ORP100S is an inhaled modified thioredoxin drug candidate that normalizes mucus in cystic fibrosis patients.
ORP100S is a modified thioredoxin with enhanced activity, increased time on target, and minimal cellular uptake, drawn from the patent-protected Theradux platform. Natural thioredoxin has a poor half-life in the circulation and is readily taken up into cells, where it can exert adverse side effects, so the protein was optimized for delivery by inhalation to its site of action on the airway surface. Key CMC milestones covering manufacturing, formulation, and stability have been achieved, and preclinical development of ORP100S is in progress.
ORP100S is an inhaled protein disulfide reductase that targets the abnormal linkages in cystic fibrosis mucus proteins linked to increased viscosity and stiffness. Unlike non-selective mucolytic drugs, it relaxes mucus gels without disrupting the polymeric structure essential for normal mucus clearance, and it retains activity across a broad airway pH range. It is being developed for patients with cystic fibrosis and other obstructive airway diseases.
