB-318 and SB-913 in adults with MPS I and MPS II, respectively, are open and enrolling subjects.

Sangamo's ZFN-mediated in vivo genome editing approach makes use of the endogenous albumin gene locus, a highly expressing and liver-specific site that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein. The ability to permanently integrate the therapeutic gene in a highly specific, targeted fashion significantly differentiates Sangamo's in vivo genome editing approach from conventional AAV cDNA gene therapy. The design of these programs is ultimately to target a population that includes pediatric patients, and it will be important in this population to be able to produce stable levels of therapeutic protein for the lifetime of the patient.

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