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Posted 20 November 2023 AM
Vertex has won the first-ever regulatory approval of a therapy based on the CRISPR gene-editing technology, with the UK's MHRA giving it the tick for two indications, sickle cell disease and transfusion-dependent beta-thalassaemia.
The treatment, previously known as exa-cel (exagamglogene autotemcel) and now branded Casgevy, may well make its way to Australia "over time" an Australian spokesperson for Vertex told Pharma in Focus.
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