ESGCT 2021 | GNT0003: AAV gene therapy for Crigler-Najjar syndrome

Lorenzo D’Antiga, MD, Hospital Papa Giovanni XXIII, Bergamo, Italy, describes the Phase I/II CareCN (NCT03466463) clinical trial evaluating GNT0003 therapy, an adeno-associated virus vector serotype 8 (AAV8) encoding hepatic UGT1A1, in patients with Crigler-Najjar syndrome (CN). In this dose-escalation study, no GNT0003-related serious adverse events were reported in the five patients treated. In patients treated with a higher dose, GNT0003 was demonstrated to be safe and restored UGT1A1 expression to levels allowing safe phototherapy withdrawal. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.