FDA clears in vivo gene editing therapy for trials in US


The US Food and Drug Administration (FDA) has cleared Intellia Therapeutics’ Investigational New Drug (IND) application for NTLA-2002 for the treatment of hereditary angioedema (HAE). 

Intellia is a clinical-stage genome editing company focused on developing potentially curative therapies using CRISPR-based technologies.  

The decision will enable the company to include the United States in the global Phase II portion of its ongoing Phase I/II 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. 

“The FDA’s acceptance of our IND application to initiate clinical evaluation of NTLA-2002 brings us one step closer to introducing a potentially paradigm-shifting treatment for people living with hereditary angioedema,” said Intellia President and Chief Executive Officer, John Leonard.  

“We are thrilled to advance the development of NTLA-2002 in the US and are working to rapidly enroll patients in the Phase II portion of the study. We look forward to presenting additional data from the first-in-human, Phase I portion of the study later this year.” 

Intellia’s multi-national Phase I/II study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE. This includes the measurement of plasma kallikrein protein levels and clinical activity as determined by HAE attack rate measures.  

The Phase I portion of the study is an open-label, single-ascending dose design used to identify two dose levels of NTLA-2002 that will be further evaluated in the randomised, placebo-controlled Phase II portion of the study.  

In 2022, Intellia reported positive interim results from the Phase I study demonstrating deep, dose-dependent reductions in plasma kallikrein and robust reductions in patient HAE attacks. 

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