VX-121
Classical PKU
Phase 3Active
Key Facts
About Vertex Pharmaceuticals
Vertex Pharmaceuticals is a premier biotechnology company that has revolutionized treatment for cystic fibrosis with its portfolio of CFTR modulators, generating billions in annual revenue. The company has successfully expanded beyond CF into gene editing with CTX001 for sickle cell disease and beta thalassemia, and is advancing programs in pain, diabetes, and other serious diseases. With a strong commercial franchise and robust pipeline, Vertex represents one of the most successful biotech companies in translating scientific innovation into life-changing medicines.
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