Intellia Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application
Thursday, March 02, 2023
Intellia Therapeutics announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for NTLA-2002 for the treatment of hereditary angioedema (HAE), enabling the company to include the United States in the global Phase 2 portion of its ongoing Phase 1/2 study. NTLA-2002 is an in vivo genome editing candidate designed to inactivate the target gene, kallikrein B1 (KLKB1), to permanently reduce plasma kallikrein protein activity and thus prevent HAE attacks after a single-dose treatment.
NTLA-2002 is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational CRISPR therapeutic candidate designed to inactivate the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. NTLA-2002 is the second investigational CRISPR therapeutic candidate to be administered systemically, by intravenous infusion, to edit disease-causing genes inside the human body with a single dose of treatment.
The FDA’s acceptance of IND application to initiate clinical evaluation of NTLA-2002 brings one step closer to introduce a potentially paradigm-shifting treatment for people living with hereditary angioedema.
