HOW DOES A DRUG GET DEVELOPED?

Stages of Development for Prime Medicine's AAV Gene Editing

The drug development process can be thought of as a series of stages, and successful drugs must pass through each stage to become available to patients.

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Studies in cells have demonstrated that prime editing technologies can interrupt the disease-causing mutations that occur within the frataxin gene. Prime editing in human cell lines and primary post-mitotic mouse cortical neurons demonstrated prime editing with varying efficiencies. Prime editing shows higher or similar efficiency and fewer by-products than homology-directed repair, has complementary strengths and weaknesses compared to base editing, and induces much lower off-target editing than Cas9 nuclease at known Cas9 off-target sites.

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News | Jan 9, 2023

Prime Medicine announces recent progress and highlights 2023 strategic priorities

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