Non‑conditioned Autologous Gene Therapy Reverses Bone Marrow Failure in Fanconi Anaemia‑A: FANCOLEN‑1 Phase 1/2 and Long‑Term Outcomes
Posted inHematology-Oncology news

Non‑conditioned Autologous Gene Therapy Reverses Bone Marrow Failure in Fanconi Anaemia‑A: FANCOLEN‑1 Phase 1/2 and Long‑Term Outcomes

The FANCOLEN‑1 trial shows that infusion of autologous FANCA‑corrected CD34+ cells without cytotoxic conditioning produced sustained engraftment and clinical improvement in a majority of treated patients with Fanconi anaemia‑A, with an acceptable short‑term safety profile and no genotoxicity detected during follow‑up.