Gene therapy is a new kind of specialty medical treatment. A patient receives a one-time injection of DNA-filled viruses, and those viruses get to work changing the way a patient’s cells react. So far, three gene therapy treatments have been approved by the U.S. Food and Drug Administration: two are CAR T-cell immunotherapy treatments used for fighting leukemia, and one treats a hereditary blindness condition. Gene therapy is now considered an experimental treatment. If it enters the standard of care, payers may be faced with a whole new universe of high-cost claims.
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