Our Gene Control Platform
Ours is the leading platform dedicated to the regulatory genome to systematically identify disease-causing alterations in gene expression and create medicines that control the expression of genes with the aim of providing a profound benefit for patients with diseases that have largely eluded other genomics-based approaches. While much of targeted drug discovery has focused on inhibiting or otherwise changing the function of abnormal genes and proteins, many more diseases could be addressed by modulating the expression of both abnormal and normal genes, turning them on, off, up or down. Our unique ability to elucidate regulatory regions of the genome allows us to home in on which genes to control, in which cells, for which patients, and how best to modulate the expression of those genes to maximize the chances of providing a profound benefit for patients.