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CareCN: A phase I/II, open-label, study to evaluate the safety and efficacy of an intravenous injection of GNT0003 (Adeno-associated Viral Vector expressing the UGT1A1 transgene) in patients with severe Crigler-Najjar syndrome requiring phototherapy

This study has been transitioned to CTIS with ID 2023-507007-60-00 check the CTIS register for the current data. Primary objectives:Dose escalation part:- To assess the safety and tolerability of an intravenous single-dose administration of GNT0003…