Adeno-Associated Viral Vector Serotype 2/8 Containing The Human Ugt1A1 Gene

2 trials 2 recruiting Orphan designation

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2 Registered trials

2 Recruiting

0 Completed trials

1 Sponsors

3 Countries

Phase II

Phase I (FiH)

Phase II: 1 Phase I (FiH): 1

Indications tested

Crigler-Najjar syndrome 2

Sponsors using this IMP

All trials

2 total

Efficacy and safety trial of GNT0003 following imlifidase pre-treatment in participants aged 16 years and older with severe Crigler-Najjar s… Recruiting

2023-510405-18-00 Phase II 1 country Genethon
Gene Therapy for Crigler-Najjar Syndrome Recruiting

2023-507007-60-00 Phase I (FiH) 3 countries Genethon

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