RICHMOND, Calif., Dec. 7, 2017 /PRNewswire/
-- Sangamo Therapeutics, Inc. (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.