Prime Medicine (PRME) investor relations material

Strategic and clinical program updates

• Successfully advanced first ex vivo cell therapy program for chronic granulomatous disease, achieving genetic cures in two patients and progressing toward a BLA filing and potential approval.

• Two in vivo programs for Wilson Disease and Alpha-1 antitrypsin deficiency are approaching the clinic, with clinical data expected in 2027; IND filings remain on track.

• Cystic fibrosis program, in partnership with the Cystic Fibrosis Foundation, targets patients unresponsive to standard care, with a hotspot editing approach to address multiple mutations.

• Collaboration with Bristol Myers Squibb on ex vivo CAR T cell therapy using PASSIGE technology is progressing, with significant preclinical milestone payments anticipated.

• Broad pipeline leverages modular LNP delivery for liver and lung programs, with favorable preclinical toxicity profiles and potential for rapid expansion to additional mutations.

Technology differentiation and delivery

• Prime Editing enables single-stranded breaks, reducing off-target effects and chromosomal translocations, and supports a wide range of edits beyond CRISPR/Cas9 and base editing.

• Proprietary modular LNP formulation used for both liver and lung programs shows promising safety and therapeutic index in preclinical studies.

• PASSIGE technology allows for large genomic insertions at precise loci, supporting both ex vivo and in vivo applications, and underpins the BMS CAR T collaboration.

• Delivery remains a key focus, with ongoing efforts to expand tissue targeting beyond liver and lung as delivery technologies mature.

Intellectual property and regulatory landscape

• Holds 10 U.S. and 19 ex-U.S. patents covering prime editing constructs, drug sequences, and delivery systems, with expectations of future royalties from other companies using prime editing.

• Arbitration with Beam Therapeutics over the Alpha-1 program is expected to conclude by late July, with confidence in operating within the Prime field.

• Regulatory flexibility and reduced CMC requirements have enabled reconsideration of commercializing the CGD program, with FDA support for approval based on transformative results in two patients.