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Open Targets @opentargets.org · 01/10/2026

Out now in Drug Discovery Today! A team at Open Targets review pathway-driven target prioritisation in drug discovery, and present a practical framework to combine pathway evidence with genomic data in early-stage prioritisation www.sciencedirect.com/science/arti...

Between the Drug Discovery Today and the Open Targets logos, a reference reads: Rusina, P. et al. 2026, Pathway-driven target prioritisation in drug discovery.

Above, a figure from the review shows pathway analysis across evidence types. Each evidence type addresses a characteristic question. Pathway analysis yields ranked candidate hypotheses rather than conclusions, and target validation is required before any of the applications shown can be supported. Applications are illustrative rather than exclusive: several evidence types bear on each.

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Open Targets @opentargets.org · 28/09/2026

The Open Targets Platform autumn (26.09) release is out now! 🍂 It's packed with genetics updates, with: 🧬 1.24 million new molecular QTL credible sets 🧬 2 new sources of gene burden data 🧬 Updates from the GWAS Catalog 🧬 Genetic constraint data for X- and Y-linked genes through gnomAD and more!

26.09 release graphic highlights some of the major updates, including 1.24 million more molecular QTL credible sets, from GTEx v10, MAGE, and IBDverse, through the eQTL Catalogue. Genes & Health and BRaVa as new sources of gene burden. 14,690 new studies from the GWAS Catalog. Genetic constraing for X- and Y- linked genes through gnomAD v4.1.1. A Platform metrics page.

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Open Targets @opentargets.org · 12/08/2026

Ambitious science can start in the most unglamorous places. In 2015, gastroenterologist Tim Raine and geneticist Carl Anderson had a casual conversation in an underground carpark, which led to the largest single-cell dataset of Inflammatory Bowel Disease (IBD)-relevant tissues

Two men smile at the camera while posing in front of the entrance to an underground car park on a sunny day.

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Open Targets @opentargets.org · 04/08/2026

6 years 646 patients 1193 samples 100 endoscopists 9 lab scientists 3 postdocs 2 PhD students 502 courier runs from Addenbrooke’s Hospital to @sangerinstitute.bsky.social IBDverse is the largest single-cell dataset of Inflammatory Bowel Disease (IBD)—and generating the data for it was no mean feat

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Open Targets @opentargets.org · 07/07/2026

The team used over 1.6 million variants in the Open Targets Platform to generate over 148,000 dose-response-like gene-phenotype relationships. The genes were then ranked for their therapeutic potential, identifying many potential new targets.

Image caption: Overview of the VIDRA framework.

(a) Data inputs. VIDRA integrates five complementary sources of genetic evidence: expression quantitative trait loci (eQTLs), protein quantitative trait loci, coding GWAS variants, AstraZeneca PheWAS variants, and ClinVar variants, together with gene-level rare variant burden tests.

(b) Variant effect estimation. Each variant contributes a point to a dose-response model, where y represents disease effect and x represents either QTL effect size or a protein function score. Gene-level burden tests provide anchoring information for loss-of-function effects. Evidence is integrated using a hierarchical Bayesian model to estimate a gene–phenotype slope.

(c) Outputs. For each gene–phenotype pair, VIDRA produces a posterior slope estimate, a posterior probability quantifying confidence in a non-zero slope, a Therapeutic Potential Score derived from a machine-learning classifier trained on approved drug targets, and a predicted direction of therapeutic modulation inferred from the slope sign.

(d) Interpretation of slope direction. The VIDRA slope captures the relationship between protein activity and disease risk across the spectrum from loss-of-function (LoF) to gain-of-function (GoF). Positive slopes indicate that increased protein activity is associated with increased disease risk, whereas negative slopes indicate that increased protein activity is protective. Directionality is inferred from the combined genetic evidence and does not require explicit GoF or LoF annotation.

