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Are there any FDA approved CRISPR therapies?

Asked by Anonymous Sep 02, 2026 0 views 1 answers
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Answered Sep 02, 2026

Yes, milestone regulatory approvals have marked the arrival of CRISPR-based gene-editing therapies in modern medicine, with regulatory agencies granting historic green lights to groundbreaking treatments. The United States Food and Drug Administration approved Casgevy (exagamglogene autotemcel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, as a revolutionary gene-therapy treatment for sickle cell disease and transfusion-dependent beta-thalassemia. This landmark approval represented the commercial validation of CRISPR/Cas9 gene-editing technology, allowing clinicians to precisely modify patient hematopoietic stem cells to produce functional fetal hemoglobin. Ongoing clinical pipelines continue to explore additional CRISPR applications for diverse genetic disorders, cancers, and rare metabolic diseases.

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