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Landmark Approval: First CRISPR Therapy Approved by FDA

ByJackie Orabone

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…

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Quick Snips: CRISPR

ByJackie Orabone

CRISPR is a powerful gene-editing tool that enables targeted therapeutic gene editing, with clinical applications for improving treatment of inherited and/or rare genetic diseases, viral infections, and arresting the progression of cancer. CRISPR gene-editing technology has the potential to revolutionize the treatment of rare genetic disorders, with the first product poised for FDA approval in…