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Vertex’s Sickle Cell Treatment Gets December Decision Date, On Track To Be First CRISPR Therapy

Rival Therapy Could Also Get Green Light By Year End

 
• By Andrew McConaghie

Vertex now looks likely to gain US approval for the first ever CRISPR gene edited therapy by the end of the year, but could face immediate competition from Bluebird Bio.

Vertex building logo
• Source: Shutterstock

Vertex looks on course to gain approval for its sickle cell disease gene therapy exagamglogene autotemcel (exa-cel) by the end of this year, with the US Food and Drug Administration having confirmed it will make its decision by 8 December. Its chief rival, bluebird bio, is not far behind with its candidates, however.

Vertex is also seeking the therapy’s approval as a treatment for transfusion-dependent beta thalassemia (TDT), but the US regulator has...

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