Non‑conditioned Autologous Gene Therapy Reverses Bone Marrow Failure in Fanconi Anaemia‑A: FANCOLEN‑1 Phase 1/2 and Long‑Term Outcomes
Highlights
• FANCOLEN‑1 is the first reported trial demonstrating sustained engraftment of autologous FANCA‑corrected haematopoietic cells in non‑conditioned patients with Fanconi anaemia‑A.
• At 2 years post‑infusion, 5 of 8 evaluable patients (62.5%) met the prespecified engraftment efficacy endpoint; median vector copies per nucleated cell were 0.18 in bone marrow and 0.06 in peripheral blood.
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