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Chemistry: Generation 2.5

IONIS-ENAC-2.5Rx is an investigational antisense medicine designed to reduce the production of epithelial sodium channel (ENaC) protein in the lung. Chronic obstructive pulmonary disease (COPD) with chronic bronchitis is characterized by sputum production and a failure of mucus transport in the presence of inflammation. ENaC reduction is believed to increase airway surface hydration and improve mucociliary clearance. In a preclinical mouse model of woodsmoke, reduction of ENaC with antisense oligonucleotides improved lung function. This drug is delivered to the lung via aerosol delivery, which results in broad distribution of antisense oligonucleotides in the lung with minimal systemic exposure.

About COPD

COPD is a chronic inflammatory lung disease that causes obstructed airflow in the lungs. It is a progressive disease and the third leading cause of death globally. There are about 16 million Americans with COPD. It occurs after chronic exposure to environmental factors such as smoke or air pollution but may also have a genetic component that predisposes certain populations to COPD. Emphysema and chronic bronchitis are the two major clinical phenotypes of COPD.

Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.