Biomedical Innovation Researchers producing an Advanced Therapy drug in the GMP cell production facility

Technology Transfer
  • Orphan Drugs
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  • Clinical Trials

ORPHAN DRUG TITLE: Autologous CD34+ cells edited with CRISPR/Cas9 and transduced with an adeno-associated virus vector serotype 6 containing the codon-optimized version of PKLR gene

EUROPEAN REFERENCE: EU/3/22/2580

YEAR: 2022

PROMOTER: CIBER

 

ORPHAN DRUG TITLE: Autologous CD34+ enriched hematopoietic stem cells genetically modified with LV vector containing RPS19 gene

EUROPEAN REFERENCE: EU/3/21/2519

YEAR: 2021

PROMOTER: CIBER

 

ORPHAN DRUG TITLE: Autologous skin equivalent graft composed of keratinocytes and fibroblasts genetically corrected by CRISPR/Cas9-mediated excision of mutation-carrying COL7A1 exon 80

EUROPEAN REFERENCE: EU/3/20/2253

YEAR: 2020

PROMOTER: CIBERER

 

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

EUROPEAN REFERENCE: EU/3/16/1753

YEAR: 2016

FDA REFERENCE: FDA #DRU-2016-5430

YEAR: 2016

PROMOTER: CIBERER

 

ORPHAN DRUG TITLE: Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene for the therapy of Pyruvate Kinase Deficiency (MERILEN)

EUROPEAN REFERENCE: EU/3/14/1330

YEAR: 2014

FDA REFERENCE: FDA #DRU-2016-5168

YEAR: 2016

PROMOTER: CIBERER

 

ORPHAN DRUG TITLE: Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for the treatment of Fanconi anaemia type A

EUROPEAN REFERENCE: EU/3/10/822

YEAR: 2010

FDA REFERENCE: FDA #DRU-2016-5193

YEAR: 2016

PROMOTER: CIBERER