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
"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
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.
Sangamo is taking a different approach. During development, a fetal form of hemoglobin is made. In infancy, it fully protects beta-thalassemia patients from developing disease symptoms. Later in childhood however, production of fetal hemoglobin ceases and is replaced by synthesis of adult-type beta-globin chains that are defective in beta-thalassemia patients. Sangamo's approach uses its proprietary ZFN genome-editing technology to enable the permanent production of therapeutic fetal hemoglobin to achieve normal levels of hemoglobin and RBCs, with the goal of eliminating, or greatly reducing, the need for chronic blood transfusions. Moreover, by performing this genome editing in HSCs isolated and returned to the same patient (so called autologous BMT), Sangamo's approach eliminates both the need for a matched donor and the risk of GvHD.
"CIRM support for this program is yet another major validation of our ZFP Therapeutics platform," said
ZFP Therapeutic® is a registered trademark of
This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references to the research and development of novel ZFP TFs and ZFNs and their applications in the treatment of beta thalassemia and sickle cell disease, receipt of funds from CIRM, partnerships with collaborators and clinical trials of ZFP Therapeutics in individuals with beta-thalassemia. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a
more detailed discussion of these and other risks, please see Sangamo's
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