Cystic Fibrosis: Discovery of CFTR Modulators
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
The Fine Print
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