Now playing catch-up to bluebird, Vertex and CRISPR send in their pitch for blood disorder cell therapy
If all things go according to plan, the FDA could start reviewing the first-ever filing for a CRISPR therapy this November.
Vertex and CRISPR Therapeutics say they have wrapped up their lengthy talks with the agency and gotten regulators on board for a rolling review beginning later this year — with the submission package due to be complete in the first quarter of 2023.
While the partners once hoped that they would be the first to bring a once-and-done therapy to the US market for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), bluebird bio beat them to the punch with the recent FDA approval of Zynteglo. The filing also marks a slight delay to earlier plans of filing in late 2022, although it wasn’t entirely unexpected.
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