Drug repurposing for nonsense-mediated Cystic Fibrosis and Hemophilia – REP-4-nmCF

Rep-4-nmCF is the continuation of a drug repurposing programme started 2 years ago by the investigators. It is set to enable the clinical development of an old drug in nonsense mediated genetic diseases. By screening a library of 1200 marketed drugs on a cell model of nonsense mutation defect, they have discovered that amlexanox, a drug used for asthma in Japan, can correct the phenotype of cells from patients having nonsense mediated cystic fibrosis. This project will confirm these results on epithelia from more patients in cystic fibrosis, and pinpoint the molecular determinants of the mode-of-action of amlexanox in nonsense mutation cells.
All together, materials and data produced by this project will support the scientific rational for a clinical trial, confirm the initial choice of cystic fibrosis and hemophilia, and improve the positions of the consortium in future negotiations with the holder of the current market authorization for the oral form of amlexanox.