RICHMOND, Calif., May 24, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. (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.
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 Edward Lanphier, Sangamo's president and CEO. "The award will assist the development of our stem cell-based ZFP Therapeutic for the potential cure of thalassemia. Importantly, this same approach can be directly applied to related hemoglobinopathies such as sickle cell disease. We look forward to working with a team of world-renowned experts in this field, including the team at Children's Hospital & Research Center Oakland and our colleagues at CIRM, to bring this treatment through IND application and into a Phase 1 clinical trial."
Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and genome editing. The Company has ongoing Phase 2 clinical trials to evaluate the safety and efficacy of a novel ZFP Therapeutic® for the treatment of HIV/AIDS. Sangamo's other therapeutic programs are focused on monogenic diseases, including hemophilia, Huntington's disease and hemoglobinopathies such as beta-thalassemia and sickle cell anemia. Sangamo's core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). Engineering of ZFPs that recognize a specific DNA sequence enables the creation of sequence-specific ZFP Nucleases (ZFNs) for gene modification and ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function. Sangamo has entered into a strategic collaboration with Shire AG to develop therapeutics for hemophilia, Huntington's disease and other monogenic diseases and has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company's website at www.sangamo.com.
ZFP Therapeutic® is a registered trademark of Sangamo BioSciences, Inc.
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 SEC (SCUR) filings, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.
SOURCE Sangamo BioSciences, Inc.