MHRA first to back Vertex and CRISPR's gene therapy Casgevy
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) today 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), developed by USA-based Vertex Pharmaceuticals (Nasdaq: VRTX) and Swiss firm CRISPR Therapeutics (Nasdaq: CRSP)
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