Reposted by Open TargetsSaez-Rodriguez Group @saezlab.bsky.social · 07/09/2026🧵 Introducing Karenina, an open-source framework to evaluate LLMs & agents: doi.org/10.64898/202... 1175
Open Targets @opentargets.org · 28/09/2026The 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! 021
Open Targets @opentargets.org · 12/08/2026Ambitious 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 172
Open Targets @opentargets.org · 04/08/20266 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 162
Open Targets @opentargets.org · 28/07/2026"Patients know what it’s like to live with these conditions on a daily basis and the impact it has on our lives. By getting involved, we can help clinicians and researchers understand what really matters to us, so future research and treatments genuinely meet our needs." fg.bmj.com/content/earl...fg.bmj.comPatient and public involvement and engagement in research: who, why, when and how to get it rightPatient and public involvement and engagement (PPIE) refers to the different ways those with lived experience, including patients, family, friends and carers, and the public engage with the design, de... 110
Open Targets @opentargets.org · 21/07/2026“We hope our framework will help drive a conceptual shift in how MASLD is studied and understood.” A new study reframes Metabolic dysfunction-Associated Steatotic Liver Disease as a continuous process, better capturing the underlying disease biology www.nature.com/articles/s42...nature.comA data-driven framework reconstructs the molecular continuum of human MASLD progression - Nature MetabolismA trajectory-based molecular framework reveals coordinated temporal shifts in metabolic, inflammatory and fibrogenic mechanisms across the human MASLD spectrum. 100
Reposted by Open TargetsEvangelia Petsalaki @epetsalaki.bsky.social · 14/07/2026Our MASLD paper is finally out @naturemetabolism.bsky.social @ebi.embl.org @opentargets.org ! Fantastic collaboration with Toni Vidal-Puig's group in the IMS, UCam (co-led), Papatheodorou @earlhaminst.bsky.social and other coauthors, led by Ioannis Kamzolas and Thodoris Koutsandreas. Check it out! 033
Open Targets @opentargets.org · 14/07/2026Out now in Nature Metabolism! 🖥️🧬 An Open Targets project has identified a set of plasma proteins that could be used to diagnose and monitor patients with Metabolic dysfunction-Associated Steatotic Liver Disease (MASLD) www.nature.com/articles/s42...nature.comA data-driven framework reconstructs the molecular continuum of human MASLD progression - Nature MetabolismA trajectory-based molecular framework reveals coordinated temporal shifts in metabolic, inflammatory and fibrogenic mechanisms across the human MASLD spectrum. 165
Open Targets @opentargets.org · 07/07/2026VIDRA is out now on medRxiv! Variant-Informed Dose-Response Analysis (VIDRA) is a computational framework which infers genetic dose-response relationships by integrating trait-associated variants across a spectrum of allele frequencies and functional consequences. www.medrxiv.org/content/10.6...medrxiv.orgGenetic dose-response modelling predicts drug mechanisms, dosing, and adverse eventsUnderstanding how changes in gene function affect disease risk is central to drug development. Genetic variants are natural perturbations of gene activity and provide an opportunity to systematically ... 100
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 100
Open Targets @opentargets.org · 30/06/2026The Open Targets Platform 26.06 release is out now! ✋ 🔴 Find out about all the updates on the blog: blog.opentargets.org/open-targets... 000
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 110
Open Targets @opentargets.org · 18/06/2026How do you ensure the stability of a platform with millions of data points updated every release? You start testing patterns In the last release of the Open Targets Platform, we implemented automated end-to-end testing into our development workflow 🧬🖥️ blog.opentargets.org/end-to-end-t...blog.opentargets.orgEnsuring stability of the Open Targets Platform: the case for automated end-to-end testingAs a platform grows in complexity, maintaining stability becomes increasingly difficult. The Open Targets Frontend Team has introduced automated end-to-end testing to ensure that core user journeys an... 000
Reposted by Open TargetsWellcome Sanger Institute @sangerinstitute.bsky.social · 15/06/2026New study into Crohn’s disease helps explain why gut inflammation persists, and could help unlock more targeted treatments for patients. Researchers analysed millions of gut cells, uncovering lasting molecular changes and key inflammatory immune cells. 👇 www.sanger.ac.uk/news_item/ce...sanger.ac.ukCellular study maps gene activity changes across more than 50 gut cell types in Crohn’s diseaseThe latest IBDverse study identifies the genes expressed in Crohn's disease, revealing a lasting 'molecular scar' in the gut lining. 182
