„Novel therapeutic approaches for the treatment of cystic fibrosis based on small molecule transmembrane anion transporters”
Project acronym: TAT-CF
The 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.
The consortium consists of 8 partners: UNIVERSIDAD DE BURGOS (SPAIN), STEINBEIS INNOVATION GGMBH (GERMANY), ISTITUTO GIANNINA GASLINI (ITALY), CONSIGLIO NAZIONALE DELLE RICERCHE (ITALY), BIONEER A/S (DENMARK, BIOPRAXIS RESEARCH AIE (SPAIN), AVIDIN LTD (HUNGARY), AGENCIA ESTATAL CONSEJO SUPERIOR DE INVESTIGACIONES CIENTIFICAS (SPAIN).
The 3 years project started on January 1st, 2016.
Avidin is the leader of WP4 (Medicinal Chemistry), in which the company coordinates the structure-activity relationship studies and evaluation of the drugability of potential anionophore compounds along with in silico optimization of selected hits, pharmacophore identification, novel synthetic modifications supporting WP1 (Chemistry) to improve the drugability of lead compounds. Our company will also participate in WP5 (Preclinical studies), where Avidin will determine ADME-tox, bioavailability, biodistribution and PK/PD in animal non-GLP studies. In WP6 (Drug delivery and formulation) Avidin will participate in determining the optimal formulation of the lead transporter compounds.