Therapeutic target discovery and validation programme & the systems microscopy initiative

The discovery and development of new drugs for human diseases is a major scientific priority worldwide, but the process is beset with challenges that make it difficult, expensive and protracted. Our work exploits advances in our understanding of the molecular circuits controlling normal and diseased cells to identify and validate new targets for the next generation of medicines. We are also developing new methods for microscopy to better understand the effect of new medicines on diseased cells.

There is a very high rate of attrition in the development of new classes of therapeutic agents that exploit our growing knowledge of the genetic basis of diseases like cancer, presenting a major impediment for academia and industry. Our ability to rapidly identify and validate of the best new therapeutic targets for drug development remains a a key challenge. To address this problem, we aim to build on the success of our recent work to develop novel approaches to systematically scrutinize cellular pathways altered in disease using large libraries encompassing diverse protein folds, and second, to extend using fragment-based and library-driven methods the discovery of chemical leads against novel molecular targets implicated in cellular regulation. In addition, our ongoing work to develop new approaches in light microscopy, such as hyper-dimensional imaging microscopy, to deconvolute complex cellular pathways will serve not only to advance understanding of disease-related derangements of these pathways, but also to validate diagnostic or therapeutic concepts for further development.