Sangamo BioSciences, Inc. To Present Data From Its ZFP Therapeutic(TM) Programs At The 9th Annual Meeting Of The American Society of Gene Therapy
BALTIMORE, June 1 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. today announced presentation of data from several of the Company's zinc finger DNA-binding protein (ZFP) Therapeutic(TM) programs at the 9th Annual Meeting of the American Society of Gene Therapy (ASGT). The meeting is being held in Baltimore, Maryland from May 31 through June 4, 2006.
Sangamo scientists and their collaborators will make a total of 9 podium and poster presentations covering several ZFP-mediated gene regulation and gene modification therapeutic programs. In addition, Sangamo collaborator Angelo Lombardo, from the San Raffaele Telethon Institute for Gene Therapy, Milan, Italy, was recognized with an Excellence in Research Award for his work described in an abstract entitled, "Towards Gene Correction of X-Linked SCID Using Engineered Zinc Finger Nucleases and Integrase Defective Lentiviral Delivery".
"The annual ASGT meeting provides an outstanding forum for sharing the latest developments in the field of gene therapy and, as in previous years, Sangamo has a significant presence," stated Edward Lanphier, Sangamo's president and CEO. "The breadth of material that we are presenting ranging from specific therapeutic programs to our internal research programs to refine and optimize our technology illustrates the potential broad utility of our science."
Philip Gregory, D.Phil., Vice President, Research at Sangamo noted, "We are presenting data for the first time on our process development progress in our ZFP Therapeutic program to disrupt the CCR5 gene for the potential treatment of HIV/AIDS. In a prize-winning abstract, our collaborators from the San Raffaele Telethon Institute in Milan will present data on the successful use of non-integrating vectors for delivery of our ZFNs for gene correction of X-linked SCID. In addition to updates across our ZFP Therapeutic programs in gene regulation, gene correction and gene disruption, several of our presentations are focused on technical advances and development of our proprietary ZFP nuclease (ZFN(TM)) technology. One presentation describes the use of our ZFN technology in targeted integration, which we believe has the potential to improve the safety, efficiency and utility of gene-based approaches for both therapeutic and enabling technology applications."
Sangamo scientists and their collaborators will make the following presentations; the abstract numbers, titles and a brief description of the data to be presented are listed below:
ZFP TF-mediated gene regulation -- Abstract #788 Engineered Zinc Finger Protein Transcription Factors as a Potential Therapy for Choriodal Neovascularization Saturday, June 3, 2006. Oral Presentation. Preclinical animal data will be presented from Sangamo's program to develop ZFP TFs for the potential treatment of age-related macular degeneration. -- Abstract #1010 Developing a Potential Pain Therapy Using Engineered Zinc Finger Protein Repressors of the Sodium Channel PN3 Saturday, June 3, 2006. Poster Presentation. Data will be presented that demonstrate the successful use of ZFP TFs to repress the expression of a well-validated pain receptor in primary neuronal cells. ZFN-mediated gene modification -- Abstract # 555 Large-Scale, Flow-Based Electroporation To Deliver Engineered Zinc Finger Protein Nucleases That Mediate High-Efficiency Disruption of the Human CCR5 Gene Friday, June 2, 2006. Poster Presentation. Data will be presented demonstrating the efficient delivery of plasmid to cells using a GMP compliant flow-based electroporation system. -- Abstract # 738 Towards Gene Correction of X-Linked SCID Using Engineered Zinc Finger Nucleases and Integrase Defective Lentiviral Delivery Saturday, June 3, 2006. Oral Presentation. -- Abstract # 758 Towards Gene Knock out Therapy for AIDS/HIV: Targeted Disruption of CCR5 Using Engineered Zinc Finger Protein Nucleases (ZFNs) Saturday, June 3, 2006. Oral Presentation. -- Abstract # 787 Engineered Fok I Heterodimers for Enhanced Zinc Finger Nuclease Specificity Saturday, June 3, 2006. Oral Presentation. Refinements of ZFN engineering will be presented that are designed to enhance the specificity of action of ZFNs. -- Abstract # 1003 Targeted Site-Specific Integration in Human Cells Using Designed Zinc Finger Nucleases Saturday, June 3, 2006. Poster Presentation. Data will be presented demonstrating the application of ZFNs for efficient targeted integration of DNA sequences. -- Abstract # 1040 Towards Gene Correction Therapy for Wiskott-Aldrich Syndrome Using Designed Zinc Finger Endonucleases Saturday, June 3, 2006. Poster Presentation. ZFP Applications -- Abstract # 387 Advantageous Properties of the piggyBac Transposon System for Gene Transfer in Human Cells Friday, June 2, 2006. Oral Presentation. 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 modification. In the case of therapeutic gene regulation, ZFP transcription factors (ZFP TFs) are being engineered to either turn on therapeutically beneficial genes or turn off the expression of disease-causing genes. For gene modification, ZFPs are being used in combination with a DNA cutting enzyme (endonuclease) functional domain to generate ZFNs that facilitate the correction of mutant gene sequences that cause disease or the disruption of genes that facilitate disease progression.
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 programs are currently in Phase 1 clinical trials for evaluation of safety in patients with diabetic neuropathy and peripheral artery disease. Other therapeutic development programs are focused on macular degeneration, ischemic heart disease, congestive heart failure, neuropathic pain, 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 sickle cell anemia, 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. Research at Sangamo is partially funded by an Advanced Technology Program (ATP) grant awarded by the National Institute of Standards and Technology (NIST). 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 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.Sangamo BioSciences, Inc.
CONTACT: Elizabeth Wolffe, Ph.D. of Sangamo BioSciences, Inc.,+1-510-970-6000, ext. 271, or firstname.lastname@example.org; or media, Justin Jacksonof Burns McClellan, Inc., +1-212-213-0006, or email@example.com, forSangamo BioSciences, Inc.
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