Deep Target Mining for the Development of First-in-Class Therapeutics to Treat COPD

Summary

• Chronic obstructive pulmonary disease (COPD) refers to a group of diseases that cause airflow blockage and breathing-related problems (See Figure 1)
• In 2019, COPD was the 3rd largest worldwide cause of death and was responsible for > 3 million deaths
• COPD develops gradually over time, often resulting from tobacco exposure from active smoking or passive exposure to secondhand smoke
• Treatment options are limited and include: bronchodilators, inhaled steroids, and PDE4 inhibitors
• Dr. Crystal, a world leading pioneering authority in the gene therapy space, is developing a novel precision medicine platform to identify causes of smoking induced COPD, and the effects of mitochondrial dysfunction in the development of COPD

Technical Overview

• Dr. Crystal and his team are leading a collaborative effort to identify novel drug targets for COPD
• The team is using in vitro and murine smoking models to focus on mitochondrial dysfunction in the lungs of COPD patients
• Dr. Crystal is also studying COPD pathogenesis using bronchoscopic brushing of small airway epithelium of non-smokers, smokers and COPD patients using a unique and extensive biobank of COPD and control samples of airway epithelial stem and progenitor cells in order to identify therapeutic targets
• The team plans to develop an in vitro phenotypic assay to determine the effects of cigarette smoke on cells and then interrogate potential targets

Market Opportunity

• COPD was the 3rd largest worldwide cause of death in 2019 and was responsible for > 3 million deaths
• In the United States, the estimated direct costs of COPD are $32 billion
• With no effective treatment, costs are predicted to increase further
• The team are working to develop a precision medicine platform to identify novel causes of smoking induced COPD, and the effects of mitochondrial dysfunction in the development of COPD
• This platform will also enable the identification of biomarkers of disease across various patient groups that could be a potentially used as a tool for diagnostics and for measuring disease progression

Partnering Opportunity

Weill Cornell Medicine is seeking an industrial partner with deep domain expertise and a presence in the COPD and respiratory space aimed at the development of novel COPD therapeutics

Figure 1: Pathology of COPD.