Respiratory Diseases

Idiopathic pulmonary fibrosis (IPF) is a progressive lung disease in which scar tissue forms in the lungs, making it increasingly difficult to breathe. Scripps Research and Calibr-Skaggs teams are developing CMR316: an inhaled, lung-targeted drug designed to stimulate stem cells in the lungs’ lower airways, known as type 2 alveolar epithelial cells (AEC2s). CMR316 works by expanding the number of AEC2s, which then mature into AEC1s—the cells responsible for gas exchange and maintaining lung function. In addition to IPF, the team is hopeful the regenerative medicine also can be used to treat other chronic pulmonary conditions, including chronic obstructive pulmonary disease (COPD) and emphysema.

Alpha-1 antitrypsin deficiency (AATD) is a genetic lung disease that affects more than 100,000 people in the United States and leads to one form of chronic obstructive pulmonary disease (COPD). Scripps Research scientists have discovered how to coax the mutated gene implicated in the disease into a correctly folded protein.

Sarcoidosis is a lung disease characterized by granulomas: tiny clumps of immune cells that form in response to inflammation. It’s the most inflammatory of the interstitial lung diseases (ILDs), a family of conditions that all involve some level of pulmonary inflammation and fibrosis. Scientists characterized a protein that can soothe the inflammation associated with sarcoidosis by regulating white blood cells—an approach that is now in clinical testing.