The main task of the Task Force for Cystic Fibrosis (TFCF) project consisted in the search for new correctors of the F508del mutant of the CFTR protein. The team identified the corrector ARN23765, a compound that partially rescues the activity of F508del-CFTR with very high potency. In 2021, following preliminary pre-clinical activities, funded by the Italian Cystic Fibrosis Research Foundation, ARN23765 was licensed to the US-based biopharmaceutical company Sionna Therapeutics.
The TFCF team shared with Sionna all the data generated on ARN23765. Based on the results obtained, and taking advantage of the data produced by the TFCF team, Sionna has discovered the compound SION-676, which was recently nominated as a development candidate. The compound SION-676 represents an improvement of ARN23765, which gathers good pharmacological potency and favourable drug-like properties.