Exagamglogene Autotemcel (CASGEVY)
Sickle Cell Disease
Key Facts
About Vertex Pharmaceuticals
Vertex Pharmaceuticals has established itself as a premier, fully integrated biopharmaceutical company with a mission to discover, develop, and commercialize innovative medicines for serious diseases. Its landmark achievement is the creation of a portfolio of CFTR modulators that treat the underlying cause of cystic fibrosis for the majority of patients, generating multi-billion dollar annual revenues. The company's strategy is to reinvest these profits to expand its therapeutic reach through a deep pipeline of 264 candidates, utilizing advanced platforms in gene editing, cell therapy, and precision medicine to address adjacent markets with significant unmet medical need.
View full company profileAbout Vertex Pharmaceuticals
Vertex Pharmaceuticals has established itself as a premier, fully integrated biopharmaceutical company with a mission to discover, develop, and commercialize innovative medicines for serious diseases. Its landmark achievement is the creation of a portfolio of CFTR modulators that treat the underlying cause of cystic fibrosis for the majority of patients, generating multi-billion dollar annual revenues. The company's strategy is to reinvest these profits to expand its therapeutic reach through a deep pipeline of 264 candidates, utilizing advanced platforms in gene editing, cell therapy, and precision medicine to address adjacent markets with significant unmet medical need.
View full company profileTherapeutic Areas
Other Sickle Cell Disease Drugs
| Drug | Company | Phase |
|---|---|---|
| Oxbryta® (voxelotor) | Cleo Life Sciences | Approved |
| CT-101 | Cetya Therapeutics | Pre-clinical |
| In Vivo Cell Therapy | GigaMune | Pre-clinical |
| Ramatroban (KAR101) | KARE Biosciences | Preclinical |
| Endari (L-glutamine) | Emmaus Life Sciences | Approved |
| Digital Model for Sickle Cell Disease | Koneksa Health | Development/Validation |
| ILX-002 | Illexcor Therapeutics | Pre-clinical |
| Epeleuton | Afimmune | Phase 2 |
| Undisclosed | Hillhurst Biopharmaceuticals | Preclinical or Phase 1 |
| CLY-124 | Cellarity | Phase 1 |
| AB1 | AkiraBio | Pre-clinical |
| Oral Decitabine/Tetrahydrouridine | Novo Nordisk | Phase 2 |