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

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.

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)

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RHEINISCH-WESTFAELISCHE TECHNISCHE HOCHSCHULE AACHEN

Germany

EU Contribution

€ 279 980,32

MAX DELBRUECK CENTRUM FUER MOLEKULARE MEDIZIN IN DER HELMHOLTZ-GEMEINSCHAFT (MDC)

Germany

EU Contribution

€ 282 004,18

BUNDESINSTITUT FUR IMPFSTOFFE UND BIOMEDIZINISCHE ARZNEIMITTEL

Germany

EU Contribution

€ 301 336

UNIVERSIDAD DE NAVARRA

Spain

EU Contribution

€ 709 162,26

IGEA SPA

Italy

EU Contribution

€ 526 291,30

UD-GENOMED MEDICAL GENOMIC TECHNOLOGIES KUTATAS-FEJLESZTESI ES SZOLGALTATO KFT

Hungary

EU Contribution

€ 347 379,67

CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE CNRS

France

EU Contribution

€ 445 411,71

3P BIOPHARMACEUTICALS SL

Spain

EU Contribution

€ 297 114,62

GENOSAFE SAS

France

EU Contribution

€ 291 901

STADT WIEN

Austria

EU Contribution

€ 78 786,12

Novartis

Germany

UNIVERSITAETSKLINIKUM AACHEN

Germany

EU Contribution

€ 453 846,56

HOTSWAP STOCKHOLM AB

Sweden

EU Contribution

€ 22 607,44

AMSTERDAM BIOTHERAPEUTICS UNIT

Netherlands

EU Contribution

€ 233 203,17

Project information

Grant agreement ID: 305134

Status

Closed project

  • Start date

    1 November 2012

  • End date

    30 April 2018

Funded under:

FP7-HEALTH

  • Overall budget:

    € 7 772 860,37

  • EU contribution

    € 5 976 298

Coordinated by:

UNIVERSITE DE GENEVE

Switzerland