RICHMOND, Calif., May 24, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced that the California Institute for Regenerative Medicine (CIRM) has granted the Company a $6.4 million Strategic Partnership Award to develop a potentially curative ZFP Therapeutic for beta-thalassemia based on the application of its zinc finger nuclease (ZFN) gene-editing technology in hematopoietic stem cells (HSCs).
The four year grant provides matching funds for preclinical work that will support an Investigational New Drug (IND) application and a Phase 1 clinical trial in transfusion-dependent beta-thalassemia patients. The grant application entitled "A Treatment for Beta-thalassemia via High Efficiency Targeted Genome Editing of Hematopoietic Stem Cells" won the highest scientific score and was the only application recommended for funding in this round of CIRM's Strategic Partnership Awards.
"Sangamo's powerful and precise ZFN-genome editing technology enables modification of a patient's own stem cells and potentially provides a safer approach to current therapies for hemoglobinopathies such as beta-thalassemia and sickle cell disease," said Mark Walters, M.D., Director of Blood and Marrow Transplantation at Children's Hospital & Research Center Oakland and a member of the clinical team that will be conducting the Phase 1 clinical trial of this ZFP Therapeutic. "We have known for some time that the persistence of fetal hemoglobin beyond the newborn stage mitigates the severity of these hemoglobin disorders. We are very pleased to have the opportunity to develop a clinical protocol that uses Sangamo's technology to permanently raise fetal hemoglobin levels in red blood cells to a sufficient degree to have a strong beneficial effect. If successful, this could eliminate the need for life-long medications and red blood cell transfusions that are currently the standard of care for these disorders."
Beta-thalassemia is a genetic disease of the blood caused by mutations in the beta-globin gene. This gene defect leads to impaired production of hemoglobin, the iron-containing protein in red blood cells (RBCs) that carry oxygen from the lungs to the tissues. Individuals with thalassemia are dependent on blood transfusions for survival as they fail to make sufficient healthy RBCs. The unmet medical need in transfusion-dependent beta-thalassemia is significant, with reduced life expectancy due to multi-organ failure caused by iron overload, blood-borne infections and other disease complications. A bone marrow transplant (BMT) of HSCs from a "matched" related donor (allogeneic BMT) is curative. However, this therapy is limited due to the scarcity of matched donors and the significant risk of graft-versus-host disease (GvHD) after transplantation of the foreign cells.