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Open Targets @opentargets.org · 30/06/2026

“The scale at which we’re doing things, and in such an unbiased way, means that anything could come out of it. You have to be open to a new theory or something completely out of the ordinary happening.” Lewis Evans is @sangerinstitute.bsky.social Senior Staff Scientist and Open Targets project lead

A man gestures at a scientific poster on a wall of posters, pointing something out to the woman standing next to him.

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Open Targets @opentargets.org · 30/06/2026

The Open Targets Platform 26.06 release is out now! ✋ 🔴 Find out about all the updates on the blog: blog.opentargets.org/open-targets...

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Open Targets @opentargets.org · 23/06/2026

“There isn’t anywhere else in the world where you’ve got genomics, computational, clinical, biological, and pharmaceutical expertise all in one group or consortium.” @mhaniffa.bsky.social discusses her experience working with partners across industry and academia as part of her Open Targets project

Next to a photo of Muzlifah Haniffa, Deputy director and head of the cellular genomics prorgamme at the Wellcome Sanger Institute, a quote reads: working through Open Targets has allowed us to partner with exactly the right people, without constraints. This kind of collaboration creates a real sense that we can accelerate progress towards clinical translation—moving faster and more effectively thanks to the breadth of expertise from many different disciplines.

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Open Targets @opentargets.org · 09/06/2026

“Open Targets has had the single biggest impact in integrating the @sangerinstitute.bsky.social and @ebi.embl.org science, above and beyond any other initiative I’ve seen in that time." 🤝

22% of active Open Targets projects involve collaborations between EMBL-EBI and the Wellcome Sanger Institute. The accompanying graphic shows shaking hands as if making a business deal, with one hand in EBI green and one in Sanger blue.

52% collaborate across academic teams within their institute. The accompanying graphic shows shaking hands, with the sleeves either two shades of EBI green or two shades of Sanger blue.

100% collaborate with Open Targets industry partners. The accompanying graphic shows shaking hands on a yellow background representing that all of them involve industry partners.

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Open Targets @opentargets.org · 01/06/2026

We're at #ESHG2026! Come say hello and find out about current Open Targets projects. See you there!

Under a banner with the ESHG 2026 graphic, a list of presenters, title sessions, and timings.

Gosia Trynka, Chair, S01. Hunting for effector genes from GWAS studies, June 13, 8:30

Chair, C17. In QTLs We Trust: Linking Genotype to Function, June 14, 10:30

Chair, S11. High-throughput perturbation methods to study disease, June 14, 14:00

Chair, S12. Blood as a window: genetic insights into health, aging, and immune function, June 15, 08:30

Tobi Alegbe, Chair, C17. In QTLs We Trust: Linking Genotype to Function, June 14, 10:30

Under a banner with the ESHG 2026 graphic, a list of presenters, title sessions, and timings.

Haerin Jang, P16.032.B Single cell eQTL mapping of immune response regulation in systemic lupus erythematosus patients, Poster viewing with authors June 13, 16:00

Yakov Tsepilov, C32.6 Integration of 100,526 public GWAS defines pervasive pleiotropy and informs drug-target selection, June 15, 11:45

Ellen McDonagh, E06.2 Understanding rare diseases with digital twins, June 15, 17:45

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Open Targets @opentargets.org · 27/05/2026

Introducing OpenSplice, an atlas of splice-altering variants and the largest systematic analysis of splicing regulatory variants OpenSplice quantifies the impact of 590,000+ variants on the splicing of 608 alternatively spliced exons, and is out now on bioRxiv 🧬🖥️ www.biorxiv.org/content/10.6...

Figure 2 from the publication: Heatmap of ΔPSI values for all 585 most completely characterized exons (>50% designed variants recovered), ordered by wild-type (WT) PSI (left color bar).

Underneath is the reference to the paper, between the bioRxiv and Open Targets logos: Quarantani, G., et al. (2026) OpenSplice: the impact of half a million mutations on the alternative splicing of 600 human exons

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Open Targets @opentargets.org · 06/05/2026

Our findings from the largest systematic analysis of complex trait associations is out now on bioRxiv! 🧬🖥️ www.biorxiv.org/content/10.6...

