In Vivo Gene Editing Emerges as Biotech’s Next Frontier

In Vivo Gene Editing Emerges as Biotech’s Next Frontier

Published Nov 16, 2025

In the last ~14 days the in vivo gene‐editing field accelerated: Azalea Therapeutics (co‐founded by Jennifer Doudna) raised US$82M Series A led by Third Rock to advance a single‐dose dual‐vector permanent genome‐editing approach targeting an in vivo CAR‐T for B‐cell malignancies, aiming for the clinic in 12–18 months; Stylus Medicine secured $85M from investors including J&J and Eli Lilly to build next‐generation in vivo genetic medicines; and Vertex ended its Verve collaboration, reclaiming a liver program while deprioritizing certain delivery platforms. These developments shift investor appetite toward clinically directed, scalable delivery solutions, raise regulatory and safety scrutiny around vectors and durability, and create near‐term catalysts to watch: IND submissions/first dosing, preclinical safety data on novel delivery vectors, and regulatory guideline updates.