Open Targets @opentargets.org · 15/06/2026Out now in Nature Genetics! The largest single-cell RNA sequencing dataset of terminal ileal biopsies profiles 1.1 million cells from over 100 Crohn’s disease patients and over 200 healthy controls. Crohn’s disease is a type of inflammatory bowel disease (IBD) www.nature.com/articles/s41...nature.comSingle-cell RNA sequencing of terminal ileal biopsies identifies signatures of Crohn’s disease pathogenesis - Nature GeneticsIBDverse is a single-cell RNA atlas of terminal ileal biopsies comprising 1.1 million cells from 111 patients with Crohn’s disease and 232 healthy participants. Analysis of the data identifies genes, ... 142
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." 🤝 141
Open Targets @opentargets.org · 03/06/2026Out now in Nature! Genetic analysis of the largest single-cell dataset of Inflammatory Bowel Disease (IBD)-relevant tissues nominates effector genes and cell types at over half of known IBD loci, including 74 for which this is the first candidate effector gene 🖥️🧬 www.nature.com/articles/s41...nature.comCell-type-resolved genetic variation shapes inflammatory bowel disease risk - NatureSingle-cell mapping of cis-expression quantitative trait loci in inflammatory bowel disease revealed distal, enhancer-enriched variants detected at the cell-type level more frequently co-loc... 141
Open Targets @opentargets.org · 01/06/2026We're at #ESHG2026! Come say hello and find out about current Open Targets projects. See you there! 020
Reposted by Open TargetsBen Lehner @benlehner.bsky.social · 28/05/2026Excited to release #OpenSplice - the impact of >500,000 mutations on the alternative splicing of 600 human exons www.biorxiv.org/content/10.6... Led by @gioia-quarantani.bsky.social + funded by @opentargets.org @sangerinstitute.bsky.social @crg.eu #cshlsymp26 #RNA2026 @rnasociety.bsky.socialbiorxiv.org 0175
Open Targets @opentargets.org · 27/05/2026Introducing 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... 1167
Reposted by Open TargetsGioia Quarantani @gioia-quarantani.bsky.social · 26/05/20261\ Excited to finally share some of my PhD work: OpenSplice! We measured how >590,000 mutations affect exon inclusion across 600 human exons, creating a resource to study splicing regulation, benchmark variant effect predictors, and support variant interpretation. 12713
Open Targets @opentargets.org · 26/05/2026Design is a team sport. Galabina Yordanova and Adedoyin Okunade share how they crafted an intuitive experience for the Perturbation Catalogue through user research and iterative design. blog.opentargets.org/designing-th...blog.opentargets.orgDesigning the Perturbation Catalogue—a UX journey through perturbation dataThe Perturbation Catalogue brings genetic perturbation data into one curated, harmonised, and discoverable platform. Once the data is in, how do you ensure it's useful and structured in a way that ans... 100
Open Targets @opentargets.org · 21/05/2026GWAS discovery is far from saturation. We need more GWAS, and more diverse GWAS 🧬🖥️ 100
Open Targets @opentargets.org · 19/05/2026How do you present complex information in an easily understandable and visually appealing way? Our web developer Graham McNeill walks you through our design process for visualising locus-to-gene scores in the Platform: blog.opentargets.org/design-proce...blog.opentargets.orgVisualising complex data in the Open Targets Platform: our design process for Locus-to-Gene scoring representationOur Shapley value-based explanations for Locus-to-Gene scores presented a design challenge of providing a diverse range of users with an intuitive ‘explanation’ of the score but also ensuring enough d... 020
Open Targets @opentargets.org · 14/05/2026"Beyond the technical aspects of large-scale data analysis, the internship gave me a clearer view of how deep biological knowledge and genetic evidence can be combined to support target discovery and safety assessment." 🖥️🧬 blog.opentargets.org/from-genetic...blog.opentargets.orgFrom genetic perturbation to drug safety: Sam Moix’s internship at Open TargetsWorking with a team at the forefront of large-scale biomedical data integration, I learned a great deal about large-scale data processing, reproducible research, human genetics, and the challenges of ... 000
Open Targets @opentargets.org · 12/05/2026Want to maximise your chance of clinical success? Gene-disease associations with: 1️⃣ Genetic support from protein-altering variants 2️⃣ Intermediate pleiotropy are 10 times more likely to result in approved medicines, balancing impactful genetic variation with genetically informed safety liability 110