Pleiotropy map of disease-associated genes arranged by chromosome around the outer ring, showing the number of unique diseases and therapy areas associated with the gene.

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Open Targets @opentargets.org · 30/03/2026

Tripso is able to learn multiple representations for each cell, each corresponding to a pre-defined gene programme This enables us to interpret cell states going beyond canonical cell type markers and annotations, and pick out biologically interesting differences in experimental conditions

Figure describing self-supervised learning of gene programs using Tripso. The first section details the base model for gene representation, then gene programme representation, and finally a global model for cell representation. Tokenized scRNA-seq count data are used as input to a transformer model, which produces contextualized gene embeddings at single-cell resolution. Curated GPs are initialized from public databases. GP-specific transformers learn latent representations for each GP based on their constituent genes. A global cell block integrates GP representations to learn a unified cell-level embedding. Optionally, an additional GP discovery transformer is defined using highly variable genes or a user-specified gene set to infer data-driven GPs.

The next section describes applications and downstream tasks, for example GP-centric representation learning, optimal transport in GP latent space to map populations across conditions, Gene-GP importance analysis, GP importance scores across groups, and identifying novel condition-specific GP. Conditions may represent health and disease, in vivo and in vitro, cell types, age groups...

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Open Targets @opentargets.org · 23/03/2026

The Open Targets Platform spring (26.03) release is out now! 🌸 Find all the details of the updates on the blog: blog.opentargets.org/open-targets...

26.03 Release highlights from the Open Targets Platform. Not covered by the next photos: update to the official Open Targets MCP.

E2G predictive features added to L2G. Context: the ENCORE-rE2G model integrates molecular features and large-scale perturbations to predict and score links between variants and transcriptional regulatory elements in a given cell. In this release, to add regulatory context to our Locus-to-Gene (L2G) machine learning model, we have used the scores to add two features: e2gMean, and e2gNeighbourhoodMean. There is a corresponding new column in the L2G Shapley visualisations on the Platform.

New data from the GWAS Catalog. In this release, we have 710 new studies from 97 publications, resulting in over 5,000 new credible sets, including the biggest study for hypothyroidism we have ingested. Also, we have updated String and Intact, as well as ClinVar and PGx through the European Variation Archive.

Find all the details of this release on the Open Targets blog: blog.opentargets.org

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Open Targets @opentargets.org · 12/03/2026

Introducing PerturbGen: a generative AI foundation model that predicts how genetic perturbations affect cellular trajectories www.biorxiv.org/content/10.6...

Screenshot of the HSPC perturbation atlas, showing genes clustered by gene programme. Clicking on one brings up predicted perturbation results for that gene.

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Open Targets @opentargets.org · 12/03/2026

They then undertook a genome-wide Perturb-seq screen targeting all expressed coding genes in the same cell type, to map the regulatory programmes of these targets.

Map of regulatory cascades as determined with TAP-seq and Perturb-seq. The top row shows 85 enhancer-like CREs with significant target genes, the second row shows TAP-seq tested genes, and the third and fourth rows show genes involved at Perturb-seq. Lines between the layers represent significant connections.

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Open Targets @opentargets.org · 03/03/2026

In cooperative game theory, Shapley values help determine how to distribute rewards among players who contribute unequally to a team’s success By implementing Shapley value-based explanations for L2G scores in the Platform, we provide a measure of which features contribute most to a given score

Locus-to-Gene widget in the Open Targets Platform showing the Shapley values for features contributing to the prioritisation of two genes in a credible set.

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Open Targets @opentargets.org · 03/03/2026

What does game theory have to do with gene predictions? Irene Lopez explains how we apply Shapley value-based explanations for Locus-to-Gene scores in the Open Targets Platform, addressing a common concern of machine learning models: explainability 🧬🖥️ blog.opentargets.org/how-we-impro...

