First gene editing therapy to treat beta thalassemia and severe sickle cell disease
5. Januar 2024 – The European Medicines Agency (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.
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