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
 

Orphan Drug: Hematopoietic stem cells modified with a lentiviral vector containing the CD18 (integrin beta 2) gene for the treatment of leukocyte adhesion deficiency type I. 

Year: 2016

EU designation number: EU/3/16/1753

EU Sponsor: Ciber

USA-FDA designation number: DRU-2016-5430

USA-FDA Sponsor: Rocket Pharmaceuticals
 

Orphan Drug: Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene for the treatment of pyruvate kinase deficiency.

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