GNT0003: AAV gene therapy for Crigler-Najjar syndrome

admin_vjr_user — Thu, 23 Dec 2021 16:24:51 +0000

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.

The unmet medical need for Crigler-Najjar syndrome

admin_vjr_user — Wed, 22 Dec 2021 18:29:32 +0000

Lorenzo D’Antiga, MD, Hospital Papa Giovanni XXIII, Bergamo, Italy, describes Crigler-Najjar syndrome (CN), an inherited liver metabolism disorder cause by a lack of hepatic uridine diphosphoglucuronate glucuronosyltransferase 1A1 (UGT1A1), which results in severe unconjugated hyperbilirubinemia that can cause irreversible neurological injury and death. The current standard-of-care is daily phototherapy, however this treatment becomes less effective with increasing age, making liver transplantation ultimately necessary. This has prompted the investigation of gene therapy approaches with the aim of finding novel, less invasive curative strategies for CN. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

Crigler-Najjar Syndrome – VJRegenMed

GNT0003: AAV gene therapy for Crigler-Najjar syndrome

The unmet medical need for Crigler-Najjar syndrome