Intellia Therapeutics, Inc. outlines its expected 2021 milestones and strategic priorities regarding HAE therapy:
“Since our founding, we set out to develop modular platform components that could serve as the engine powering an expansive portfolio of curative therapeutics. We have paved a rapid and reproducible development path for both in vivo and engineered cell therapies to address serious genetic diseases and cancers. Over the next 12 months, we anticipate first-in-human regulatory submissions for NTLA-2002,” says Intellia President and CEO John Leonard, M.D.
NTLA-2002 aims to prevent attacks and eliminate the current, significant treatment burden for people living with HAE after a single course. 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.
- Intellia plans to submit an IND or equivalent regulatory application in the second half of 2021.
- The Company is applying insights gained from NTLA-2001 to expedite clinical development of NTLA-2002.