CFTR corrector
- Rescues folding and trafficking so mutant CFTR reaches the cell surface at all
- Distinct from a POTENTIATOR, which holds an already-surfaced channel open
- Corrector alone does little; the pairing is the therapy
- Rescues folding and trafficking so mutant CFTR reaches the cell surface at all
- Distinct from a POTENTIATOR, which holds an already-surfaced channel open — the two are given together for that reason
- Corrector alone does little; the pairing is the therapy
Drugs with this mechanism (2)
- Lumacaftor — CFTR corrector paired with ivacaftor; a strong 3A4 inducer that undercuts its own partner.
- Tezacaftor — CFTR corrector used in cystic-fibrosis combinations; improves mutant protein trafficking rather than directly opening the channel.
All mechanisms · Open CFTR corrector in the interactive console ↗