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Sangamo BioSciences Announces First Presentation Of Data From ZFP Therapeutic Program For Treatment Of Sickle Cell Disease And Beta-Thalassemia At American Society Of Hematology Meeting
Date:12/9/2013

RICHMOND, Calif., Dec. 9, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the presentation of preclinical data from its ZFP Therapeutic program for the treatment and potential cure of both sickle cell disease (SCD) and beta-thalassemia. The data demonstrate that Sangamo's approach, using its proprietary and highly specific zinc finger nuclease (ZFN) gene-editing technology, enables permanent increase in the expression of fetal gamma-globin in adult red blood cells (RBCs). This increase restores the normal balance of globin proteins that together form the oxygen-carrying hemoglobin of RBCs. In addition, the pre-clinical studies demonstrate that this can be accomplished at clinical-scale reproducibly achieving high levels (up to 80 percent) of gene editing in hematopoietic stem cells (HSCs).

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The study was carried out in collaboration with scientists in the laboratory of George Stamatoyannopoulos, M.D., Dr.Sci., Professor of Medicine and Genome Sciences at the University of Washington. The data were presented at the 55th Annual Meeting of the American Society of Hematology (ASH), which is being held in New Orleans.

"These preclinical data demonstrate that Sangamo's precise ZFN genome editing technology enables a unique approach that can be used to treat, and potentially cure, both sickle cell disease and beta-thalassemia," said Mark Walters, M.D., Director of Blood and Marrow Transplantation at Children's Hospital & Research Center Oakland, and a leader of one of the clinical teams that will be conducting the first Phase 1 clinical trial of this ZFP Therapeutic in transfusion-dependent beta thalassemia patients. 
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