Prime Medicine (PRME) Corporate presentation summary

Prime editing technology and platform

• Prime editing offers versatile, permanent correction of genetic alterations without double-strand breaks or bystander edits, supporting curative potential for a broad range of diseases.

• The platform is modular, enabling rapid adaptation across programs and leveraging regulatory and manufacturing synergies.

• Extensive intellectual property covers all permutations of prime editing, including delivery technologies and therapeutics.

• No detectable off-target editing, large deletions, or translocations observed in lead programs, supporting a strong safety profile.

• New regulatory models facilitate platform-based approvals, accelerating clinical development.

Pipeline and clinical progress

• Lead programs target Wilson Disease (PM577) and Alpha-1 Antitrypsin Deficiency (PM647), with IND/CTA filings planned for 2026 and initial clinical data expected in 2027.

• PM359 for chronic granulomatous disease (CGD) demonstrated rapid engraftment, restored function, and no serious adverse events, with a BLA filing planned.

• Cystic fibrosis programs are advancing with support from the Cystic Fibrosis Foundation, aiming to address over 93% of patients through hotspot and PASSIGE strategies.

• Strategic partnerships, including with Bristol Myers Squibb, expand reach into immunology and oncology, with significant milestone and royalty potential.

• The pipeline includes liver, lung, immunology, and oncology indications, with additional programs in neurological and large indications planned.

Disease focus and clinical data

• Wilson Disease and AATD represent large, genetically defined markets with high unmet need and no approved curative therapies.

• PM577 efficiently corrected the H1069Q mutation in Wilson Disease models, restoring copper homeostasis and demonstrating normal copper clearance.

• PM647 corrected AATD mutations in vivo, restoring healthy AAT protein levels at clinically relevant doses.

• Cystic fibrosis efforts focus on both hotspot correction and super exon insertion (PASSIGE), with in vitro and in vivo optimization underway.

• CAR-T programs leverage multiplex editing and non-viral manufacturing, enabling precise, efficient, and safe cell therapies.