Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
AATD Program
- Development stage
- Preclinical
- Lead developer
- Prime Medicine
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
Prime Medicine's AATD Program is an investigational gene-editing therapy designed to treat Alpha-1 Antitrypsin Deficiency (AATD) by precisely correcting the underlying genetic cause. Utilizing proprietary Prime Editing technology delivered via liver-targeted lipid nanoparticles (LNPs), the program aims to edit the E342K (Pi*Z) mutation in the *SERPINA1* gene. This correction restores the production of functional wild-type M-AAT protein, potentially addressing both the pulmonary and hepatic manifestations of the disease. Preclinical studies in humanized mouse models have demonstrated high levels of precise gene correction (up to 72%) and restoration of serum AAT to normal physiological levels. The program is currently in the IND-enabling stage, with clinical trial applications expected in mid-2026.
- Other names
- Prime Medicine-SERPINA1-Prime Editor (gene editing)-LNP delivery-Alpha-1 Antitrypsin DeficiencyAlpha-1 Antitrypsin Deficiency ProgramAlpha1 Antitrypsin Deficiency ProgramAlpha 1 Antitrypsin Deficiency ProgramSERPINA1 Prime Editing ProgramSERPINA-1 Prime Editing ProgramSERPINA 1 Prime Editing Program
02
Targets
SERPINA1 Z (Alpha-1-antitrypsin Z variant protein)
Beyond the preview
Go deeper on AATD Program.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on AATD Program.