Sangamo BioSciences Announces Presentation of ZFP Therapeutic Data From Nerve Regeneration Program at American Society for Neural Therapy and Repair Meeting

Statistically Significant Improvements in Function in Spinal Cord Injury
Model With ZFP TF Treatment

RICHMOND, Calif., May 3 /PRNewswire-FirstCall/ -- Sangamo BioSciences,
Inc. (Nasdaq: SGMO) announced today the presentation of preclinical data
from its ZFP Therapeutic(TM) program in nerve regeneration at the
Fourteenth Annual Conference of the American Society for Neural Therapy and
Repair. The data generated, in a model of spinal cord injury (SCI),
demonstrate that treatment of the spinal cord at the time of injury with a
VEGF ZFP TF had a statistically significant effect on recovery of hind-limb
function as well as a number of other measures of nerve integrity and
The work was carried out in the laboratory of Dr. Michael Fehlings who
holds the Krembil Chair in Neural Repair and Regeneration at the Krembil
Neuroscience Center, Toronto Western Hospital and the Division of
Neurosurgery, Faculty of Medicine, University of Toronto, Ontario, Canada.
Dr. Fehlings is a Christopher Reeve Foundation Scientific Advisory Council
member and a leading expert in the molecular mechanisms and treatment of
In collaboration with Dr. Fehlings and his colleagues, Sangamo is
evaluating a zinc finger DNA-binding protein transcription factor (ZFP
TF(TM)), designed to upregulate the expression of the gene encoding
vascular endothelial growth factor (VEGF-A) in spinal cord injury (SCI)
models. In addition to its effects on angiogenesis or blood vessel growth,
VEGF-A has been demonstrated to have direct neurotrophic and
neuroprotective properties in several models that assess nerve integrity
and health. In addition, Sangamo is currently developing SB-509, a plasmid
formulation of this same ZFP TF, for the treatment of diabetic neuropathy
and has two ongoing Phase 2 trials in this area.
Dr. Fehlings and his colleagues observed that administration of the ZFP
TF into the spinal cord at the time of injury produced measurable VEGF ZFP
TF and increased levels of all of the major isoforms of the VEGF protein.
They observed a corresponding neuroprotective effect with a statistically
significant decrease in nerve fiber degradation and post-injury nerve cell
death. They also noted an increase in blood vessel density around the
injury. Most importantly, in a severe model of SCI where animals are
paraplegic post- injury, they observed a statistically significant
(p<0.0001) improvement of hind-limb function over time.
"I am very impressed by the improvements that we were able to generate
through treatment of acutely injured spinal cord with the VEGF ZFP TF,"
stated Dr. Fehlings. "In earlier studies we had observed clear increases in
VEGF protein levels and good evidence of a neuroprotective effect of the
ZFP TF. In these more recent experiments we have seen evidence of improved
functional outcomes that are quite impressive compared with any other
therapeutic approach that we have evaluated including stem cell therapies.
We look forward to continuing these studies in collaboration with Sangamo
and believe that this VEGF-activating ZFP TF may hold promise as a unique
therapy for spinal cord injury and other forms of neural injury."
"Dr. Fehling's studies, particularly the experiments that demonstrate
an improvement in hind-limb function, are very encouraging," said Dale
Ando, M.D. Sangamo's vice president of therapeutic development and chief
medical officer. "The outcomes of these studies are consistent with other
data from a variety of models that suggest that our ZFP TF activator of
VEGF expression has regenerative effects on nerves and a positive effect on
the health of nerves injured by both mechanical means such as in spinal
cord injury and the toxicity that is a function of diabetes. We are very
pleased to continue to work with Dr Fehlings who is world-renown for his
work on SCI and has significant clinical trial experience in this area."
S. Kaye Spratt, Ph.D., Director, Preclinical Development at Sangamo
BioSciences, Inc., will chair a session on novel therapies at the meeting.
About Spinal Cord Injury
Spinal Cord Injury (SCI) is damage to the spinal cord that results in a
loss of function such as mobility or feeling. The National Spinal Cord
Injury Statistical Center (NSCISC) estimates that there are approximately
11,000 new cases each year primarily in young adults. The spinal cord is
the major bundle of nerves that carries nerve impulses to and from the
brain to the rest of the body. The spinal cord does not have to be severed
in order for a loss of function to occur. In fact, in most people with SCI,
the spinal cord is intact, but the damage to it results in loss of
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. Phase 1 clinical trials are
ongoing to evaluate a ZFP Therapeutic for peripheral artery disease. Other
therapeutic development programs are focused on cancer and HIV/AIDS,
neuropathic pain, nerve regeneration, ischemic heart 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(TM)) that can control gene expression
and, consequently, cell function. Sangamo is also developing
sequence-specific ZFP Nucleases (ZFN(TM)) for therapeutic gene modification
as a treatment for a variety of monogenic diseases, such as X-linked SCID
and hemophilia, and for infectious diseases, such as HIV. Sangamo has
established several Enabling Technology Agreements with companies to apply
its ZFP Technology to enhance the production of protein pharmaceuticals.
For more information about Sangamo, visit the company's web site at
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 ZFNs as ZFP Therapeutics, applications of Sangamo's ZFP TF
technology platform 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, uncertainties relating to
the initiation and completion of stages of ZFP Therapeutic clinical trials,
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 10-Q. Sangamo BioSciences, Inc. assumes no
obligation to update the forward-looking information contained in this
press release.