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Prime Medicine (PRME) investor relations material

Prime Medicine Evercore ISI 8th Annual HealthCONx Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Evercore ISI 8th Annual HealthCONx Conference summary2 Dec, 2025

Advances in gene editing technologies

  • Prime editing is described as a versatile and safer genome editing method, enabling precise and permanent corrections, including large insertions and multiple mutation fixes, surpassing the limitations of CRISPR and base editing.

  • Base editing allows single-letter changes with lower off-target effects than CRISPR but is limited in scope and can cause bystander edits within the editing window.

  • Prime editing uses a reverse transcriptase and a DNA template in the guide RNA, enabling correction of various mutation types and larger gene insertions, as demonstrated in ex vivo CAR T cell therapies.

Off-target analysis and safety

  • Extensive in vitro and in vivo assays are used to assess off-target editing, with no evidence of off-target effects in the lead program for chronic granulomatous disease.

  • Assays are developed internally and applied to each drug candidate before IND-enabling studies, ensuring no off-target liability.

  • Both in vitro and in vivo testing are performed, including tissue-specific analysis to confirm targeted editing.

Strategic focus and pipeline prioritization

  • Leadership changes in 2024 led to a strategic refocus on programs with high technical and commercial success probability, reducing the pipeline from 18 programs to a core set.

  • Current priorities include Wilson disease, alpha-1 antitrypsin deficiency, cystic fibrosis, and an ex vivo CAR T program with BMS.

  • Ocular, hearing loss, neurological, and some ex vivo HSC programs were deprioritized, with potential for future partnerships, especially in neuro and liver indications.

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Frequently asked questions

Prime Medicine, Inc. is a biotechnology company specializing in next-generation gene editing technologies aimed at developing curative therapies for a broad range of genetic diseases. The company utilizes its proprietary Prime Editing platform, a highly precise and versatile method of correcting disease-causing mutations in DNA, targeting nearly 90% of known genetic mutations. Prime Medicine's therapeutic focus includes areas like hematology, immunology, liver, ocular, and neuromuscular diseases. The company is headquartered in Cambridge, Massachussetts, and its shares are listed on the NASDAQ.

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