Table 1.

Overview of current (pre)clinical treatments to restore CFTR function

Compound	Developmental stage	Mode of action
ABBV-2222	Clinical; phase 2	Corrector
ABBV-3067	Clinical; phase 2	Potentiator
ELX-02	Clinical; phase 2	Read-through
PTI-801	Clinical; phase 2	Corrector
PTI-808	Clinical; phase 2	Potentiator
PTI-428	Clinical; phase 2	Amplifier
ABBV-3067	Clinical; phase 1	Potentiator
MRT5005	Clinical; phase 1	mRNA (inhaled)
RPL554	Clinical; phase 1	Phosphodiesterase 3/4 inhibitor
VX-121	Clinical; phase 1	Corrector
VX-561	Clinical; phase 1	Potentiator
ARCT-032	Pre-clinical	mRNA (inhaled)
ARCT-032	Pre-clinical	mRNA (inhaled)
SPIRO-2101	Pre-clinical	Gene therapy (inhaled)
SPIRO-2102	Pre-clinical	Gene therapy (inhaled)
4D-710	Pre-clinical	Gene therapy (inhaled)