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Sangamo BioSciences Announces Presentation of New Data from ZFP Therapeutic® Program in Hemophilia B at American Society for Hematology Meeting
Date:12/13/2011

ZFN-mediated repair of the human Factor IX gene, substantially expanding the potential of the approach.

The study was conducted 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.

In a second study presented at the meeting, ZFN gene disruption was used to generate a next generation cancer immunotherapy by enhancing the targeted killing activity and safety profile of the product.

Abst. No. 667 - TCR Gene Editing Results in Effective Immunotherapy of Leukemia without the Development of GvHD (Oral Session: 801)

Cancer immunotherapy uses CD8 T-cells that have been engineered to express high avidity T-cell receptor (TCR) genes isolated from tumor-specific lymphocytes.  The engineered CD8 T-cells are then able to attack the tumor. Problems can arise with this approach because the expression of the CD8 T-cell's own TCR gene interferes with expression of the inserted tumor-specific TCR gene.  This interaction limits the potency of this cellular therapy but, more importantly, it can also make the cells "self-reactive" leading to graft versus host disease (GvHD).

In this study, ZFNs were used to disrupt the native TCR genes in these tumor-directed CD8 T-cells resulting in an enhanced immunotherapeutic product with potent cancer cell-killing activity and the elimination of GvHD in a mouse model.  These studies were performed in the laboratory of Chiara Bonini, M.D., Head of the Experimental Hematology Unit, San Raffaele Hospital, Milan, in collaboration with Luigi Naldini, Head of TIGET, San Raffaele Hospital, and Sangamo scientists.

"We continue to develop our ZFP Therapeutic pipeline and, on the strength of our success in mouse models, have advanced ou
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SOURCE Sangamo BioSciences, Inc.
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