vchal/iStock via Getty Images
The U.S. Food and Drug Administration (FDA) cleared Intellia Therapeutics’ (NASDAQ:NTLA) application seeking to start a trial of CRISPR-based gene editing therapy, NTLA-2002 in the U.S. to treat hereditary angioedema (HAE).
The clearance of the investigational new drug (IND) the company to include the U.S. in a global phase 2 portion of its ongoing phase 1/2 study.
Intellia noted that NTLA-2002 is an in vivo genome editing therapy aimed to inactivate the target gene kallikrein B1 (KLKB1) to permanently reduce plasma kallikrein protein activity and thereby prevent HAE attacks after a single-dose treatment.
HAE is a disorder characterized by recurrent episodes (attacks) of severe swelling of the skin and mucous membranes.
“We are thrilled to advance the development of NTLA-2002 in the U.S. and are working to rapidly enroll patients in the Phase 2 portion of the study. We look forward to presenting additional data from the first-in-human, Phase 1 portion of the study later this year,” said Intellia’s President and CEO John Leonard.
The company had reported initial data from the phase 1/2 trial in September 2022.
Intellia noted that the phase 2 portion of the study recently began patient screening outside the U.S.
NTLA +9.01% to $44.42 premarket March 3