Sangamo Presents Data From ZFP Therapeutic Program for Treatment Of Sickle Cell Disease And Beta-Thalassemia at ASH

Sangamo BioSciences

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).#ASH13