Drug intelligence / Profile preview

PM647

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

Overview

PM647 is an investigational prime editing-based gene therapy developed by Prime Medicine for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). It utilizes precision gene editing technology to correct the underlying mutation in the SERPINA1 gene within hepatocytes, thereby restoring the production of functional alpha-1 antitrypsin protein and potentially addressing both the pulmonary and hepatic manifestations of the disease. The therapy is delivered using a liver-targeted lipid nanoparticle (LNP) system. PM647 was nominated as a development candidate in August 2025, with clinical trials anticipated to begin following IND/CTA filings in mid-2026.

Targets

Pi (Inorganic phosphate)

Beyond the preview

Go deeper on PM647.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

Connect 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 PM647.

Explore the full profile