Drug targets with human genetic support are ~2.6x more likely to reach approval. A Regeneron geneticist on using natural mutations as ready-made human trials, from CCR5 to HAO1 knockouts

An r/biotech post by u/works-in-progress relays a claim: drug targets with "human genetic support" are about 2.6 times more likely to be approved than those without—stemming from an argument by Regeneron geneticists that naturally occurring human mutations can be treated as "ready-made human trials" (from CCR5 to HAO1 knockouts), to avoid animal models missing human biology. Methodological caveats appeared in the comments: u/mkb96mchem (17▲) cautioned that this statistic is not necessarily purely driven by a "genetic support → approval" causal effect, and that genetic support may merely be a proxy variable for "already well-validated/already drugged targets"; u/alpha_as_f-ck noted that the source of "next-wave targets" from large-scale paired health records is drying up; u/Conscious_Cell1825, u/pacificjunction, and others added clarifications about genetic redundancy and "2.6x is a hazard ratio, not a guarantee." Factually, "targets with genetic support have about 2.6 times the success probability of those without" is indeed a real published finding: Minikel et al., Nature 2024, 629:624-629 (doi:10.1038/s41586-024-07316-0). As speculation, the skepticism about "causal direction/proxy confounding" is a reasonable statistical calibration, not a refutation of the finding.

Checked ✔ Verified: "Targets with human genetic support have about 2.6 times the success probability of those without" comes from