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...
