CASGEVY™ (exagamglogene autotemcel)
Transfusion-Dependent Beta Thalassemia
Key Facts
About CRISPR Therapeutics
CRISPR Therapeutics' mission is to develop curative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. Its landmark achievement is the approval of CASGEVY™ (exa-cel), the world's first CRISPR-based therapy, developed with Vertex Pharmaceuticals for sickle cell disease and beta thalassemia. The company's strategy leverages a fully integrated R&D and manufacturing infrastructure to advance a diversified clinical pipeline, including allogeneic CAR-T therapies for oncology and programs in regenerative medicine. CRISPR Therapeutics aims to sustain its first-mover advantage by expanding the applications of gene editing across multiple therapeutic areas.
View full company profileAbout CRISPR Therapeutics
CRISPR Therapeutics' mission is to develop curative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. Its landmark achievement is the approval of CASGEVY™ (exa-cel), the world's first CRISPR-based therapy, developed with Vertex Pharmaceuticals for sickle cell disease and beta thalassemia. The company's strategy leverages a fully integrated R&D and manufacturing infrastructure to advance a diversified clinical pipeline, including allogeneic CAR-T therapies for oncology and programs in regenerative medicine. CRISPR Therapeutics aims to sustain its first-mover advantage by expanding the applications of gene editing across multiple therapeutic areas.
View full company profileTherapeutic Areas
Other Transfusion-Dependent Beta Thalassemia Drugs
| Drug | Company | Phase |
|---|---|---|
| Exagamglogene Autotemcel (CASGEVY) | Vertex Pharmaceuticals | Approved |