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BEAM-302 update shows durable biomarker effects in early AAT-deficiency trial

Company / institutional announcement · Beam Therapeutics

Source published: · Event / reporting date: 2026-09-08

Site published: 2026-11-03 · Updated: 2026-11-03

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What changed

Beam presented updated open-label Phase 1/2 data for BEAM-302, an LNP-delivered in vivo base-editing treatment for alpha-1 antitrypsin deficiency. Follow-up extended to 18 months after one dose, with increased functional AAT and reduced mutant Z-AAT and circulating polymers. Dosing is underway in a pivotal cohort. These biomarker findings do not yet establish controlled evidence of improved lung or liver clinical outcomes.

Why it may matter

This program connects RNA/LNP delivery with durable gene correction, making pivotal-cohort results, long-term safety, and organ-level outcomes important next milestones.

The interpretation above is analysis, not a conclusion attributed to the original source.

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