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NTLA-3001
- Development stage
- Discontinued
- Lead developer
- Intellia Therapeutics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
**NTLA-3001** was an investigational, wholly owned **in vivo CRISPR/Cas9 targeted gene-insertion therapy** developed by Intellia Therapeutics for **alpha-1 antitrypsin deficiency-associated lung disease**. The systemically administered lipid nanoparticle-based treatment was designed to insert a functional copy of the **SERPINA1** gene, enabling durable production of normal functional alpha-1 antitrypsin protein after a single administration. It entered a Phase 1/2 study in 2024, but Intellia discontinued the program on January 9, 2025 as part of a pipeline prioritization and strategic reorganization.
- Other names
- AAV8-hSERPINA1AAV-8-hSERPINA1AAV 8-hSERPINA1AAV8-hAATAAV-8-hAATAAV 8-hAATAAV8-SERPINA1AAV-8-SERPINA1AAV 8-SERPINA1
02
Targets
SERPINA1 (Alpha-1-antitrypsin)
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