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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

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 plerixafor+G-CSF mobilized HSCs with the novel lentiviral vector, accompanied by comprehensive and groundbreaking safety and efficacy patient monitoring studies.

Call for proposal

FP7-HEALTH-2012-INNOVATION-1
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Coordinator

CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS-CIEMAT
Address
Avenida Complutense 40
28040 Madrid
Spain

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Activity type
Research Organisations
Administrative Contact
Ana Collados Martín-Posadillo (Ms.)
EU contribution
€ 689 659

Participants (10)

ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
France
EU contribution
€ 498 120
Address
3 Avenue Victoria
75000 Paris

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Activity type
Research Organisations
Administrative Contact
Alix Pillot (Ms.)
UCL Elizabeth Garrett Anderson Institute for Women’s Health
United Kingdom
EU contribution
€ 493 800
Address
Gower Street
WC1E 6BT London

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Greta Borg-Carbott (Mrs.)
SERVICIO MADRILENO DE SALUD
Spain
EU contribution
€ 293 760
Address
Plaza Carlos Trias Bertran 7
28020 Madrid

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Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Juan Ignacio De Juan Fernandez (Mr.)
INSTITUT CATALA DE LA SALUT
Spain
EU contribution
€ 268 368
Address
Gran Via De Les Corts Catalanes 587
08007 Barcelona

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Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Joan Comella (Dr.)
ASSOCIATION GENETHON
France
EU contribution
€ 630 918,75
Address
Rue De L Internationale 1 Bis
91002 Evry

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Activity type
Research Organisations
Administrative Contact
Jean-Philippe Marin (Mr.)
Université Paris Diderot-Paris 7
France
EU contribution
€ 264 600
Address
Rue Thomas Mann
F-75205 / 13 Paris

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Anne Bonvalet (Ms.)
UNIVERSIDAD AUTONOMA DE BARCELONA
Spain
EU contribution
€ 259 845
Address
Calle Campus Universitario Sn Cerdanyola V
08290 Cerdanyola Del Valles

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Francisca Rabadán Sotos (Ms.)
Eurofins Genomics Europe Sequencing GmbH
Germany
EU contribution
€ 654 192
Address
Jakob Stadler Platz 7
78467 Konstanz

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Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Janet Kenklies (Mrs.)
DEUTSCHES KREBSFORSCHUNGSZENTRUM HEIDELBERG
Germany
EU contribution
€ 271 680
Address
Im Neuenheimer Feld 280
69120 Heidelberg

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Activity type
Research Organisations
Administrative Contact
Ina Krischek (Dr.)
INNOVACION DESARROLLO Y TRANSFERE
Spain
EU contribution
€ 1 055 227
Address
Calle Hermosilla, Numero 48, 3 Derecha
28001 Madrid

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Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Gema Roque (Mrs.)