Vertex Pharmaceuticals/CRISPR Therapeutics’ cell-based gene therapy exagamglogene autotemcel (exa-cel) and Reata Pharmaceuticals’ omaveloxolone are among the latest drugs on which the European Medicines Agency is due to decide this week whether they should be granted EU-wide marketing approval.
Exa-cel is a CRISPR/Cas9 genome-edited cell therapy indicated for the treatment of the genetic conditions, sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), while omaveloxolone is for treating the