A UK Hub to Catalyse Open Target Discovery.

Ageing societies and diseases of modern living require truly novel, efficacious and safe medicines. Increasing investment in R&D has not improved the efficiency of this endeavor. There is an urgent need for novel, validated drug targets. To facilitate this, the Wellcome Trust and other organisations have sponsored large-scale genome-wide association and sequencing studies in patients, identifying thousands of genes linked to human disease, some of which may become targets for pioneer drug disco very. However, this genetic output is not widely exploited because of the lack of data and research tools to prioritise targets. The SGC was created by the Wellcome Trust to systematically solve 3-D structures of novel human proteins, and subsequently to develop novel inhibitors for a subset of these proteins (epigenetic probes). The free availability of data and reagents has amplified its impact and stimulated research in industry and academic labs. This proposal addresses the bottleneck in translation of genetic associations to therapeutic strategies. We will build capacity and infrastructure to create early discovery tools (Target Enabling Packages) for genes nominated by genetics, disease biology and clinical experts. These tools, including protein structures, assays, and initial chemical matter, will provide much-needed links between genome sciences and target discovery.