Prime Medicine (PRME) Goldman Sachs 45th Annual Global Healthcare Conference summary

Company progress and technology overview

• Prime Editing technology has advanced rapidly, moving from a single lab to an open IND in under four years.

• The platform demonstrates robust, versatile, and safe gene editing with minimal off-target effects.

• Transitioning to a clinical-stage company, with clinical data expected next year.

• Pipeline has become more focused, with clear guidance on upcoming programs.

Differentiation and intellectual property

• Prime Editing can correct over 90% of known pathological mutations, handling both small and large edits.

• PASSIGE technology enables insertion of large DNA segments at precise locations.

• Prime Editing's IP is robust, with broad patents originating from the inventors and covering key aspects of the technology.

Clinical development and CGD program

• First IND targets chronic granulomatous disease (CGD), a severe childhood-onset immune disorder.

• Current standard of care is allogeneic bone marrow transplant, which is risky and often limited by donor availability.

• Prime Editing offers autologous transplants with corrective edits, aiming for a one-time cure.

• CGD was prioritized due to technical success, ex vivo delivery simplicity, and high unmet need.

• Patients are easily identified and concentrated in major centers, facilitating trial enrollment.