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

2026-02-23 pharmacology and therapeutics Title + abstract only

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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 contrast to genome-wide association study (GWAS) support, which confers an increase in ...

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

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