EMEA-003001-PIP01-21-M01 - paediatric investigation plan
Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified with the lentiviral vector IDUA LV, encoding for the human α-L-iduronidase (IDUA) gene (OTL-203)
PIPHuman
Orchard Therapeutics (Netherlands) B.V.
regulatory@orchard-tx.com
Tel. +44 (0) 20 3808 8286