A landmark gene-editing treatment for sickle cell disease moves closer to reality

The Food and Drug Administration (FDA) is considering approving a therapy that uses CRISPR gene editing to treat sickle cell disease, marking the first time gene editing could move from the lab to clinical use. The therapy, called “exa-cel,” has shown promising results in preventing painful episodes in sickle cell disease patients. The FDA’s committee of advisers is reviewing the scientific evidence, focusing on long-term safety and potential unintended “off-target” effects of the treatment. A decision on the therapy is expected by December 8. Sickle cell disease disproportionately affects individuals of African, Middle Eastern, and Indian descent and has a severe impact on patients’ lives, often leading to early mortality. If approved, this therapy could be life-changing for those affected.