RICHMOND, Calif., July 21, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the publication of a study demonstrating the use of zinc finger nucleases (ZFNs) to produce genetically modified pigs. The study, which was published in the Proceedings of the National Academy of Sciences (PNAS), represents a significant advancement in the development of improved, less immunogenic animal tissue as a source for transplant into humans.
Transplantation is considered to be the best treatment option for thousands of patients every year whose own organs are damaged or diseased. However, the supply of human organ and tissues available for transplantation is insufficient to satisfy the demand.
"This work is a major advance because it provides an efficient method of knocking out any desired gene in the pig. ZFN-mediated genome editing can be used to make porcine cells and tissues less immunogenic and more suitable for transplantation into humans," said Richard Insel, M.D., Chief Scientific Officer for the Juvenile Diabetes Research Foundation (JDRF) who was not associated with the study. "In the example of type 1 diabetes, pancreatic islets can be replaced by transplanting functioning ones into a patient. However, we face a severe shortage of human cadaver organs in the U.S. and cannot satisfy the transplantation needs of people with the disease. Using ZFN-modified pigs as a source of tissues for human transplantation may prove to be a promising solution to the shortage of donated human organs for diabetes and other diseases."
As part of their mission to ultimately find a cure for type 1 diabetes, JDRF has helped pioneer islet cell transplantation research since the 1970s.
The paper, entitled "Efficient Generation of a Biallelic Knockout in Pigs Using Zinc-Finger Nucleases," describes a general method for the generation of animals in which a chosen gene is specifically removed or "knocked out" from the pig's genome. In the published example, Sangamo scientists and their collaborators in the laboratory of Prof. Dr. Heiner Niemann of the Institute of Farm Animal Genetics in Germany, knocked out both copies of the 1,3-galactosyl transferase (GGTA-1) gene from pig cells and used these to produce cloned animals that lack the target gene. Knockout of the GGTA-1 gene has been shown to lead to significantly improved organ survival in a pig-to-baboon organ transplantation model.
"Sangamo's mission is to develop novel ZFP Therapeutics to address unmet medical needs and make paradigm-shifting therapeutic solutions a reality," stated Edward Lanphier, Sangamo's president and chief executive officer. "This proof of concept study lays the foundation for the use of our validated ZFP platform technology to modify animal organs for human transplantation."
Efficient Generation of a Biallelic Knockout in Pigs using Zinc-Finger Nucleases.
Hauschild J, Petersen B, Santiago Y, Queisser AL, Carnwath JW, Lucas-Hahn A, Zhang L, Meng X, Gregory PD, Schwinzer R, Cost GJ, Niemann H.
Proc Natl Acad Sci U S A. 2011 108 (29) 12013-12017.
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. 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 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 enable the generation of tissues and organs for xenotransplantation, the therapeutic potential of ZFP technology to address unmet medical needs, 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 assumes no obligation to update the forward-looking information contained in this press release.
|SOURCE Sangamo BioSciences, Inc.|
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