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Vertex, CRISPR prep filing for gene-editing blood disorder therapy

The rolling application for exagamglogene autotemcel (exa-cel) – formerly known as CTX001 – will start in November, while a filing in Europe is also expected by the end of the year, said the two partners.

The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

It involves an ‘ex vivo’ use of gene-editing, where the technology is used to modify a patient’s own cells outside the body to make foetal haemoglobin (HbF), which can serve as a substitute to regular haemoglobin in both SCD and thalassaemia.

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