Other Results

 

Orphan Drugs

Date: 9/11/2017
Drug name:Células madre hematopoyéticas modificadas con un vector lentiviral que contiene el gen CD18 (ITGB2) 
More information: FDA 16-5453
Number EU: 
Description: Deficiencia de adhesión leucocitaria tipo I

Date: 14/10/2016
Drug name: Células madre hematopoyéticas modificadas con un vector lentiviral que contiene el gen CD18 (ITGB2)  
More information: EMA/OD/154/16
Number EU: (EU/3/16/1753)
Description: Deficiencia de adhesión leucocitaria tipo I

Date: 23/06/2016
Drug name:  Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene for the treatment of pyruvate kinase deficiency
More information: FDA designation number: DRU-2016-5168
Number EU: 
Description: Anemia hemolítica por deficiencia de piruvato quinasa de los glóbulos rojos

Date: 02/05/2016
Drug name: Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for the treatment of Fanconi anaemia type A.
More information: FDA Designation request no. 16-5193
Description: Anemia de Fanconi

 

Date: 22/08/2014
Drug name: Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene for the treatment of pyruvate kinase deficiency.
More information: Sponsor CIBERER 
http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2014/10/human_orphan_001420.jsp&mid=WC0b01ac058001d12b
Number EU: EU/3/14/1330
Description: http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2014/10/WC500174547.pdf

 

Date: 18/01/2011
Drug name: ORPHA260287: Lentiviral vector carrying the Fanconi anemia-A (FANCA) gene for the treatment of Fanconi anemia type A.
More information: Orphan number: ORPHA260287
Number EU: EU/3/10/822
Description: This orphan designation (EU/3/10/822) was granted by the European Commission to the Centre for Biomedical Network Research on Rare Diseases (CIBERER), Spain, for lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for the treatment of Fanconi anaemia type A.

Patents

Patent: "GENE THERAPY FOR PATIENTS WITH FANCONI ANEMIA"
Priority application number: US201662412028
Inventors: Juan Bueren, Guillermo Guenechea, Julian Sevilla, Paula Rio, África González, José Carlos Segovia, Susana Navarro, Juan Antonio Casado
Type: Otros países; USA
Priority date: 24/10/2016
Patent family: 26/07/2017
Abstract: Acuerdo de licencia con Rocket Pharma en Anemia de Fanconi

Patent: "Compositions and methods for enhanced gene expression of PKLR"
Priority application number: RTWI-001/00US (326219-2001)
Inventors: Jose C. Segovia, María Garcia-Gomez, Susana Navarro, Nestor W. Meza, Juan A. Bueren, María Garcia-Bravo
Type: Otros países; USA
Priority date: 20/04/2016
Patent family: 
Abstract: HOLDER ENTITY: CIEMAT OTHER COUNTRIES WHICH THE PATENT HAS BEEN EXTENDED TO: COMPANIES THAT ARE LICENSING IT: ROCKET PHARMACEUTICALS

Patent: "Knock-in Induced Pluripotent Stem Cells (iPSCs) derived from peripheral blood mononuclear cells and uses thereof".
Priority application number: EP15382545
Inventors: José Carlos Segovia
Type: Europea
Priority date: 05/11/2015
Patent family: 08/03/2016
Abstract: Acuerdo de cooperación, desarrollo y licencia con Rocket Pharmaceuticals

Patent: "Generation of genetically corrected disease-free induced pluripotent stem cells"
Priority application number: US20090181287P
Inventors: A. Raya, JA. Bueren, JC. Izpisúa-Belmonte
Type: Otros países; US
Priority date: 27/05/2009
Patent family: 2021484
Abstract: A method for preparing a genetically corrected induced pluripotent stem cell is provided. The method includes transfecting a genetically diseased non-pluripotent cell with a nucleic acid encoding a disease correcting gene to form a genetically corrected non pluripotent cell. The genetically corrected non-pluripotent cell is transfected with a nucleic acid encoding an OCT4 protein, a nucleic acid encoding a SOX2, KLF4 and cMYC to form a genetically corrected transfected non-pluripotent cell.

