ENTCF001 - Cystic Fibrosis

Cystic fibrosis (CF) is a progressive genetic disease that affects the lungs, pancreas, and other organs. In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional. This defect impairs chloride ion transport across epithelial cell membranes, leading to the production of abnormally thick and viscid mucus. This viscous mucus accumulates in the respiratory and gastrointestinal tracts, resulting in chronic pulmonary infections, progressive lung damage, and obstructive lung disease. In the digestive system, mucus obstructs pancreatic ducts, leading to malabsorption of nutrients and pancreatic insufficiency. 

While a number of treatments such as CFTR modulators have significantly improved the management of CF, significant unmet needs remain. There is a lack of a definitive cure, and existing treatments are not effective for all mutations of the CFTR gene. 

Entos is developing ENTCF001 for the treatment of cystic fibrosis. Leveraging the FOCUS-AI system, Entos has developed a pulmonary-focused Fusogenix PLV formulation that can deliver a functional copy of the CFTR gene to the lungs and other affected organs.