reni-cel
Reni-cel is Editas Medicine's ex vivo gene-editing therapy for sickle cell disease and beta thalassemia that uses CRISPR/Cas12a — rather than the more common Cas9 — to edit patient-derived blood stem cells and reactivate fetal hemoglobin production. After the edited cells are reinfused following conditioning chemotherapy, elevated fetal hemoglobin compensates for defective adult globin, preventing red blood cell sickling and reducing transfusion dependence. Editas positions reni-cel as a next-generation alternative to CASGEVY, with the hypothesis that Cas12a provides a distinct precision profile at the BCL11A enhancer locus.
Upcoming catalysts
Programs
Sickle Cell Disease
Sickle cell disease causes red blood cells to sickle and obstruct blood vessels due to a point mutation in HBB; reactivating fetal hemoglobin (HbF) compensates for defective adult hemoglobin, the same mechanism pursued by CASGEVY and BEAM-101. Editas uses CRISPR/Cas12a for the BCL11A enhancer edit rather than Cas9; Phase 1/2 data including HbF %, VOC rates, and transfusion independence are being presented at ASH 2026 to inform the pivotal trial design.