VRT-325 is a novel and potent CFTR-F508del corrector which is able to modify the channel function. The deletion of Phe-508 (F508del) constitutes the most prevalent cystic fibrosis-causing mutation. VRT-325 has been shown to cause a significant, if small, rescue of the mature, complex glycosylated form of the ΔF508 CFTR molecule. The VRT-325 compound appears to work by directly binding to the ΔF508 molecule and by promoting interactions between the two halves of the ΔF508 CFTR molecule.

纯度：≥98%

CAS：815592-21-3