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CASGEVY™: A Gene-Edited Therapy for Sickle Cell Disease
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Vertex Pharmaceuticals secures CHMP positive opinion for CASGEVY™, a CRISPR/Cas9 gene-edited therapy for severe sickle cell disease and transfusion-dependent beta thalassemia, potentially marking the first genetic therapy for eligible patients in the EU.
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How does CASGEVY™'s gene-editing technology differ from traditional therapies for sickle cell disease?
How might the approval of CASGEVY™ impact the landscape of gene-edited therapies and genetic medicine?
What are the potential implications of CASGEVY™'s approval in the EU for the treatment of genetic diseases?
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