Background

Sickle cell disease (SCD) is a genetic blood disorder that results in the formation of sickled red blood cells (RBC). Sickled RBC obstruct blood vessels, causing vaso-occlusive crisis/events (VOC/VOE) - acute pain in the lower back or limbs, acute chest syndrome and stroke are frequent complications. SCD is one of the most common inherited blood disorders, resulting in > $1.1 billion in healthcare expenditure/yr in the U.S. alone. There are ~110,000 affected patients in the USA and a similar number in Europe. There are 15-20 million patients worldwide with SCD.

Currently, the only established cure is through a bone marrow transplant (BMT) from a matched donor, which is not commonly performed due to the risks involved and the difficulty