Interventions of Interest:

  • exagamglogene autotemcel (“exa-cel”, Vertex Pharmaceuticals and CRISPR Therapeutics)
  • lovotibeglogene autotemcel (“lovo-cel”, bluebird bio)

Sickle cell disease (SCD) is an inherited blood disorder caused by a genetic mutation of the HBB gene responsible for the β-globin component of hemoglobin; hemoglobin is the protein in red blood cells that carries oxygen. ICER will be examining the comparative clinical and value of two gene therapies: exagamglogene autotemcel (“exa-cel”, Vertex Pharmaceuticals) and lovotibeglogene autotemcel (“lovo-cel”, bluebird bio) for sickle cell disease.

For questions, please contact Kelsey Gosselin, Program Manager, at

View the Key Stakeholder List.