Table 4 Approved CRISPR-based medicine
From: Between hope and reality: treatment of genetic diseases through nucleic acid-based drugs
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 |
