FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia | CRISPR Therapeutics

– First CRISPR gene-editing filings to be accepted for review by FDA – – FDA grants Priority Review for severe sickle cell disease (SCD) and Standard Review for transfusion-dependent beta thalassemia (TDT) – – PDUFA target action date of December 8, 2023 , for SCD and March 30, 2024 , for TDT –

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