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
- Date: Thursday, April 20, 2017 @ 2:00-3:00pm ET
- Fee: Free to Attend
- Download Peter's Slides
The Fine Print
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