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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