Drug intelligence / Profile preview

recombinant alpha-1 antitrypsin

Development stage
Phase 2
Lead developer
Sanofi
Modality
Recombinant Proteins and Enzymes
Administration
Intravenous, Inhalation (dry Powder), Topical
01

Overview

**Recombinant alpha-1 antitrypsin** is a genetically engineered form of the serine protease inhibitor alpha-1 antitrypsin, produced from non-human sources (such as yeast or bacteria) to avoid the limitations and risks associated with plasma-derived products. It functions primarily by irreversibly inhibiting neutrophil elastase and other serine proteases, protecting tissues (especially lung parenchyma) from enzymatic degradation. Recombinant AAT is investigated for treatment of alpha-1 antitrypsin deficiency (AATD), which predisposes to early-onset emphysema, as well as in inflammatory and immune-mediated diseases such as graft-versus-host disease, type 1 diabetes, rheumatoid arthritis, and others due to its anti-inflammatory and immunomodulatory effects. Compared to plasma-derived AAT, recombinant formulations aim to improve safety (no blood-borne pathogen risk) and supply, and allow for alternative administration routes (e.g., inhaled/dry powder). Variant molecules like INBRX-101 and fusion proteins (AAT-IgG1 Fc) are designed to prolong half-life or enable less frequent dosing. Topical formulations (e.g., Dermolastin) have also been investigated for dermatologic indications. Recombinant AAT is still in development and not yet approved as a medication for AATD or other conditions[3][4][1][5][2].

Brand names
Dermolastin
Other names
rAATrecombinant alpha 1-antitrypsinrecombinant human alpha-1 proteinase inhibitor
02

Targets

ELANE (Human Neutrophil Elastase)CTSG (Cathepsin G)PRTN3 (Proteinase 3)PRSS1 (Trypsin family)

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