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Open Targets Platform is a free, openly licensed resource that integrates public datasets to build and score links between drug targets (genes and proteins) and diseases, so researchers can prioritise targets for further study. It is run by the Open Targets partnership and is available as a web interface, an API and bulk downloads. This page covers what it holds, how its data may be reused and how to cite it, as read from the Platform’s own documentation on 2026-11-08.
What Open Targets is
Open Targets is a public-private partnership, active since 2014, whose stated aim is to give researchers a single starting point for building therapeutic hypotheses. Its main product is the Open Targets Platform, which the documentation describes as a tool that supports systematic identification and prioritisation of potential therapeutic drug targets. The Platform annotates targets, diseases and phenotypes, drugs, variants, studies and credible sets, and calculates target-disease association scores from the evidence it integrates.
The word “Open” refers to access and reuse: the Platform is free to use, its pipeline and infrastructure code is open source, and its data carry an open licence (see below).
Who runs it and what it covers
The Open Targets website lists seven partners: EMBL-EBI, Genentech, GSK, MSD, Pfizer, Sanofi and the Wellcome Sanger Institute. The Platform’s 2025 publication in Nucleic Acids Research acknowledges a slightly different set of supporting organisations and Wellcome Trust funding, so cite the partner list to the page you took it from.
According to that publication, the Platform integrates 23 public evidence sources spanning germline and somatic variation, perturbation (including CRISPR screens such as Project Score and CRISPRbrain), pathways, known drugs and the literature. Further details it reports:
- Literature mining covers roughly 440 million entities drawn from more than 14.7 million publications.
- Pharmacogenetics data from PharmGKB are included, and direction-of-effect annotation is provided for eight evidence sources.
- Data from Open Targets Genetics, the post-GWAS resource, have been incorporated into the Platform, including FinnGen freeze 6 and GTEx v8 splicing QTLs.
- Gene burden results added 2,584 gene-disease associations.
Releases are quarterly, according to the Platform documentation.
How to access and use it
The documentation names three access routes. This page does not reproduce click-by-click steps because the documentation does not publish them as a numbered procedure.
- Web interface. Search for a target or disease in the Platform at platform.opentargets.org and browse the associated evidence.
- API. A GraphQL API, released under the Apache 2.0 licence, for programmatic queries.
- Data downloads. Bulk datasets for local analysis; the 2025 paper also reports a listing as an Azure Open Dataset.
Two interface features introduced in the 2025 paper are worth knowing about. Associations on the Fly lets users re-weight evidence sources, exclude known drugs, pin targets and upload their own target lists. The Target Prioritisation view shows a traffic-light heatmap across clinical precedence, tractability, doability and safety. The paper also reports literature summaries generated with GPT-4o-mini and links to ProtVar. Questions go to the Open Targets Community forum.
Licence and reuse
For research administrators and data managers the reuse terms matter as much as the content. The licence page of the documentation sets out three layers:
- Platform data: CC0 1.0, a public-domain dedication (compare the Creative Commons licence family).
- Platform code, including the GraphQL API: Apache 2.0.
- Underlying sources: each integrated source keeps its own licence, listed in a table in the documentation. Examples given there include ChEMBL (CC BY-SA 3.0), GTEx (CC BY 4.0) and gnomAD (CC0 1.0).
The documentation states that the source providers agreed to unrestricted use of their data through the Platform. If you redistribute a derived dataset, check the per-source table so that any attribution or share-alike conditions of individual sources are met.
How to cite Open Targets
The citation page asks users to cite the latest Platform publication: Buniello A. et al. (2025), “Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery”, Nucleic Acids Research 53(D1), D1467, available at academic.oup.com/nar/article/53/D1/D1467/7917960. Because the Platform is updated every quarter, also record the release version you queried in your methods section so the analysis can be reproduced.
Strengths and limits
Strengths
- Free access, open data licence and open-source code, which simplifies compliance with funder open-data expectations.
- Evidence from genetics, perturbation screens, pathways, known drugs and literature in one place, with three access routes.
- Transparent scoring that users can re-weight in the interface.
Limits
- Editorial interpretation: association scores are a way to rank and organise evidence for hypothesis generation; they are not clinical validation of a target.
- Quarterly release cycle: results can change between releases, so version your queries.
- Licence heterogeneity: CC0 for the Platform does not remove the conditions of individual sources.
Related resources
Open Targets integrates several resources that are also useful on their own. The pages below are CASRAI guides to some of them; they are complementary rather than direct substitutes.
| Resource | Relationship to Open Targets | CASRAI page |
|---|---|---|
| ChEMBL | One of the integrated sources (CC BY-SA 3.0 per the Platform licence table) | ChEMBL |
| PharmGKB (ClinPGx) | Pharmacogenetics data integrated into the Platform | PharmGKB |
| GWAS Catalog | Separate genetics resource; the Platform incorporates post-GWAS data from Open Targets Genetics | GWAS Catalog guide |
| DrugBank | Separate drug knowledge resource | DrugBank guide |
| DepMap | Separate cancer dependency resource | DepMap guide |
For repositioning work that starts from known drugs, see also drug repurposing.
FAQ
What is Open Targets?
A public-private partnership, active since 2014, that runs the Open Targets Platform, a free resource for identifying and prioritising potential drug targets by integrating public evidence on target-disease links.
Is Open Targets free to use?
Yes. The documentation describes the Platform as free, with data under CC0 1.0 and code under Apache 2.0. Individual underlying sources keep their own licences.
What happened to Open Targets Genetics?
The 2025 Platform paper reports that Open Targets Genetics post-GWAS data have been incorporated into the Platform.
Can I download the data?
Yes. Bulk data downloads are one of the three documented access routes, alongside the web interface and the API.
How often is it updated?
The documentation says the Platform is actively maintained with quarterly updates.
Sources
All read 2026-11-08.
