Prime Medicine (PRME) investor relations material

Strategic priorities and capital allocation

• Focused on programs with high probability of early de-risking, unmet medical need, and strong commercial potential, especially two liver programs: Wilson’s disease and Alpha-1 antitrypsin deficiency.

• Cystic fibrosis program is advancing with funding from the Cystic Fibrosis Foundation; delivery remains a challenge but progress is ongoing.

• Ex vivo CAR-T cell therapy collaboration with Bristol Myers Squibb is progressing, with potential for future proprietary and partnered programs in neurological and other diseases.

• Long-term vision includes leveraging Prime Editing’s unique capabilities for neurological and other high-value indications.

• Capital allocation is focused on core programs, with future expansion possible as opportunities arise.

Differentiation and technology platform

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

• The technology enables writing multiple base pairs, expanding the range of treatable diseases beyond what base editing or CRISPR can achieve.

• Regulatory outlook is favorable due to the platform’s ability to restore wild-type protein without bystander edits, potentially easing approval pathways.

Clinical and preclinical program updates

• First clinical program in chronic granulomatous disease showed high editing efficiency and strong biomarker response, but limited commercial opportunity led to no further investment.

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

• High editing efficiency demonstrated in multiple tissue types; delivery is the main focus for in vivo applications, with lipid nanoparticles used for liver targeting.