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Sangamo BioSciences Receives Michael J. Fox Foundation Funding to Develop Novel Drug for the Treatment of Parkinson's Disease
Date:7/1/2010

forward to collaborating with Sangamo to further develop this promising therapeutic approach."

Sangamo's technology platform permits the development of highly specific ZFP TFs that can be used to regulate genes at the DNA level. Using a ZFP TF, a gene can be activated in a patient's own cells in its natural cellular context. Use of a ZFP TF to activate the GDNF gene in the brain — as compared to addition of a GDNF cDNA or the recombinant protein —allows the delivery of a more physiologically relevant dose of the growth factor which may be a safer and more effective alternative. This is of particular significance when targeting potent natural growth factors such as GDNF, where sufficient but not super-physiological levels of the therapeutic protein are required to achieve efficacy.

"We are very pleased that The Michael J. Fox Foundation continues to support these IND-enabling studies as part of our program to develop a novel ZFP Therapeutic for PD," said Edward Lanphier, Sangamo's president and CEO. "ZFP TF-driven gene activation underpins our lead ZFP Therapeutic (SB-509) which has been evaluated in four Phase 2 clinical trials for diabetic peripheral neuropathy and demonstrated both clinical efficacy and an excellent safety profile. Preclinical success in PD models with the ZFP TF activator of GDNF will support the evaluation of this approach in the clinic.  Moreover, it would demonstrate the potential for ZFP TF use in the brain and set the stage for the further application of our powerful technology platform to other genes that may have a therapeutic benefit in PD."

About Parkinson's Disease

Parkinson's disease is a chronic, progressive disorder of the central nervous system and results from the loss of cells in a section of the brain called the substantia nigra. These cells produce
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SOURCE Sangamo BioSciences, Inc.
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