First gene editing therapy to treat beta thalassemia and severe sickle cell disease

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Summary

EMA recommended approval of Casgevy, the first gene-editing therapy using CRISPR/Cas9 for transfusion-dependent beta-thalassemia and severe sickle cell disease …

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Diseases / indications

• Beta-thalassemia
• Sickle Cell Disease

Drugs mentioned

• Casgevy (exagamglogene autotemcel)

Snippet

EMA has recommended approval of the first medicine using CRISPR/Cas9, a novel gene-editing technology. Casgevy (exagamglogene autotemcel) is indicated for the treatment of transfusion‑dependent beta thalassemia and severe sickle cell disease in patients 12 years of age and older for whom haematopoietic stem cell transplantation is appropriate and a suitable donor is not available.