CRISPR Base Editing for AATD
Idiopathic Pulmonary Fibrosis · Base editing corrects genetic mutations causing lung disease without DNA double-strand breaks
Mechanism
Base editing corrects genetic mutations causing lung disease without DNA double-strand breaks
Development stage
Phase I (Beam/Prime)
Evidence level
Level 2 (Phase I)
Developer
Beam Therapeutics / Prime Medicine
Overview
Beam testing base editing for AATD (alpha-1 antitrypsin deficiency) in US/UK/Australia. Prime Medicine planning AATD trial 2026. Lung SORT LNPs enable CRISPR delivery to lung epithelium. Corrected CFTR mutations in patient bronchial cells. Direct crossover path to correcting IPF genetic drivers.
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Medical disclaimer. This page summarizes research about CRISPR Base Editing for AATD for information only. It is not medical advice. Verify approval status and evidence with the FDA and a pulmonologist.