Tiziano Bandiera, Ph.D. - "Drug discovery outside the pharmaceutical industry: an Italian example"
Feb 06, 2017
from 03:00 PM to 04:30 PM
|Where||Tigem Auditorium Vesuvius|
|Contact Name||Luis Galietta|
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The discovery and development of a new drug is a challenging, time-consuming and expensive process, which develops through a series of steps and requires multidisciplinary skill sets. Although the whole process is almost exclusively carried out in mid-sized and big Pharma companies, the discovery phase can be performed also in small biotechs and academic laboratories. To carry out the discovery phase, academic institutions need to bring together researchers and technologies from different disciplines. This can be done by creating drug discovery centers or by joining forces among institutions having complementary expertise.
The presentation will first outline the activities that are carried out in the discovery phase of potential new drugs. An example of academic drug discovery will then be described, the Task Force for Cystic Fibrosis (TFCF) project. This project aims at the discovery of new modulators of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein for the treatment of cystic fibrosis (CF). Originating from mutations in the CFTR protein, CF is a fatal genetic disease affecting approximately 1 in ca. 2500 live births in the Caucasian population. The most frequent mutation among patients with CF is deletion of phenylalanine 508 (F508del), which causes a defective maturation and impaired gating of the CFTR chloride channel. The maturation defect can be treated with compounds known as correctors, whereas the gating defect can be overcome by compounds called potentiators. Two CFTR modulators have been approved for the treatment of CF, but only one of them, the potentiator ivacaftor, shows significant efficacy in ca. 5% of CF patients. The need exists, therefore, to find new drugs, in particular new correctors, that allow a greater number of CF patients to be more effectively treated. The preliminary results of the CFTR project toward this goal will be presented.