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33rd Annual North American Cystic Fibrosis Conference

S03--ET-NT-GMS: *Fixing What Modulators Won't

Oct 31, 2019 2:00pm ‐ Oct 31, 2019 3:45pm


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.

Learning Objectives:

  • 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.

Symposium Chair(s):

Symposium Speaker(s):

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