CFTR CORRECTOR PROGRAM FOR CF
Cystic Fibrosis Overview
Cystic Fibrosis (CF) is a chronic, genetic disorder that causes the body to produce unusually thick, sticky mucus that obstructs the lungs and digestive tract. This sticky mucus leads to infections in the lung and inhibits the pancreas from producing natural enzymes used in digestion and overall bodily health. There are approximately 30,000 patients with CF in the US and 70,000 world-wide. In the past, CF patients died at an early age. However, significant advances have been made in research and medical treatments for CF, both increasing life-expectancy and improving quality of life. Today, over 45% of CF patients are over the age of 18 with a median life expectancy in the mid-30s. Despite this progress, there remains an unmet need to extend and improve upon the lives of these patients.
Parion CFTR Corrector Program
CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most prevalent disease-causing mutation in CFTR, the deletion of phenylalanine 508 (DF508), causes the CFTR protein to be mis-folded, ultimately resulting in the loss of CFTR function, dehydration of the mucus membranes, and impairment of pulmonary, GI, and pancreatic function.
Parion is developing a novel series of small molecule CFTR correctors targeted at restoring the proper folding and stability to the DF508 CFTR protein. The Parion CFTR correctors are being developed as oral agents intended to treat all organs affected by CF.