FTX-6058
Sickle Cell Disease
Phase 1bDevelopment Paused
Key Facts
About Fulcrum Therapeutics
Fulcrum Therapeutics is pioneering a novel approach to drug discovery by targeting the mechanisms that control gene expression to treat genetically defined diseases. The company's lead program, losmapimod, is in Phase 3 development for facioscapulohumeral muscular dystrophy (FSHD), a debilitating muscle disorder with no approved therapies. Fulcrum's platform leverages functional genomics to identify master regulator proteins that can be targeted with small molecules, aiming to correct imbalanced gene expression and address the underlying cause of disease. The company's strategy focuses on high-unmet-need rare diseases where its technology can deliver transformative treatments.
View full company profileTherapeutic Areas
Other Sickle Cell Disease Drugs
| Drug | Company | Phase |
|---|---|---|
| Decitabine combination | Novo Nordisk | Phase 2 |
| CTX001 | Vertex Pharmaceuticals | Phase 3 |
| DISC-3405 | Disc Medicine | Phase 1 |
| Mitapivat | Agios Pharmaceuticals | Phase 2/3 |
| BIVV003 | Sangamo Therapeutics | Phase 1/2 |
| Oxbryta® (voxelotor) | Cleo Life Sciences | Approved |
| CT-101 | Cetya Therapeutics | Pre-clinical |