RICHMOND, Calif., Dec. 5, 2016 /PRNewswire/ -- Sangamo BioSciences,
Inc. (Nasdaq: SGMO), the leader in therapeutic genome editing,
announced the presentation of preclinical and manufacturing data
that support SB-525, its gene therapy program for hemophilia A, at
the 58th Annual Meeting of the American Society of
Hematology (ASH) being held in San Diego,
CA, from December 3-6,
2016.
"We have developed an improved gene therapy vector for treatment
of hemophilia A which we believe is highly competitive, and we
remain on track to file an Investigational New Drug (IND)
application for our clinical program by the end of 2016," said
Sandy Macrae, M.B., Ch.B., Ph.D.,
Sangamo's president and chief executive officer. "This
program illustrates Sangamo's drug development capabilities beyond
our ZFP platform. Moreover, it demonstrates the ability of our
R&D and technical operations teams to rapidly develop a program
from research data, through positive preclinical results in
relevant animal models to a clinical program fully supported by a
scalable manufacturing process. We have strengthened our clinical
development capabilities with recent senior management changes and
are committed to consistent achievement of planned milestones for
our four clinical programs. We look forward to initiating a
clinical trial in 2017 to evaluate SB-525."
The development of adenovirus associated vector (AAV) cDNA gene
therapy approaches for hemophilia A has been challenging due to a
number of factors, including the large size of the native human
Factor 8 gene (hF8), low levels of human Factor VIII protein
(hFVIII) expression from conventional promoters, and low yields of
vector in large scale manufacturing processes. Through an iterative
process Sangamo scientists have developed an improved AAV construct
that expresses an hF8 cDNA cassette (SB-525). A single
intravenous administration of SB-525 results in the expression of
significant levels of hFVIII in mice and non-human primates (NHPs)
and correction of the bleeding defect in a mouse model of
hemophilia A. The construct also resulted in greatly improved AAV
yields during vector manufacturing.
Importantly, dosing studies in NHPs which evaluated SB-525
manufactured at GMP-clinical scale demonstrated a robust and
reproducible dose response curve and the most potent dose response
in NHPs thus far disclosed for an hF8 cDNA gene therapy
program. In these animals, mean hFVIII levels ranged from 5%
of normal at the lowest dose to 230% at the highest (AAV doses in
the 6 x 1011 – 6 x 1012 vgs/kg range). The
therapeutic levels of hFVIII that were observed in these studies
support starting clinical doses in the E11 vg/kg range.
About Sangamo
Sangamo BioSciences, Inc. is focused on
Pioneering Genetic Cures™ for monogenic and infectious diseases by
deploying its AAV-based gene therapy platform, and therapeutic
genome editing and gene regulation platforms based on its novel
zinc finger DNA-binding protein technology. The Company's
proprietary zinc finger nuclease (ZFN)-mediated in vivo genome
editing approach is focused on monogenic diseases, including
hemophilia and lysosomal storage disorders MPS I and MPS II.
Sangamo has initiated a Phase 1/2 clinical trial for hemophilia B,
the first in vivo genome editing application cleared by the
FDA. In addition, Sangamo has Phase 1/2 and Phase 2 clinical
programs in HIV/AIDS (SB-728). The Company has a strategic
collaboration with Bioverativ, Biogen's planned spin-off company
for rare blood disorders, for hemoglobinopathies, including sickle
cell disease and beta-thalassemia, and with Shire International
GmbH to develop therapeutics for Huntington's disease. 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.
This press release may contain forward-looking statements
based on Sangamo's current expectations. These forward-looking
statements include, without limitation, the research and
development of novel AAV-based gene therapy vectors for
treatment of hemophilia A, therapeutic applications of Sangamo's
ZFP technology platform in hemoglobinopathies and other diseases,
the anticipated timing of IND application filing and initiation of
clinical trials for the hemophilia A program, and the potential of
clinical dosage to drive the expression
of therapeutic levels of Factor VIII expression. 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 BioSciences, Inc.
assumes no obligation to update the forward-looking information
contained in this press release.
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SOURCE Sangamo BioSciences, Inc.