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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

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.
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Coordinator

CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS-CIEMAT

Address

Avenida Complutense 40
28040 Madrid

Spain

Activity type

Research Organisations

EU Contribution

€ 689 659

Administrative Contact

Ana Collados Martín-Posadillo (Ms.)

Participants (10)

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ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS

France

EU Contribution

€ 498 120

UNIVERSITY COLLEGE LONDON

United Kingdom

EU Contribution

€ 493 800

SERVICIO MADRILENO DE SALUD

Spain

EU Contribution

€ 293 760

INSTITUT CATALA DE LA SALUT

Spain

EU Contribution

€ 268 368

ASSOCIATION GENETHON

France

EU Contribution

€ 630 918,75

UNIVERSITE PARIS DIDEROT - PARIS 7

France

EU Contribution

€ 264 600

UNIVERSITAT AUTONOMA DE BARCELONA

Spain

EU Contribution

€ 259 845

Eurofins Genomics Europe Sequencing GmbH

Germany

EU Contribution

€ 654 192

DEUTSCHES KREBSFORSCHUNGSZENTRUM HEIDELBERG

Germany

EU Contribution

€ 271 680

INNOVACION DESARROLLO Y TRANSFERE

Spain

EU Contribution

€ 1 055 227

Project information

Grant agreement ID: 305421

Status

Closed project

  • Start date

    1 January 2013

  • End date

    31 December 2018

Funded under:

FP7-HEALTH

  • Overall budget:

    € 7 014 945,20

  • EU contribution

    € 5 380 169,75

Coordinated by:

CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS-CIEMAT

Spain

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