Sangamo BioSciences Announces Presentation of New Data from ZFP Therapeutic® Program in Hemophilia B
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Sangamo BioSciences, Inc. announced the presentation of new data demonstrating the ability to permanently correct a disease gene in an adult mouse model of hemophilia B using systemic delivery of zinc finger nucleases (ZFNs) at the 53nd Annual Meeting of the American Society of Hematology (ASH).
"We have demonstrated functional correction of a human gene for the clotting factor, Factor IX, with a single, systemic administration of ZFNs in an animal model of disease," said Geoff Nichol, M.B, Ch.B., Sangamo's executive vice president of research and development. "Our approach enables permanent correction of the genetic defect responsible for hemophilia B. This circumvents the problems of traditional gene-addition approaches that uncouple the gene from its normal regulatory mechanism and which may result in gene silencing and random gene insertion and potentially lead to malignancy or other unintended consequences."
