Prime Medicine (PRME) 44th Annual J.P. Morgan Healthcare Conference summary

Technology and Platform Overview

• Prime Editing enables precise, permanent genome modifications without double-strand breaks, supporting large deletions, insertions, and correction of various mutations for broad disease applicability, addressing up to 90% of genetic diseases.

• Demonstrated curative potential in ex vivo and in vivo models, with strong safety data showing no detectable off-target or bystander edits, large deletions, or translocations in lead programs.

• The platform's modularity allows rapid adaptation for various indications, leveraging shared delivery systems and manufacturing processes across programs.

• Robust intellectual property covers foundational and product-level innovations, with 6 U.S. and 12 ex-U.S. issued patents, and a commitment to defend and enforce IP rights.

• Regulatory frameworks are evolving, allowing multiple mutations under one IND and leveraging studies across programs for efficiency, with new models paving the way for platform-based approvals.

Pipeline Focus and Clinical Development

• Strategic review narrowed focus from 18 to a few high-value programs, prioritizing technical feasibility, commercial opportunity, and near-term clinical endpoints.

• Lead in vivo programs target Wilson disease and alpha-1 antitrypsin deficiency, with IND/CTA filings planned for the first half of the year and mid-2026, and proof-of-concept data expected in 2027.

• Cystic fibrosis program is advancing with support from the Cystic Fibrosis Foundation, aiming for in vivo proof-of-concept data in 2026 and addressing over 93% of patients through hotspot and PASSIGE strategies.

• PM359 in CGD demonstrated rapid engraftment, restored function, and no serious adverse events, providing proof-of-concept for curative therapies.

• Pipeline is designed for modularity, enabling rapid adaptation across mutations and diseases within the same tissue.

Market Opportunity and Competitive Landscape

• Wilson disease and alpha-1 antitrypsin deficiency represent multi-billion-dollar orphan indications, with the potential to address tens of thousands of patients globally.

• Prime Editing offers a differentiated approach, especially for transversion mutations in Wilson disease and for restoring wild-type protein in alpha-1 antitrypsin deficiency.

• Competitive landscape includes gene therapy, base editing, and RNA editing, but Prime Editing is positioned as potentially best-in-class due to precision and one-time treatment potential.