Born in late 2009 at UniPA’s STEBICEF Department , the research group formed when Professor Andrea Pace invited Professors Aldo Di Leonardo, Ivana Pibiri, Laura Lentini, Raffaella Melfi and Marco Tutone to collaborate on discovering small heterocyclic compounds capable of promoting readthrough of premature stop codons in the CFTR gene.
In 2014, the Italian Cystic Fibrosis Foundation formally backed the collaboration under project FFC#1/2014, funding systematic in vitro assays in cell cultures and follow-up in vivo studies in animal models. Further grants in 2017 (FFC#3/2017) and 2020 (FFC#6/2020) allowed the team to refine their chemistry and validate three lead molecules NV848, NV914 and NV930 for efficient readthrough activity.
By 2016 these molecules were secured under an international patent and licensed to a pharmaceutical partner for drug development. This milestone marked the transition from academic discovery to preclinical pipeline.
In 2022, the group became a key partner in the HEAL ITALIA project funded by Italy’s PNRR under NextGenerationEU, and obtained complementary PRIN support to expand its translation readthrough inducing drug (TRID) platform into Duchenne muscular dystrophy, inherited ocular diseases, and primary immunodeficiencies.
Today the group operates in STEBICEF’s modern facilities and counts over a dozen researchers—organic chemists, computational chemists, cell biologists, geneticists, pharmacologists and clinical collaborators—working together under one roof.
