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Transposon-based, targeted ex vivo gene therapy to treat age-related macular degeneration (AMD)

Objective

Age-related Macular Degeneration (AMD), a neurodegenerative disease of the retina, is a major cause of blindness in elderly people. Due to the aging population, AMD has been referred to as a “time bomb” in society. In the exudative form of AMD, high levels of vascular endothelial cell growth factor (VEGF) and low levels of pigment-epithelial derived factor (PEDF), an inhibitor of vascularization and a neuroprotective factor produced by retinal pigment epithelial (RPE) cells result in subretinal neovascularization and retinal pigment cell degeneration. The current treatment by monthly injections of anti-VEGF antibodies is only effective for ~30% of patients. To avoid the severe side effects, high costs and the overall continuing burden on health care associated with monthly antibody injections, inducing a higher level of PEDF expression to inhibit neovascularization would be a viable therapeutic alternative. TargetAMD will subretinally transplant genetically modified, patient-derived, Iris Pigment Epithelial cells that overexpress PEDF to provide a long-lasting cure of AMD. Stable PEDF gene delivery will be based on the non-viral Sleeping Beauty transposon system, which combines the efficacy of viral delivery with the safety of naked DNA plasmids. Academic scientists and SME partners will produce innovative gene delivery technologies, reagents and devices to be translated into a simple and safe gene therapeutic treatment for exudative AMD. Experienced clinicians will perform one clinical trial, comprising isolation and PEDF-transfection of a patient’s pigment epithelial cells and implantation of transfected cells into the patient during a single, 60-minute surgical session. This project will bring a significant enhancement on quality of life to AMD patients, highlight the synergistic power of academic, clinical and industrial cooperation to the scientific arena, and open new markets for novel products for clinical applications of transposon-based gene therapy to industry.

Call for proposal

FP7-HEALTH-2012-INNOVATION-1
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

Coordinator

UNIVERSITE DE GENEVE
Address
Rue Du General Dufour 24
1211 Geneve
Switzerland
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 1 707 273,65
Administrative Contact
Gabriele Thumann (Prof.)

Participants (14)

RHEINISCH-WESTFAELISCHE TECHNISCHE HOCHSCHULE AACHEN
Germany
EU contribution
€ 279 980,32
Address
Templergraben 55
52062 Aachen
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Ernst Schmachtenberg (Prof.)
MAX DELBRUECK CENTRUM FUER MOLEKULARE MEDIZIN IN DER HELMHOLTZ-GEMEINSCHAFT (MDC)
Germany
EU contribution
€ 282 004,18
Address
Robert Rossle Strasse 10
13125 Berlin
Activity type
Research Organisations
Administrative Contact
Iwan Meij (Dr.)
BUNDESINSTITUT FUR IMPFSTOFFE UND BIOMEDIZINISCHE ARZNEIMITTEL
Germany
EU contribution
€ 301 336
Address
Paul-ehrlich-strasse 51-59
63225 Langen
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Stefanie Prokopp
UNIVERSIDAD DE NAVARRA
Spain
EU contribution
€ 709 162,26
Address
Campus Universitario Edificio Central
31080 Pamplona
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Cristina Patsouris
IGEA SPA
Italy
EU contribution
€ 526 291,30
Address
Via Parmenide 10A
41012 Carpi Mo
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Giuditta Scuotri (Dr.)
UD-GENOMED MEDICAL GENOMIC TECHNOLOGIES KUTATAS-FEJLESZTESI ES SZOLGALTATO KFT

Participation ended

Hungary
EU contribution
€ 347 379,67
Address
Nagyerdei Krt 98
4032 Debrecen
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Gábor Zahuczky (Dr.)
CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE CNRS
France
EU contribution
€ 445 411,71
Address
Rue Michel Ange 3
75794 Paris
Activity type
Other
Administrative Contact
Marine Moguen-Toursel (Ms.)
3P BIOPHARMACEUTICALS SL
Spain
EU contribution
€ 297 114,62
Address
Calle Mocholi 2 Poligono Mocholi
31110 Noain Navarra
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Pablo Aranda (Dr.)
GENOSAFE SAS
France
EU contribution
€ 291 901
Address
Rue De L'internationale 1
91000 Evry
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Séverine Pouillot (Dr.)
STADT WIEN
Austria
EU contribution
€ 78 786,12
Address
Rathaus
1010 Wien
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Ulrike Stolba (Dr.)
Novartis

Participation ended

Germany
Address
Roonstrasse 25
90429 Nurnberg
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Stefan Scheidl (Dr.)
UNIVERSITAETSKLINIKUM AACHEN
Germany
EU contribution
€ 453 846,56
Address
Pauwelsstrasse 30
52074 Aachen
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Volker Legewie (Mr.)
HOTSWAP STOCKHOLM AB

Participation ended

Sweden
EU contribution
€ 22 607,44
Address
Landsvagen 39
172 63 Sundbyberg
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Oliver Trepte (Dr.)
AMSTERDAM BIOTHERAPEUTICS UNIT
Netherlands
EU contribution
€ 233 203,17
Address
Louwesweg 6
1066EC Amsterdam
Activity type
Research Organisations
Administrative Contact
Bastiaan Nuijen (Dr.)