SCI tissue preservation

Sangamo BioSciences, Inc. announced the presentation of preclinical data from its ZFP Therapeutic(TM) program in nerve regeneration at Neuroscience 2007, the 37th Annual Meeting of the Society for Neuroscience. A statistically significant effect on both recovery of hind-limb function and spinal cord tissue preservation was demonstrated in a severe model of spinal cord injury (SCI) following treatment at the time of injury with a zinc finger DNA-binding protein activator of vascular endothelial growth factor (VEGF ZFP TF).

"We are excited by the statistically significant improvements in measures of both nerve health and, more importantly, nerve function after treatment with our VEGF ZFP TF," said Dale Ando, M.D. Sangamo's vice president of therapeutic development and chief medical officer. "Data generated in 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 increases the density of blood vessels in and around the treated tissue.

We believe that these effects will prove therapeutically important in conditions that involve nerve damage including diabetic neuropathy, ALS, stroke and traumatic injuries to the brain, spinal cord and other nerves. We are very pleased to collaborate with Dr. Michael Fehlings, senior author of this study, who is world-renown for his work on SCI and has significant clinical trial experience in this area."

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. In addition, the Company plans to initiate two new Phase 2 clinical trials to evaluate this ZFP Therapeutic in stem cell mobilization and amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease. ZFP activation of VEGF-A has been demonstrated to stimulate angiogenesis or blood vessel growth, to have direct neurotrophic and neuroprotective properties in several models that assess nerve integrity and health, and may play an important role in stem cell mobilization and thereby tissue repair.

Data Reported in the Neuroscience 2007 Presentation

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 protein and increased levels of all 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 as well as a statistically significant improvement in spinal cord tissue preservation at the site of injury (P<0.005). They also noted an increase in blood vessel density around the injury. Importantly, in this 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.

"In our studies, Sangamo's VEGF ZFP TF has shown remarkable positive improvements in all measured parameters of this model," stated Dr. Fehlings, M.D. Ph.D., senior author of the study. "The data obtained in our preclinical model appear highly encouraging and we look forward to continuing to work with Sangamo to move this program forward into clinical studies. We believe that this VEGF-activating ZFP TF holds considerable promise as a unique therapy for spinal cord injury and other forms of neural injury."

Source - Sangamo BioSciences

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