VRTX

Vertex Pharmaceuticals Inc / Ma

A global biotech developing transformative medicines for serious diseases, Vertex Pharmaceuticals is best known for its cystic fibrosis franchise — KALYDECO, ORKAMBI, SYMDEKO, TRIKAFTA, and ALYFTREK — which treats most people with CF in key Western markets. The company also makes CASGEVY, a CRISPR gene-editing therapy for sickle cell disease and beta thalassemia, and JOURNAVX, a non-opioid pain signal inhibitor for acute pain. Its pipeline targets kidney disease, chronic pain, and type 1 diabetes.