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Drug

NTLA-2001

In clinical development
Emerging researchLate-stage development

Also known as nexiguran ziclumeran.

RxNorm2666903
1
Research papers
2
Active clinical trials
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)

Clinical trial2026-04-16Results expected Q3 2027 · ClinicalTrials.gov

MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Clinical trial2026-03-27Results expected Q4 2027 · ClinicalTrials.gov

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)

Clinical trial2025-09-12Completed · ClinicalTrials.gov

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.

Research2021-06-26The New England journal of medicine

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
NTLA-2001
Aliases & brands
nexiguran ziclumeran
RxNorm CUI
2666903

Clinical trials

3 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
3
registered trials across all phases
LATEST COMPLETION 2025
2
Active studies
2
Recruiting
2
Late-stage (III+)
1
Completed
PHASE DISTRIBUTIONn = 3
Phase 11Phase 32

Research activity

1 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Most influential

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.

The New England journal of medicine · 2021 · 1,256 cites
Recent papers

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.

The New England journal of medicine · 2021 · 1,256 cites
Major research themes2
CRISPR-Cas Systems1Gene Editing1
Journals, researchers & institutions
Top journals
  • The New England journal of medicine1
Leading researchers
  • Amaral A1
  • Boyd AP1
  • Cehelsky JE1
  • Fontana M1
  • Gane E1
  • Gillmore JD1
  • Gutstein DE1
  • Harari O1
Leading institutions
free-text, unnormalised
  • From the National Amyloidosis Centre1

Related drugs

1 matches

Drugs sharing diseases, protein targets and literature with this one. Ranked by graph evidence (shared targets + diseases weighted above co-mentions).

Liposomes

Studied across the same disease area as NTLA-2001 in the shared literature.

1 shared disease1 shared paper
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials