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Vertex/CRISPR Prepare To Take Exa-Cel To Regulators For SCD, TDT

 
• By Alaric DeArment

The first gene-edited therapy will be submitted for sickle cell disease and transfusion-dependent beta thalassemia; it will compete with bluebird in both indications, but analysts anticipate blockbuster sales in excess of $2bn.

Vertex and CRISPR plan to start a rolling submission of exa-cel, previously CTX001, in SCD and TDT in November • Source: Shutterstock

Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics AG’s submission plans for exagamglogene autotemcel (exa-cel) in sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) will likely place it neck-and-neck with competitor bluebird bio’s investigational gene therapy for SCD, not to mention its approved TDT therapy. Analysts anticipate approval, successful launch and blockbuster sales.

Vertex/CRISPR said 27 September that they plan to start a rolling submission of exa-cel to the US Food and Drug...

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