Drug discovery faces significant challenges — 90% of drugs entering Phase 1 clinical trials never reach the market. Open Targets is a public-private partnership that integrates human genetics and genomics data to systematically address these challenges through the Open Targets Platform (platform.opentargets.org).

The Platform integrates data from over 20 diverse sources to build and score gene-disease associations, supporting evidence-based target prioritisation for drug discovery. This year, the Platform underwent a major expansion, incorporating the most comprehensive set of genetic associations to date and requiring significant advances in both data infrastructure and user-facing interfaces.

This update includes ancestry-specific fine-mapping and colocalisation of GWAS and molecular QTL data from the GWAS Catalog, eQTL Catalogue, FinnGen, and UK Biobank Pharma Proteomics Project. Over 3.5 million credible sets were generated, yielding more than 1 million gene-disease associations supported by over 3 million evidence items.

To surface this complexity, new interactive interfaces were designed and built: variant-centric pages display credible sets, colocalisation results, and clinical and pharmacogenomic annotations for over 7.8 million variants. New study and credible set pages allow researchers to explore GWAS and QTL evidence in detail. Enhanced machine learning–based Locus-to-Gene (L2G) assignments further refine gene prioritisation, identifying more than 1 million credible sets with L2G score >0.5.

These frontend-driven enhancements unify evidence from common and rare variant studies within a single explorable interface, enabling researchers to build stronger causal links between genes and diseases and supporting data-driven drug target identification.

Co-authors: Polina Rusina, Ellen M McDonagh, David G Hulcoop, Yakov Tsepilov, Szymon Szyszkowski, Xiangyu Jack Ge, Daniel Considine, Wei Wen Vivien Ho, Tobi Alegbe, Annalisa Buniello, Ricardo Esteban Martinez Osorio, James D Hayhurst, Graham McNeill, Irene Lopez, Helena Cornu, Javier Ferrer, David Ochoa, Daniel Suveges