Prime Medicine (PRME) Morgan Stanley 24th Annual Global Healthcare Conference summary

Advances in gene editing technologies

• Prime Editing enables all 12 base changes and large gene insertions, surpassing earlier CRISPR and base editing methods in versatility and safety.

• PASSIGE technology allows for near full gene insertion, expanding therapeutic possibilities.

• Prime Editing minimizes off-target effects and chromosomal rearrangements compared to previous technologies.

Delivery challenges and organ targeting

• Effective gene editing delivery to the liver is established, but expanding to organs like the brain, heart, and kidney remains challenging.

• Early preclinical data show promising delivery to the eye and ear, with lung delivery advancing through cystic fibrosis programs.

• Success in brain delivery could unlock significant new therapeutic opportunities within 5–10 years.

• Lung delivery is progressing, with LNP, AAV, and HSV technologies showing promise for cystic fibrosis.

Regulatory environment and FDA interactions

• FDA has shown flexibility, allowing approval filings based on data from as few as two patients in rare diseases.

• Recent leadership stability at CBER and CDER is expected to support continued progress in gene editing approvals.

• Constructive and ongoing dialogue with the FDA is reported, with a focus on unmet needs and rare indications.