The gene editing race between Editas Medicine and Vertex Pharmaceuticals with CRISPR Therapeutics has heated up as both companies released positive data for their sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) hopeful treatments. Editas Medicine unveiled early data from the RUBY and EdiTHAL trials on Friday, demonstrating the potential of its gene editor EDIT-301 in treating SCD and TDT. Meanwhile, Vertex Pharmaceuticals and CRISPR Therapeutics announced that the FDA had accepted the BLAs for exagamglogene autotemcel (exa-cel), their investigational CRISPR-based, gene-edited therapy for these indications. The partners also revealed that 16 out of 17 evaluable SCD patients met the primary endpoint of freedom from vaso-occlusive crises (VOC) for at least 12 consecutive months following exa-cel treatment. Both Editas and Vertex/CRISPR will present their respective data at the ongoing European Hematology Association Congress.
Keywords: Gene Editing, Editas Medicine, Vertex Pharmaceuticals, CRISPR Therapeutics, Sickle Cell Disease
Editas Medicine has presented initial data from two trials showcasing how its gene editor EDIT-301 can treat sick