Patent: "Vectores de expresión que comprenden el promotor del gen PKLR humano y su uso para la elaboración de composiciones farmacéuticas destinadas a terapia génica somática con expresión específica en células eritroides"
Priority application number: P20070001024
Inventors: JC Segovia-Sanz, NW Meza; JA Bueren, E Almarza-Novoa
Type: Española
Priority date: 17/04/2007
Patent family: PCT/ES2008/070069
Abstract: Expression vectors comprising the promoter of the human PKLR gene and its use for the preparation of pharmaceutical compositions intended for somatic gene therapy, with specific expression in erythroid cells. The present invention relates to the use of regulatory sequences from the PKLR gene promoter in the production of vectors, with the exception of vectors comprising adeno-associated virus, for use in the preparation of pharmaceutical compositions intended for somatic gene therapy in erythroid tissue .

Patent: "Generation of gene therapy vectors with physiological expression levels of transgenes driven by the proximal promoter of the vav1 oncogene"
Priority application number: ES20060001477
Inventors: E Almarza, M Aldea, G Guenechea, JC Segovia, JA Bueren
Type: Española
Priority date: 01/06/2006
Patent family: PCT/EP2007/055368
Abstract: The present invention relates to the use of the HS1 promoter of the vav oncogene in the production of vectors selected from the groups of integrative vectors and non- integrative, non-plasmid vectors, for use in the preparation of pharmaceutical compositions intended for somatic gene therapy. By generating vectors that contain the HS1 promoter of the vav oncogene, it was possible to generate vectors in which the marker transgene or therapeutic transgene is expressed at moderate, but stable, levels in various cell lines both in vitro and in vivo.

Patent: "Use of adipose derived mesenchymal stem cells for the treatment of graft versus host disease"
Priority application number: EP20050380266
Inventors: MA González de la Peña, JA. Bueren, RM Yáñez-González, ML Lamana-Luzuriaga, D Büscher y A Beraza-Pérez
Type: Europea
Priority date: 07/12/2005
Patent family: PCT/EP2006/069426 
Abstract: The present invention relates to the use of a particular type of adipose tissue derived mesenchymal stem cells (AD-MSCs), which exert immunosuppressive properties, in the manufacture of a pharmaceutical composition for the prevention and treatment of the graft-versus-host disease (GVHD) produced after allogeneic hematopoietic stem cell transplantation.

Clinical Trials

Title: "Ensayo clínico FaseI/II para evaluar la seguridad y eficacia de la Infusión de células CD34+ autólogas transducidas con un vector lentiviral portador del gen FANCA (medicamento huérfano) para pacientes con Anemia de Fanconi del Subtipo A"

PI: Juan A. Bueren

Starting year: 2013; End year: 2016

Reference: Nº Eudra CT 2011-006100-12 Promoter: Julián Sevilla, Hospital del Niño Jesús (Madrid)

Phase: Phase II  

 

Title: "Ensayo clínico Fase II para evaluar la seguridad y eficacia de la movilización y colecta de células CD34+ tras tratamiento con plerixafor y filgrastim en pacientes con anemia de Fanconi para su posterior transducción con un vector lentiviral portador del gen FANCA y reinfusión en el paciente"

PI: Juan A. Bueren y Cristina Díaz de Heredia

Starting year: 2012; End year: 2016

Reference: Nº Eudra CT 2011-006197-88 Promoter: Cristina Díaz de Heredia

Phase: Phase II

Title: "Phase Ib/IIa, escalating dose, single-blind, clinical trial to assess the safety of the intravenous administration of expanded allogeneic adipose-derived mesenchymal stem cells (eASCs) to refractory rheumatoid arthritis (RA) patients"

PI: Marina Inmaculada Garín Ferreira

Starting year: 2012; End year: 2014

Reference: Nº Eudra CT 2010-021602-37 Promoter: TiGenix, S.A.U.

Phase: Phase I

 

Title: "Ensayo clínico de terapia génica para el control de la enfermedad del injerto contra el huésped en pacientes mayores de 45 años sometidos a trasplante alogénico de progenitores hematopoyéticos"

PI: Juan A. Bueren

Starting year: 2006; End year: 2008

Reference: PI061005; Promoter: Instituto de Salud Carlos III. Fondo de Investigación Sanitaria

Phase: Phase II