MHRA first to back Vertex and CRISPR's gene therapy Casgevy

16 November 2023

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)

Casgevy is the first medicine to be licensed that uses the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020.

CASGEVY has been authorized for the treatment of eligible patients 12 years of age and older with SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available. There are an estimated 2,000 patients eligible for Casgevy in the UK.

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