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diponecaftor
- Development stage
- Phase 2
- Lead developer
- Fair Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Diponecaftor is an investigational oral **triple CFTR-modulator therapy** comprising dirocaftor, posenacaftor, and nesolicaftor. It combines a CFTR potentiator intended to improve channel gating, a CFTR corrector intended to improve folding and trafficking of mutant CFTR protein, and a CFTR amplifier intended to increase CFTR protein production. Originally developed through the Proteostasis Therapeutics CFTR-modulator program and subsequently advanced by Fair Therapeutics, diponecaftor is being evaluated for cystic fibrosis, particularly in adults with rare and ultra-rare CFTR variants selected using patient-derived intestinal organoid responses.
- Brand names
- PTI-808PTI808PTI 808PTI-801PTI801PTI 801PTI-428PTI428PTI 428
- Other names
- dirocaftor + posenacaftor + nesolicaftorDirocaftor/Posenacaftor/Nesolicaftor
02
Targets
CFTR (Cystic fibrosis transmembrane conductance regulator)
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