EDIT-301: An investigational gene editing medicine for the treatment of severe sickle cell disease and transfusion-dependent beta thalassemia.
In patients with severe sickle cell disease, sickle-shaped red blood cells lead to vaso-occlusive events, inflammation, chronic hemolytic anemia, and ischemic organ damage.
EDIT-301, a potential one-time, durable treatment, drives early correction of anemia and clinically meaningful improvements in patients living with severe sickle cell disease and transfusion-dependent beta thalassemia.