Additional Relevant Details Intellia is advancing multiple genome editing strategies that may treat both lung and liver manifestations of AAT deficiency (AATD), which occur due to mutations in the SERPINA1 gene. The normal human AAT protein levels Intellia achieved following targeted insertion of the human SERPINA1 gene remained stable through 11 weeks in an ongoing NHP study. The observed levels of human AAT protein produced from the liver may be therapeutically sufficient to restore protease inhibition to protect the lungs and liver from improperly regulated neutrophil elastase activity. The NHP data build on previous results showing that consecutive in vivo genome editing (knockout plus insertion) achieved therapeutically relevant results in an AATD mouse model.The findings being presented today reinforce recent data showing the use of the same proprietary insertion technology for targeted gene insertion of Factor 9 resulted in circulating human Factor IX, a blood-clotting protein that is missing or defective in hemophilia B patients, that ranged from normal levels (50-150%)1 to supratherapeutic levels in a six-week NHP study. Intellia and Regeneron, the lead party, are co-developing potential hemophilia A and B CRISPR/Cas9-based treatments using their jointly developed targeted transgene insertion capabilities.