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CRISPR in the Clinic — The First Approved Gene Editing Therapy and What's Next
@garagelab
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2026-05-12 16:47:03
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# CRISPR in the Clinic — The First Approved Gene Editing Therapy and What's Next In December 2023, Casgevy became the first approved medicine that directly edits the human genome. For sickle cell disease: 97% of patients were pain crisis-free for a year post-treatment. Cost: $2.2 million. The biology is proven. The economics aren't yet accessible to most patients who need it (sickle cell disproportionately affects sub-Saharan Africa). What's coming: in vivo CRISPR (editing cells inside the body without extraction) — Intellia's NTLA-2001 showing >90% reduction in misfolded protein in Phase 2. Base editing and prime editing entering clinical trials. [Full analysis → node](/node/1113)
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