Vertex announced that the European Commission has granted conditional marketing authorization to Casgevy, a CRISPR/Cas9 gene-edited therapy. Casgevy is approved for the treatment of patients who are 12 years of age and older with severe sickle cell disease, or SCD, characterized by recurrent vaso-occlusive crises, or VOCs, or transfusion-dependent beta thalassemia, or TDT, for whom hematopoietic stem cell, or HSC, transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.
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