Open Targets @opentargets.org · 07/05/2026How can you compare cells across conditions? We chat to Marie Moullet about Tripso, a self-supervised transformer model she co-developed for single cell data analyses going beyond canonical cell type markers and annotations to understand cell states blog.opentargets.org/how-the-lotf...blog.opentargets.orgHow the Lotfollahi lab created Tripso to interpret single cell dataMarie Moullet describes Tripso—Transformers for learning Representations of Interpretable gene Programs in Single-cell transcriptOmics—a method for single cell data analyses that goes beyond canonical... 100
Open Targets @opentargets.org · 06/05/2026Our findings from the largest systematic analysis of complex trait associations is out now on bioRxiv! 🧬🖥️ www.biorxiv.org/content/10.6... 111
Reposted by Open TargetsDavid Ochoa @d0choa.bsky.social · 01/05/2026🧬 New preprint: "The Human Pleiotropic Map of GWAS Associations and Therapeutic Implications" Why do some genetically supported drug targets succeed in the clinic while others fail? Across 100,526 GWAS, the same evidence flags constrained gene functions rarely safe to modulate. 1198
Reposted by Open TargetsEurope PMC @europepmc.org · 23/04/2026What a conversation! 🙌 Thank you to everyone who joined "Closed Licences, Closed Doors" with @knowledgerights21.org Do restrictive licences protect your research from AI, or just limit its impact? Couldn't make it? Watch the recording 👇 youtu.be/BprlLw6CRyI #OpenScience #AcademicSky 🧪youtu.beClosed licences closed doors: Open licensing, AI, and the future of research impactYouTube video by EuropePMC 121
Reposted by Open TargetsEurope PMC @europepmc.org · 13/04/2026📣Tomorrow: Closed Licences, Closed Doors webinar! Join us and @knowledgerights21.org to find out about open licensing, AI, and the future of research impact! 🗓️ 14 April | ⏰ 10:00 BST/11:00 CEST There's still time to register 👉 embl-org.zoom.us/meeting/regi... #OpenScience #AcademicSky 🧪embl-org.zoom.usWelcome! You are invited to join a meeting: Closed licences, closed doors: Open licensing, AI, and the future of research impact. After registering, you will receive a confirmation email about joining...Open licensing and copyright are important aspects of publishing research, but what do they mean in practice? Creative Commons (CC) licences offer a standardised framework for sharing research outputs... 031
Open Targets @opentargets.org · 30/03/2026🧬🖥️ Introducing TRIPSO: a transformer network to help interpret single cell data analyses by learning multiple predefined or data-driven gene programme-specific embeddings Tripso was developed by a team across the Lotfollahi, Haniffa, Gottgens and Vento labs www.biorxiv.org/content/10.6...biorxiv.org 110
Reposted by Open TargetsEurope PMC @europepmc.org · 25/03/2026The reveal is here! Meet your experts: 🎤 Presentations: Dr. David Hulcoop @opentargets.org Dr. Benjamin White @knowledgerights21.org Dr. Summer Rosonovski #EuropePMC 💬 Panel: Melissa Harrison #EuropePMC @ebi.embl.org Dr. Kiera McNeice Research Transparency Manager* Dr. Bastian Drees @embl.org 132
Reposted by Open TargetsDavid Ochoa @d0choa.bsky.social · 23/03/2026The Open Targets Platform spring 🌼 release is out — and this one marks the beginning of something we've been building towards for a while. 143
Open Targets @opentargets.org · 23/03/2026The 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... 021
Reposted by Open TargetsEurope PMC @europepmc.org · 19/03/2026Closed licences, closed doors. We're hosting a webinar with @kr21.bsky.social exploring how licensing shapes real-world research impact, from @opentargets.org to pandemic response. Speaker panel reveal soon 👀 🗓️ 14 April ⏰ 10:00 BST 🔗 embl-org.zoom.us/meeting/regi... #OpenScience #AcademicSky 🧪embl-org.zoom.usWelcome! You are invited to join a meeting: Closed licences, closed doors: Open licensing, AI, and the future of research impact. After registering, you will receive a confirmation email about joining...Open licensing and copyright are important aspects of publishing research, but what do they mean in practice? Creative Commons (CC) licences offer a standardised framework for sharing research outputs... 177
Open Targets @opentargets.org · 12/03/2026Introducing PerturbGen: a generative AI foundation model that predicts how genetic perturbations affect cellular trajectories www.biorxiv.org/content/10.6... 130
Open Targets @opentargets.org · 12/03/2026Out now on bioRxiv! 🧬🖥️ Dewi Moonen, @anniquec.bsky.social and a team led by Lars Steinmetz and Daniel Schraivogel at EMBL have mapped the downstream regulatory networks of thousands of immune disease-relevant variants in CD4+ T cells www.biorxiv.org/content/10.6...biorxiv.org 1143
