Sickle Cell Disease Gene Therapies Using CRISPR Approach Possible FDA Approval
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Sickle cell disease causes red blood cells to warp into crescent shapes that get stuck in blood vessels, leading to pain, organ damage, and possibly death.
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Vertex Pharmaceuticals' exa-cel, developed with CRISPR Therapeutics, edits genes to treat sickle cell and could soon be the first CRISPR-based treatment approved by the FDA.
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Exa-cel removes stem cells, uses CRISPR to snip out the mutated sickle cell gene, then returns the cells to the patient to produce normal hemoglobin.
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The treatment requires chemotherapy and extended hospital stay, but did not have unwanted CRISPR gene edits in trials.
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Competitor Bluebird Bio also has a sickle cell gene therapy awaiting potential FDA approval in December, but the high costs of these treatments is generating discussion.
