Professor Marcus Mall is chair of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité-Universitätsmedizin Berlin, Germany. He is a physician-scientist and specialist in pediatric pulmonology and one of the founding directors of the German Center for Lung Research (DZL). His research focusses on cystic fibrosis, which to date remains the most common fatal hereditary lung disease worldwide.
Mall has made several groundbreaking contributions to unraveling of disease mechanisms underlying cystic fibrosis including the development of the first animal model with cystic fibrosis-like lung disease. Building on his basic research on cystic fibrosis, he played a key role in translating research findings into the development of small molecules (so called CFTR modulators) that target the underlying molecular defect in patients. Unraveling of the genetic defect in the CFTR gene and disease mechanisms that cause cystic fibrosis along the translational value chain has led to the development of a highly effective triple combination therapy of small molecules that targets the underlying molecular defect and leads to unprecedented improvement in lung function and quality of life in the majority of patients with this devastating disease.