Evidence map›Paper›PMID 42461796›Full record

ArticlePLoS genetics2026

A genomic-led strategy to anticipate drug safety effects.

Brian R Ferolito, Andrea R V R Horimoto, Kai Gravel-Pucillo, Daniel J Golden, Hesam Dashti, Claudia Giambartolomei, Danielle Rasooly, Rachael Matty, Liam Gaziano, Yakov Tsepilov and 20 more

Abstract read
In one paragraph

Article in PLoS genetics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

30 authors.

Brian R FerolitoMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.ORCID https://orcid.org/0000-0003-3208-6446
Andrea R V R HorimotoDepartment of Genetics, University of North Carolina School of Medicine, Chapel Hill, North Carolina, United States of America.ORCID https://orcid.org/0000-0002-8573-5158
Kai Gravel-PucilloMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.ORCID https://orcid.org/0009-0004-9754-9688
Daniel J GoldenMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.
Hesam DashtiThe Novo Nordisk Foundation Center for Genomic Mechanisms of Disease, Broad Institute of MIT and Harvard, Cambridge, Massachusetts, United States of America.
Claudia GiambartolomeiHealth Data Science Centre, Human Technopole, Milan, Italy.ORCID https://orcid.org/0000-0003-2786-1225
Danielle RasoolyMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.ORCID https://orcid.org/0000-0001-7715-1809
Rachael MattyMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.
Liam GazianoMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.ORCID https://orcid.org/0000-0003-2059-8081
Yakov TsepilovOpen Targets Genetics, Open Targets, Hinxton, United Kingdom.
Lauren CostaMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.
Nicole KosikMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.ORCID https://orcid.org/0000-0003-1384-7035
Harris IoannidisEuropean Molecular Biology Laboratory, European Bioinformatics Institute, Hinxton, United Kingdom.ORCID https://orcid.org/0000-0003-3470-6055
Mohd KarimGenomic Discovery, Variant Bio, Seattle, Washington, United States of America.
Giovanna WinickiMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.ORCID https://orcid.org/0009-0001-4977-9225
Fiona HunterEuropean Molecular Biology Laboratory, European Bioinformatics Institute, Hinxton, United Kingdom.
Claudia LangenbergPrecision Healthcare University Research Institute, Queen Mary University of London, London, United Kingdom.ORCID https://orcid.org/0000-0002-5017-7344
John C WhittakerMRC Biostatistics Unit, University of Cambridge, Cambridge, United Kingdom.ORCID https://orcid.org/0000-0002-3529-2379
Million Veteran Program
Tianxi CaiDepartment of Biomedical Informatics, Harvard Medical School, Boston, Massachusetts, United States of America.
Gina M PelosoMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.
Barbara ZdrazilEuropean Molecular Biology Laboratory, European Bioinformatics Institute, Hinxton, United Kingdom.ORCID https://orcid.org/0000-0001-9395-1515
Maya GhoussainiRegeneron Genetics Center, Regeneron Pharmaceuticals, Tarrytown, New York, United States of America.
Andrew R LeachLifeArc, Accelerator Building, Open Innovation Campus, Stevenage, United Kingdom.
Sumitra MuralidharDepartment of Veterans Affairs, Office of Research and Development, Washington District of Columbia, United States of America.
Ines A SmitEuropean Molecular Biology Laboratory, European Bioinformatics Institute, Hinxton, United Kingdom.
Juan P CasasBiomarker Development/Translational Medicine, Novartis Institutes for Biomedical Research, Cambridge, Massachusetts, United States of America.
J Michael GazianoMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.
Kelly ChoMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.
Alexandre C PereiraMillion Veteran Program (MVP) Coordinating Center, Veterans Affairs Healthcare System, Boston, Massachusetts, United States of America.ORCID https://orcid.org/0000-0002-7782-5540

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Safety-related issues account for approximately 25% of failures in new drug discovery programs. On top of that, many are discovered during post-marketing surveillance, significantly limiting drug utility and application. To proactively address these concerns, we developed a genetics-led strategy leveraging Mendelian Randomization (MR) across large-scale genetic datasets from the Million Veteran Program, FinnGen, and UK Biobank. By mapping genetic variants associated with gene expression and protein abundance to 1,449 harmonized human phenotypes, we systematically identified potential adverse drug reactions (ADR). Our extensive MR analysis, encompassing 16,915 protein-coding genes, demonstrated the capacity to predict hundreds of known ADR for approved medications, with approximately 40% corroborated by FDA Adverse Event Reporting System (FAERS) data. Additionally, we found significant enrichment of identified gene-mechanism pairs in clinical trials terminated early due to safety concerns, highlighting the clinical utility of genetics-informed safety prediction. Notably, immune-related pathways were prominently associated with ADR, indicating particular sensitivity within immune modulation targets. Our comprehensive atlas, integrating genetic evidence with pharmacological mechanisms, provides a robust predictive framework for anticipating drug safety, potentially enhancing decision-making in drug development and pharmacovigilance. An interactive web interface allowing filtering by gene, phenotype, drug phase, and mechanism of action is available at https://shiny.parse-health.org/safety/.

Indexed as

Drug-Related Side Effects and Adverse ReactionsGenomicsAdverse Drug Reaction Reporting SystemsDrug DiscoveryHumansPhenotype

Identifiers

PMID42461796
PMCPMC13375020

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.