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).
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 55 th 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. "The modification process is extremely efficient and scalable. We look forward to conducting a clinical study that employs Sangamo's technology in a patient's own stem cells to potentially provide a safer approach than current therapies, and eliminate the need for life-long medications and red blood cell transfusions that are currently the standard of care for these disorders."SCD and beta-thalassemia are genetic diseases of the blood caused by mutations in the beta-globin gene. In SCD, the gene defect in beta-globin results in an abnormal hemoglobin which causes the RBCs to develop a sickle, or crescent, shape. These abnormal RBCs are stiff and sticky and can block blood flow in the small blood vessels of the limbs and organs resulting in pain, organ damage and an increased risk of infection. The gene defect responsible for beta-thalassemia leads to impaired production of hemoglobin, the iron-containing protein in red blood cells (RBCs) that carry oxygen from the lungs to the tissues, and results in a failure to make healthy RBCs. Individuals with thalassemia are therefore dependent on blood transfusions for survival.