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AAT
AAT (Alpha-1 Antitrypsin) is a protease inhibitor that replaces deficient endogenous alpha-1 antitrypsin to protect lung tissue from enzymatic degradation.
AAT is an alpha-1 antitrypsin replacement therapy that replenishes deficient serum levels of the protease inhibitor to prevent progressive lung and liver damage. Used for Alpha-1 antitrypsin deficiency with emphysema, Alpha-1 antitrypsin deficiency with progressive lung disease.
At a glance
| Generic name | AAT |
|---|---|
| Also known as | Alpha-1 proteinase inhibitor, Respreeza |
| Sponsor | Rabin Medical Center |
| Drug class | Protease inhibitor replacement therapy |
| Target | Alpha-1 antitrypsin (serum protein replacement) |
| Modality | Small molecule |
| Therapeutic area | Pulmonology / Hepatology |
| Phase | FDA-approved |
Mechanism of action
Alpha-1 antitrypsin deficiency leads to unopposed neutrophil elastase activity in the lungs, causing progressive tissue destruction. AAT therapy restores the protease-antiprotease balance by supplying exogenous alpha-1 antitrypsin, which inhibits neutrophil elastase and other serine proteases, thereby slowing lung parenchymal damage and decline in pulmonary function.
Approved indications
- Alpha-1 antitrypsin deficiency with emphysema or chronic obstructive pulmonary disease (COPD)
Common side effects
- Infusion reactions
- Headache
- Fatigue
- Dizziness
Key clinical trials
- Role of Genetic Factors in the Development of Lung Disease
- A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1) (PHASE1)
- A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) (PHASE1, PHASE2)
- mHealth Behavior Study (NA)
- Effects of Auricular Acupressure Versus Intermittent Dietary Restriction in Children With Gastric Heat and Dampness Obstruction (NA)
- Gene Therapy for Alpha 1- Antitrypsin Deficiency (PHASE1)
- A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD) (PHASE1)
- Study to Evaluate the Pharmacokinetics and Safety of Oral Decitabine and Cedazuridine in Cancer Patients With Hepatic Impairment (PHASE1)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |