At the presentation of the financial results for the first quarter ended 31 March 2020, Intellia Therapeutics, Inc. President and CEO John Leonard, M.D. says:
“Our mission is to develop curative, CRISPR/Cas9-based treatments for severe diseases. I am grateful to our team for remaining steadfast in our commitment to patients and putting us in the position to deliver on our goals despite the challenging circumstances presented by the global COVID-19 pandemic. We continue to advance the nomination of NTLA-2002 as our development candidate for the treatment of HAE.”
Intellia has nominated its third development candidate, NTLA-2002 for the treatment of HAE. Building on the modular lipid nanoparticle (LNP) delivery system developed for the ATTR (transthyretin amyloidosis) program, NTLA-2002 is designed to knock out the prekallikrein B1 (KLKB1) gene in the liver after a single course of treatment. As part of an ongoing durability study of its lead LNP formulation in support of NTLA-2002, Intellia has now demonstrated six months of sustained therapeutically relevant reduction of serum kallikrein levels and activity following a single dose in non-human primates. Intellia expects to submit an Investigational New Drug (IND) or IND-equivalent for NTLA-2002 in the second half of 2021.
NTLA-2002 is subject to an option by Regeneron to enter into a Co/Co agreement, which must be exercised within a limited time period after development candidate selection. Intellia would be the lead party if the option is exercised.