Vertex Pharmaceuticals: EC Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVYTM, for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

European Commission Grants Conditional Marketing to Vertex Pharmaceuticals CASGEVY

Tuesday Feb 13, 2024, Vertex Pharmaceuticals (VRTX) announced that the European Commission has granted conditional marketing authorization to CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy.

CASGEVY is being approved for patients 12 years of age and older with severe sickle cell disease (SCD) characterized by recurrent vaso . . .

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