EMA – Casgevy: First gene editing therapy to treat beta thalassemia and severe sickle cell disease
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.
More information on the EMA Website.
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vroby (31 mai 2024). EMA – Casgevy: First gene editing therapy to treat beta thalassemia and severe sickle cell disease. ELSIBI. Consulté le 16 janvier 2025 à l’adresse https://doi.org/10.58079/11rmw
