Prime Medicine (PRME) H.C. Wainwright 28th Annual Global Investment Conference summary

Technology overview and differentiation

• Prime Editing enables versatile and precise genome edits, surpassing CRISPR and base editing in scope and safety.

• Utilizes reverse transcriptase and RNA templates to directly write genetic changes, allowing correction of various mutation types and large gene inserts.

• Demonstrates minimal off-target effects and bystander editing compared to other technologies.

• PASSIGE technology enables large, specific gene insertions, enhancing versatility.

Program updates and clinical progress

• Lead program in CGD has treated initial patients, with plans to treat more in in vivo programs.

• Wilson disease program leverages LNP platform for multiple mutations, with regulatory feedback allowing multiple mutations under one IND.

• Alpha-1 program targets regulatory filing in Q3, with clinical data expected in 2027.

• CGD BLA filing targeted for first half of next year, with FDA showing flexibility on requirements.

Clinical trial design and readouts

• Wilson disease trial uses radio-labeled copper PET scans and multiple biomarkers to assess copper metabolism and treatment efficacy.

• PET scans before and after dosing help determine readiness to discontinue standard of care.

• Minimal additional work required for new mutations in Wilson disease due to platform similarities.