Two emerging gene therapies for SCD, lovotibeglogene autotemcel (lovo-cel) from bluebird bio and exagamglogene autotemcel (exa-cel) from Vertex Pharmaceuticals and CRISPR Therapeutics, are anticipated to receive regulatory decisions by December 2023. Both use an autologous HSCT approach in which a patient’s own stem cells are genetically modified ex vivo to inhibit sickling of the blood cells. Lovo-cel uses a lentiviral vector to insert a modified β-globin gene variant into cells, whereas exa-cel employs CRISPR gene editing technology to increase production of fetal hemoglobin.

The Institute for Clinical and Economic Review (ICER) evaluated lovo-cel and exa-cel as 1-time gene therapies for SCD. This report presents the summary of our systematic literature review and cost-effectiveness analysis and highlights the key policy recommendations discussed at the California Technology Assessment Forum (CTAF) public meeting on July 27, 2023.