What patient population participated in the VX-809 study?
VX-809 was studied in CF patients who have two copies of the Delta F508 CF gene mutation. The Delta F508 mutation leads to a CFTR protein that does not fold correctly. Because the defective CFTR is not the right shape, it does not make it to the surface of the cell. The purpose of VX-809 is to help the protein fold into the right shape, so it can move to its proper place at the cell surface.