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MHRA Authorizes First CRISPR/Cas9 Gene-Edited Therapy CASGEVY

To treat sickle cell disease and transfusion-dependent beta thalassemia in eligible patients 12 years and older.

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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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