AAV Gene Editing
Prime Editing is a next-generation gene editing technology that acts like a DNA word processor, with the power to search and replace genetic sequences at their exact location in the genome, all without making double-strand breaks in DNA. Prime Editors are designed to produce precise edits and have editing activity across many different tissues, organs, and types of cells. Prime Editors work broadly across almost all types of genetic mutations at the natural genomic location, while minimizing unwanted DNA modifications. In the case of FA, prime editing will be used to delete the GAA repeat sequence in the frataxin gene.
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.
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.