Sangamo BioSciences Research Collaborators Awarded Grant From California Institute for Regenerative Medicine to Develop Zinc Finger Nuclease-Based Stem Cell Therapy for HIV/AIDS

Wed Oct 28, 2009 4:02pm EDT
 
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Sangamo BioSciences Research Collaborators Awarded Grant From California
Institute for Regenerative Medicine to Develop Zinc Finger Nuclease-Based Stem
Cell Therapy for HIV/AIDS
$14.5 million award to bring ZFP HIV therapy to the clinic






RICHMOND, Calif. Oct. 28 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc.
(Nasdaq: SGMO) announced today that the California Institute for Regenerative
Medicine (CIRM) has granted a $14.5 million Disease Team Research Award to
develop an AIDS-related lymphoma therapy based on the application of its zinc
finger nuclease (ZFN) gene-editing technology in stem cells.  The four year
grant supports an innovative research project conducted by a multidisciplinary
team of investigators led by John Zaia, M.D. the Aaron D. and Edith Miller
Chair in Gene Therapy and chair of virology, City of Hope.  The grant
application entitled "Zinc Finger Nuclease-Based Stem Cell Therapy for AIDS"
won the highest score of all grants CIRM received in this 1st round of Disease
Team Research Award funding.

"Sangamo scientists have developed a ZFN-mediated gene-editing technology that
has been demonstrated to make hematopoietic stem cells and mature immune
system cells resistant to HIV infection," said Dr. Zaia.  "This will be the
first test of whether hematopoietic stem cells made HIV resistant using
Sangamo's technology can correct the disease.  If successful, our work could
open the door to ZFN-based cell therapies for other important diseases."

Patients homozygous for a natural mutation (the delta-32 mutation) in the CCR5
gene are resistant to HIV infection by blocking the ability of the virus to
enter a cell. Building on this observation, a study published in the New
England Journal of Medicine in 2009 reported a potential "cure" in an AIDS
patient with leukemia after receiving a bone marrow transplant from a
"matched" donor with this delta-32 CCR5 mutation. This approach transferred
the hematopoietic stem cells (HSC) residing in the bone marrow from the
delta-32 donor, and provided a self-renewable and lifelong source of
HIV-resistant immune cells. After transplantation, this patient was able to
discontinue all anti-HIV drug treatments, CD4 counts increased, and the viral
load dropped to an undetectable level, demonstrating effective transplantation
of protection from HIV infection. 

This CIRM Disease Team Research Award proposes an approach to modify a
patient's own HSC to circumvent the need to find matched donors that carry the
delta-32 CCR5 mutation and while providing a renewable and long-lasting source
of HIV-resistant cells. Specifically, the grant funds the development of a ZFN
approach to treat AIDS patients by first isolating their HSC, modifying them
using CCR5-specific ZFNs, and then re-infusing them to reconstitute the immune
system with CCR5-negative, HIV-resistant immune cells.

"We are delighted that this research proposal was chosen for funding by CIRM,"
commented Dr. Philip Gregory, Sangamo's chief scientific officer and vice
president, research.  "This grant brings together a team of world-renowned
experts to develop this novel ZFN-based stem cell therapy for AIDS-related
lymphoma through to the clinic.  We look forward to working with the team
which includes Paula Cannon, Ph.D., associate professor of Molecular
Microbiology & Immunology, Keck School of Medicine of the University of
Southern California, who has carried out extensive pre-clinical research using
our technology in stem cells, and Dr. Zaia and his colleagues at City of Hope
who are pioneers in hematopoietic cell transplantation." 

"CIRM support for this program is a major validation of our ZFP Therapeutics
platform both scientifically and financially and we are very pleased to be
part of the exceptional team that received the highest score of all of the
grants reviewed by CIRM," said Edward Lanphier, Sangamo's president and CEO. 

The CIRM Disease Team Research Awards will fund actively managed
multidisciplinary teams engaged in milestone-driven translational research for
the development of stem cell-based therapies. The mission of these teams will
be to conduct the necessary research and regulatory activities to prepare and
file a complete, well supported Investigational New Drug Application (IND)
with the Food and Drug Administration (FDA) (and, if desired, other regulatory
agencies), to enable Phase I clinical testing.

About Sangamo
Sangamo BioSciences, Inc. is focused on the research and development of novel
DNA-binding proteins for therapeutic gene regulation and modification.  The
most advanced ZFP Therapeutic(TM) development program is currently in Phase 2
clinical trials for evaluation of safety and clinical effect in patients with
diabetic neuropathy and ALS. Sangamo also has two Phase 1 clinical trials to
evaluate safety and clinical effect of a ZFP Therapeutic for the treatment of
HIV/AIDS. Other therapeutic development programs are focused on cancer,
neuropathic pain, nerve regeneration, Parkinson's disease and monogenic
diseases.  Sangamo's core competencies enable the engineering of a class of
DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs).  By
engineering ZFPs that recognize a specific DNA sequence Sangamo has created
ZFP transcription factors (ZFP TF) that can control gene expression and,
consequently, cell function.  Sangamo is also developing sequence-specific ZFP
Nucleases (ZFN) for gene modification.  Sangamo has established strategic
partnerships with companies in non-therapeutic applications of its technology
including Dow AgroSciences, Sigma-Aldrich Corporation and several companies
applying its ZFP technology to engineer cell lines for the production of
protein pharmaceuticals. For more information about Sangamo, visit the
company's web site 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, references to the research and development of novel ZFP TFs and
ZFNsand their applications in the treatment of HIV/AIDS, receipt of funds from
CIRM, strategic partnerships with collaborators and clinical trials of ZFP
Therapeutics.  Actual results may differ materially from these forward-looking
statements due to a number of factors, including technological challenges,
Sangamo's ability to develop commercially viable products and technological
developments by our competitors.  See the company's SEC filings, and in
particular, the risk factors described in the company's 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.




SOURCE  Sangamo BioSciences, Inc.

Elizabeth Wolffe, Ph.D. of Sangamo BioSciences, Inc., +1-510-970-6000, ext.
271, ewolffe@sangamo.com

 

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