rAAV8-hUGT1A1

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

An open-label, phase 2 trial to evaluate the efficacy and safety of a single intravenous administration of GNT0003 (an adeno-associated viral vector expressing the UGT1A1 transgene) following imlifidase pre-treatment in participants aged 16 years and older with severe Crigler-Najjar syndrome requiring daily phototherapy and presenting pre-existing anti-AAV8 antibodies.