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Landmark Approval: First CRISPR Therapy Approved by FDA

Today, the US Food and Drug Administration (FDA) approved exagamglogene autotemcel (exa‑cel), the first CRISPR-based gene therapy for the treatment of sickle cell disease (SCD). Exa‑cel was co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics. This is a landmark approval that paves the way not only for better outcomes for patients with SCD, but for many…