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Phase 3 study of NTLA-2001 in transthyretin amyloidosis with cardiomyopathy
NL-OMON59355Phase 3Recruiting
- Sponsor
- Intellia Therapeutics
- Start
- Nov 2024
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial in transthyretin amyloidosis (ATTR) comparing ziclumeran with placebo (2 arms). Primary endpoint: Composite outcome of cardiovascular mortality and cardiovascular events. Started Nov 2024; status: recruiting.
02
Drugs
nexiguran ziclumeran
03
Conditions
transthyretin amyloidosis (ATTR)
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