Open access platform accelerates drug discovery and development


A web interface to help researchers find therapeutic targets for new and repurposed medicines is now available from the Centre for Therapeutic Target Validation

The Centre for Therapeutic Target Validation (CTTV) has launched a new web platform for life-science researchers that helps them to identify therapeutic targets for new and repurposed medicines.

Establishing the validity of an association between a disease and biological target for a drug can be extremely challenging and expensive. To address this challenge, the CTTV, a collaboration between GSK, the Wellcome Trust Sanger Institute and the European Bioinformatics Institute (EMBL-EBI), has created a web-based platform ( that draws on public data resources to help drug discovery.

This state-of-the-art web interface will allow researchers to access thousands of target profiles summarising the evidence for the involvement of a specific gene product with a disease. ‘The huge growth in human genetic and genomic data has transformed research in a very short space of time, but there is a gap between basic and translational research,’ explains Jeff Barrett, Director of the CTTV. ‘We have learned so much about the links between genes, tissue function and disease, and we need solid partnerships such as the CTTV to make sure that knowledge makes it to the people who make medicines. The Target Validation platform is all about enabling communities to work together, making the hand-off from basic research to drug discovery smoother.’

The Target Validation Platform provides a single, robust infrastructure that integrates high-level information from key sources of evidence covering common and rare disease genetics, somatic mutations in cancer, tissue and cellular expression patterns, information mined from published scientific literature, approved drugs, reaction pathways and animal models. Co-designed with biologists, it is easy to search by target, disease or therapeutic area.

‘Our ability to identify and validate effective targets can mean the difference between developing a new medicine successfully or wasting valuable time and resources pursuing dead ends,’ says Lon Cardon, Senior Vice President of GSK R&D. ‘The collaborative work under way at the Centre for Therapeutic Target Validation provides a real opportunity for us to make a profound impact on attrition and, even more importantly, to tackle unexpected pathways for new medicine. The launch of this new open access platform is a major step in how we’ll truly open up the discoveries made through this collaboration to benefit the broader scientific community.’

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‘We worked very closely with biologists working in different environments to make sure the platform would be both useful and intuitive,’ says Ian Dunham, Scientific Director of the CTTV. ‘We’ve now launched a service that provides evidence for more than 21,800 targets, with more than 8800 structured disease and clinical phenotype terms – but we expect those numbers to grow substantially as we integrate CTTV experimental project data.’