Cystic Fibrosis

Cystic fibrosis, also known as mucoviscidosis, is a genetically inherited, chronic disease and is progressive in nature. The onset of cystic fibrosis typically occurs in early childhood or, rarely, at birth. The primary symptoms of cystic fibrosis include breathing difficulties, high salt content in the sweat, and secretion of abnormally viscous mucus. The principal indicators of cystic fibrosis in patients are pancreatic insufficiency, pancreatitis, chronic bronchitis, adolescent diabetes, male sterility, and very rarely liver cirrhosis or intestinal obstruction. The most usual forms of cystic fibrosis are those with respiratory complications, difficulties related to digestion, and anomalies in height and growth. The mortality and morbidity of a patient are dependent on the degree of bronchopulmonary involvement.

Significant unmet need remains for cystic fibrosis (CF) treatment options that are effective against biofilms and avoid resistance

Mucus build-up in airways causes chronic chest infections

• Thick mucus unable to pass through airways
• Build-up causes bacteria growth and chronic infections

---

Current Treatment Landscape

Current therapies focus on:

• Correcting the function of the defective protein made by CF gene
• Prevention/Treatment of lung infections
• Loosen/remove mucus from lungs
• Prevention/treatment for blockages in the intestines
• Providing adequate nutrition
• Controlling dehydration

---

Methods include:

• Physical therapies, antibiotics, mucolytics, pancreatic enzymes, CFTR modulators, oxygen therapy and ultimately lung transplant
• Major problem with drug-resistant bacteria – increasingly difficult to treat with antibiotics

80K+ people affected in US & Europe, most die before reaching the age of 40