Prime Medicine announced FDA clearance of the IND for PM647, its in vivo Prime Editor for Alpha-1 Antitrypsin Deficiency (AATD), enabling a global Phase 1/2 trial. The study will assess safety and early efficacy of a one-time IV infusion, starting with lung manifestations and expanding to liver disease. A large PiZZ patient base and platform reuse of PM577a's LNP could support future upside if early results are favorable.
