European regulator ready to review curative sickle cell disease therapy
The European Medicines Agency is to review a regulatory submission from Swiss gene editing specialist CRISPR Therapeutics (Nasdaq: CRSP) and partner Vertex Pharmaceutical (Nasdaq: VRTX).
The companies have submitted for approval of the gene therapy candidate exagamglogene autotemcel (exa-cel) formerly known as CTX001, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
The submission is backed by positive data from two global Phase III studies, which tested exa-cel)= as a potential one-time curative treatment for people with SCD or TDT.
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