FDA approves gene editing

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The U.S. Food and Drug Administration (FDA) has approved the use of gene-editing therapies for the treatment of certain diseases. The first cell-based gene therapies, Casgevy and Lyfgenia, were approved for the treatment of sickle cell disease in patients 12 years and older. Casgevy, in particular, is the first FDA-approved treatment to utilize CRISPR/Cas9, a type of genome editing technology. This technology allows for precise editing of DNA, enabling the removal, addition, or replacement of DNA where it was cut1. In January 2024, the FDA approved the use of Casgevy, a therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics, for treating transfusion-dependent beta thalassemia, a serious genetic disorder that hinders the production of hemoglobin. This marked the second major U.S. regulatory approval for the emerging gene-editing technology4511. Casgevy uses the CRISPR gene-editing technology to modify patients' blood cells and transplant them back into the patient, triggering an increase in the production of healthy red blood cells4. The therapy requires administration through authorized treatment centers with experience in stem cell transplantation11. Despite these advancements, there are still challenges to be addressed in the field of genome editing. These include the development of new tools capable of introducing genomic modifications in a more targeted manner, and addressing off-target effects, which can lead to potential genomic toxicity, genome instability, and even carcinogenesis312. The approval of these therapies represents a significant advancement in the field of gene therapy, particularly for individuals with rare diseases where current treatment options are limited1. However, the high cost of these therapies and the requirement for specialized treatment centers may limit their accessibility105.
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