RICHMOND, Calif., Aug. 31, 2017 /PRNewswire/ -- Sangamo
Therapeutics (Nasdaq: SGMO) this afternoon is presenting
preclinical data demonstrating the Company's engineering
capabilities in T cell genome editing using zinc finger nucleases
(ZFNs). Gary Lee, Ph.D., Senior
Director, Genome Editing at Sangamo, is delivering the
presentation, "Engineering T Cells for Cancer Therapeutics," at the
Immuno-Oncology Summit in Boston.
"We believe that next generation cellular immuno-oncology
treatments will be allogeneic, 'off-the-shelf', genome edited T
cell therapies," said Dr. Lee. "Developing allogeneic T cell
therapies requires engineering with genome editing technology in
order to 'knock-out' the proteins that may cause the body to reject
the treatment and to 'knock-in' the targeting moiety that enables
the T cell to find and destroy a particular cancer cell. We believe
the higher cell modification efficiency and simultaneous knock-out
of multiple endogenous genes via ZFN-mediated genome editing have
the potential to provide more consistent and better regulated
TCR/CAR expression and activity, improved anti-tumor activity and
durability, as well as a stronger safety profile."
Dr. Lee's presentation highlights Sangamo's cell therapy
expertise and provides data from the Company's recent preclinical
research toward the development of allogeneic T cell
immunotherapies utilizing ZFN editing techniques:
- Elimination of endogenous T cell receptor (TCR) expression by
knock-out of the TCR alpha constant locus (TRAC) with greater than
90% efficiency
- Elimination of human leukocyte antigen (HLA) Class I proteins
by knock-out of b2-microglobulin (B2M) with greater than
90% efficiency
- Co-delivery double knock-out of TRAC and B2M with greater than
90% efficiency
- Targeted integration of a green fluorescent protein (GFP)
expression cassette into either the TRAC or B2M locus with ~90%
efficiency with double knock-out of TCR and HLA Class I
- CD19 chimeric antigen receptor (CAR) integrated into either the
TRAC or B2M locus demonstrated strong antigen specific killing with
a clear dose response
- No evidence of off-target cleavage in human primary T
cells
Through the company's legacy T cell genome editing programs,
Sangamo has accumulated robust clinical expertise in therapeutic T
cell development and manufacturing of clinical grade materials. In
approximately 100 HIV patients treated with ZFN-edited T cells,
Sangamo has demonstrated persistent engraftment and established a
strong safety profile for the duration of observation (3 years post
infusion).
"Sangamo is uniquely positioned in the emerging field of
immuno-oncology with our ZFN gene editing platform, which we
believe is ideally suited for allogeneic or autologous approaches,
and with our ex vivo clinical and manufacturing experience
in cell engineering and development," said Dr. Sandy Macrae, CEO of Sangamo. "Our strategy in
oncology is to advance our T cell editing platform in collaboration
with partners with the appropriate development and
commercialization expertise."
Dr. Lee's slides are available on the Presentations +
Publications page of the technology section of Sangamo's
website.
About Sangamo's Zinc Finger Nucleases
Sangamo's
proprietary genome editing technology is based on a naturally
occurring class of proteins called zinc finger DNA-binding proteins
(ZFPs) which recognize and bind to specific sequences of DNA.
Sangamo can engineer these naturally occurring ZFPs to bind to
virtually any chosen DNA sequence. By combining ZFPs with nucleases
(DNA cutting enzymes) to create zinc finger nucleases (ZFNs),
Sangamo can harness the powerful targeting capabilities of zinc
fingers to edit the human genome, specifically knocking out a DNA
sequence or adding a new gene in a precise location.
In 2017, Sangamo scientists have reported on recent advancements
in design and engineering which have enhanced the profile of ZFNs
across three important criteria for the development of therapeutic
genome editing: Precision, Efficiency and Specificity. With
thousands of zinc finger modules in the Sangamo library and an
array of linkers attaching the zinc fingers and the FOK1 nuclease
domain, Sangamo is able to assemble highly specific ZFN pairs for
virtually any chosen target site. For any given 20-base pair window
in the genome, Sangamo has on average 450 functionally distinct ZFN
modules to evaluate. Sangamo believes that the very high design
density of the Company's ZFN library has operational advantages in
choosing a final ZFN pair with an optimal profile to advance into
potential clinical development.
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. For more information
about Sangamo, visit www.sangamo.com.
Forward Looking Statements
This press release
contains forward-looking statements, including, but not limited to,
statements related to Sangamo's expectations for cellular
immuno-oncology treatments, the clinical and therapeutic potential
of Sangamo's ZFN gene editing platform, Sangamo's strategy to
advance its T cell editing platform in collaboration with partners,
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: gene therapy product candidate
development and the inherent uncertainty of clinical success,
including the risks that Sangamo and/or its collaborators may
encounter unanticipated toxicity or adverse events or fail to
demonstrate efficacy in clinical development; Sangamo's substantial
dependence on the clinical success of its lead therapeutic
programs; the initiation, enrollment and completion of the stages
of its clinical trials; technological challenges; the lengthy and
uncertain regulatory approval process; Sangamo's ability to develop
commercially viable products; technological developments by its
competitors and others in the gene therapy and/or cellular
immuno-oncology treatment fields; Sangamo's dependence on
collaborations to further the development of its technology
platforms, including Sangamo's potential inability to successfully
enter into new collaborations with third parties on acceptable
terms, or at all, in order to advance its T cell editing platform.
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 June 30, 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.