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RepAIR1: AIR-001 in Alpha-1 Antitrypsin Deficiency
NCT07431112Phase 1Recruiting
- Sponsor
- AIRNA Pharmaceuticals
- Start
- Mar 2026
- Primary completion
- Nov 2028
- Modality
- Antisense DNA, Gene Editing, Gene Silencing
- Countries
- 9
01
Overview
Phase 1, open-label trial of AIR-001 in alpha-1 antitrypsin deficiency (AATD) (10 cohorts), sponsored by AIRNA Pharmaceuticals. Primary endpoints: Number of participants with treatment-emergent adverse events at Day 169, Incidence of laboratory abnormalities and shifts from baseline at Day 169 and 2 more. Started Mar 2026; status: recruiting.
02
Drugs
AIR-001
03
Conditions
alpha-1 antitrypsin deficiency (AATD)
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