Vertex Pharmaceuticals

Vertex Pharmaceuticals discovers, develops and commercializes pharmaceutical products for treatment of cystic fibrosis, cancer, inflammatory bowel, autoimmune disease and neurological disorders. Vertex’s product Trikafta was approved by the USFDA in 2019 for treatment of cystic fibrosis. Other approved cystic fibrosis medicines from Vertex include Symdeko, Orkambi and Kalydeco.

Vertex and CRISPR Therapeutics are collaborating with their treatment, CTX001, made from a patient’s own stem cells harvested and edited using CRISPR technology to increase fetal hemoglobin levels to treat transfusion-dependent beta thalassemia and sickle cell disease.