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A gene therapy has received clearance for use in American patients, one of at least two approvals the Food and Drug Administration processed in a short stretch this week.
Gene therapy, in plain terms, is a one-time medical intervention that modifies the genetic instructions inside a patient's cells rather than delivering a drug the body absorbs on a recurring schedule.
The treatment that cleared the FDA was developed by Ultragenyx. Merck and Moderna's cancer vaccine drew separate attention in the same stretch, with both companies describing it as potentially field-changing.
A cancer vaccine trains the immune system to identify and attack tumor cells rather than prevent an infection. A new FDA commissioner nominee also entered the news this week.
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