EUROFANCOLEN

Phase I/II Gene Therapy Trial of Fanconi anemia patients with a new Orphan Drug consisting of a lentiviral vector carrying the FANCA gene: A Coordinated International Action

 Coordinatore CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS-CIEMAT 

 Organization address address: Avenida Complutense 22
city: MADRID
postcode: 28040

contact info
Titolo: Ms.
Nome: Ana
Cognome: Collados Martín-Posadillo
Email: send email
Telefono: +34 91 34 66 096
Fax: +34 91 34 66 480

 Nazionalità Coordinatore Spain [ES]
 Totale costo 7˙002˙945 €
 EC contributo 5˙380˙169 €
 Programma FP7-HEALTH
Specific Programme "Cooperation": Health
 Code Call FP7-HEALTH-2012-INNOVATION-1
 Funding Scheme CP-FP
 Anno di inizio 2013
 Periodo (anno-mese-giorno) 2013-01-01   -   2017-12-31

 Partecipanti

# participant  country  role  EC contrib. [€] 
1    CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS-CIEMAT

 Organization address address: Avenida Complutense 22
city: MADRID
postcode: 28040

contact info
Titolo: Ms.
Nome: Ana
Cognome: Collados Martín-Posadillo
Email: send email
Telefono: +34 91 34 66 096
Fax: +34 91 34 66 480

ES (MADRID) coordinator 689˙659.00
2    INNOVACION DESARROLLO Y TRANSFERENCIA DE TECNOLOGIA SA

 Organization address address: CALLE HERMOSILLA 48-3 IZQ
city: Madrid
postcode: 28003

contact info
Titolo: Mrs.
Nome: Gema
Cognome: Roque
Email: send email
Telefono: +34 91 577 40 14

ES (Madrid) participant 1˙055˙227.00
3    GATC BIOTECH AG

 Organization address address: JAKOB STADLER PLATZ 7
city: KONSTANZ
postcode: 78467

contact info
Titolo: Mrs.
Nome: Janet
Cognome: Kenklies
Email: send email
Telefono: 4975320000000
Fax: 497532000000

DE (KONSTANZ) participant 654˙192.00
4    ASSOCIATION GENETHON

 Organization address address: RUE DE L INTERNATIONALE 1 BIS
city: EVRY
postcode: 91002

contact info
Titolo: Mr.
Nome: Jean-Philippe
Cognome: Marin
Email: send email
Telefono: +33 1 69 47 25 72
Fax: +33 1 69 47 19 46

FR (EVRY) participant 630˙918.75
5    ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS

 Organization address address: 3 Avenue Victoria
city: PARIS
postcode: 75004

contact info
Titolo: Ms.
Nome: Alix
Cognome: Pillot
Email: send email
Telefono: +33 1402 74613
Fax: +33 1448 41788

FR (PARIS) participant 498˙120.00
6    UNIVERSITY COLLEGE LONDON

 Organization address address: GOWER STREET
city: LONDON
postcode: WC1E 6BT

contact info
Titolo: Mrs.
Nome: Greta
Cognome: Borg-Carbott
Email: send email
Telefono: +44 20 31083033

UK (LONDON) participant 493˙800.00
7    SERVICIO MADRILENO DE SALUD

 Organization address address: PLAZA CARLOS TRIAS BERTRAN 7
city: MADRID
postcode: 28020

contact info
Titolo: Mr.
Nome: Juan Ignacio
Cognome: De Juan Fernandez
Email: send email
Telefono: 34915035900

ES (MADRID) participant 293˙760.00
8    DEUTSCHES KREBSFORSCHUNGSZENTRUM

 Organization address address: Im Neuenheimer Feld 280
city: HEIDELBERG
postcode: 69120

contact info
Titolo: Dr.
Nome: Ina
Cognome: Krischek
Email: send email
Telefono: +49 6221 422700
Fax: +49 6221 422708

DE (HEIDELBERG) participant 271˙680.00
9    INSTITUT CATALA DE LA SALUT

 Organization address address: GRAN VIA DE LES CORTS CATALANES 587
city: BARCELONA
postcode: 8007

contact info
Titolo: Dr.
Nome: Joan
Cognome: Comella
Email: send email
Telefono: +34 93 489 40 63
Fax: +34 93 489 41 02

ES (BARCELONA) participant 268˙368.00
10    UNIVERSITE PARIS DIDEROT - PARIS 7

 Organization address address: RUE THOMAS MANN 5
city: PARIS
postcode: 75205

contact info
Titolo: Ms.
Nome: Anne
Cognome: Bonvalet
Email: send email
Telefono: +33 1 57 27 55 59
Fax: +33 1 57 27 66 62

FR (PARIS) participant 264˙600.00
11    UNIVERSITAT AUTONOMA DE BARCELONA

 Organization address address: Campus UAB -BELLATERRA- s/n
city: CERDANYOLA DEL VALLES
postcode: 8193

contact info
Titolo: Ms.
Nome: Francisca
Cognome: Rabadán Sotos
Email: send email
Telefono: +34 93 581 3752

ES (CERDANYOLA DEL VALLES) participant 259˙845.00

Mappa


 Word cloud

Esplora la "nuvola delle parole (Word Cloud) per avere un'idea di massima del progetto.

patients    hematopoietic    fa    hscs    recent    cell    vectors    disease    correction    gene    safe    lentiviral    hsc    cells    genetic    therapy    vector   

 Obiettivo del progetto (Objective)

'Fanconi anemia (FA) is a rare inherited syndrome characterized by the early development of bone marrow failure and increasing predisposition to cancer with age. Allogeneic hematopoietic cell transplantation (alloHCT) is the only curative therapy for hematopoietic manifestations of FA, although associated with complications arising from myeloablation, graft versus host disease and increased incidence of squamous cell carcinoma. The genetic correction of autologous hematopoietic stem cells (HSC) with lentiviral vectors constitutes a recent and safe alternative for the treatment of different genetic diseases affecting mature cells from different tissues and/or committed progenitors of the hematopoietic system. One of the key features of FA that make it a unique disease for gene therapy approaches rely on the characteristic proliferation defect that is already evident in the very primitive HSCs. Thus, a marked survival advantage would be expected from corrected HSCs, potentially allowing normalization of hematopoiesis in the absence or after mild conditioning. Difficulties in the collection of sufficient numbers of HSC from FA patients and the use of sub-optimal transduction protocols with gammaretroviral vectors limited the success of FA gene therapy trials conducted 10 years ago in the USA. Our innovative approach to develop for the first time an efficient and safe gene therapy of FA is based on two recent innovations: 1) Discovery of potent HSC mobilizers, such as plerixafor, and 2) Development of a new lentiviral vector by members of this Consortium, designed as Orphan Drug by the EC in December 2010. The principal objective of this Project is, therefore, the development of a multicentric Phase I/II gene therapy trial for FA-A patients, based on the genetic correction of plerixaforG-CSF mobilized HSCs with the novel lentiviral vector, accompanied by comprehensive and groundbreaking safety and efficacy patient monitoring studies.'

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