Our Pioneering Approach
Rooted in a deep understanding of how genes are controlled, our approach represents a new and unexploited field for drug discovery and development. Targeted drug discovery to date has focused largely on inhibiting or otherwise changing the function of abnormal genes and proteins. But many more diseases could be treated by controlling the expression of both abnormal and normal genes. Until recently, however, scientists lacked the tools to study regulatory regions of the genome that control the expression of genes.
We are now at a pivotal point in time where a confluence of scientific and technological advances is bringing into view what was once beyond our reach. Building on the seminal work of our scientific founders, we have built the leading platform dedicated to systematically analyzing regulatory regions of the genome to develop small-molecule medicines that control the expression of genes critical to a given cell.