Prime Medicine wins FDA IND clearance for PM647 in alpha-1 antitrypsin deficiency
PRME•Prime Medicine received FDA clearance for an investigational new drug application for PM647, enabling a Phase 1/2 trial in adults with alpha-1 antitrypsin deficiency. Initial clinical data are expected in 2027.
1. Trial plans
The global, single-arm, open-label study will evaluate a one-time intravenous infusion. Initial enrollment will target patients with lung-only disease; a separate cohort with significant liver disease is planned following tolerability.
