Thousands of gene variants linked to human disease sit on a shelf, unused, because no one has built the tools to turn them into drug targets. This matters because the current system for discovering new medicines is broken. Despite rising investment, the number of effective new drugs has not kept pace. Large genetic studies have identified thousands of genes connected to diseases, but researchers lack the basic resources—protein structures, chemical probes, and assays—to decide which genes are worth pursuing as drug targets. The SGC has already shown that making such tools freely available accelerates research in both academia and industry. If this hub succeeds, it will create "Target Enabling Packages" for genes nominated by geneticists and clinicians. These packages—including protein structures and initial chemical compounds—will provide the missing link between genome science and drug discovery. The impact will be felt in the early-stage research infrastructure that quietly determines which drug targets get pursued, potentially shortening the path from genetic discovery to clinical trials.
View original technical description
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.
Plain English summaries and category classifications on this site are generated by AI and may not perfectly reflect the original research.
Is something wrong? Let us know