American company Sangamo BioSciences, Inc. has announced the presentation of preclinical data from its ZFP Therapeutic programme 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).The study 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.
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 conducting the first Phase 1 clinical trial of the ZFP Therapeutic in transfusion-dependent beta, said: “These preclinical data demonstrate that Sangamo’s precise Edward Lanphier, Sangamo’s president and CEO., said ”These data demonstrate the versatility, specificity and high level of efficiency of Sangamo’s ZFP genome-editing technology.”
ZFN genome editing technology enables a unique approach that can be used to treat, and potentially cure, both sickle cell disease and beta-thalassemia. SCD and beta-thalassemia are genetic diseases of the blood caused by mutations in the beta-globin gene.