RICHMOND, Calif., May 24 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today that data from research and preclinical programs focused on the development of zinc finger DNA-binding protein (ZFP) Therapeutics were described in fifteen presentations given by Sangamo scientists and collaborators at the 13th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). The meeting was held in Washington, DC from May 17-22, 2010.
“Sangamo’s ZFP technology provides an exciting new approach for the development of novel human therapeutics,” said Luigi Naldini, M.D., Ph.D., Director of the San Raffaele Telethon Institute for Gene Therapy, Milan, and the principal investigator on several papers presented at the meeting. “ZFP nucleases (ZFNs) enable us to edit the human genome with unprecedented specificity and efficiency. This opens the way to correction of inherited mutations, a potentially revolutionary approach as it can restore a gene’s function while preserving its natural regulation, likely overcoming the risks associated with random gene addition approaches. We are using this technology to investigate novel therapies for monogenic diseases and cancer.”
The presentations made at the ASGCT meeting covered research and preclinical data from Sangamo’s programs and collaborations in human therapeutics and technology development in primary human cells and stem cells. Therapeutic areas included ZFN-based approaches to infectious diseases such as HIV/AIDS and cytomegalovirus (CMV), monogenic diseases such as sickle cell anemia and epidermolysis bullosa, and oncology. Positive preclinical data were also presented from experiments using Sangamo’s gene regulation technology to up-regulate the vascular endothelial growth factor-A (VEGF-A) gene in an animal model of traumatic brain injury. Philip Gregory, D. Phil., Sangamo’s vice president of research and chief scientific officer, made a presentation entitled “Stem Cell Modification with Zinc Finger Nucleases,” and chaired a session devoted to stem cell modification. All abstracts for the meeting are available online at http://www.asgct.org/am10/
“Sangamo’s technology has the potential to revolutionize the field of cell and gene therapy,” stated Barrie Carter, Ph.D., a member of Sangamo’s scientific advisory board and the current President of ASGCT. “ZFN technology enables an efficient and precise process for making changes to the DNA sequence of a cell, which is necessary for therapeutic applications of gene-editing. ZFN-based therapeutics are already being evaluated by Sangamo in human clinical trials in HIV/AIDS and brain cancer. As this technology functions at the DNA level, it can be applied to address numerous diseases for which target genes have been identified.”
“The data presented at this meeting highlight the breadth of potential applications for ZFP Therapeutics,” said Dale Ando, M.D., Sangamo’s vice president of therapeutic development and chief medical officer. “Our ZFP Therapeutic platform is maturing. We have completed four Phase 2 clinical trials, have an ongoing Phase 2b trial of our ZFP activator of VEGF-A, SB-509, in diabetic neuropathy and have three ongoing trials of our ZFN-based technology. At the same time, with our collaborators, we continue to define and develop new therapeutic opportunities.”
About Sangamo
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 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 two Phase 1 clinical trials to evaluate safety and clinical effect of a treatment for HIV/AIDS and another Phase 1 trial to evaluate safety and clinical effect of a treatment for recurrent glioblastoma multiforme. Other therapeutic development programs are focused on neuropathic pain, nerve regeneration, Parkinson’s disease 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) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFN) 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/.
This press release may contain forward-looking statements based on Sangamo’s current expectations. These forward-looking statements include, without limitation, the research and development of novel ZFP TFs and ZFNs as ZFP Therapeutics and therapeutic applications of Sangamo’s ZFP technology platform to specific human diseases. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to whether clinical trials will validate and support tolerability and efficacy of ZFP Therapeutic approaches, technological challenges, Sangamo’s ability to develop commercially viable products and technological developments by our competitors. See Sangamo’s SEC filings, and in particular, the risk factors described in the Company’s Annual Report on Form 10-K and 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.
