Editas Medicine Reports Promising in Vivo Gene Editing Data for Sickle Cell Disease and Beta Thalassemia Treatment

Editas Medicine reported new in vivo data showing therapeutically relevant levels of HBG1/2 promoter editing in hematopoietic stem cells in non-human primates using a single dose of its proprietary targeted lipid nanoparticle. The data demonstrated a mean on-target editing level of 58% in stem cells, exceeding the predicted editing threshold for therapeutic benefit. This approach is being developed to treat sickle cell disease and beta thalassemia.