FFC#07/2015

Aminoarylthiazole derivatives as correctors of the chloride transport defect in novel cystic fibrosis: computer assisted drug design, synthesis and biological evaluation

FFC#07/2015

Aminoarylthiazole derivatives as correctors of the chloride transport defect in novel cystic fibrosis: computer assisted drug design, synthesis and biological evaluation

PRINCIPAL INVESTIGATOR

Enrico Millo (CEBR, Centro Eccellenza Ricerca Biomedica, Università di Genova)

Partner

Elena Cichero (Dip. di Farmacia, Sezione di Chimica Medica – Scuola di Scienze Mediche e Farmaceutiche, Università di Genova)

RESEARCHERS

8

CATEGORY

AREA 1 Therapies to correct the underlying defect

DURATION

1 year

GOAL

€ 40.000 €

RESULTS

The study allowed to better explore the structure-activity relationship exhibited within AATs and the reference corrector VX-809.The results provided novel information on AATs and led the identification of some molecules with a particular ability to rescue CFTR-F508del. This approach reveal the possibility to generate libraries of molecules particularly suited to be offered to industry research for development of pharmacological treatment of CFTR-F508del patients.

OTHER RESULTS

FFC #3/2024

Two molecules are effective in activating Heat Shock Proteins and enhancing the action of CFTR correctors with the F508del mutation in vitro.

FFC#5/2024

Some peptide nucleic acids (PNAs) re-sensitise Pseudomonas aeruginosa to the antibiotic meropenem in vitro and reduce its virulence.

FFC#1/2023

Tezacaftor, one of the components of Kaftrio, induces an accumulation of dihydroceramides both in vitro and in vivo in animal models