Human Pathobiology
The principle that drug discovery should be grounded in human disease biology rather than animal model biology. A central theme of Session I, championed especially by Alistair Henry (UCB).
The Argument
- The mouse will keep telling you a clean, wrong story
- F: the human peripheral blood signature showed IL-17F driving the late phase of inflammation — mouse showed nothing
- Bimekizumab was built because human pathobiology won the internal argument at UCB against affinities and receptor stoichiometry
- With FDA evaluation of New Approach Methods (NAMs), this stops being aspirational
Tools
- Organoids and tissue explants
- Patient-derived material
- Human peripheral blood profiling
- Transcriptome back-translation from treated patients
- iPSC-derived neurons (used in SARM1 and Alzheimer’s studies)
Cross-References
- New Approach Methods (NAMs) — the regulatory and technical infrastructure
- Back-translation — using approved drugs as hypothesis generators
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