Transferrin receptor-targeted anti-amyloid antibody enhances brain delivery and mitigates ARIA.

Pizzo, Michelle E; Plowey, Edward D; Khoury, Nathalie; Kwan, Wanda; Abettan, Jordan; DeVos, Sarah L; Discenza, Claire B; Earr, Timothy et al. · Science · 2025

basic_science · Level V

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Abstract

Amyloid-related imaging abnormalities (ARIA), side effects of anti-amyloid drugs seen in magnetic resonance imaging of the brain, are a major safety concern in patients with Alzheimer's disease. We developed an antibody transport vehicle (ATV) targeting transferrin receptor (TfR) for brain delivery of anti-amyloid-β protein (anti-Aβ) using asymmetrical Fc mutations (ATV<sup>cisLALA</sup>) that mitigates TfR-related liabilities and retains effector function when bound to Aβ. Administration of ATV<sup>cisLALA</sup>:Aβ in mice exhibited broad brain distribution and enhanced parenchymal plaque target engagement. This biodistribution reduced ARIA-like lesions and vascular inflammation. Taken together, ATV<sup>cisLALA</sup> has the potential to improve the next generation of Aβ immunotherapy through enhanced biodistribution mediated by transport across the blood-brain barrier.

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