Reposted by Open TargetsCarlos Cruz @carcruz.dev · 15/02/2026Finally I can properly explore the mcp-ui pattern. Pretty interesting how the development of new components (especially complex ones) will change dramatically in how data is loaded into them. Exiting times🔥 building a demo using @opentargets.org components. Ref: mcpui.dev 011
Open Targets @opentargets.org · 03/03/2026What 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... 152
Open Targets @opentargets.org · 04/02/2026🧬🖥️ Our latest publication reveals a shift in strategies to discover novel drug targets over the past 20 years: after 2015, biomedical evidence supporting a target-disease indication increasingly appears before the approval year www.nature.com/articles/s41...nature.comTemporal trends in evidence supporting novel drug target discovery - Nature CommunicationsHere the authors implement a comprehensive timestamping across millions of pieces of biomedical evidence supporting target–disease associations in the Open Targets Platform and analyse trends in evide... 111
Open Targets @opentargets.org · 02/02/2026Open Targets Platform data is now available on @amazonwebservices.bsky.social through the Open Data Program! registry.opendata.aws/opentargets/ 070
Reposted by Open TargetsEMBL-EBI @ebi.embl.org · 27/01/2026🔬New Perturbation Catalogue update! With a redesigned interface, richer datasets and powerful analysis workflows, it’s now easier than ever to explore genetic perturbation data. www.ebi.ac.uk/perturbation... #Bioinformatics #FunctionalGenomics @training.ebi.embl.org @opentargets.org 141
Open Targets @opentargets.org · 27/01/2026The 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... 1115
Open Targets @opentargets.org · 26/01/2026🧬🖥️ "This integration is a great example of bringing leading AI to platforms and data that are already widely used across the industry.” — Jonah Cool, Head of Life Sciences Partnerships, Anthropic Find out more about the Open Targets Platform MCP server: blog.opentargets.org/official-ope...blog.opentargets.orgIntroducing the official Open Targets Platform Model Context ProtocolWe have released the first iteration of a dedicated MCP server to bring our systematic target discovery capabilities to Large Language Model (LLM)-based workflows. 000
Reposted by Open TargetsDavid Ochoa @d0choa.bsky.social · 23/01/2026The needle in the haystack problem: spotting novel drug targets among redundant evidence. Our solution? A time-series novelty metric for Open Targets Platform associations. Thanks to Coté Falaguera & @opentargets.org partners for making this happen. 033
Open Targets @opentargets.org · 22/01/2026Out now in Nature Communications! 🧬🖥️ An Open Targets team developed a metric for the novelty of a target in the context of a disease, according to current available knowledge. This allows drug discovery scientists to easily identify potentially novel targets www.nature.com/articles/s41...nature.comTemporal trends in evidence supporting novel drug target discovery - Nature CommunicationsHere the authors implement a comprehensive timestamping across millions of pieces of biomedical evidence supporting target–disease associations in the Open Targets Platform and analyse trends in evide... 131
Open Targets @opentargets.org · 21/01/2026Out now! 🧬 Loss of CHD1 and MAP3K7 creates an acquired cancer vulnerability, highlighting these genes as potential biomarkers of immune checkpoint blockade response Find out more: www.cell.com/cell-reports...cell.comCRISPR screens in the context of immune selection identify CHD1 and MAP3K7 as mediators of cancer immunotherapy resistanceImmunotherapies only provide benefit for a subset of individuals with cancer, highlighting the need for biomarkers of response. Using whole-genome CRISPR-Cas9 screens, Watterson et al. map genetic dep... 000
Reposted by Open TargetsWellcome Sanger Institute @sangerinstitute.bsky.social · 20/01/2026New biomarkers for cancer immunotherapy have been identified. 🔎 Researchers found that loss of two genes, CHD1 and MAP3K7, makes tumours more likely to respond to immunotherapy. The findings could help support more personalised cancer treatment 👇 bit.ly/4sMBTq2bit.lyImmunotherapy success could be predicted with new biomarkersScientists pinpoint two cancer genes that when deleted, improve the vulnerability of tumour cells to immunotherapy. 145
Open Targets @opentargets.org · 20/01/2026We're recruiting 3 roles through @sangerinstitute.bsky.social 👩🔬🧬💻 NEW: ➡ Postdoc with @carlanderson.bsky.social ➡ Technical Specialist with @bayraktarlab.bsky.social ALSO: ➡ (Senior) Postdoc in Mo Lotfollahi’s lab More information in the thread See all available jobs: opentargets.org/jobsopentargets.orgJobs - Open TargetsOpen Targets is a public-private initiative to generate evidence on the validity of therapeutic targets based on genome-scale experiments and analysis. 110