Strategic Partnership Award from California Institute for Regenerative Medicine
SGMO receives $6.4 Million Strategic Partnership Award From California Institute for Regenerative Medicine (CIRM) to develop ZFP Therapeutic® for Beta-thalassemia funding for IND application and clinical trial of curative approach on the application of its zinc finger nuclease (ZFN) gene-editing technology in hematopoietic stem cells (HSCs).
The 4 year grant provides matching funds for preclinical work that will support an IND application and a P1 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.
SGMO 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. SGMO's approach enables 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), SGMO's approach eliminates both the need for a matched donor and the risk of GvHD.
The Bottom Line: Adding to our coverage list as SGMO's 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. the persistence of fetal hemoglobin beyond the newborn stage mitigates the severity of these hemoglobin disorders. 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."
SGMO closed on 5/23/13 at $7.43 on a low volume <450> day and had traded as high as $10.80 on 5/3/13. This is definitely an actionable event and I project a $0.10 to $0.20 jump for SGMO even in a DOWN and vacation weekend market on the non-dilutive CIRM funding - it might even spill over into Tuesday. The 50 day moving average is $9.28 followed by the 200 at a low of $8.10.
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 (allogeneicBMT) is curative.
Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.