A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)

This is a Phase 1, open-label, single ascending dose (SAD) and multiple dose (MD) study of AIR-001 in participants with alpha-1 antitrypsin deficiency (AATD) due to PiZZ genotype.

• : 1. Male or female participants >18 years and 10 kPa defined by screening liver elastography, historical liver biopsy showing ≥ F3 fibrosis (METAVIR or comparable scoring system), or established diagnosis of hepatic cirrhosis. 8. Any of the following screening laboratory abnormalities: 1. Alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma-glutamyl transferase (GGT) > 3 x upper limit of normal (ULN) 2. Total bilirubin > ULN (note: for participants with documented Gilbert's syndrome and direct bilirubin ≤ ULN , exclusion criterion is total bilirubin is > 2.5 mg/dL) 3. INR > ULN (for participants taking stable doses of anticoagulants, the exclusion criterion is INR > 3.0) 4. Platelet count ≤ 150 k/μL 5. Estimated glomerular filtration rate (eGFR) ≤ 60 mL/min/1.73m² by Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) equation 6. Urine Albumin-to-Creatinine Ratio > 300 mg/g 7. Urine Protein-to-Creatinine Ratio > 500 mg/g 9. Prolonged QT interval on electrocardiogram (ECG), defined as QTcF ≥ 450ms (men) or ≥ 470ms (women) 10. ECG findings at screening that render measurements of QT interval imprecise. 11. History of congestive heart failure, serious cardiac arrythmias requiring anti-arrhythmic medications or unexplained black-outs or fainting episodes with a suspected cardiac origin 12. Positive…

Use the source registry for the full inclusion and exclusion criteria before discussing referral or enrolment.