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Aerosolized, Recombinant Alpha 1-Antitrypsin
- Development stage
- Phase 2
- Lead developer
- Takeda
- Modality
- Recombinant Proteins and Enzymes
- Administration
- Inhalation
01
Overview
Alpha 1-antitrypsin (A1AT) is a serpin protein that functions as a protease inhibitor, primarily protecting tissues from the destructive effects of neutrophil elastase, an enzyme released by inflammatory cells. Alpha 1-antitrypsin Deficiency (A1ATD) is a genetic disorder characterized by low levels of A1AT, leading to conditions such as emphysema and liver damage. "Respriva" is believed to be a code name for an aerosolized, recombinant form of Alpha 1-Antitrypsin (rAAT) under investigation for the treatment of Alpha 1-Antitrypsin Deficiency. This recombinant protein is designed to be delivered via inhalation to augment A1AT levels in the lungs, thereby inhibiting neutrophil elastase and preventing further tissue degradation. The development of this drug was initiated by Baxalta, which is now part of Shire.
- Other names
- Alpha 1-AntitrypsinAlpha1-AntitrypsinAlpha-1-AntitrypsinA1ATA-1ATA 1AT
02
Targets
ELANE (Human Neutrophil Elastase)
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