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Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

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Trial Profile

Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 04 Feb 2026

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At a glance

  • Drugs Nexiguran-ziclumeran (Primary)
  • Indications Transthyretin-related hereditary amyloidosis
  • Focus Adverse reactions; First in man; Pharmacodynamics; Pharmacokinetics; Proof of concept
  • Sponsors Intellia Therapeutics

Most Recent Events

  • 30 Jan 2026 Status changed from active, no longer recruiting to completed.
  • 11 Nov 2025 According to an Intellia Therapeutics media release, results were shared today in a late-breaking oral presentation at the American Heart Association (AHA) Scientific Sessions 2025 in New Orleans, Louisiana.
  • 11 Nov 2025 Results presented in an Intellia Therapeutics media release.

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