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 Prepare To Take Exa-Cel To Regulators For SCD, TDT
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.
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