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 across a broad range of diseases.

• The platform is modular, enabling rapid adaptation for different mutations and indications, and leverages a strong intellectual property estate with 10 U.S. and 21 ex-U.S. patents.

• Proprietary lipid nanoparticle (LNP) delivery systems are used across programs, with modular components and targeting ligands to optimize delivery to specific tissues.

• Extensive off-target analysis shows no detectable off-target edits, large deletions, or translocations in lead programs, supporting a favorable safety profile.

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

Clinical and pipeline progress

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

• PM577a efficiently corrects the H1069Q mutation in Wilson Disease, restoring copper metabolism in preclinical models; PM647 restores healthy AAT protein in AATD models.

• The pipeline includes programs for Cystic Fibrosis (CF), with both hotspot and PASSIGE approaches, and ex vivo therapies for Chronic Granulomatous Disease (CGD) and CAR-T cell therapies.

• Proof-of-concept clinical data for PM359 in CGD demonstrated rapid engraftment, restored neutrophil function, and no serious adverse events.

• Strategic partnerships with the Cystic Fibrosis Foundation and Bristol Myers Squibb provide funding and collaboration for CF and CAR-T programs, respectively.

Market opportunity and business strategy

• Addressable markets for lead programs are substantial, with Wilson Disease and AATD affecting tens to hundreds of thousands of patients globally.

• Platform modularity enables rapid expansion to additional mutations and indications, leveraging regulatory and manufacturing synergies.

• The BMS partnership includes $110 million upfront, over $3.5 billion in potential milestones, and royalties on net sales for ex vivo CAR-T products.

• Pro forma cash and equivalents of $108.8M as of June 2026 provide runway into 2027, supporting ongoing development and expansion.

• The strategy focuses on internal pipeline growth in core areas and external partnerships to access expertise and expand into new therapeutic areas.