ASGCT 2025: Physicians and scientists raced to make a bespoke gene editing therapy for an infant who urgently needed one in months - a first hand account, and what this means for drug development

As published in NEJM today, UPenn's Kiran Musunuru describes how the team used base editing in a matter of months to treat an infant with CPS1 deficiently, and his thoughts on what the learnings of this case are for drug development.