Sickle cell disease refers to a group of genetic red blood cell (RBC) disorders characterized by the presence of an abnormal hemoglobin, called hemoglobin S (HbS) or sickle hemoglobin, in the RBCs. The sickle cell disease market is evolving with significant advancements in treatment options. Although novel agents like Adakveo and Endari have been introduced to the market, they have seen limited adoption due to modest efficacy or regulatory setbacks. The approvals of gene therapies such as Vertex Pharmaceuticals' Casgevy and bluebird bio's Lyfgenia marks a paradigm shift in the treatment of sickle cell disease, but uptake has so far been modest due high costs, significant treatment burdens, and logistical complexities. Payer pressure, infrastructure limitations, and global disparities in access remain significant barriers to widespread adoption of advanced therapies.

This Datamonitor Healthcare report contains a Disease Analysis module.