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 www.sangamo.com.
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.