Gene editing companies Editas Medicine, Vertex Pharmaceuticals and CRISPR Therapeutics are all racing to develop treatments for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. Editas recently unveiled early data from its RUBY and EdiTHAL trials, demonstrating the potential of its gene editor EDIT-301 in treating these diseases. However, Vertex and CRISPR also released positive data for their exagamglogene autotemcel (exa-cel) treatment, with the FDA accepting their BLAs for these indications. Both Editas and Vertex/CRISPR will present their respective data at the ongoing European Hematology Association Congress. The SCD race also includes bluebird bio, which filed a BLA for its gene therapy candidate lovotibeglogene autotemcel (lovo-cel) in April 2023.
The three gene editing companies are competing to develop treatments for sickle cell disease and transfusion-dependent beta thalassemia. Editas has revealed early data from trials showcasing the effectiveness of their gene editor EDIT-301 in treating these diseases. Meanwhile, Vertex Pharmaceuticals and CRISPR Therapeutics have also released positive data on their exagamglogene autotemcel