From BBB crossing to biological effect: How do we evaluate brain biodistribution, target engagement, and efficacy?
10 Sept 2026
Preclinical Models
- How do we determine whether a biologic has crossed the BBB—and distinguish true parenchymal delivery from vascular or nonspecific signal? Is this important?
- Once in the brain, how do we characterize where a therapeutic distributes across regions, cell types, and subcellular compartments? Do we need to?
- Do we need to connect brain biodistribution with evidence of target engagement and downstream phenotypic efficacy? How?
- What roles, if any, should conventional histology, tissue clearing, 3D imaging, and complementary molecular assays play in building a convincing evidence package?
- How should these approaches evolve when moving from rodents to large-animal models—and ultimately toward clinical translation? What regulatory or GLP considerations are there?
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