PF-AtlasTreatments › CRISPR Base Editing for AATD/Lung Disease

CRISPR Base Editing for AATD/Lung Disease

Base editing corrects genetic mutations causing lung disease without DNA double-strand breaks

Clinical research — In human clinical trials (Phase I–III). How PF-Atlas grades evidence.
Development stage
Phase I (Beam/Prime)
Evidence level
Level 2 (Phase I)
Mechanism class
Base editing corrects genetic mutations causing lung disease without DNA double-strand breaks
Timeline
3-5 years
Risk profile
Medium
Developer
Beam Therapeutics / Prime Medicine
Key researchers
D. Liu

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.

How CRISPR Base Editing for AATD/Lung Disease fits into IPF treatment

CRISPR Base Editing for AATD/Lung Disease is classified as Level 2 (Phase I) at the Phase I (Beam/Prime) stage. Explore how it compares to other options and where it sits in the development pipeline:

Other IPF treatments

Medical disclaimer. This page summarizes published research and PF-Atlas model output about CRISPR Base Editing for AATD/Lung Disease for information only. It is not medical advice or a treatment recommendation. Drug availability, approval status and evidence change — verify with a pulmonologist and official sources (FDA, ClinicalTrials.gov) before any decision.