Forbes November 2, 2023
Joshua Cohen

Next month, the Food and Drug Administration will decide on the regulatory approval of two gene therapies for sickle cell disease. Exa-cel and lovo-cel are potentially one-time treatment options for SCD patients burdened by unmet needs.

The two treatments—exa-cel (exagamglogene autotemcel) and lovo-cel (lovotibeglogene autotemcel)—attack SCD at its genetic root. In the case of exa-cel,* a patient’s blood stem cells are extracted and then edited via CRISPR/Cas9 to express fetal hemoglobin and subsequently returned to the patient’s body. CRISPR/Cas9 is a component of the bacterial immune system that can be used to cut and edit DNA. As such, it is called a gene editing tool.

Lovo-cel works differently. It inserts a functional human beta-globin gene into a patient’s own hematopoietic...

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