Two Sickle Cell Disease Gene Therapies May Soon Be Approved By FDA

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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