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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: Active, no longer recruiting
Phase of Trial: Phase I

Latest Information Update: 23 May 2025

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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

  • 18 May 2025 According to an Intellia Therapeutics media release,positive two-year follow-up data from this study were shared in an oral presentation on Sunday, May 18 at the 2025 Peripheral Nerve Society (PNS) Annual Meeting in Edinburgh, United Kingdom.
  • 18 May 2025 Results presented in the Intellia Therapeutics Media Release
  • 08 May 2025 According to an Intellia Therapeutics media release, the company expects to present longer-term data from both ATTR-CM and ATTRv-PN patients in the second half of 2025.

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