Tag Sickle Cell Disease

FDA has approved CASGEVY™, a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises.

Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced today that FDA has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).…

MHRA has granted conditional marketing authorization for CASGEVY™.

Vertex Pharmaceuticals and CRISPR Therapeutics announced that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) has 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…