RICHMOND, Calif., Jan. 10, 2017 /PRNewswire/
-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), the leader in
therapeutic genome editing, today announced the presentation of key
improvements to its technology platform for engineering highly
specific zinc finger nucleases (ZFNs) for therapeutic genome
editing applications. The presentation was delivered January 9, 2017 by Edward
Rebar, Ph.D., Sangamo's vice president, technology, an
invited speaker at the Keystone Symposium on Precision Genome
Engineering, which is being held this week in Breckenridge, CO.
The data demonstrate genome editing of therapeutic gene targets
in clinically relevant cell types, including high levels of
targeted modification (80%) of the BCL11A enhancer in hematopoietic
stem and progenitor cells at clinical scale with no significant
off-target activity. Off-target activity was assessed using
state of the art assays involving unbiased oligonucleotide capture
methods followed by deep sequencing.
"Based on a human DNA-binding motif, we believe ZFNs are the
most advanced, flexible and precise genome editing technology
available, and, as our scientists have demonstrated, they can be
further engineered to refine their specificity and potency for
therapeutic use," said Sandy Macrae,
M.B., Ch.B., Ph.D., Sangamo's CEO. "The new architectures and
strategies that we describe represent a substantial improvement in
our capabilities while retaining full compatibility with our
existing genome editing platform. The modifications enable us to
target any investigator chosen site in the genome with enhanced
precision and a reduction in off-target cleavage to levels that are
at or below the limit of detection even under conditions of very
high on-target activity. Importantly, these results are achievable
at clinical scale, using clinical delivery conditions, and in
clinically relevant cell types."
ZFNs can be engineered to introduce a precise double strand
break at any chosen location in the genome to correct, delete or
add genes. The advances described in Dr. Rebar's talk include new
linkers for connecting individual fingers to each other or to the
DNA cleavage domain, which enables greater configurational
diversity and increases the number of sites in the genome that can
be targeted. Modifications of key framework residues in the
zinc fingers are also presented that can selectively suppress
off-target cleavage activity by >100 fold.
"These engineering advances are exciting because they enable
development of extremely specific nucleases for therapeutic
applications that are not limited by the targeting constraints
inherent to other cleavage platforms," said Dr. Rebar. "These new
architectures will also accelerate development of highly active and
specific ZFNs, resulting in much faster identification of
therapeutic leads."
In addition to the Keystone Symposium, Sangamo is also
participating this week in the JP Morgan Healthcare Conference
being held in San Francisco.
Sangamo CEO Sandy Macrae will
present an overview of the Company's strategy, clinical development
programs, and research on January 11th,
2017 at 4:30 pm Pacific Time.
A live webcast of Dr. Macrae's presentation will be accessible
through a link on the Investors + Media section of the company's
website, www.sangamo.com.
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 is also
conducting 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, the advantages and capabilities of
improved ZFN engineering in therapeutic applications, the potential
of modified ZFN technology to enhance clinical development and
identification of therapeutic leads, and the ability of the new ZFN
architectures to enable specific modification of DNA with high
efficiency without significant off-target event. 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.