The U.S. Food and Drug Administration is expected to approve exa-cel, a gene-editing treatment for sickle cell disease. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, exa-cel would be the first medicine in the U.S. to use CRISPR gene-editing technology. The treatment aims to alleviate the symptoms of sickle cell disease by editing a person’s DNA using CRISPR technology, thereby turning on a protein called fetal hemoglobin to help red blood cells maintain their healthy shape. Approval of exa-cel would be a significant milestone, offering relief to the approximately 100,000 people in the U.S. affected by sickle cell disease.
While the treatment could be a breakthrough for patients, it also presents challenges for the American health-care system. Wall Street anticipates that the treatment could cost around $2 million per patient, and tens of thousands of people could be eligible. Additionally, exa-cel would only be available at specialized health-care facilities, limiting its accessibility. Despite these potential hurdles, exa-cel has shown promising results in clinical trials, with participants experiencing a significant reduction in pain crises. The FDA’s approval of exa-cel would not only provide hope to patients with sickle cell disease but also pave the way for other gene-editing treatments in the future.
