CASGEVY
CASGEVY (exagamglogene autotemcel) is an ex vivo gene therapy co-developed by CRISPR Therapeutics and Vertex Pharmaceuticals that uses CRISPR/Cas9 to edit patients' own blood stem cells, providing a functional cure for sickle cell disease and transfusion-dependent beta thalassemia. The therapy harvests a patient's stem cells, disables the BCL11A enhancer — a genetic switch that keeps protective fetal hemoglobin silenced after birth — then reinfuses the edited cells after conditioning chemotherapy, reactivating fetal hemoglobin to compensate for defective adult hemoglobin. CASGEVY is the first FDA-approved CRISPR-based medicine and the first curative option for these hemoglobin disorders outside of allogeneic stem cell transplant.
Upcoming catalysts
Programs
Sickle Cell Disease
Sickle cell disease affects ~100,000 Americans — predominantly of African or Hispanic descent — causing painful vaso-occlusive crises, organ damage, stroke, and a median life expectancy roughly 20 years shorter than the general population; the only prior curative option was allogeneic stem cell transplant from a matched donor. CASGEVY is the first approved CRISPR therapy; this program tracks 5-year durability with data at ASH 2026 to establish the robustness of VOC elimination and fetal hemoglobin persistence needed for broad payer coverage.