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To treat sickle cell disease and transfusion-dependent beta thalassemia in eligible patients 12 years and older.
November 16, 2023
By: Kristin Brooks
Managing Editor, Contract Pharma
Vertex Pharmaceuticals and CRISPR Therapeutics were granted conditional marketing authorization for CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) from the Medicines and Healthcare products Regulatory Agency (MHRA). CASGEVY is authorized for the treatment of eligible patients 12 years and older with SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom a...
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