The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type I diabetes and alpha-I antitrypsin deficiency.

In December 2022, Vertex announced a global collaboration with Entrada Therapeutics focused on discovering and developing intracellular Endosomal Escape Vehicle (EEV) therapeutics for myotonic dystrophy type 1 (DM1).