Vertex receives chmp positive opinion for the first crispr/cas9 gene-edited therapy, casgevy™ (exagamglogene autotemcel), for the treatment of sickle cell disease and transfusion-dependent beta thalassemia

Boston--(business wire)--vertex pharmaceuticals incorporated (nasdaq: vrtx) announced today 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 genetic therapy for.
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