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Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene

EU orphan designation number: EU/3/14/1257   
Active ingredient: Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene
Indication: Treatment of recombination-activating gene 1 deficient severe combined immunodeficiency
Sponsor: Prof. F.J.T.Staal
Leiden University Medical Center, building 1, room L3-35, P.O. Box 9600, 2300 RC Leiden, Nederland

   Public summary of scientific opinion    

European Commission proceduresGoto top of the page

Close date procedure Procedure type EMEA number Decision summary publ decision docs annex
28/03/2014 Orphan designation EMA/OD/188/13 (2014)2115 of 26/03/2014