The available therapeutic treatments are not effective on rare CFTR gating mutations, so the research group aims to identify new effective modulators for CFTR variants characterized by reduced channel opening. The synthesized compounds will be tested to verify the enhancement activity on a series of defects – both common and rare – of CFTR functionality and on pharmacologically correct class II/III mutations. The most active compounds will be tested, alone or in combination with other known and in preclinical phase CFTR modulators, to confirm the induced ability to open the channel. Through the use of special chemical probes, the researchers also intend to analyze the binding site between CFTR and the new potentiators.

XIX Convention FFC Ricerca – download here a brief presentation of the project