Sickle cell disease (SCD) is one of the most common inherited blood disorders worldwide, impacting millions of people globally and most commonly affecting individuals of African, Mediterranean, Middle Eastern, and Indian descent. Despite its prevalence, SCD has historically received less research funding and therefore fewer treatment advances compared to other genetic conditions, contributing to longstanding disparities in care and outcomes.
SCD affects red blood cells, causing them to become rigid and “sickle”-shaped. These cells can block blood flow, leading to severe pain episodes, organ damage, and reduced life expectancy. One of the most widely used medications, hydroxyurea, helps the body produce more fetal hemoglobin—a form of hemoglobin that prevents red blood cells from sickling—but it does not work for everyone.