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Intellia Therapeutics Initiates Phase 1/2 Trial of NTLA-3001 for AATD-Associated Lung Disease

  • Intellia Therapeutics has launched a Phase 1/2 clinical trial (NCT06622668) to assess NTLA-3001 in adults with Alpha-1 Antitrypsin Deficiency (AATD)-associated lung disease.
  • The study will evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in AATD patients.
  • Phase 1 is an open-label, single-arm, ascending dose study to determine the safety and optimal dose of NTLA-3001, while Phase 2 is a dose expansion study to further assess safety and clinical activity.
  • NTLA-3001's impact on pulmonary function will be initially evaluated in Phase 2, offering a potential new therapeutic avenue for AATD-related lung conditions.
Intellia Therapeutics has commenced a Phase 1/2 clinical trial (NCT06622668) to investigate NTLA-3001 as a potential treatment for lung disease associated with Alpha-1 Antitrypsin Deficiency (AATD). The trial aims to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in adult patients diagnosed with AATD-related lung conditions. This study marks a significant step forward in exploring new therapeutic options for individuals affected by this genetic disorder.

Trial Design and Objectives

The clinical trial is structured into two phases. Phase 1 is designed as an open-label, single-arm, ascending dose study. This initial phase will focus on characterizing the safety profile and activity of NTLA-3001, with the primary goal of identifying an appropriate dose for further evaluation in Phase 2. Subsequently, Phase 2 will proceed as an open-label, dose expansion study. This phase aims to further characterize the safety and clinical activity of NTLA-3001, while also providing an initial assessment of the drug's effect on clinical measures of pulmonary function.

NTLA-3001 and AATD

Alpha-1 Antitrypsin Deficiency (AATD) is a genetic condition that can lead to severe lung disease. NTLA-3001 represents a novel therapeutic approach, and this trial is designed to provide critical data on its potential to address the unmet medical needs of AATD patients. The trial's endpoints include assessing changes in pulmonary function tests and other relevant clinical measures. The initiation of this trial signifies an important milestone in the development of new treatments for AATD-associated lung disease.
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Reference News

[1]
Clinical trial: Alpha-1 Antitrypsin Deficiency, AATD, (NCT06622668) - CRISPR Medicine
crisprmedicinenews.com · Nov 3, 2024

Study to assess NTLA-3001's safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics in adults wi...

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