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.
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.