Overview
- Peer-reviewed phase 1 data report 82% achieving very deep remission and 64% remaining disease-free for up to three years after BE-CAR7.
- The study treated nine children and two adults with refractory T-ALL at Great Ormond Street and King’s College Hospitals in London.
- BE-CAR7 uses donor T cells modified by base editing to create universal CAR T cells that eliminate CD7-positive T cells, enabling a subsequent stem-cell transplant to restore immunity.
- Investigators said short-term toxicities such as low blood counts, rashes and cytokine release syndrome were manageable, with infection risk highest before immune recovery.
- Great Ormond Street Hospital Charity will fund treatment for 10 additional patients as teams advance larger studies and routes to wider access.