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Sangamo BioSciences Announces Data Presentation of First In Vivo Demonstration of ZFN-mediated Gene Correction Via Systemic Delivery at American Society for Hematology Meeting
Date:12/7/2010

ent," 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
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SOURCE Sangamo BioSciences, Inc.
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