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Sangamo BioSciences Provides Summary of Data Presented At the 7th Annual Meeting of the American Society of Gene Therapy

Posted on: Monday, 7 June 2004, 06:00 CDT

RICHMOND, Calif., June 7 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. announced today a summary of data presented at the 7th Annual Meeting of the American Society of Gene Therapy (ASGT) in Minneapolis. Sangamo scientists gave a total of 16 presentations highlighting the Company's proprietary zinc finger technology. Sangamo also hosted a scientific symposium entitled, "Zinc Finger DNA-Binding Proteins for Gene Regulation and Gene Correction: A Novel Class of Human Therapeutics." The symposium featured presentations by some of the foremost thought leaders in the area of cell and gene therapy, including; Professor Sir Aaron Klug of the Laboratory of Molecular Biology, Cambridge UK; Dr. Frank Giordano of Yale University Medical School, Dr. Don Kohn of Children's Hospital Los Angeles, the current president of the ASGT and Dr. Matt Porteus of the University of Texas Southwestern Medical Center. Zinc finger DNA-binding proteins, or ZFPs are a naturally occurring class of DNA transcription factors. Sangamo is developing ZFPs for two significant therapeutic areas: gene regulation and gene correction.

"The data presented by our scientists and collaborators demonstrate the breadth and versatility of our zinc finger technology," said Edward Lanphier, Sangamo's president and CEO. "We believe that the specificity and potency of our zinc finger approach may have important therapeutic implications for the future treatment of many debilitating diseases including cardiovascular disease, diabetic neuropathy, congestive heart failure and neuropathic pain. We were also honored and delighted to have hosted world renown authorities at our scientific symposium focused on the potential use of ZFPs as novel therapeutics in gene regulation and gene correction."

Study Highlights

Gene Regulation

Sangamo is using ZFPs for therapeutic gene regulation by turning on therapeutically beneficial genes or by turning off the expression of disease- causing genes. Data were presented detailing the use of this technology in stem cell differentiation, in the treatment of neuropathic pain, diabetic neuropathy and cardiovascular disease.

Data from a rat model of diabetic neuropathy (DN) were presented demonstrating that a single treatment with a ZFP transcription factor (ZFP TF) designed to activate the expression of vascular endothelial growth factor (VEGF-A) resulted in a dose dependent, statistically significant increase in limb nerve function compared with the untreated limb. VEGF has been shown to have positive effects on the maintenance of nerve function and for the restoration of nerve integrity. There are currently no pharmaceutical therapies approved by the FDA for the treatment of diabetic neuropathy. Sangamo expects to file an investigational new drug (IND) application in the second half of this year for a ZFP TF designed to upregulate the expression of VEGF-A for the treatment of DN.

This week, the first data were presented demonstrating the potential importance of ZFPs in improving cardiac function in congestive heart failure. Study results show that engineered ZFPs can successfully repress Phospholamban (PLN), a critical regulator of cardiac homeostasis and muscle contractility. Repression of PLN has been shown to improve cardiac contractile properties in animal models of congestive heart failure.

The study of the molecular mechanisms triggering neuropathic pain has identified several genes that are over-expressed in sensory neurons. Sangamo is using ZFP TFs to specifically repress the expression of three of these targets; the Vanilloid Receptor 1 (VR1), a non-selective cationic channel that responds to thermal, pH and capsacin stimulation; the Tyrosine kinase A receptor (TrkA), shown to be involved in chronic spinal cord injury, and the sodium channel Nav1.8 (PN3), expression of which has been found to be increased in patients with neuropathic pain symptoms. Data were presented from experiments with rat cells treated with ZFP TFs that showed a reduction of VR1 expression of 70-80% at the RNA level and 50% at the protein level. Similar data were demonstrated for TrkA.

In the area of stem cell research, Sangamo scientists presented data showing that either the up or down regulation of a single gene in mouse stem cells results in the production of differentiated cells with the intended characteristics. Demonstrating the control of stem cell fate provides an important foundation for applications in regenerative medicine.

Gene Correction

Sangamo is using ZFPs in combination with DNA cutting enzymes (endonucleases) to facilitate the correction of mutant or disease-causing gene sequences in cells. Data were presented this week on the Company's recent efforts in the areas of X-linked severe combined immunodeficiency (X-linked SCID) and sickle cell anemia (SCA). Both of these diseases are caused by a mutation in a single gene. In this study, results showed that using high affinity, highly-specific zinc finger nucleases (ZFNs), gene correction efficiencies can be obtained as high as 18% at an endogenous gene in a model cell system and efficiencies of greater than 1% have been obtained in CD34+ cells, the cell type that will be targeted in patients.

About Sangamo

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 http://www.sangamo.com/ or http://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, ZFNs, the therapeutic applications of Sangamo's ZFP technology platform and the filing of an IND application and initiation of clinical trials. 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.

Sangamo BioSciences, Inc.

CONTACT: Elizabeth Wolffe, Ph.D. Sangamo BioSciences, Inc.+1-510-970-6000, ext. 27, ewolffe@sangamo.com; Kathy Jones-Nugent, Ph.D.,media, or John Nugent, investors, both of Burns McClellan, Inc.,+1-212-213-0006, for Sangamo BioSciences, Inc.

Web site: http://www.sangamo.com/http://www.expressinglife.com/

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