Our science
of clinical programmes fail
Dowden & Munro,
Nat Rev Drug Discov, 2019
Drug mechanisms with genetic support have 2.6 greater probability of success
Minikel et al., Nature, 2024
The problem
The success rate of clinical trials is notoriously low: 90% of all clinical programmes that are started fail, often because the new drug is not safe and effective. Open Targets was founded to change that.
What we do
Clinical trials backed by genetic data show higher probabilities of success. Through large-scale genetic and functional genomics experiments combined with deep understanding of real-world data from patients and the application of innovative computational techniques, we aim to systematically build a mechanistic understanding of target biology in context so that researchers can select the best targets for drug discovery.
We work pre-competitively to bridge the gap between pharmaceutical companies and not-for-profit research institutes, allowing the partners to apply their combined knowledge and expertise to our research projects.
Since its founding, Open Targets consortium has made significant contributions to understanding the relationships between targets and diseases, established leading informatics tools such as the Open Targets Platform, and contributed to global initiatives such as the Cancer Dependency Map.
Linking targets to diseases &
improving target selection for drug discovery
Our research programme generates and analyses data to connect targets to diseases, assesses the strength of
that evidence, and prioritises the most promising candidates for drug discovery.
We combine large-scale genomic experiments with high-throughput functional assays in the most relevant
cellular systems, working across therapeutic areas of common interest to our partners: Oncology,
Neurodegeneration, and Immunology & Inflammation with significant unmet need.
Our pre-publication data is initially shared within our partnership, but
ultimately, our results and data are shared openly with the broader scientific community and feed into the
Open Targets Platform where they can be analysed alongside other public data.
Target Identification
Discovering targets with significant associations to disease biology, using systematic approaches that assess candidates in a comprehensive genome-wide context.
Target Prioritisation
Refine candidate target lists based on the association evidence and additional translational and disease metrics, to focus effort on high quality drug discovery opportunities.
Showcase projects
Project Score (Cancer Dependency Map)
In an early consortium project an Open Targets team systematically disrupted every gene in over 300 cancer cell models, prioritising 628 potential drug targets. Notably, they nominated WRN as an essential gene in microsatellite unstable cancers, commonly seen in colon, ovarian, and endometrial cancers. This effort “Project Score” was made public by sharing through the Cancer Dependency Map project.
Read moreNeuroFlux
Using whole genome CRISPR screens in cells with autosomal lysosomal dysfunction, this project identifies targets which could reestablish normal levels of autophagy or lysosomal activity. Autophagy and lysosomal pathway dysregulation is a hallmark of dementias including Alzheimer' disease.
Read moreIBDverse
This project conducts single-cell analysis of Inflammatory Bowel Disease (IBD) tissues. The IBDverse project generated the largest single-cell datasets of IBD-relevant samples, spanning million of cells from hundreds of individuals, allow us to compare the effects of disease, location and genetics on cellular expression. This work was co-sponsored by the Crohns & Colitis Foundation.
Read moreTranslational informatics and data generation pipelines
Our translation informatics team brings together the data relevant to understand target-disease association so that drug discovery scientists can explore it systematically, simply and seamlessly. We cover many data types relevant to human disease biology and target identification, using expertise from Open Targets partners and beyond.
Open Targets Platform
Open Targets Platform brings to life our approach to target identification and prioritisation. This open source resource integrates public data relevant to the association between targets and diseases and provides additional analysis, data and tools for target prioritisation. The ongoing development and evolution of the Platform is a major consortium goal, bringing features, data and analysis to our community.
Launch Platform
Bioinformatics services and tools launched by Open Targets
Several databases and methods that are now central to bioinformatics research were launched through Open Targets projects. Some examples include:
eQTL Catalogue
The eQTL Catalogue aims to provide uniformly processed gene expression and splicing QTLs from all available public studies on human. This solves a major bottleneck in functional genomics: the lack of standardisation among scattered, heterogeneous public datasets needed to link GWAS loci to underlying causal genes.
Explore
Perturbation Catalogue
With the advent of new experimental technologies genetic perturbation data is rapidly expanding. The Perturbation Catalogue brings together data from multiple different types of genetic perturbation experiments, making it easier for researchers to study how modifying genes or proteins affects biological function across various biological and molecular contexts.
Explore
GWAS Catalog
A consistent, searchable, visualisable and freely available database of SNP-trait associations, and one of the largest repositories of full genome-wide summary statistics. The capability for researchers to deposit and then access summary statistics for GWAS was a joint project that Open Targets and the GWAS Catalog delivered.
Explore
ProtVar
To aid the mechanistic interpretation of genetic variation with respect to disease Open Targets worked with the Uniprot team to develop ProtVAR. This resource investigates SNV missense variation in humans by bringing together structural, functional, population, and disease annotations and sharing their evaluation with the community.
Explore