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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.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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