Base Editing: A Strong Contender in Cell and Gene Therapy

Base editors, DNA double-strand break (DSB)-independent genome modification agents, have been recognized alongside prime editors as key technologies to watch this year,¹ and advances to date have been encouraging. Although these technologies have yet to enter the clinic, the first candidate therapy received investigational new drug approval from the U.S. Food and Drug Administration in 2021—less than five years after the first base editor was described.²