Sangamo Announces EMA Recommendation of Orphan Medicinal Product Designation for Investigational Genome Editing Treatments for MPS I and MPS II

RICHMOND, Calif., Dec. 7, 2017 /PRNewswire/ -- Sangamo Therapeutics (NASDAQ: SGMO) announced today 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 In Vivo Genome Editing Approach
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.

About Sangamo Therapeutics
Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients’ lives using the company’s industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is conducting Phase 1/2 clinical trials in Hemophilia A and Hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has an exclusive, global collaboration and license agreement with Pfizer Inc. for gene therapy programs for Hemophilia A, with Bioverativ Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington’s disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences. For more information about Sangamo, visit the Company’s website at www.sangamo.com.

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SOURCE Sangamo Therapeutics, Inc.