Non-Malignant Hematologic Diseases

We are working to develop approaches that use genome editing in hematopoietic stem cells to help treat non-malignant hematological diseases. We are researching approaches to potentially treat genetic disorders such as beta thalassemia and sickle cell disease. We are also actively looking at other potential opportunities to develop medicines for diseases where we believe gene editing of hematopoietic stem cells may be beneficial.