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Vertex Receives CHMP Positive Opinion For The First CRISPR/Cas9 Gene-Edited Therapy CASGEVY

Por: RTTNews Health December 15, 2023

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Vertex Pharmaceuticals Inc. () announced Friday that the European Medicines Agency's (EMA's) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).If approved, exa-cel would be the only... + full article



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