NTLA-3001 is a potential one-time gene editing treatment that may normalize AAT protein levels and halt the progression of lung disease associated with alpha-1 antitrypsin deficiency (AATD) NTLA-3001 is Intellia's first wholly owned CRISPR-based in vivo targeted gene insertion candidate to advance into the clinic On track to dose the first patient in 2H 2024 "NTLA-3001 is a groundbreaking in vivo CRISPR-based gene insertion candidate designed to durably produce functional AAT protein at normal levels after ...