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Approved CRISPR-based medicine
| Trade name (drug name) | Approval year | Strategy | Targeted gene | Disease | Company |
|---|---|---|---|---|---|
| Casgevy™ (Exagamglogene autotemcel) | 2023 | Ex vivo-Autologous transfusion | BCL11A | β-thalassemia Sickle Cell Disease | Vertex Pharmaceutical |
