The first patient has been dosed with NTLA-2002, Intellia Therapeutics, Inc’s in vivo CRISPR/Cas9 genome editing candidate being developed as a single-dose therapy to prevent attacks in people living with HAE. NTLA-2002 is a systemically administered therapy designed to inactivate the target gene kallikrein B1 (KLKB1) to reduce plasma kallikrein activity and thus prevent HAE attacks.
“With the progress of our first-in-human clinical study evaluating NTLA-2002 for people living with HAE, we look forward to beginning clinical testing as we aim to develop a single-dose treatment for these patients”, says Intellia President and CEO John Leonard, M.D.
Intellia’s multi-national Phase 1/2 study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE. This includes the measurement of kallikrein protein levels and activity as determined by HAE attack rate measures. The Phase 1 portion of the study is an open-label, single-ascending dose design used to identify up to two dose levels of NTLA-2002 that will be further evaluated in the randomized, placebo-controlled Phase 2 portion of the study. This Phase 1/2 study will identify the dose of NTLA-2002 for use in future studies.