diponecaftor (Proteostasis Therapeutics)

Diponecaftor is an investigational oral fixed-dose triple CFTR-modulator therapy comprising dirocaftor, posenacaftor, and nesolicaftor. Dirocaftor is a CFTR potentiator that increases chloride-channel gating, posenacaftor is a CFTR corrector that improves mutant CFTR folding and trafficking, and nesolicaftor is a CFTR amplifier that increases CFTR biosynthesis through PCBP1-mediated stabilization of CFTR mRNA. Fair Therapeutics is developing diponecaftor for adults with cystic fibrosis caused by rare and ultra-rare CFTR variants selected using patient-derived intestinal-organoid testing. The Phase IIb CHOICES study evaluates the therapy in this population. ([fairtherapeutics.com](https://www.fairtherapeutics.com/science))

Other names

DIR/POS/NESDirocaftor/Posenacaftor/Nesolicaftordiponecaftor