“We are progressing NTLA-2002 for the treatment of HAE, to a regulatory submission next year”, says President and CEO John Leonard, M.D., at the presentation of Intellia Therapeutics, Inc. operational highlights and financial results for the third quarter ended 30 September 2020.
Third Quarter 2020 and Recent Operational Highlights – HAE Program:
NTLA-2002 is a wholly owned, in vivo development candidate for the treatment of HAE. Today, Intellia announced results from its completed non-human primate (NHP) study of its lead lipid nanoparticle (LNP) formulation for NTLA-2002. Following a single dose, the knockout of the prekallikrein B1 (KLKB1) gene resulted in a year-long therapeutically relevant reduction of serum kallikrein protein levels and activity. Building on Intellia’s modular LNP delivery system, NTLA-2002 is designed to knock out the KLKB1 gene in the liver after a single course of treatment. This approach is expected to prevent improperly regulated bradykinin production and therefore reduce HAE attacks. During the third quarter, the Company initiated Good Laboratory Practices (GLP) toxicology studies in preparation for an IND or IND-equivalent submission for NTLA-2002, which remains on track for the second half of 2021.