Implications of the FDA’s new plausible mechanism framework for the development of a personalized in vivo prime editing platform

The authors report initial proof-of-concept studies supporting a customizable prime editing platform geared to the treatment of 7 urea cycle disorders and other liver-centered disorders, as well as the outcome of a formal meeting with the FDA to discuss an “umbrella-of-umbrellas” clinical trial under the new plausible mechanism framework.