The first phase of the project has shown very promising results. A group of compounds with CFTR-DF508 corrector potential comparable to or greater than that of known correctors has been identified. Some highly significant potentiators have also been identified. Based on these results, FFC Ricerca has decided to proceed to Phase II, securing the necessary funding.

In the second phase, the hit-to-lead phase (from the initial active compound to the lead compound), which will last one year (from March 2015 to February 2016), the work of the IIT chemists will begin. For each initial active compound (hit), analogous compounds will be generated, in which various types of minor modifications will be made to the initial chemical structure. The new compounds, in groups of 20-30, will be tested on cells in the Gaslini laboratory to determine which of the modifications has had a potentially beneficial effect on the compound’s activity. These results will be crucial in guiding the synthesis of new compounds at the IIT, in the search for modulators and potentiators with progressively improved characteristics.

Project continues in Phase 3.