There’s could be some big news ahead for CRISPR Therapeutics (CRSP). 

Along with Vertex Pharmaceuticals (VRTX), the company’s exagamglogene autotemcel (exa-cel) could see approval for the treatment of sickle cell disease and transfusion dependent beta thalassemia (TDT) shortly. 

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If successful, the treatment could be substantial. After all, there are limited treatment options for beta-thalassemia or sickle cell disease. Right now, patients typically rely on blood transfusions to manage these conditions. Also, exa-cel is a one-time treatment that could provide a much-needed cure for those conditions.  And, we have to consider that if successful, CRISPR could see validation and optimism.


Ian Cooper