RICHMOND, Calif., May 16, 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-525, the Company's clinical stage cDNA gene
therapy candidate for hemophilia A, which is being developed as
part of an exclusive, global collaboration and license agreement
with Pfizer Inc. (NYSE: PFE). 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
assures that questions and issues are resolved quickly, often
leading to earlier drug approval and access by patients.
SB-525 uses a recombinant adeno-associated virus (rAAV) to
deliver a human Factor VIII cDNA construct and proprietary,
synthetic liver-specific promoter to the nucleus of liver cells
with a single infusion. The therapy is designed as a single
treatment strategy intended to provide continuous, therapeutic
expression of Factor VIII protein.
SB-525 has already received Orphan Drug designation from the
FDA. The FDA has cleared an Investigational New Drug application
for this program, and a Phase 1/2 clinical trial evaluating SB-525
in adults with hemophilia A is expected to open and begin screening
subjects for enrollment by the end of the second quarter 2017. Data
from this study are expected in late 2017 or early 2018.
About Hemophilia A
Hemophilia A is a monogenic, rare bleeding disorder in which the
blood does not clot normally. It is caused by mutations in the F8
gene which encodes Factor VIII clotting protein that helps the
blood clot and stop bleeding when blood vessels are injured.
Individuals with this mutation experience bleeding episodes after
injuries and spontaneous bleeding episodes that often lead to joint
disease such as arthritis. According to the Centers for Disease
Control and Prevention, hemophilia occurs in about one of every
5,000 male births, with an estimated 20,000 males in the U.S.
living with the disorder.
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 advancing Phase 1/2 clinical programs
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 may contain forward-looking
statements based on Sangamo's
current expectations.
These forward-looking statements
include, without limitation references
relating to the benefit of Fast Track designation to accelerate
regulatory approval of SB-525, 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, and the expected
timing of initiating clinical trials of SB-525 and the release of
data from these trials. Actual results may
differ materially from these
forward-looking statements due to
a number of factors,
including uncertainties relating to
substantial dependence on the clinical success of lead
therapeutic programs, 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.