Intellia Therapeutics, Inc. reports operational highlights and financial results for the fourth quarter and year ended 31 December 2020.
“Intellia’s achievements in 2020 reflect important progress on both our full-spectrum strategy and our mission to deliver curative genome editing treatments for people with severe diseases”, says Intellia President and CEO John Leonard, M.D. “We are on track to submit first-in-human regulatory applications to begin clinical studies of NTLA-2002 for HAE, and we plan to nominate at least one new development candidate from our research portfolio.”
NTLA-2002 aims to prevent attacks for people living with HAE after a single course of treatment. Intellia is applying its modular LNP delivery system to develop NTLA-2002 to knock out the KLKB1 gene in the liver to permanently reduce plasma kallikrein activity. This approach is expected to provide continuous suppression of kallikrein activity and eliminate the significant treatment burden associated with currently available therapies for HAE patients.
Intellia commenced clinical manufacturing activities to support the Company’s plans to submit an IND or equivalent regulatory application in the second half of 2021.