Company: PRME
Filing Date: 2025-03-18
Form Type: 8-K
Source: 0001193125-25-056122
Chunk: 1

Company: Prime Medicine, Inc.
Filing Date: 2025-03-18
Form: 8-K
Item: Item 8.01
Chunk 1
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Item 8.01      Other Events.  

On March 18, 2025, the Company unveiled a preclinical program for the treatment of alpha-1antitrypsin deficiency (“ AATD”), the next program within its liver franchise.

The AATD program leverages the Company’s universal liver lipid nanoparticle (“ LNP”) to edit the E342K (“ Pi*Z”) mutation in the SERPINA1 gene, the prevalent disease-causing mutation in AATD, restoring the mutated protein sequence back to wild-type M protein, with the potential to treat both lung- and liver-associated disease.

In the Company’s initial in vivodata, LNP delivery of Prime Editors targeting the Pi*Z (E324K) mutation demonstrated up to 72% precise correction of the SERPINA1 gene in the hepatocytes of fully humanized mice. Importantly, this restored over 95% of serum AAT to the corrected isoform, with healthy AAT(“ M-AAT”)protein in the serum at levels well above 20µ M, indicating restoration ofM-AATto normal levels in a humanized mouse model.

The Company is advancing its AATD program through the final stages of lead optimization and expects to file an investigational new drug (“ IND”) and/or clinical trial application (“ CTA”) in mid-2026.