Gene editing · Gene replacement: AAT (SERPINA1) · developed by Intellia Therapeutics
NTLA-3001 is an in vivo CRISPR/Cas9 gene editing therapy delivered by AAV for the treatment of alpha-1 antitrypsin deficiency (AATD), a genetic lung disease caused by deficiency of the AAT protein.
Summary written with AI from the sources listed at the end of this page.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT06622668 | Phase 1/2 | Lung Disease | Withdrawn | NTLA |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
AATDAlpha-1 Antitrypsin DeficiencyAlpha-1 Antitrypsin Deficiency-associated Lung DiseaseLung DiseasePulmonary Disease
Updated Oct 3, 2026. Something wrong or missing? Report it on the NTLA page.
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