RICHMOND, Calif., May 03, 2007 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. 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 health.
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 SCI.
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 produce d 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 function.
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 http://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 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.
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