CTX001
Sickle Cell Disease
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.
View full company profileAbout 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.
View full company profileTherapeutic Areas
Other Sickle Cell Disease Drugs
| Drug | Company | Phase |
|---|---|---|
| Decitabine combination | Novo Nordisk | Phase 2 |
| DISC-3405 | Disc Medicine | Phase 1 |
| Mitapivat | Agios Pharmaceuticals | Phase 2/3 |
| FTX-6058 | Fulcrum Therapeutics | Phase 1b |
| BIVV003 | Sangamo Therapeutics | Phase 1/2 |
| Oxbryta® (voxelotor) | Cleo Life Sciences | Approved |
| CT-101 | Cetya Therapeutics | Pre-clinical |