RICHMOND, Calif., Sept. 29 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. announced today that the company has been granted research funding from the Cystic Fibrosis Foundation as part of an award obtained by collaborator Dr. Eric Sorscher, M.D., Professor in the Departments of Medicine and Physiology and Biophysics at the University of Alabama at Birmingham (UAB) and Director of the Gregory Fleming James Cystic Fibrosis Research Center. Sangamo will receive research funding over a period of 2 years to generate important cell lines for cystic fibrosis (CF) research using its zinc finger DNA-binding protein (ZFP) technology.
"The cystic fibrosis research community could benefit enormously from the technology pioneered by Sangamo BioSciences," noted Dr. Sorscher. "Currently there is a paucity of cell lines that provide appropriate expression of the mutant protein (deltaF508 CFTR) that is the cause of the majority of cases of CF. Sangamo's ZFN-mediated gene modification technology is the best method to specifically engineer human cells so that they make deltaF508 CFTR from the native promoter. These new lines will be important in the study of the biology of the CF defect and for drug discovery."
Research scientists frequently use cultured cell lines to learn about protein function. In the proposed research funded by CFF, Sangamo scientists, in collaboration with CF investigators in a national consortium, will use ZFP nuclease (ZFN) technology to introduce the deltaF508 mutation into the CFTR gene in well-characterized cell lines. These new cell lines would express the mutant CFTR from the cell's own promoter, providing a new resource that will be invaluable to the CF scientific community at large.
"The Cystic Fibrosis Foundation has sought the best and the brightest scientists to join in its mission of improving the length and quality of life for people with CF while also pursuing the cure," noted Robert J. Beall, Ph.D., President and CEO, Cystic Fibrosis Foundation. "Funding research, such as this project undertaken by Sangamo, and facilitating the collaboration of academia and industry, are significant steps along this path."
"We welcome the opportunity to use our technology to assist The Cystic Fibrosis Foundation in their quest for resources to aid research that will benefit patients suffering from CF," said Edward Lanphier, chief executive officer of Sangamo. "We believe our technology is uniquely suited to generate cell lines that will facilitate the search for a cure for cystic fibrosis and may also be used to develop novel therapeutics to treat patients suffering from other genetic diseases."
About Cystic Fibrosis
The deltaF508 mutation in the CFTR (cystic fibrosis transmembrane conductance regulator) gene is responsible for approximately 90% of cystic fibrosis cases. This defective gene causes the body to produce abnormally sticky mucus that clogs the lungs and leads to life-threatening lung infections. These thick secretions also obstruct the pancreas, preventing digestive enzymes from reaching the intestines to help break down and absorb food. The predicted survival of persons with CF in the United States is 35 years.
People with CF have a variety of symptoms including: very salty-tasting skin; persistent coughing, at times with phlegm; wheezing or shortness of breath; an excessive appetite but poor weight gain; and greasy, bulky stools. CF is a genetic disease affecting approximately 30,000 children and adults in the United States or in approximately one of every 3,500 live births, which translates to about 1,000 new cases of CF each year. More than 80 percent of patients are diagnosed by age three; however, nearly 10 percent of newly diagnosed cases are age 18 or older.
About Sangamo BioSciences, Inc.
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 programs are currently in Phase I clinical trials for evaluation of safety in patients with diabetic neuropathy and peripheral artery disease. Other therapeutic development programs are focused on macular degeneration, ischemic heart disease, congestive heart failure, neuropathic pain, and infectious 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 therapeutic gene modification as a treatment for a variety of monogenic diseases, such as sickle cell anemia, and for infectious diseases, such as HIV. 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 therapeutic applications of Sangamo's ZFP technology platform. 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 10-Q. Sangamo BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.
Sangamo BioSciences, Inc.
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