FDA approves world’s first CRISPR gene-editing drug for sickle-cell disease
Gabrielle M. Etzel
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The Food and Drug Administration has approved the world’s first medicine employing CRISPR gene-editing technology to treat sickle cell disease, giving thousands of Americans new hope to treat the painful disease.
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research.
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The new treatment, Casgevy, developed by Vertex Pharmaceuticals and Crispr theraputics, was approved on Friday in a long-anticipated move that tackles genetic disorders by turning off or replacing specific genes.
Approximately 100,000 Americans are affected by sickle cell disease, which is the result of an inherited genetic mutation that produced a dysfunctional form of the protein hemoglobin, which carries oxygen in the bloodstream.
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Casgevy targets the specific gene that causes the dysfunction, switching it off and allowing for the production of normal hemoglobin proteins.
“Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited,” said Verdun.
This is a developing story.