Prime Medicine obtained FDA IND approval for PM-647, an investigational in vivo Prime Editor targeting α-1 antitrypsin deficiency (AATD). The first-in-human Phase I/II trial will evaluate a one-time intravenous infusion in adults with lung and liver manifestations of AATD. Prime editing represents a gene-editing approach designed to introduce targeted genetic changes with reduced DNA double-strand breaks, and IND clearance moves the program into clinical dose exploration. For AATD, a disease with limited mechanism-based options, this is a step toward potentially durable correction strategies rather than lifelong augmentation therapy.