Further translational development would benefit from: (1) chronic dosing studies in AD rodent models assessing both biomarker modulation (cerebral Aβ, p‐tau) and cognitive outcomes using Morris water maze or novel object recognition tests; (2) bioavailability studies quantifying brain parenchyma concentrations after oral administration; and (3) clinical pilot studies evaluating CSF biomarkers in mild cognitive impairment patients. The gene discussed is MAPT; the disease is Alzheimer disease.