THE HAGUE, The Netherlands, Sept. 23 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO - News) announced today that Dr. Casey Case, Sangamo's vice president of research, will present data on a novel approach for the generation of cell lines for high throughput small molecule screening. The cell lines, employing Sangamo's proprietary zinc finger DNA binding protein transcription factor (ZFP TF) technology, are engineered to upregulate the expression of endogenous genes of therapeutic importance thereby providing a "workaround" solution to cDNA patents. The data are being presented at the 8th Annual Meeting of the Society for Biomolecular Screening.
A basic tenet of patent law is that genes and proteins cannot be patented as they occur "in nature", within the context of the cell or organism. A patent can only be granted if the gene or protein has been isolated so that it can be used outside its original context i.e. if the gene has been cloned or the protein purified. Endogenous genes, therefore, cannot be patented. Sangamo's ability to engineer ZFP TFs that can activate or repress an endogenous chromosomal gene does not use isolated or purified cDNA of the gene target.
Small molecule screening is used by the pharmaceutical industry to rapidly test large libraries of chemical compounds for their effects on cellular proteins that have been identified to have a role in a disease process. This type of assay enables the efficient identification of viable drug candidates. A common step in screening efforts is to overexpress the target gene by using a cDNA encoding that gene in a cell-line of interest. This approach has become more complicated in recent years as the cDNAs for more and more genes have been patented. In the application developed by Sangamo scientists, cell lines can be engineered using gene-specific ZFP TFs that will effect overexpression of proteins that have been implicated in specific diseases. By employing customized cell lines in which the expression of an endogenous gene of interest and thus its protein is upregulated by a ZFP TF, pharmaceutical researchers are able to more quickly assess and quantify the impact of various chemical compounds on that target without infringing a cDNA patent on that gene.
"The intellectual property surrounding a large number of well-validated gene targets has prevented many companies from pursuing drug development on these clinically important genes. We believe our ability to regulate endogenous genes is ideally suited to provide pharmaceutical companies with a viable workaround to these types of patents," said Edward Lanphier, Sangamo's president and chief executive officer. "The data that we are presenting, using a G-protein coupled receptor as an example, demonstrate that our engineered ZFP TFs very effectively activate endogenous genes and that ZFP TF cell lines function well under actual screening conditions. Moreover, this approach can be applied to any gene target in any cell type."... |
