The gene editing race between Editas and Vertex, CRISPR heats up as both companies release positive data on their respective gene therapies for sickle cell disease and transfusion-dependent beta thalassemia. Editas unveiled early data from its RUBY and EdiTHAL trials, demonstrating the potential of its gene editor EDIT-301 in treating these diseases. However, Vertex Pharmaceuticals and CRISPR Therapeutics also released positive data on their exagamglogene autotemcel (exa-cel) therapy, cementing their frontrunner status in the field. Both companies 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 lovotibeglogene autotemcel (lovo-cel) therapy in April 2023 after being placed under partial clinical hold by the FDA in December 2021 due to safety concerns. Many other players have dropped out of the race recently including Graphite Bio, Novartis, Intellia, and Sanofi.
Keywords: gene editing race, Editas Medicine, Vertex Pharmaceuticals, CRISPR Therapeutics, exagamglogene autotemcel
In a bid to treat sick