Cystic Fibrosis: Discovery of CFTR Modulators

April 20, 2017
Session 4 of the 2017 Drug Design and Delivery Symposium


Molecules in a cross-hair with a #4

How are modern day chemists seeking to treat cystic fibrosis? Join Peter Grootenhuis of Vertex Pharmaceuticals as he delivers a brief overview of the drug discovery approaches and challenges to identify small molecules CFTR modulators. These compounds improve the trafficking, processing and/or restore the function of the mutant CFTR protein and address the primary cause of cystic fibrosis.

What You Will Learn

  • How misfolded mutant CFTR is partially ‘fixable’ by a combination of complementary drugs
  • Why an open mind is required when working with CFTR modulators
  • Examples of multiple HTS campaigns and medicinal chemistry efforts using phenotypic assays and cultured primary human cells from CF patients were essential to the discovery of two CFTR modulator drugs

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