OS001

OS001 is a first-in-class regenerative gene therapy being developed by OmniSpirant for the treatment of cystic fibrosis (CF). The therapy utilizes the proprietary OmniSomes platform, which consists of bioengineered stem cell-derived extracellular vesicles (exosomes) that are surface-engineered to deliver a functional copy of the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene directly to lung airway epithelial cells. Administered via inhalation as an aerosol, OS001 is designed to efficiently penetrate the thick mucus barrier characteristic of CF lungs to restore CFTR expression and function regardless of the patient's specific genetic mutation. In addition to gene delivery, the stem cell-derived exosomes possess inherent anti-inflammatory, regenerative, antimicrobial, and antifibrotic properties, potentially offering a comprehensive treatment for both the underlying genetic cause and the chronic lung damage associated with CF.

Other names

OmniSpirant-CFTRCFTR-EGFPOmniSomes-delivered CFTR