RICHMOND, Calif., Jan. 23 /PRNewswire/ -- Sangamo BioSciences, Inc. announced today that it has been awarded funding by The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support the development of a ZFP Therapeutic(TM) to treat Parkinson’s disease (PD). The $950,000 award will be paid over a period of two years.
Sangamo will develop zinc-finger DNA-binding protein transcription factors (ZFP TFs(TM)) 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.
“We are enthusiastic about Sangamo’s novel approach to GDNF as it fits squarely within our desire to promote alternative therapeutic strategies for this and other promising molecules,” said Brian Fiske, PhD, associate director of research programs for MJFF. “The work is of particular interest given that Sangamo already has similar technology in clinical trials for diabetic neuropathy and peripheral artery disease and, with a positive result, could proceed rapidly into clinical trials for PD.”
Sangamo’s technology platform permits the development of highly specific ZFP TFs that can be used to regulate or modify 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 -- may allow the delivery of a more physiologically relevant dose of the growth factor. This may be 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 potency with safety.
“We are very pleased that The Michael J. Fox Foundation is recognizing and supporting the potential of Sangamo’s unique technology with this award,” said Edward Lanphier, Sangamo’s President and CEO. “We believe that our approach to therapeutic gene regulation, which mimics the natural regulation of the gene in the body, has advantages over other approaches that add GDNF as either the gene or the recombinant protein. Success in this project would establish the validity of the use of ZFP TFs in the brain and set the stage for the application of this powerful technology 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 $90 million in research to date, either directly or through partnerships.
About Sangamo BioSciences, Inc.
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 a Phase 2 clinical trial for evaluation of safety 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 ischemic heart disease, neuropathic pain, cancer and infectious 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 and therapeutic applications of Sangamo’s ZFP technology platform. 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 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.
Sangamo BioSciences, Inc.
CONTACT: Elizabeth Wolffe, Ph.D. of Sangamo BioSciences, Inc.,+1-510-970-6000, ext. 271, or ewolffe@sangamo.com, or Justin Jackson(media) of Burns McClellan, Inc., 212-213-0006, or jjackson@burnsmc.com
Web site: http://www.sangamo.com/
