A pioneering public-private research initiative between GlaxoSmithKline, the European Bioinformatics Institute (EMBL-EBI) and the Wellcome Trust Sanger Institute has formed to harness the power of “big data” and genome sequencing to improve the success rate for discovering new medicines. The new Center for Therapeutic Target Validation (CTTV) will aim to address a wide range of human diseases and will share its data openly in the interests of accelerating drug discovery.

The CTTV aims to use the almost daily advances in cutting-edge genetic research to help researchers in that crucial first step in exploring new medicines—finding where to start. Target validation is about clearly defining the role that a biological process plays in disease before developing a new drug to tackle it. Currently, an estimated 90% of compounds entering clinical trials fail to demonstrate the necessary efficacy and safety requirements, never reaching patients as medicines. This often is because the biological target for a drug is not well understood.

Ewan Birney, associate director and senior scientist at EMBL-EBI, has been appointed interim head of the CTTV. Birney, who brings knowledge about genomics and bioinformatics, will develop a work program to steer the research activities of the center’s scientists.

“The Center for Therapeutic Target Validation is a transformative collaboration to improve the process of discovering new medicines,” said Birney. “The pre-competitive nature of the center is critical: the collaboration of EMBL-EBI and the Sanger Institute with GSK allows us to make the most of commercial R&D practice, but the data and information will be available to everyone.”

CTTV scientists will combine their experience to explore and interpret large volumes of data from genomics, proteomics, chemistry and disease biology. The new approach will complement existing methods of target validation, including analysis of published research on known biological processes, preclinical animal modeling and disease epidemiology.

This new collaboration draws on the diverse, specialized skills from scientific institutes and the pharmaceutical industry. Scientists from the Wellcome Trust Sanger Institute will contribute their understanding of the role of genetics in health and disease, and EMBL-EBI, a global provider focused on the analysis and dissemination of biological data, will provide bioinformatics-led insights on the data and integrate huge streams of different varieties of experimental data. GSK will contribute experience in disease biology, translational medicine and drug discovery.

A cornerstone of the collaboration is an agreement among the collaborators that sequence data and information gathered within the CTTV will be shared to benefit the broader scientific community, after basic quality control checks to ensure consistency with the data-sharing guidelines of both institutes. The center also will seek publication of data and information arising from CTTV projects in peer-reviewed scientific journals. Once the center is fully established, the collaborators will actively seek to attract new interest from other companies and academic institutions in the center with the aim of expanding its activities.

The CTTV will be supported by up to 50 researchers drawn from the three founding organizations and will be based on the Wellcome Trust Genome Campus near Cambridge, U.K. Researchers will use EMBL-EBI’s Innovation and Translation suite, which received substantial support from the U.K. government in 2012, and the laboratories of the Wellcome Trust Sanger Institute. The CTTV has been established with significant contributions of resource, skills and platform technologies from each of the three founding organizations and a multi-million dollar contribution by GSK to fund an initial wave of projects.

Patrick Vallance, president of pharmaceuticals R&D at GSK, said, “Target validation is one of the greatest challenges in drug discovery. We need to understand better the mechanisms in our body related to disease to improve how we can develop the most effective medicines. By changing our business model, taking a more open-minded approach to sharing information and forging collaborations like the Center for Therapeutic Target Validation, we believe there is an opportunity to accelerate the development of innovative new medicines.”

Professor Sir Mike Stratton, director of the Wellcome Trust Sanger Institute, said, “Advances in genomics have led to a rapid increase in the availability of drug targets, providing enormous opportunity but also posing the problem of how we best convert this new knowledge into medicines. The challenge we now address is to identify those new targets with the greatest relevance to human disease which, in turn, will undoubtedly increase the speed and efficiency in which new medicines can be developed.”