MINNEAPOLIS, Minn., June 4 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today that the Company sponsored a scientific symposium on Thursday, June 3, at the 7th Annual Meeting of the American Society of Gene Therapy (ASGT) in Minneapolis. The Symposium, entitled "Zinc Finger DNA-Binding Proteins for Gene Regulation and Gene Correction: A Novel Class of Human Therapeutics", focused on advances in ZFP Therapeutics (TM); zinc finger DNA-binding proteins (ZFPs) that are being developed to treat and possibly cure human diseases.
The symposium was opened and chaired by Professor Sir Aaron Klug of the MRC Laboratory of Molecular Biology, Cambridge, U.K. Among his many honors, Dr. Klug was awarded the Nobel Prize for Chemistry in 1982 for his development of crystallographic electron microscopy and his structural elucidation of biologically important nucleic acid protein complexes. Dr. Klug's most recent work has focused on understanding the molecular interactions between ZFPs and the DNA sequences to which they bind. His presentation described the structural basis for the versatility of the ZFP motif as a protein that can be engineered and used in a modular fashion to bind novel DNA sequences.
Philip Gregory, D. Phil., Sangamo's senior director of research, described the translation of these structural findings into a robust technology platform. Dr. Gregory provided examples highlighting the Company's ability to design and engineer ZFPs capable of binding to a specific DNA sequence within any gene of interest. He summarized some of the potential applications of the technology in gene regulation and gene correction. Additionally, he presented data that demonstrate both the specificity and potency that can be achieved with ZFP transcription factors (ZFP TFs).
Frank Giordano, M.D., assistant professor of medicine at Yale University Medical School described the use of ZFP TFs for therapeutic gene regulation of cardiovascular disease targets describing data obtained in therapeutic programs for peripheral artery disease and congestive heart failure. He presented preclinical animal data that showed that a ZFP Therapeutic designed to activate the expression of vascular endothelial growth factor (VEGF A) was able to induce angiogenesis, increase blood flow to the limbs and consequently prevent limb loss in advanced age mice, a model of peripheral artery disease. He also presented the first in vivo data from Sangamo's program to develop a ZFP TF to shut off the expression of phospholamban (PLN) for treatment of congestive heart failure. These data show that ZFP-mediated repression of endogenous PLN results in augmentation of calcium reuptake and improved contractile function of ZFP TF-treated rat cardiac myocytes.
A fourth presentation by Matt Porteus, M.D. Ph.D., assistant professor of pediatrics and biochemistry at The University of Texas Southwestern Medical Center at Dallas described the molecular development of zinc finger nucleases (ZFNs) for targeted gene correction. Dr. Porteus described his own primary studies, now published in Science, which highlight the potential of the gene correction approach. Dr. Porteus also presented early progress in the development of ZFNs for gene correction of the mutation in the b-globin gene that causes sickle cell anemia.
Don Kohn, M.D., current president of the American Society of Gene Therapy, gave the final presentation of the symposium. Dr. Kohn is professor of Pediatrics and Molecular Microbiology and Immunology at U.S.C. Keck School of Medicine and Children's Hospital Los Angeles. A leader in the clinical application of gene therapy, Dr. Kohn presented potential approaches for treating X-linked SCID focusing on Sangamo's most advanced therapeutic gene correction program. He presented data demonstrating ZFP-mediated gene correction efficiencies as high as 18% at an endogenous gene in a model cell system and greater than 1% in CD34+ cells, the cell type that will be targeted in patients. He concluded his presentation by outlining the steps for bringing ZFP-mediated gene correction towards clinical trials.
A webcast of the symposium presentations is available via a link on the Sangamo BioSciences website in the Investor Relations section under "Company Overview".
About Zinc Finger DNA Binding Proteins
Zinc Finger DNA Binding Proteins (ZFPs) are a naturally occurring class of DNA binding proteins. The DNA recognition and binding function of ZFPs can be engineered and thus directed to a targeted sequence of DNA. This permits the delivery of a variety of functional domains to a gene-specific location. ZFPs are being developed for two significant therapeutic applications: gene regulation and gene correction. In the case of therapeutic gene regulation, ZFPs are being engineered to either turn on therapeutically beneficial genes or turn off the expression of disease-causing genes. For gene correction, ZFPs are being used in combination with a DNA cutting enzyme (endonuclease) functional domain to facilitate the correction of mutant DNA sequences in cells.
Sangamo BioSciences, Inc is focused on the research and development of novel transcription factors for therapeutic gene regulation and repair. The company's most advanced therapeutic development program, which is currently in a Phase I clinical trial, involves the use of transcription factors for the treatment of peripheral artery disease. Other therapeutic development programs are focused on ischemic heart disease, cancer, neuropathic pain, and monogenic diseases. Sangamo's core competencies enable the engineering of a class of transcription factors known as zinc finger DNA binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNA sequence Sangamo has created ZFP transcription factors (ZFP TFs) that can control gene expression and consequently, cell function. Sangamo is also developing sequence-specific ZFP nucleases (ZFNs) for therapeutic gene correction as a treatment and possible cure for a variety of monogenic diseases such as severe combined immunodeficiency and sickle cell anemia. For more information about Sangamo, visit the company's web site at www.sangamo.com or www.expressinglife.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, clinical trials 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 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 assumes no obligation to update the forward-looking information contained in this press release.
For Sangamo BioSciences Inc.
Elizabeth Wolffe, Ph.D.
Manager, Corporate Communications
Burns McClellan, Inc.
Tricia Morsch (media)
Michelle Levine (investors)
Copyright 2013 Sangamo BioSciences, Inc.