First In human Successful CRISPR/Cas9 Gene Therapy (Exagamglogene Autotemcel) in a Heart Transplant Recipient with Beta-Thalassemia Major (TTS 2026) - Sep 17, 2026 - Pres time: Sep 21, 2026; 04:04 PM - 04:09 PM; Location: Room C4.11; 294; "Post-transplant, he was maintained on immunosuppression including tacrolimus and mycophenolate mofetil...He underwent myeloablative conditioning in Aug 2025 with intravenous Busulfan from Day -7 to Day -4, dynamically adjusted to a target AUC of 900 micromol-min/L via pharmacokinetic monitoring... This first-in-human case establishes the feasibility and safety of administering myeloablative CRISPR/Cas9-edited gene therapy in a highly immunosuppressed, recent heart transplant recipient. Curing the underlying beta-thalassemia and achieving transfusion independence definitively eliminates the primary source of iron overload, thereby protecting the cardiac allograft. Crucially, this success highlights that a history of solid organ transplantation and concurrent immunosuppression should no longer serve as exclusion criteria, paving the way for future patients with severe hemoglobinopathies to access life-saving gene therapies."
Clinical • First-in-human • Gene therapy • P1 data • Atrial Fibrillation • Beta-Thalassemia • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Gene Therapies • Genetic Disorders • Heart Failure • Hematological Disorders • Hepatology • Solid Organ Transplantation • Transplant Rejection • Transplantation • CD34
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