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 wide range of diseases.

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

• Extensive intellectual property covers all permutations of prime editing, with 10 U.S. and 19 ex-U.S. issued patents.

• New regulatory models and platform-based approvals are paving the way for accelerated clinical development.

• The technology is designed for edits ranging from small base pair swaps to large gene insertions or inversions.

Pipeline and clinical programs

• Lead liver programs target Wilson Disease (PM577) and Alpha-1 Antitrypsin Deficiency (PM647), with IND/CTA filings expected in H1 and mid-2026, respectively, and initial clinical data in 2027.

• PM359 for Chronic Granulomatous Disease (CGD) has shown proof-of-concept clinical data, with plans for BLA submission.

• Cystic Fibrosis programs are advancing with support from the Cystic Fibrosis Foundation, targeting both hotspot mutations and super exon insertion strategies.

• Ex vivo CAR-T programs are being developed in collaboration with Bristol Myers Squibb, leveraging PASSIGE technology for multiplex editing.

• The pipeline is expanding into immunology, oncology, and additional genetic and non-genetic diseases.

Disease focus and market opportunities

• Wilson Disease and AATD represent large, genetically defined liver disease markets, with over 20,000 and 200,000 patients in the US/EU, respectively.

• Prime editing efficiently corrects key mutations in Wilson Disease (H1069Q, R778L) and AATD, restoring normal protein levels and function in preclinical models.

• Cystic Fibrosis affects ~40,000 people in the US, with no curative therapy; prime editing could address over 93% of patients.

• CGD is a rare, life-threatening disease; PM359 demonstrated rapid engraftment and restoration of neutrophil function in clinical studies.

• The addressable market for Wilson Disease alone is multi-billion dollar, with consistent disease presentation across geographies.