With 23 base editors in the clinic, technology’s potential comes into focus

Few emerging therapeutic modalities have demonstrated as strong an early clinical track record as base editing. Of the nearly two dozen programs to reach the clinic, 12 have delivered initial data, all successfully producing the intended gene edit and showing initial signs of clinical activity. Only one program has required adjustments to improve safety.

In conversation with BioCentury, David Liu, whose lab at the Broad Institute of MIT and Harvard invented base and prime editing technologies, noted that these tools are doing for patients what researchers hoped they would. Setbacks observed so far, he said, have largely been linked to the conditioning regimens used for ex vivo programs or the delivery method for in vivo programs...