Pulmonary Diseases

Targeting the Lungs to Breathe Easier

Our Focus

Cystic fibrosis (CF) lung disease

Cystic fibrosis (CF) is the most common fatal inherited disease in the United States and Europe, and it results from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CF causes impaired lung function, inflammation, and bronchiectasis and is commonly associated with repeat and persistent lung infections due to the inability to clear thickened mucus from the lung, often resulting in frequent exacerbations and hospitalizations. Current treatments that address the CFTR gene works for 90% of the CF population; however, the improvement in lung function is incomplete and is variable. In addition, ~5% of the population cannot tolerate CFTR modulators. Current research is ongoing for the final 15% not amenable to modulators, and for the remaining population with incomplete response on modulators.

Pulmonary Clinical Trials

About 4D-710 for the treatment of cystic fibrosis lung disease

4D-710 is being evaluated in the currently-enrolling AEROW clinical trial, a Phase 1/2 dose-escalation and dose-expansion clinical trial in patients with cystic fibrosis who are ineligible for CFTR modulator therapy or who have discontinued therapy due to adverse effects. The primary endpoints of the study are safety, tolerability, and definition of the maximum-tolerated dose and/or Phase 2 dose level. Secondary endpoints include assessments of clinical activity, including quality-of-life and lung function, plus exploratory endpoints on the feasibility of detecting transgene transfer and CFTR transgene expression as measured in bronchoscopic biopsies and brushings.

Cystic Fibrosis and Genetic Medicines

Patient Advocacy Collaborators

Cystic Fibrosis Foundation

The mission of the Cystic Fibrosis Foundation is to cure cystic fibrosis and to provide all people with cystic fibrosis the opportunity to lead long, fulfilling lives by funding research and drug development, partnering with the cystic fibrosis community, and advancing high-quality, specialized care.

Language courtesy of Cystic Fibrosis Foundation

Emily’s Entourage

Emily’s Entourage is an innovative 501(c)3 foundation that accelerates research for individuals in the final 10% of the CF population that do not benefit from existing mutation-targeted therapies, including those with nonsense mutations of CF. Since 2011, Emily’s Entourage has awarded millions of dollars in research grants, launched a now-acquired CF gene therapy company, developed a patient registry and clinical trial matchmaking program to accelerate clinical trial recruitment, and led worldwide efforts to drive high-impact research and drug development.

Language courtesy of Emily’s Entourage