Prime Medicine (PRME) Morgan Stanley 23rd Annual Global Healthcare Conference summary

Strategic priorities and capital allocation

• Focused on de-risking and prioritizing programs with high success probability and unmet need, especially in liver diseases and cystic fibrosis.

• Wilson’s disease and alpha-1 antitrypsin deficiency (AATD) are prioritized due to delivery feasibility and commercial opportunity.

• Cystic fibrosis program is advancing with external funding, while ex vivo CAR-T cell therapy is progressing through a collaboration.

• Long-term value expected from neurological and other indications, leveraging unique Prime Editing capabilities.

• Vision includes creating value over the next five to ten years, with potential future proprietary or partnered programs.

Technology differentiation and regulatory landscape

• Prime Editing offers safer, more precise editing than CRISPR-Cas9, avoiding double-stranded breaks and off-target effects.

• Enables insertion of multiple base pairs, expanding the range of treatable diseases.

• Regulatory environment is increasingly supportive, with new FDA pathways for rare and difficult-to-treat indications.

• Prime Editing’s ability to restore wild-type protein without bystander edits is a key differentiator.

• Reduced off-target effects may ease regulatory discussions and approvals.

Clinical and preclinical program updates

• Chronic granulomatous disease program showed strong biomarker and clinical responses but is discontinued due to limited commercial potential.

• Plans to discuss potential regulatory paths with the FDA for this program within six months.

• High editing efficiency demonstrated in human cells and preclinical models, with delivery being the main challenge for broader application.

• IND submissions for Wilson’s disease and AATD targeted for first half and mid-2026, respectively, with data expected in 2027.

• Preclinical data for both programs show high editing efficiency and strong phenotypic correction at low doses.