CAMBRIDGE (MASSACHUSETTS) (dpa-AFX) - Vertex Pharmaceuticals (VRTX), Thursday announced a reimbursement agreement with the Italian Medicines Agency that allows eligible transfusion-dependent beta thalassemia and severe sickle cell disease patients to access the CRISPR/Cas9 gene-edited therapy CASGEVY.
CASGEVY, a non-viral ex vivo CRISPR/Cas9 gene-edited cell therapy, is designed to produce high levels of fetal hemoglobin in red blood cells, reducing or eliminating vaso-occlusive crises for SCD patients and transfusion needs for TDT patients.
VRTX is currently trading at $387.05, down $0.10 or 0.03 percent on the Nasdaq.
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