Overview
- Phase I data published in the New England Journal of Medicine and presented at ASH report very deep remissions in 82% of participants and 64% remaining disease-free, with the earliest patients now about three years off treatment.
- The BE‑CAR7 approach uses base editing to create universal donor CAR T cells that target CD7 on leukemic T cells, enabling rapid clearance of disease before a planned stem-cell transplant.
- The first-in-human program was led by Great Ormond Street Hospital and University College London with treatments delivered at GOSH and King’s College Hospital to both children and adults with relapsed or refractory T‑ALL.
- Toxicities were generally manageable, though clinicians flagged frequent viral reactivations and infection risks during immune reconstitution, as well as occasional CD7 antigen loss that could limit durability.
- Following the results, Great Ormond Street Hospital Charity committed support to treat approximately 10 additional patients, while investigators emphasize ongoing safety monitoring and the need for larger trials.