Interventions of Interest:

  • betibeglogene autotemcel (Zynteglo/LentiGlobin, bluebird bio)

Beta thalassemia is an inherited blood disorder caused by a genetic mutation of the HBB gene that leads to reduced or absent synthesis of the β-globin proteins of hemoglobin, components of red blood cells responsible for carrying oxygen. When β-globin is markedly reduced or absent, it leads to ineffective production and increased destruction of red blood cells, manifesting in clinically significant anemia.

ICER postponed a previous beta thalassemia assessment.

For questions, please contact Maggie O’Grady, Program Manager, at

View the Key Stakeholder List.