Overview

• The antibody briquilimab targets CD117 to clear host hematopoietic stem cells without chemotherapy or radiation toxicity.
• All three pediatric participants achieved donor cell engraftment within two weeks and near-complete donor dominance by day 30, maintained at nearly 100% after two years.
• Alpha/beta T-cell depletion and CD34+ stem cell enrichment enabled successful transplants using half-matched parental donors while reducing graft-versus-host disease risk.
• Full Phase I outcomes published in Nature Medicine confirmed safety and rapid engraftment in fragile Fanconi anemia patients with no graft rejection.
• Investigators have launched a multicenter Phase II trial and plan to evaluate the antibody-based protocol in other inherited marrow failure syndromes such as Diamond-Blackfan anemia.