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CRISPR Therapeutics
Overview
The world's first approved CRISPR/Cas9 gene-editing medicine (exagamglogene autotemcel). A one-time therapy that edits the BCL11A gene in a patient's own blood stem cells to reactivate fetal hemoglobin, treating sickle cell disease and transfusion-dependent beta thalassemia. Partnered with Vertex Pharmaceuticals.
Approved across 9+ jurisdictions and priced at ~$2.2M per treatment. In July 2026 the FDA expanded the US label to patients ages 2+. Uptake is ramping — >500 cumulative patient starts and >75 US treatment centers — though gated by cell-collection and manufacturing timelines.