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.
Programmable and highly flexible
High fidelity and specificity
High editing efficiency
Minimal off-target activity
Potential for improved gene function through in situ editing
Edits in multiple clinically relevant rapidly dividing, non-dividing, mammalian, non-mammalian cell types and organs
No DNA double-strand breaks
Validated by multiple independent research laboratories worldwide
Prime Editing can correct almost all types of gene mutations and can be used to modify gene-regulatory sequences. These edits can be made in therapeutically relevant cells and organs, including specialized terminally-differentiated cells. A single Prime Editor can correct the individual mutations found across patients, meaning that Prime Editing can potentially address more than 90% of known disease-causing genetic mutations.
Prime Editing occurs with high fidelity, making the correct edit at the exact target site with minimal off-target activity. Prime Editing does not cause double-strand breaks and does not affect cell viability, which may contribute to better patient outcomes, fewer side effects and overall improved safety.
Prime Editing makes a permanent correction at the natural place in the genome. As a result, the corrected gene returns to physiologic regulation. This can be especially important where gene dosage is critical to protein function. Other approaches may not faithfully restore gene function. These advantages can provide patients with long-lasting benefit, complete correction and a durable cure.
Prime Editing can be delivered via multiple modalities including RNA, DNA, and RNA–protein complexes, unlocking opportunities for genetic medicine. By taking advantage of proven delivery technologies, we can choose the best method to deliver Prime Editing depending on where it needs to go in the body. This flexibility provides opportunities to fix the underlying causes of genetic disorders, treat common diseases, and target cancers with treatments that are easily distributed, manufactured at scale, and re-dosed if necessary.