A poker player lifts the corner of their cards on a table strewn with chips and playing cards, revealing a pair of kings.

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Open Targets @opentargets.org · 02/02/2026

Open Targets Platform data is now available on @amazonwebservices.bsky.social through the Open Data Program! registry.opendata.aws/opentargets/

Where to access Open Targets Platform data?
Web interface, API and API playgrounds, Data downloads, Google BigQuery public dataset, Microsoft Azure Open Dataset, and new: AWS Open Data

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Open Targets @opentargets.org · 27/01/2026

The Perturbation Catalogue is live! 🧬🔎🖥️ It aims to bring genetic perturbation data into one curated, harmonised, and discoverable platform. Take a look! www.ebi.ac.uk/perturbation...

Perturbation Catalogue home page showing key metrics

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Open Targets @opentargets.org · 12/01/2026

Introducing the official Open Targets Platform Model Context Protocol 🧬🖥️ In partnership with @anthropic.com, we’ve created an MCP server making Open Targets Platform data accessible to AI-powered tools and agents Try it out and watch out for future releases: mcp.platform.opentargets.org/mcp

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Open Targets @opentargets.org · 18/12/2025

The Wellcome Genome Campus lit up for the holidays ✨ 📸 Helena Cornu

A brightly lit Christmas tree at night, with a building in the background. Pathways leading to the building are illuminated with lights dotted on each side.

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Open Targets @opentargets.org · 08/12/2025

#OnThisDay 10 years ago, we launched the Open Targets Platform! 🎂 🧬🖥️

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Open Targets @opentargets.org · 24/11/2025

The recently-announced Open Psychiatry Project is the first initiative to jointly analyse UK mental health research data Findings from this project will be integrated into the Open Targets Platform to help identify and prioritise the most promising therapeutic targets.

Quote accompanying a photo of Ellen McDonagh, Open Targets Translational Informatics Director and EMBL-EBI co-lead for the Open Psychiatry Project: The Open Psychiatry Project will translate the power of omics data into identifying potential new treatments for mental health conditions. This is the first major initiative to bring together mental health data for multiple stakeholders in an accessible way, and analysing this information collectively is a key step towards finding new targets for drug development.

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Open Targets @opentargets.org · 23/10/2025

And that's the Open Targets Hackathon done! 🧬🖥️ Thank you to everyone who joined in person and online, and who contributed to make this event a success Particular thanks to our sponsors: Gates Foundation, Google Cloud, and @thehyvenl.bsky.social 🎉

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Open Targets @opentargets.org · 22/10/2025

Open Targets Hackathon Day 1! Following our official kick off yesterday, there was some incredible energy and focus in the room Thanks to our sponsors Gates Foundation, Google Cloud, and @thehyvenl.bsky.social for making this possible!

People are sat in groups, working at laptops arranged. They are engaged in individual and small group work. The room looks busy, with whiteboards covered in writing along the back wall.

In a busy meeting room, participants are seated at white tables working on laptops, working independently or in groups.

Three people are engaged in conversation while looking at a whiteboard covered with diagrams and notes. A blue banner or bunting is visible above.

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Open Targets @opentargets.org · 21/10/2025

The Open Targets Hackathon is today! 🎉 We're super excited to kick off this event celebrating 10 years of the Open Targets Platform Thank you to Google Cloud for sponsoring our compute and Hariprasad Radhakrishnan for mentoring our teams and sharing this video — made with Google Veo 3! 🖥️🧬

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Open Targets @opentargets.org · 16/10/2025

At #ASHG25 and want to find out more about the largest and most comprehensive systematic analysis of common variation in humans? Go find Daniel at poster board 1014T!

A man stands beside a poster board labelled 1014

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Open Targets @opentargets.org · 14/10/2025

We're at #ASHG2025! Come say hello and find out more about Open Targets projects 👋

Find us at ASHG 2025. The graphic lists speakers in the Featured Symposium, beyond transcriptomics: can imaging bridge the gap in variant interpretation, and poster presenters.

