Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today that research led by Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics (MIMG) and Pediatrics, the Director of the UCLA Human Gene Medicine Program and member of the Broad Stem Cell Research Center, and Philip Gregory, D. Phil., Sangamo's chief scientific officer and vice president, research, has been selected to receive a $486,000 Doris Duke Innovations in Clinical Research Award from the Doris Duke Charitable Foundation. The grant, which will be paid over three years, will support an innovative research project conducted by Dr. Kohn and Sangamo scientists and titled "Beta-globin Gene Correction in Hematopoietic Stem Cells for Sickle Cell Disease."
"ZFN-mediated gene editing is an exciting and now proven technology with the potential to provide a solution for sickle cell anemia and other hemoglobinopathies," said Dr. Kohn. "Despite the fact that sickle-cell anemia was the first genetic disorder for which a molecular cause was identified and one in which every patient has the same gene mutation, the lack of treatment options makes the disease a hugely important problem worldwide. This novel approach will develop the use of efficient ZFN-mediated correction of the sickle mutation in the human beta-globin gene in stem cells from patients with sickle cell disease rather than traditional approaches of gene replacement therapy."
The Doris Duke Innovations in Clinical Research Award (ICRA) provides seed funding for early stage, multi-disciplinary clinical research projects. The 2009 ICRA grants will support a range of approaches to improving the health of patients with sickle cell disease. The research proposed by Dr. Kohn and Sangamo is one of five projects selected to receive grants from the foundation out of a total of 81 applications from 52 teams of investigators for the 2009 ICRA competition.
"We are very pleased that our research proposal was chosen for this prestigious award," commented Dr. Philip Gregory. "Dr. Kohn is a pioneer in the development of hematopoietic stem cell therapies for monogenic diseases and we look forward to continuing our collaboration to develop a novel stem cell therapeutic approach to sickle cell disease."
Sangamo BioSciences, Inc.