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Register of orphan medicinal products


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Product information

Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene

EU orphan designation number: EU/3/17/1955   
Active ingredient: Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene
Indication: Treatment of mucopolysaccharidosis type I
Sponsor: TxCell S.A.
Les Cardoulines Ht1, Allee De La Nertiere, 06560 Valbonne, France

   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
19/01/2018 Orphan designation EMA/OD/167/17 (2018)376 of 17/01/2018
2/05/2018 Change of name and/or address of sponsor
12/06/2018 Transfer of orphan designation EMA/OD/167/17/T/01 (2018)3810 of 8/06/2018
18/02/2019 Transfer of orphan designation EMA/OD/0000003576 (2019)1370 of 12/02/2019