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Phase 1/2 Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-3001 in Participants with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease

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Trial Profile

Phase 1/2 Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-3001 in Participants with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease

Status: Discontinued
Phase of Trial: Phase I/II

Latest Information Update: 20 Feb 2025

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At a glance

  • Drugs NTLA-3001 (Primary)
  • Indications Alpha 1-antitrypsin deficiency
  • Focus Adverse reactions; First in man
  • Acronyms AATD

    • Most Recent Events

    • 14 Feb 2025 According to an Intellia Therapeutics media release, Intellia investors filed a complaint against the company. According to the complaint, company initially expressed confidence in its timeline for this study and expected to dose the first patient in the 2H2024. However, the complaint alleges that Intellia did not disclose the declining demand for viral-based editing, which ultimately led to the discontinuation of NTLA-3001 as part of a company reorganization announced on January 9, 2025.
    • 14 Feb 2025 Status changed from withdrawn prior to enrolment to discontinued due to shifting priorities and declining demand for viral-based editing.
    • 17 Jan 2025 Planned End Date changed from 1 May 2030 to 9 Jan 2025.

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