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Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-2002 for the Treatment of Hereditary Angioedema
NTLA-2002 is the first single-dose genome editing therapeutic candidate designed to prevent attacks in people living with HAE to enter clinical studyNTLA-2002
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"NTLA-2002 is the first single-dose genome editing therapeutic candidate designed to prevent attacks in people living with HAE to enter clinical studyNTLA-2002 is Intellia’s second in vivo CRISPR genome editing therapeutic candidate; program to leverage platform insights gained from ongoing development of NTLA-2001 for transthyretin (ATTR) amyloidosisOn track to initiate patient enrollment by year-end CAMBRIDGE, Mass., Oct. 06, 2021 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, today announced the authorization of its Clinical Trial Application (CTA) by the New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) to initiate a Phase 1/2 study evaluating NTLA-2002 for the treatment of adults with hereditary angioedema (HAE). HAE is a genetic disorder characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. NTLA-2002 is a systemically administered single-dose CRISPR/Cas9-based therapeutic candidate designed to inactivate the target gene Kallikrein B1 (KLKB1) to permanently reduce plasma kallikrein activity and thus prevent HAE attacks. “We look forward to initiating this year our first-in-human study of NTLA-2002 for people living with HAE, a debilitating disorder that causes frequent, potentially life-threatening attacks,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We believe NTLA-2002 has the potential to be a curative therapy for patients with HAE by providing continuous suppression of plasma kallikrein activity following a single dose and eliminating the significant treatment burden associated with currently available HAE therapies. This study of NTLA-2002 leverages early insights from our ATTR amyloidosis program, where we established proof-of-concept for our modular in vivo genome editing platform with interim Phase 1 data earlier this year. The NTLA-2002 program represents the second systemic in vivo CRISPR genome editing therapy candidate to enter human clinical trials.” The Phase 1/2 study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Typ...