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.
The skinny
The skinny isn't ready yet — notes appear once the transcript is processed.