Intellia Therapeutics ( NTLA Quick Quote NTLA - Free Report) announced the initiation of a phase I/II study to evaluate its investigational CRISPR/Cas9 therapy NTLA-2002 as a single-dose therapy to prevent angioedema attacks in patients with hereditary angioedema (HAE). The study will identify the dosage of NTLA-2002 for use in future studies.
Intellia designed the study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE.
While the phase I portion of the study will identify upto two doses of NTLA-2002, the phase II will evaluate the doses already identified in the phase I portion.
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Following initiation of the phase I/II study, NTLA-2002 is the first single-dose CRISPR/Cas9 therapy, designed to prevent attacks in HAE patients, to enter clinical studies.
A rare genetic disorder, HAE is marked by severe inflammatory attacks in various body organs and tissues, which can be painful and even life-threatening. Despite the current treatment options, which require chronic administration for disease control, HAE attacks still occur. Per management, one in 50,000 people is affected by HAE.
NTLA-2002, which utilizes the CRISPR/Cas9 technology, will be administered systematically via intravenous infusion. The therapy is designed to inactivate the kallikrein B1. This will lead to the reduction of plasma kallikrein activity and in turn, should prevent HAE attacks. A reduction in kallikrein activity is clinically validated in preventing HAE attacks.
NTLA-2002 is Intellia’s second candidate to move to the clinical studies. NTLA’s first candidate to enter into clinical development is NTLA-2001, which is being evaluated in a phase I study for transthyretin amyloidosis with polyneuropathy (ATTRv-PN). Intellia plans to present interim data from the study in first-quarter 2022.
Zacks Rank & Other Stocks to Consider
Intellia presently carries a Zacks Rank #2 (Buy). Other top-ranked stocks in the biotech/drug sector include
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