FDA charts path for approval of individualized gene editing therapies
The FDA has outlined steps drugmakers can take to seek regulatory approval for gene editing medicines tailored to fix a single person's disease-causing mutation. The announcement is part of an ongoing ...
The Food and Drug Administration (FDA) has released draft guidance that aims to expedite the approval process for personalized gene therapies, a development that is being welcomed by rare disease advocates. This move comes as breakthroughs in gene therapies have captured the attention of researchers and pharmaceutical companies, who believe these personalized medicines could significantly benefit patients with rare mutations.
Paul Scribner, vice president of community programs and education at the PKD Foundation, highlighted the significance of the FDA's initiative at a recent rare disease event.
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