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OS005
- Development stage
- Preclinical
- Lead developer
- OmniSpirant
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Inhalation
01
Overview
OS005 is a preclinical-stage therapeutic candidate being developed by OmniSpirant for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The drug utilizes OmniSpirant's proprietary OmniSome platform, which involves bioengineered stem cell-derived extracellular vesicles (exosomes) designed for inhaled delivery. OS005 is engineered to deliver a corrective therapeutic payload—likely the Alpha-1 Antitrypsin (AAT) protein or the mRNA encoding it—directly to the lung epithelium and alveolar macrophages. By delivering the functional protein or its genetic instructions to the site of disease, OS005 aims to restore the protease-antiprotease balance in the lungs, thereby preventing the progressive tissue destruction and emphysema associated with AATD.
02
Targets
SERPINA1 (Alpha-1-antitrypsin)ELANE (Human Neutrophil Elastase)
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