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