The two speakers are Gosia Trynka and Helena Kilpinen. The four poster presenters are: Catherine Sutherland, Haerin Jang, Annalisa Buniello, and Daniel Considine.

Four Open

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Open Targets @opentargets.org · 03/10/2025

CLOSING: Postdoc in @francescoiorio.bsky.social's lab at @humantechnopole.bsky.social Apply through LinkedIn by 5 October: www.linkedin.com/jobs/view/43... #scienceJobs #AcademicSky #postdocJobs

Closing job ad for a postdoc in CRISPR meta-analytics and AI, from Open Targets and the Human Technopole. The role is in the Iorio Lab, salary up to 40k euros, apply by 5 October.

The description reads: The Perturbation Catalogue project aims to create an online repository of data from post-perturbational experiments. You will focus on a meta-analytics project, developing computational methods for the reproducibility assessment, harmonisation and analysis or inter-study CRISPR datasets, aggregating data and developing and using innovative AI-based methods for therapeutic target prioritisation pipelines.

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Open Targets @opentargets.org · 02/10/2025

They found an interesting variant in the USP18 locus: a trans-eQTL that increases the expression of 50 interferon response genes. This variant also colocalises with a GWAS signal for systemic lupus erythematosus (SLE)

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Open Targets @opentargets.org · 18/09/2025

This new data can provide insights about the function of genetic variants by linking them to likely regulatory elements that affect the transcriptional activity of nearby genes, and is available to browse on our variant pages

An example of an intervals widget for a variant in the Open Targets Platform

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Open Targets @opentargets.org · 04/09/2025

Last call to submit a project for the Open Targets Hackathon! Submit a project by 12 September: docs.google.com/forms/d/e/1F... We will prioritise projects where the project leads have signed up to attend the hackathon. Registrations are currently full but you can sign up to the waiting list

Open Targets Hackathon: one week left to submit a project

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Open Targets @opentargets.org · 31/07/2025

Google Cloud is sponsoring the compute for the Open Targets hackathon! There is now a waiting list to attend the event in person, but project submissions and online registrations are still open. The two-day hackathon will explore data, analyses, and tools for target discovery

Text reads: Open Targets hackathon, 21-22 October 2025, compute sponsored by Google Cloud

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Open Targets @opentargets.org · 29/07/2025

Meet Carlos Cruz! Carlos joined the Open Targets Platform team in 2021 🖥️🧬 @carcruz.dev, what is your role in Open Targets?

Carlos Cruz smiles at the camera from a bridge over a small river in a European town on a cloudy day.

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Open Targets @opentargets.org · 10/07/2025

We have now deprecated Open Targets Genetics — it has been superseded by the Open Targets Platform The team said goodbye to Open Targets Genetics, through the medium of memes Enjoy!

Meme: Jeremy Clarkson and Richard Hammond comparing two cars. In the first image, Jeremy points to one labelled "Open Targets Genetics" saying, This is brilliant. In the second image, he points to another labelled "Merged Open Targets Platform" saying, "But I like this".

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Open Targets @opentargets.org · 09/07/2025

Open Targets Genetics has now been deprecated It has been superseded by the Open Targets Platform

Comic with four squares. Death, a skull in a black cloak, carrying a scythe, takes Open Targets Genetics by the hand, saying "It's time to go". Open Targets Genetics asks, "Was I a good platform?", and Death says "yes. They leave together, side by side.

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Open Targets @opentargets.org · 08/07/2025

We also undertake an all vs all study comparison, allowing you to view new studies in the context of all previously established GWAS and QTL information GWAS and eQTL colocalisations for these credible sets further support the link between ESR1 and osteoporosis Have you explored the data?

