During the early stages of the project (Phases 1, Phase 2, and Phase 3), two families of compounds, PP and SH, were identified and shown to exhibit significant corrective activity on mutated CFTR protein function. Some of these compounds demonstrated dual activity, acting both as correctors and potentiators; this finding will require further investigation through additional analyses of the interactions between the compounds and the mutated CFTR protein.

The study will continue with the optimization of compound–CFTR interactions, supported by computational approaches such as molecular docking. At this stage of the project, the pharmacokinetic properties (absorption, distribution, metabolism, and excretion) of a selected panel of compounds will also be evaluated through in vivo studies. In addition to the collaboration with the Foundation’s Primary Cell Culture Service, certain preclinical evaluation assays will be outsourced to a Contract Research Organization (CRO) to ensure high-quality analytical standards.