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Trial intelligence / Profile preview
Long-term follow-up of subjects treated with NTLA-2001
2023-507385-11-00Phase 3Authorised
- Sponsor
- Intellia Therapeutics
- Start
- May 2024
- 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
- Countries
- 4
01
Overview
Phase 3, open-label trial of nexiguran ziclumeran in hereditary transthyretin amyloidosis (hATTR) and transthyretin amyloidosis (ATTR) (2 arms), sponsored by Intellia Therapeutics. Primary endpoints: Incidence of treatment-related serious adverse events and Incidence of protocol-specified adverse events of special interest. Planned start May 2024; status: authorised.
02
Drugs
nexiguran ziclumeran
03
Conditions
hereditary transthyretin amyloidosis (hATTR)transthyretin amyloidosis (ATTR)
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