Other Results

 

Orphan Drugs

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

 

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: "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