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)
Latest Information Update: 23 May 2025
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.