Areas of Research and Impact
We have a diversified pipeline that is supported by our underlying platform
We believe that our CRISPR technology has the potential to treat a variety of blood diseases, including non-malignant hematologic diseases.
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.