RICHMOND, Calif., July 1 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today that it has
Sangamo has developed zinc finger DNA-binding protein transcription factors (ZFP TFs) to activate the expression of glial cell line-derived neurotrophic factor (GDNF), a potent neurotrophic factor that has shown promise in preclinical testing to slow or stop the progression of Parkinson's disease. In earlier preclinical studies also funded by MJFF and carried out by Sangamo scientists and their collaborators, a ZFP TF activator of GDNF was shown to be neuroprotective, resulting in positive functional improvements in a validated rat model of PD.
"We are very enthusiastic about this novel ZFP Therapeutic approach to Parkinson's disease," said Krystof Bankiewicz, M.D., Ph.D., Professor of Neurosurgery and Neurology at the University of California, San Francisco (UCSF), whose group is collaborating with Sangamo on the preclinical animal studies. "We believe that GDNF is an excellent therapeutic target for PD. However, using a ZFP TF to turn on the expression of the native GDNF gene in the brain rather than adding GDNF protein or a copy of the GDNF gene, enables the achievement of physiological levels of this potent neuroprotective factor. Based on our earlier success with a ZFP activator of GDNF in the rat model we look 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 dopamine, a chemical messenger responsible for transmitting signals within the brain. Loss of dopamine causes critical nerve cells in the brain, or neurons, to fire out of control, leaving patients unable to direct or control their movement in a normal manner. The symptoms of Parkinson's may include tremors difficulty maintaining balance and gait, rigidity or stiffness of the limbs and trunk, and general slowness of movement (also called bradykinesia). Patients may also eventually have difficulty walking, talking, or completing other simple tasks. Symptoms often appear gradually yet with increasing severity and the progression of the disease may vary widely from patient to patient. There is no cure for Parkinson's disease. Drugs have been developed that can help patients manage many of the symptoms; however, they do not prevent disease progression.
About The Michael J. Fox Foundation
Founded in 2000, The Michael J. Fox Foundation for Parkinson's Research is dedicated to ensuring the development of a cure for Parkinson's disease within this decade through an aggressively funded research agenda. The Foundation has funded over $179 million in research to date, either directly or through partnerships.
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™ development program is currently in a Phase 2b clinical trial for evaluation of safety and clinical effect in patients with diabetic neuropathy and a Phase 2 trial in ALS. Sangamo also has two Phase 1 clinical trials to evaluate safety and clinical effect of a treatment for HIV/AIDS and another Phase 1 trial to evaluate safety and clinical effect of a treatment for recurrent glioblastoma multiforme. Other therapeutic development programs are focused on 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 and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company's website 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 and therapeutic applications of Sangamo's ZFP technology platform, including treatment of Parkinson's disease. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. See Sangamo's SEC filings, and in particular, the risk factors described in Sangamo's Annual Report on Form 10-K and its most recent report on Form 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.
SOURCE Sangamo BioSciences, Inc.
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