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MAGNITUDE: NTLA-2001 in Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)
jRCT2053250023Phase 3Recruiting
- Sponsor
- Intellia Therapeutics
- Start
- Jun 2025
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
01
Overview
Phase 3, randomised, double-blind trial in transthyretin amyloidosis (ATTR), hereditary transthyretin amyloidosis (hATTR) and acquired transthyretin amyloidosis (ATTRwt) comparing NTLA-2001 55 mg with Placebo (2 arms). Primary endpoint: Composite cardiovascular outcome. Started Jun 2025; status: recruiting.
02
Drugs
nexiguran ziclumeran
03
Conditions
transthyretin amyloidosis (ATTR)hereditary transthyretin amyloidosis (hATTR)acquired transthyretin amyloidosis (ATTRwt)
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