Prime Medicine (PRME) investor relations material

Prime editing technology and platform

• Prime Editing enables permanent correction of genetic alterations without double-strand breaks or bystander edits, offering versatility across a wide range of genome edits and disease targets.

• The platform is modular, allowing rapid adaptation for different mutations and indications, leveraging shared delivery and manufacturing processes.

• Extensive intellectual property covers all permutations of Prime Editing, including CRISPR-Cas enzymes, guide RNAs, and reverse transcriptase.

• New regulatory models and platform-based approvals are being pursued to accelerate clinical development.

Clinical pipeline and development programs

• Lead liver programs target Wilson Disease (PM577a) and Alpha-1 Antitrypsin Deficiency (PM647), with clinical trials initiating in 2026 and initial data expected in 2027.

• PM359 for Chronic Granulomatous Disease (CGD) has shown proof-of-concept with rapid engraftment, restored neutrophil function, and no serious adverse events; BLA submission is planned.

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

• Ex vivo CAR-T programs in collaboration with Bristol Myers Squibb leverage multiplex editing and non-viral manufacturing for oncology and autoimmune indications.

Preclinical and clinical data highlights

• PM577a efficiently corrects the H1069Q mutation in Wilson Disease, restoring copper metabolism and wild-type protein levels in animal models.

• PM647 corrects the Pi*Z mutation in AATD, restoring healthy AAT protein levels at clinically relevant doses.

• Prime Editors show no detectable off-target editing, large deletions, or translocations in lead programs, supporting a strong safety profile.