BioCentury
ARTICLE | Discovery & Translation

Taking ‘n-of-1’ base editing therapies into broader populations: Q&A with David Liu

Liu’s newest tech targets a class of mutations rather than a specific gene for cross-disease activity

October 22, 2025 11:22 PM UTC

Recent technology advancements promise to move base editing beyond highly personalized medicines to drugs that work across many rare diseases, with the Broad Institute’s David Liu once again at the forefront.

Today, developing a base editing therapy still requires bespoke design and intensive coordination across academic and clinical teams. Cross-sector efforts are often the only way to bring new gene-editing solutions to patients whose disorders are too rare to attract traditional industry investment...