RE:Cystic Fibrosis and VertexJust for clarification regarding Vertex, CF, & gene therapy:
The current Vertex medications available on market are not "gene therapy".
However, Vertex is trying to get involved with fundamental research aimed at CF gene therapy. (not yet clinical study trials & not close - still some time to go) .
For CF patients, there is some hope for a cure due to some 'limited success" in new breakthrough gene therapy approaches (not involving Vertex) but unfortunately this is still many trials & years away.
Gene therapy, in general, is more complicated than it seems.
As mentioned by saltaja, PLI aims to treat some CF side effects.
GLTA