NTLA-2003

NTLA-2003 is an investigational **in vivo CRISPR-based gene editing therapy** from **Intellia Therapeutics** being developed for **alpha-1 antitrypsin deficiency-associated liver disease**. The program is designed as a **knockout** approach that uses **CRISPR/Cas9** delivered by **lipid nanoparticles** to edit hepatocytes and inactivate the **SERPINA1** gene, with the goal of reducing production of the misfolded mutant alpha-1 antitrypsin protein that accumulates in the liver and drives hepatic injury. It is a wholly owned Intellia program and, based on company pipeline materials and public reporting, remained a preclinical candidate that entered IND-enabling work in 2023 before later reports indicated Intellia halted further preclinical activities in favor of another AATD program.