EMEA-002021-PIP01-16-M01 - paediatric investigation plan
rAAV8 viral vector encoding the human UGT1A1 transgene (rAAV8-hUGT1A1)
PIPHuman
Key facts
- Active Substance
- rAAV8 viral vector encoding the human UGT1A1 transgene (rAAV8-hUGT1A1)
- Therapeutic area
- Congenital, familial and genetic disorders
- Decision number
- P/0228/2024
- PIP number
- EMEA-002021-PIP01-16-M01
- Pharmaceutical form(s)
- Concentrate for solution for infusion
- Condition(s) / indication(s)
- Treatment of Crigler-Najjar syndrome
- Route(s) of administration
- Intravenous use
- Contact for public enquiries
Genethon
E-mail: clinical_development@genethon.fr
Tel: +33 169472828- Decision type
- PM: decision on the application for modification of an agreed PIP
- Decision date