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				Sangamo BioSciences Introduces ZFP-Mediated Gene Correction as a New Therapeutic Platform at American Society of Gene Therapy Meeting RICHMOND, Cailf., June 9 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) today announced the presentation of preliminary data from their new therapeutic initiative -- the development of their zinc finger DNA binding protein (ZFP) technology for the treatment and potential cure of human diseases caused by genetic defects. Sangamo scientists have demonstrated the ability to use ZFPs to facilitate the "correction" of a mutant DNA sequence within a therapeutically relevant gene laying the technical foundation to address monogenic human diseases. These data were presented on Saturday, June 7 at the 6th Annual Meeting of the American Society of Gene Therapy. “We are very excited about our newest ZFP Therapeutic program -- ZFP-Mediated Gene Correction,” said Edward Lanphier, Sangamo’s president and chief executive officer. “Using this approach we may be able to permanently correct a mutated gene sequence and provide normal gene function. This technology could be used to treat and potentially cure a number of genetic diseases including severe combined immunodeficiency, sickle cell anemia, Gaucher's disease, hemophilia and potentially many others.” “We are moving rapidly to develop our ZFP-Mediated Gene Correction platform,” added Tyler Martin M.D., Sangamo’s vice president of development. “The potential benefits for patients with monogenic disease are enormous. These patients have an extreme need for effective therapeutics and have often been neglected in the past. We are working very hard to rapidly develop this technology so that these benefits can be realized.” Sangamo scientists design engineered ZFPs that recognize and bind to a specific DNA target sequence within a gene. By linking these ZFPs to the functional domain of a restriction enzyme such as Fok I, very precise cuts can be made in the target sequence. Cutting the DNA sequence of a gene close to the site of a mutation facilitates permanent replacement of the stretch of DNA containing the mutation with a corrected version of the sequence by a process known as homologous recombination. It has previously been shown that the generation of a double strand break in DNA by a restriction endonuclease increases the rate of homologous recombination. The ability to use this knowledge for correcting endogenous genes with genetic errors has previously been limited by the inability to cut the DNA at the specific site of the mutation. Sangamo’s ZFP technology provides the necessary tools for precisely targeting the functional domain of a restriction endonuclease to any therapeutically relevant genetic mutation. “Gene correction is an excellent application of our ZFP technology and provides new clinical opportunities while leveraging our existing core competency,” commented Dr. Carl O. Pabo, Sangamo’s senior vice president and chief scientific officer. “This technical approach is consistent with the same basic principles we have always employed -- the ability to engineer a ZFP that will specifically bind to a chosen DNA sequence and target a functional domain, in this case a restriction endonuclease or DNA cutting enzyme, to that exact sequence. These new results are very exciting and may eventually lead to a treatment and cure of many human genetic disorders.” Sangamo is the exclusive worldwide licensee of several patents related to ZFP-linked restriction enzymes (US Patent No. 5,436,150; European Patent 682,699) and to methods for targeted DNA cleavage (US Patent No. 6,265,196). In the presentation, authored by Sangamo scientists Michael Holmes, Jeff Miller, Andrew Jamieson, Ya-Li Lee, Sheldon Augustus, Fyodor Urnov, and Carl Pabo and titled “Targeting In Vivo Gene Correction Using Designed DNA-Binding Proteins” data are shown from both in vitro and cellular systems that are being used to optimize this technique for use in therapeutic applications. About Sangamo Sangamo BioSciences, Inc., of Richmond, CA, is focused on the research and development of novel transcription factors for the regulation of gene expression. The company’s most advanced therapeutic development program involves the use of transcription factors for the treatment of coronary artery disease and peripheral arterial disease. Other therapeutic development programs are focused on cancer, neuropathic pain, ophthalmic and infectious diseases. Sangamo’s proprietary technology enables the engineering of transcription factors known as zinc finger DNA-binding proteins, or ZFPs. By engineering ZFPs so that they can recognize a specific gene, Sangamo has created ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function. The company is developing ZFP TFs as a fundamentally enabling technology for commercial applications in human therapeutics, pharmaceutical discovery and plant agriculture. For more information about Sangamo, visit the company’s web site at 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 applications of Sangamo’s ZFP TF Therapeutic programs. 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 their most recent 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release. SOURCE Sangamo BioSciences, Inc. 06/09/2003 CONTACT: Elizabeth Wolffe, Ph.D. of Sangamo BioSciences, Inc., +1-510-970-6000, ext. 271, ewolffe@sangamo.com Web site: http://www.sangamo.com (SGMO) back to top
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