GWAS colocalisation metrics for overlapping sets from GWAS studies for one of the credible sets linking ESR1 to osteoporosis

MolQTL colocalisation metrics for overlapping sets from GWAS studies for one of the credible sets linking ESR1 to osteoporosis

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Open Targets @opentargets.org · 08/07/2025

@anuragverma.bsky.social, Huffman, Rodriguez, Conery, Liu et al. (2024) conducted Genome Wide Association Studies with over 635,000 participants, identifying more than 26,000 variant-trait associations across 1270 traits

Screenshot of the publication in Science, titled: Diversity and scale, genetic architecture of 2068 traits in the VA Million Veteran Program

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Open Targets @opentargets.org · 08/07/2025

🧬 The latest Open Targets Platform release includes the first genetic evidence for ESR1 in osteoporosis ESR1 is a known target for osteoporosis, with approved drugs such as Estradiol, but this is the first time we have genetic evidence for this association platform.opentargets.org/disease/EFO_...

Osteoporosis associations page on the Open Targets Platform showing GWAS associations evidence for ESR1

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Open Targets @opentargets.org · 02/07/2025

Hackathon registrations are now open! www.opentargets.org/hackathon Join us in person or online on October 21-22, 2025 to explore data, analyses, and tools for target discovery. Know someone who might be interested? Send this to them or tag them below!

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Open Targets @opentargets.org · 01/07/2025

Out now in iScience! @itreg.bsky.social in Annie Speak's lab created an in vitro model of T cell exhaustion to help identify and target new genes relevant to improving the immune response against cancer cells www.cell.com/iscience/ful...

Graphical abstract for the publication. The top section illustrates the in vitro model of T cell exhaustion, in which naive CD8 T cells OT-I are chronically stimulated with their cognate antigen— OVA peptide. This is followed by temporal phenotypic characterisation. This model recapitulates many critical hallmarks of exhaustion, including expression of canonical surface markers—ROS, PD1, CD39, 2B4 and LAG3— impaired proliferation, reduced cytokine production, decreased cytotoxic granule release, and metabolic alterations. The second section shows the identification of a gene signature of exhaustion between in vitro, mouse, and human TILs. Two in vivo models validate these results and establish a gene signature shared by in vitro and in vivo exhausted states. Critically, this signature is observed in tumour infiltrating T cells from multiple human tumour types, validating the translational potential of this model for discovering therapies.

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Open Targets @opentargets.org · 26/06/2025

Preprint out today! A team led by @tobioinformatics.bsky.social and Bradley Harris in @carlanderson.bsky.social ‘s lab has created the largest single-cell atlas of IBD tissues to date www.medrxiv.org/content/10.1...

UMAP of the 9 populations and 86 cell types identified after quality control and clustering

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Open Targets @opentargets.org · 24/06/2025

A rundown of the new features and data in the Open Targets Platform 25.06 release! What are you most excited about?

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Open Targets @opentargets.org · 15/05/2025

Open Targets Genetics will be deprecated on 9 July 2025 You will no longer be able to access genetics.opentargets.org. An archive of the data will be accessible through the EMBL-EBI FTP We are happy to help with questions or concerns on the Open Targets Community: community.opentargets.org

Text reads: Announcement, Open Targets Genetics will be deprecated on 9 July 2025

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Open Targets @opentargets.org · 08/05/2025

🎂 The Open Targets Platform turns 10 this year! To celebrate this milestone, join us in person or online for a two-day hackathon exploring data, analyses, and tools for target discovery 🖥️🧬 You can already submit a project idea: www.opentargets.org/hackathon Registrations will open in June

Open Targets hackathon, 21-22 October 2025, Submit a project

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Open Targets @opentargets.org · 06/05/2025

Join us for a live Q&A on the Open Targets Platform, and the genetics data in our latest release! 🧬🖥️ We will review and prioritise questions submitted in advance: forms.gle/EA75gDdNwjxk... Register: embl-org.zoom.us/webinar/regi...

Submit your questions for the Open Targets Platform Q&A session, 13 May, 3-4pm (UK Time)

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