FDA-approved gene editing

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The U.S. Food and Drug Administration (FDA) has recently approved the first gene-editing therapies for the treatment of sickle cell disease, a genetic condition affecting at least 100,000 Americans2. The therapies, Casgevy and Lyfgenia, utilize CRISPR/Cas9, a type of genome editing technology, to modify patients' hematopoietic (blood) stem cells12. Casgevy, developed jointly by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first FDA-approved therapy utilizing CRISPR/Cas912. This technology can be directed to cut DNA in targeted areas, enabling the ability to accurately edit (remove, add, or replace) DNA where it was cut. The modified blood stem cells are then transplanted back into the patient where they engraft (attach and multiply) within the bone marrow and increase the production of fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery. In patients with sickle cell disease, increased levels of HbF prevent the sickling of red blood cells1. The FDA's approval of these therapies is considered a significant milestone in the field of gene-editing and for the treatment of sickle cell disease24. However, concerns have been raised about the accessibility of these breakthrough treatments due to their high cost, estimated at several million dollars per patient, and the complexity of the procedure, which many hospitals may not be equipped to provide48. The FDA regulates human genome editing under the existing framework for biological products, which includes gene therapy products13. Clinical studies of gene therapy in humans require the submission of an Investigational New Drug (IND) application prior to their initiation in the United States, and marketing of a gene therapy product requires submission and approval of a biologics license application (BLA)9. The FDA provides guidance to sponsors developing human gene therapy products incorporating genome editing (GE) of human somatic cells, including recommendations on product design, product manufacturing, product testing, preclinical safety assessment, and clinical trial design37.
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