- The stock price of Intellia Therapeutics Inc (NASDAQ: NTLA) increased over 5% during intraday trading today. This is why it happened.
The stock price of 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 – increased over 5% during intraday trading today. Investors have been responding positively to the company recently reporting 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 known as 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. And 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.
The Phase 1/2 study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE. And 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 and single-ascending dose design used to identify up to 2 dose levels of NTLA-2002 that will be further evaluated in the randomized and 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.
Beyond New Zealand, Intellia has submitted additional regulatory applications in other countries as part of its ongoing, multi-national development approach for NTLA-2002.
“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. 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.”
— Intellia President and Chief Executive Officer John Leonard, M.D.
Disclaimer: This content is intended for informational purposes. Before making any investment, you should do your own analysis.
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