Prime Medicine unveils preclinical AATD program with up to 72% gene correction, IND/CTA expected mid-2026

Prime Medicine, Inc.

AATD program targets E342K (Pi*Z) mutation in SERPINA1 gene using LNP-delivered Prime Editors.
In humanized mice, up to 72% precise correction of SERPINA1 in hepatocytes, restoring >95% serum AAT to corrected isoform.
Serum M-AAT levels well above 20µM, indicating restoration to normal levels in mouse model.
Company expects to file IND and/or CTA for AATD program in mid-2026.

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