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

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.
Improving Outcomes in Mismatched Unrelated Donor Stem Cell Transplants With Post-Transplant Cyclophosphamide

Improving Outcomes in Mismatched Unrelated Donor Stem Cell Transplants With Post-Transplant Cyclophosphamide

Post-transplant cyclophosphamide (PTCy) for graft-versus-host disease prophylaxis significantly improves 1-year survival and lowers severe GVHD rates in mismatched unrelated donor peripheral blood stem cell transplants, expanding access for underrepresented patient groups.