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European Patent Office Grants 2nd Charpentier/Doudna Patent Covering CRISPR-Cas9 Gene Regulation ApplicationsERS Genomics announced today that the European Patent Office (EPO) has granted Dr. Emmanuelle Charpentier, ERS Genomics' co-founder, together with the University of California and University of Vienna, its second EU patent with very broad claims covering the use of CRISPR-Cas9 technology for gene regulation. The claims are directed to compositions and uses of a chimeric version of the Cas9 protein, most often associated with use in regulation of gene expression as opposed to direct editing of the genetic code itself. The patent covers uses in both cellular and non-cellular settings, including use in bacteria, plants, animals, and cells from vertebrate animals such as humans. "Going beyond gene editing, this second patent addresses yet another set of important applications of the CRISPR technology, which are becoming widely used in new drug discovery efforts and other areas of research," commented Eric Rhodes, CEO of ERS Genomics. "This form of CRISPR is broadly enabling as it allows researchers to selectively repress or activate genes of interest to determine their function. It is thenewest tool being used to discern the role of genes in disease, ultimately leading to new therapeutic applications." The most popular embodiments of this form of the CRISPR technology are known as CRISPR-i (inhibitory CRISPR) and CRISPR-a (activating CRISPR). Both make use of a modified form of the Cas9 protein in which its cutting function is inactivated while leaving its targeting capability intact. This modified form of Cas9 can then be linked to any number of protein subdomains that have the ability to impair or enhance the expression of the gene to which the Cas9 complex targets them. The approach is widely used in combination with large libraries of single guide RNAs (sgRNAS) and known as 'sgRNA screening' to identify genes involved in particular cell processes, including their role in causing disease.
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