CFTR Modulators currently help multiple organs by increasing the function of the CFTR protein.

Gene Therapy helps the lungs with a less frequent medication, but is further behind in terms of trial development.

In the future could gene therapy be used in conjunction with CFTR modulators?


Currently approved with Kalydeco: G551D

Currently in trials with Kalydeco: Other Gating, R117H, Residual Function

Currently in trials with VX809, VX661 & N6022: F508del

Previously in trials with Ataluren: Nonsense mutations

N30s compounds improved F508del expression and channel function.

N30s compounds have the potential to improve a number of factors including: chloride channel function, inflammation and lung perfusion.

N30 is conducting a clinical trial with F508del homozyotes.

N30 has started a phase 1b/2a trial with homozygous F508del patients.

They are trialling a compound called N6022, which is both anti-inflammatory and a CFTR corrector.

The trial is being held in the US with CF patients aged 18 and older.

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