RICHMOND, Calif., July 13, 2017 /PRNewswire/
-- Sangamo Therapeutics, Inc. (NASDAQ: SGMO) announced today
that the U.S. Food and Drug Administration (FDA) has granted Fast
Track designation to SB-318 and SB-913, the Company's clinical
stage in vivo genome editing product candidates for the
treatment of Mucopolysaccharidosis Type I (MPS I) and MPS II,
respectively. The FDA's Fast Track designation is designed to
facilitate the development and expedite the review of drugs and
biologics to treat serious conditions and fill an unmet medical
need. Once a drug receives Fast Track designation, early and
frequent communication with the FDA is encouraged throughout the
development and review process. The frequency of communication is
designed to ensure that questions and issues are resolved quickly,
potentially leading to earlier drug approval and access by
patients.
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 and Rare
Pediatric Disease designations from the FDA. The FDA has cleared an
Investigational New Drug application for these programs, and Phase
1/2 clinical trials evaluating SB-318 and SB-913 in adults with MPS
I and MPS II, respectively, are open and screening subjects for
enrollment.
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.
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.
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 has open 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.
Forward-Looking Statements
This press release contains forward-looking statements,
including, but not limited to, statements related to the potential
therapeutic applications of, and target populations for, Sangamo's
gene therapy and ZFP technology platforms, including the potential
of Sangamo's technology to treat hemophilia and lysosomal storage
disorders, the potential benefits of Fast Track designation, and
other statements that are not historical facts. These
forward-looking statements are based on Sangamo's current plans,
objectives, estimates, expectations and intentions and inherently
involve significant risks and uncertainties. Actual results and the
timing of events could differ materially from those anticipated in
such forward-looking statements as a result of these risks and
uncertainties, which include, without limitation, risks and
uncertainties associated with: the lengthy and uncertain regulatory
approval process, including the risks that Fast Track designation
may not lead to an expedited development, review or approval
process, and that such designation does not increase the likelihood
that Sangamo's product candidates will receive regulatory approval;
Sangamo's substantial dependence on the clinical success of its
lead therapeutic programs; the initiation, enrollment and
completion of stages of its clinical trials; whether Sangamo's
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 its competitors. A more detailed discussion of
these and other risks and uncertainties may be found under the
caption "Risk Factors" and elsewhere in Sangamo's SEC filings and
reports, including Sangamo's Quarterly Report on Form 10-Q for the
quarter ended March 31, 2017 and
future filings and reports by Sangamo. Sangamo assumes no
obligation to update the forward-looking information contained in
this press release.
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SOURCE Sangamo Therapeutics, Inc.