“Novel therapeutic approaches for the treatment of cystic fibrosis based on small molecule transmembrane anion transporters”
This project will develop an innovative therapeutic approach for the treatment of Cystic Fibrosis (CF). This condition originates from the defective function of the CFTR protein, a chloride and bicarbonate permeable transmembrane channel. This project will evaluate small molecules capable of facilitating the transmembrane transport of anions such as chloride and bicarbonate and will thus enable CF treatment by replacing the missing CFTR anion permeation activity. This represents an unexplored path in the treatment of CF and a paradigm shift with respect to current strategies searching for a cure for CF. Instead of focusing on the development of mutation-specific treatments, we plan to develop a therapy applicable to CF patients, regardless of the type of mutation they harbor. Thus, this therapeutic approach overcomes the limitation of current mutation-specific treatments and is applicable to CF patients in general.
To achieve this goal we have set up a comprehensive program to validate a research concept and complete the preclinical development of a new lead compound, making it ready for early clinical development. A multidisciplinary team of qualified researchers have been assembled to bring to conclusion a truly translational project from the synthesis of new compounds to validation on animal models.