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Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-318 Genome Editing Treatment for MPS I

Mittwoch, 11.01.2017 13:30 von PR Newswire

PR Newswire

RICHMOND, Calif., Jan. 11, 2017 /PRNewswire/ -- Sangamo Therapeutics, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SB-318, a genome editing product candidate for the treatment of Mucopolysaccharidosis Type I (MPS I), a rare lysosomal storage disorder. Orphan drug designations are granted to drugs and biologics intended to treat rare diseases. The designation provides incentives to advance development of rare disease drugs and for commercialization of those drugs that progress to approval.

MPS I is caused by mutations in the gene encoding the alpha-L-iduronidase (IDUA) enzyme. Using Sangamo's zinc finger nuclease (ZFN) genome editing technology, SB-318 is designed as a single treatment strategy intended to provide stable, continuous production of the IDUA enzyme for the lifetime of the patient.

In 2017, Sangamo plans to conduct the first ever in vivo genome editing clinical trials including Phase 1/2 studies for three lead programs: SB-318 for the treatment of MPS I; SB-913 for the treatment of MPS II, another rare lysosomal storage disorder; and SB-FIX for the treatment of hemophilia B, a rare blood disease. Data from these studies and from a planned clinical trial for a fourth lead program, SB-525, a gene therapy approach for hemophilia A, are expected in late 2017 or early 2018.

Sangamo's In Vivo Genome Editing Approach
Sangamo's ZFN-mediated in vivo genome editing approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic 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 product. 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 approaches. Ultimately, the target population for these programs will include 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. With such a large capacity for protein production (approximately 15g/day of albumin), targeting and co-opting only a very small percentage of the albumin gene's capacity is sufficient to produce the needed replacement protein at therapeutically relevant levels with no significant effect on albumin production.

About Sangamo
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's proprietary zinc finger nuclease (ZFN) in vivo genome editing approach is being evaluated in Phase 1/2 clinical trials to treat hemophilia B and lysosomal storage disorders MPS I and MPS II. Sangamo also plans this year to conduct a Phase 1/2 clinical trial to evaluate its AAV cDNA human Factor 8 gene therapy approach, SB-525, to treat hemophilia A. Sangamo has a strategic collaboration with Biogen, Inc. for hemoglobinopathies, including sickle cell disease and beta-thalassemia, and with Shire plc to develop therapeutics for Huntington's disease. In addition, Sangamo has Phase 1/2 and Phase 2 clinical programs in HIV/AIDS (SB-728). It has established strategic partnerships with companies in non-therapeutic applications of its technology, including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the Company's website at

Forward Looking Statements
This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation references relating to research and development of therapeutic applications of Sangamo's gene therapy and ZFP technology platforms, the potential of Sangamo's technology to treat hemophilia and lysosomal storage disorders, the expected timing of clinical trials and the release of data from these trials, the impact of Sangamo's clinical trials on the field of genetic medicine and the benefit of orphan drug status. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's SEC filings, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo Therapeutics, Inc. assumes no obligation to update the forward-looking information contained in this press release.


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SOURCE Sangamo Therapeutics, Inc.