Orphan Drugs - Terapias innovadoras
Orphan Drug: Autologous CD34+ enriched hematopoietic stem cells genetically modified with LV vector containing RPS19 gene.
Year: 2021
EU designation number: EU/3/21/2519
EU Sponsor: Ciber
Year: 2016
EU designation number: EU/3/16/1753
EU Sponsor: Ciber
USA-FDA designation number: DRU-2016-5430
USA-FDA Sponsor: Rocket Pharmaceuticals
Year: 2014
EU designation number: EU/3/14/1330
Sponsor EU: Ciber
USA-FDA designation number: DRU-2016-5168
USA-FDA Sponsor: Rocket Pharmaceuticals
Orphan Drug: Lentiviral vector carrying the Fanconi anemia-A (FANCA) gene for the treatment of Fanconi anemia type A.
Year: 2010
EU designation number: EU/3/10/822
Sponsor EU: Ciber
USA-FDA designation number: DRU-2016-5193
USA-FDA Sponsor: Rocket Pharmaceuticals