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."
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