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Vertex/CRISPR Are First To US FDA With A CRISPR Gene-Editing Therapy For Sickle Cell Disease

 
• By Jessica Merrill

The companies completed a rolling BLA filing for exagamglogene autotemcel (exa-cel) for sickle cell disease and beta thalassemia, a decade after the discovery of CRISPR.

milestone
The filing of exa-cel marks a milestone for CRISPR • Source: Shutterstock

Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics AG are positioned to be first to market with a CRISPR gene-edited therapy for sickle cell disease (SCD). The companies announced on 3 April the completion of a rolling biologics license application (BLA) to the US FDA for an autologous, ex vivo CRISPR/Cas9 gene-edited therapy, exagamglogene autotemcel (exa-cel), for sickle cell disease and transfusion-dependent beta-thalassemia (TDT).

Vertex/CRISPR are seeking a priority review, which would shorten the FDA review window from 12 months to eight months

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