Vertex -Additional Small Molecules

We’re investigating a portfolio of other small molecule medicines aimed at treating the underlying cause of CF. This includes CFTR potentiators, which are designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across cell membranes, helping to hydrate and clear mucus from the airways. We’re also researching additional small molecules to address the trafficking and processing defect of the CFTR protein to enable it to move through cells and reach the surface.