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NTLA-3001 in Alpha-1 Antitrypsin Deficiency-Associated Lung Disease
2023-508138-33-00Phase 2Completed
- Sponsor
- Intellia Therapeutics
- Start
- Nov 2024
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Countries
- 3
01
Overview
Phase 2, open-label, single-arm trial of NTLA-3001 in alpha-1 antitrypsin deficiency emphysema and emphysema, sponsored by Intellia Therapeutics. Primary endpoint: Treatment-emergent adverse events. Started Nov 2024; status: completed.
02
Drugs
NTLA-3001
03
Conditions
alpha-1 antitrypsin deficiency emphysemaemphysema
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