Currently available modulator therapies target the defective CFTR protein to restore function. Patients with genotypes that are refractory to treatment with extant, and on the horizon, modulators are a challenge for these small molecule approaches - as nonsense and other changes can prevent production of the CFTR protein, which these modulators target. This symposium will focus on emerging science and technologies that point a way forward for this population. The theme will be to: 1) discuss the cells that require CFTR restoration, 2) new ways to deliver non-small molecule payloads to those cells, 3) methods to evaluate success, and 4) an example of such a non-traditional approach in development.
Summarize the cell types necessary to restore CFTR.
Evaluate delivery methods for restoration of CFTR.
Identify outcomes for success.
Note: Speaker Daniel Seigwart's presentation was not recorded at his request.