RICHMOND, Calif., Oct. 1 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. 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.”
About Sickle Cell Anemia and Sickle Cell Disease
Sickle cell anemia is a serious disease in which the body makes sickle-shaped red blood cells. This condition is caused by a mutation in the beta-globin gene which leads to the production of abnormal hemoglobin protein. Normal red blood cells are disc-shaped and move easily through blood vessels. Sickle-shaped cells are stiff and sticky and tend to form clumps and get stuck in the blood vessels blocking blood flow to the limbs and organs. Blocked blood vessels can cause pain, serious infections, and organ damage.
Many of the more than 70,000 people living with sickle cell disease in the United States - most of whom are African-American - face a lifetime of painful, debilitating and expensive health problems, with a much-shortened life expectancy. Sickle cell disease takes an even heavier toll in Africa, where approximately 200,000 babies are born with the disease each year. The only FDA-approved drug available for treating sickle cell disease is the anticancer drug hydroxyurea.
About the Doris Duke Charitable Foundation
The mission of the Doris Duke Charitable Foundation is to improve the quality of people’s lives through grants supporting the performing arts, environmental conservation, medical research and the prevention of child maltreatment, and through preservation of the cultural and environmental legacy of Doris Duke’s properties. Since 1998, the foundation’s Medical Research Program has committed approximately $185 million to strengthen and support clinical research, which advances the translation of basic biomedical discoveries into new treatments, preventions and cures for human diseases. To learn more about the program or to receive competition announcements, visit http://www.ddcf.org/mrp.
About Sangamo
Sangamo BioSciences, Inc. is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic(TM) development program is currently in Phase 2 clinical trials for evaluation of safety and clinical effect in patients with diabetic neuropathy and ALS. Sangamo also has two Phase 1 clinical trials to evaluate safety and clinical effect of a ZFP Therapeutic for the treatment of HIV/AIDS. Other therapeutic development programs are focused on cancer, neuropathic pain, nerve regeneration, Parkinson’s disease and monogenic diseases. Sangamo’s core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNA sequence Sangamo has created ZFP transcription factors (ZFP TF(TM)) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFN(TM)) for gene modification. Sangamo has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences, Sigma-Aldrich Corporation and several companies applying its ZFP technology to engineer cell lines for the production of protein pharmaceuticals. For more information about Sangamo, visit the company’s web site at www.sangamo.com.
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 sickle cell disease, strategic partnerships with collaborators and clinical trials of ZFP Therapeutics. Actual results may differ materially from these forward-looking statements due to a number of factors, including technological challenges, Sangamo’s ability to develop commercially viable products and technological developments by our competitors. See the company’s SEC filings, and in particular, the risk factors described in the company’s 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.
CONTACT: Elizabeth Wolffe, Ph.D., of Sangamo BioSciences, Inc.,
+1-510-970-6000, ext. 271, ewolffe@sangamo.com
Web site: http://www.sangamo.com/
