Article

Retrospective evaluation of human genetic evidence for clinical trial success using Mendelian randomization and machine learning

2026-02-23

Abstract excerpt

Human genetics has become a cornerstone of drug target discovery, yet the value of Mendelian randomization (MR) for predicting clinical success remains uncertain. Here, we systematically evaluated MR across 11,482 target–indication pairs with documented Phase II clinical outcomes to assess its utility for drug development. We find that MR statistical significance alone does not enrich for Phase II success, in cont...

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Literature Corpus work: 70d7fcee-3f6e-5d25-bc33-b4c43a5f71d9
DOI: 10.64898/2026.02.19.26346536

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