Article: CRISPR technology one step closer to approval in a new therapy

EMA validates Vertex's CRISPR application, clearing barrier on road to landmark approval

FierceBiotech published an article yesterday noting:

“The candidate, exagamglogene autotemcel (exa-cel), is an autologous cell therapy treatment for sickle cell disease and transfusion-dependent beta thalassemia. By using CRISPR-Cas9 to edit a patient’s own hematopoietic stem cells, the partners modify cells to produce high levels of fetal hemoglobin. One-time treatment with exa-cel has freed patients from transfusions and vaso-occlusive crises.

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Exa-cel, formerly known as CTX001, has PRIME designation from the EMA, making it eligible for a priority review that usually takes 150 days, rather than the typical 210 days. The timeline suggests exa-cel could win approval in Europe later this year and become the first marketed CRISPR-Cas9_treatment."