Cystic fibrosis (CF) is a genetic disorder that is caused by loss-of-function mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which in combination with the epithelial sodium channel (ENaC), regulates the movement of salt and water in various organ systems.


In particular, CF has a profound effect on the lungs and respiratory tract, characterized by dehydration of the airway surface and reduced mucus clearance – the lung’s principle mechanism for maintaining a clean environment. The mucus becomes thick and sticky, progressively accumulating into obstructions that block airflow and result in recurrent colonization of the airways by viruses and bacteria. This condition leads to frequent, acute lung infections, chronic inflammation, and impaired lung function. The long-term result of the disease is progressive, permanent tissue damage and scarring (fibrosis) in the lung. No cure for CF is known, although several treatments have been approved to address the underlying cause of the disease for some CFTR mutations.

Despite the positive advances, more work is needed to ensure effective therapies are available for each of the approximately 75,000 individuals worldwide who are living with CF.

Spyryx is committed to that effort.

Additional Resources and Disease Information

The Cystic Fibrosis Foundation

Leading patient advocacy organization promoting disease awareness, drug development and treatment options for individuals living with CF

CF Roundtable

Newsletter for adults living and dealing with Cystic Fibrosis