Vertex and CRISPR Therapeutics announced authorization of first CRISPR/Cas9 gene-edited therapy by UK MHRA for treatment of Sickle Cell Disease

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On Nov. 16, 2023, Vertex and CRISPR Therapeutics announced that the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for CASGEVYル (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

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