Key Information
Sickle cell disease (SCD) is a hereditary blood disorder caused by a point mutation in the beta-globin gene that produces abnormally shaped red blood cells. Instead of being flexible and disc-shaped, the cells become curved like a sickle.
SCD affects approximately 100,000external link, opens in a new tab people in the United States and millions more worldwide. Patients often experience vaso-occlusive episodes, hemolytic anemia, and progressive organ damage.
Two FDA-approved gene therapies introduced in December 2023 now offer the first potential curative treatment options for eligible patients. Despite being the most common inherited blood disorder globally, SCD has historically received less clinical attention and research funding than its disease burden warrants.