About Cystic Fibrosis:
Cystic fibrosis (CF)is the most common life-limiting autosomal recessive disease among people of mostly European heritage. In the United States, approximately 30,000 individuals have CF and most are diagnosed by six months of age. Life expectancy for people with CF depends largely upon access to health care. In 1959, the median age of survival of children with CF was only six months1. Thanks to important therapeutic developments life expectancy in the western world is now close to 36 years.
CF is an inherited disease caused by mutations in the gene that makes a protein called CFTR (cystic fibrosis transmembrane regulator). CF patients have a defect in chloride permeability and secondarily sodium conductance in sweat glands and epithelial tissue. These defects cause their epithelial cells to produce a thick, sticky mucus rather than a watery mucus that normally acts as a lubricant that lines the passages of the body’s organs. Consequently, CF patients have difficulty in breathing and the thick mucus prevents efficient removal of bacteria from the lungs. This leads to problems such as repeated bacterial lung infections and lung damage.2 Problems with digesting food due to mucus blockage of digestive enzymes from the pancreas into the intestine also occurs. Other organs can also be affected, including the liver, intestines, sinuses and sex organs.
Therapies target mucus production and bacterial exacerbations of CF in the lungs, among other things. Some therapies are designed to rectify the CFTR membrane transporter gene or its faulty protein product.1 NB-402 will impact the life quality of the CF patient by interacting with the membranes and killing gram-negative pathogens that cause infections in the lungs of CF patients.