RICHMOND, Calif., Feb. 8, 2017 /PRNewswire/ -- Sangamo
Therapeutics, Inc. (Nasdaq: SGMO), the leader in therapeutic genome
editing, today announced upcoming oral and poster presentations at
the 13th Annual WORLDSymposium™ Meeting being
held in San Diego, CA from
February 13 - 17, 2017.
Members of the Company's research and development team will
present additional preclinical disease model data from its SB-318
and SB-913 therapeutic in vivo genome editing programs for
MPS I and MPS II, new preclinical data on an alternative treatment
modality using lipid nanoparticle delivery of zinc finger nucleases
(ZFNs), as well as preclinical data on gene therapy and
ZFN-mediated in vivo genome editing approaches for Fabry
disease.
- Liver-based expression of the human alpha-galactosidase A
gene (GLA) in a murine Fabry model results in continuous
supra-physiological enzyme activity and effective substrate
reduction – Marshal W. Huston, PhD,
Sangamo Therapeutics
Oral Presentation: Basic Science I, 10:45am PT, Tuesday, Feb.
14, 2017
Poster Presentation: 4:30p – 6:30pm
PT, Tuesday, Feb. 14, 2017
[Poster #151]
- ZFN-mediated in vivo genome editing results in phenotypic
correction in murine MPS I and MPS II models – Russell DeKelver, PhD, Sangamo
Therapeutics
Oral Presentation: Translational Research I, 9:15am PT, Wednesday, Feb.
15, 2017
Poster Presentation: 4:30p – 6:30pm
PT, Tuesday, Feb. 14, 2017
[Poster #71]
- In vivo genome editing via non-viral delivery of zinc finger
nucleases results in supraphysiological levels of human iduronate
2-sulfatase in adult mice – Anthony
Conway, PhD, Sangamo Therapeutics
Poster Presentation: 4:30p – 6:30pm
PT, Wednesday, Feb. 15, 2017
[Poster #532]
About SB-318 and SB-913
SB-318 and SB-913 are being evaluated in Phase 1/2 clinical trials
as single treatment therapies for MPS I and MPS II, lysosomal
storage disorders caused by mutations in the genes encoding the
alpha-L-iduronidase (IDUA) and iduronate 2-sulfatase (IDS) enzymes,
respectively. SB-318 and SB-913 are based on Sangamo's zinc finger
nuclease (ZFN)-mediated in vivo genome editing approach and
are designed to produce continuous, lifelong therapeutic levels of
IDUA or IDS.
Preclinical data for the two programs demonstrated stable,
continuous production of active, human IDUA (hIDUA) or hIDS enzymes
from the liver, which were secreted into circulation and taken up
by various secondary tissues, including the liver, spleen, kidneys,
lungs, heart, muscle and brain of MPS I or MPS II mice after a
single administration of SB-318 or SB-913, respectively. This
resulted in a significant increase in hIDUA or hIDS enzyme activity
and reduction of glycosaminoglycan (GAG) biomarkers in the plasma
and secondary tissues. Treated mice also demonstrated preserved
cognitive function when assessed using a Barnes Maze, which evaluates spatial learning
and memory.
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 Phase 1/2 clinical programs in
hemophilia A and B, and lysosomal storage disorders MPS I and MPS
II. Sangamo has a strategic collaboration 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.
This press release contains forward-looking statements
regarding Sangamo's current expectations. These forward looking
statements include, without limitation, references to preclinical
data from SB-318 and SB-913 programs and the potential of these
programs to treat MPS I and MPS II. These statements are not
guarantees of future performance and are subject to certain risks,
uncertainties and assumptions that are difficult to predict.
Factors that could cause actual results to differ include, but are
not limited to, the early stage of ZFP Therapeutic development, the
lengthy and uncertain regulatory approval process, uncertainties
related to the timing of initiation and completion of clinical
trials, whether clinical trial results will validate and support
the safety and efficacy of ZFP Therapeutics, and the ability to
establish strategic partnerships. Further, there can be no
assurance that the necessary regulatory approvals will be obtained
or that Sangamo and its partners will be able to develop
commercially viable gene-based therapeutics. Actual results may
differ from those projected in forward-looking statements due to
risks and uncertainties that exist in Sangamo's operations and
business environments. These risks and uncertainties are described
more fully in Sangamo's Annual Reports on Form 10-K and Quarterly
Reports on Form 10-Q as filed with the Securities and Exchange
Commission. Forward-looking statements contained in this
announcement are made as of this date, and Sangamo undertakes no
duty to update such information except as required under applicable
law.
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SOURCE Sangamo Therapeutics, Inc.