RICHMOND, Calif., Dec. 7, 2010 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today the presentation of preclinical data that demonstrate the ability to permanently correct a disease gene in an animal using systemic delivery of zinc finger nucleases (ZFNs). The presentation was one of four made by Sangamo's collaborators at the 52nd Annual Meeting of the American Society of Hematology (ASH), which is being held in Orlando, Florida.
Abst.# 647 "Manipulating Higher Order Chromatin Structure of the Beta-Globin Locus by Targeted Tethering of a 'looping' Factor" Oral Session: Thalassemia and Globin Gene Regulation: Regulation of Globin Gene Expression
These data demonstrate the use of zinc finger DNA-binding proteins (ZFPs) as a tool to dissect the role of the physical structure of a chromosome in gene regulation.
Abst.# 3764 "Editing Human Lymphocyte Specificity for Safe and Effective Adoptive Immunotherapy of Leukemia" Poster Session: Gene Therapy and Transfer: Poster II
The study describes a ZFN-based method for improving the potency, specificity and safety of T-cell immunotherapy approaches to cancer immunotherapy.
Abst.# 3766 "HLA and TCR Knockout by Zinc Finger Nucleases: Toward "off-the-Shelf" Allogeneic T-Cell Therapy for CD19+ Malignancies" Poster Session: Gene Therapy and Transfer: Poster II
These data provide proof of concept for the use of ZFN-based gene modification to develop 'off-the-shelf' cell-based immunotherapies to treat individuals of any HLA type.
Abst.# LBA-5 "Phenotypic Correction of a Mouse Model of Hemophilia B by In Vivo Genetic Correction of the F9 Gene" Oral Session: Late-Breaking Abstracts Session
"These data represent important progress in the advancement of systemic ZFN-based therapeutics towards clinical development," said Dale Ando, M.D, Sangamo's vice president of therapeutic development and chief medical officer. "Using a mouse model of hemophilia B, we have demonstrated successful functional correction of a human gene for the clotting factor, Factor IX, by direct systemic delivery into the animal rather than by delivery into cells ex vivo. Our ZFN approach, which enables permanent correction of the disease-related gene in situ, circumvents the problems of traditional gene-addition approaches that result in random insertion and the loss of normal regulation of the gene which may lead to malignancy or other unintended consequences."
Scientists demonstrated efficient ZFN-mediated correction of a defective human Factor IX gene in a mouse model of hemophilia B by delivering ZFNs and a corrected donor DNA sequence directly into the animals. Stable levels of protein made from the corrected human gene could be measured in the plasma of the treated animals and resulted in the restoration of normal rates of blood clotting for the period of the study. The study was carried out in the laboratory of Katherine High, M.D., Investigator, Howard Hughes Medical Institute, Professor of Pediatrics, University of Pennsylvania School of Medicine and Director, Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia, in collaboration with Sangamo scientists.
"These data represent a fundamental step forward and provide important proof of concept that ZFNs can be delivered efficiently in vivo and have potential therapeutic value for the treatment of human disease," stated Edward Lanphier, Sangamo's president and chief executive officer. "Our ZFN technology platform is broadly applicable and has been demonstrated to enable permanent gene modification, as in our CCR5 HIV clinical programs and in the studies presented this week at ASH. We believe that this technology, which can be applied to any disease-relevant gene, provides a unique and valuable therapeutic approach to the many human monogenic diseases that are currently classified as unmet medical needs."
Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic® development program is currently in a Phase 2b clinical trial for evaluation of safety and clinical effect in patients with diabetic neuropathy and a Phase 2 trial in ALS. Sangamo also has a Phase 1 / 2 clinical trial and two ongoing Phase 1 clinical trials to evaluate the safety and efficacy for the treatment of HIV/AIDS as well as a Phase 1 trial for the treatment for recurrent glioblastoma multiforme. Other therapeutic programs are focused on Parkinson's disease, monogenic diseases, neuropathic pain and nerve regeneration. 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 TFs) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for gene modification. Sangamo has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company's website at http://www.sangamo.com/.
ZFP Therapeutic® is a registered trademark of Sangamo BioSciences, Inc.
This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, the potential of ZFNs to treat human monogenic diseases, 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 the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's SEC filings, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.
SOURCE Sangamo BioSciences, Inc.