The Food and Drug Administration (FDA) is nearing approval of a groundbreaking CRISPR-based therapy for sickle cell disease, marking a historic shift from lab experimentation to clinical application. The FDA, set to decide by December 8, is scrutinizing the therapy, named “exa-cel,” which has demonstrated exceptional effectiveness in alleviating excruciating pain episodes for 29 out of 30 patients studied over 18 months. Despite promising results, concerns persist about potential long-term effects due to unintended DNA edits. The therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, involves modifying patients’ cells using CRISPR and reintroducing them to produce fetal hemoglobin, restoring normal red blood cell function and significantly improving patients’ lives.
Sickle cell disease, affecting millions worldwide, especially among individuals of African, Middle Eastern, and Indian descent, causes debilitating pain, organ damage, and premature death. Although the therapy’s success offers hope, hurdles such as its estimated $2 million cost per patient and its complexity, requiring bone marrow transplants and sophisticated medical facilities, raise accessibility concerns.
Experts debate the therapy’s practicality, emphasizing the urgent need for affordability and widespread availability to truly impact the lives of those affected by this debilitating genetic disorder. Efforts are underway to address these challenges, ensuring this innovative treatment becomes accessible to those who need it most.
Read more: https://www.npr.org/sections/health-shots/2023/10/31/1208041252/a-landmark-gene-editing-treatment-for-sickle-cell-disease-moves-closer-to-realit
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