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ENDOSCAPE, a clinically applicable non-viral gene delivery technology

Objective

Gene therapy is one of the most promising treatment options for future advanced therapies in a broad range of diseases. Successful gene delivery requires the recognition of target cells as well as cytosolic and nucleosolic uptake of the gene. Currently, non-viral based gene delivery such as transfection reagents are only suitable for in vitro applications, and clinical gene therapeutics delivery is accomplished via viral vectors, which still has major safety concerns as well as complex and costly manufacturing procedures, preventing future implementation for the treatment of diseases with large patients groups.

In the last 15 years, a class of secondary plant metabolites has been discovered that selectively mediates endosomal escape and cytoplasmic delivery of macromolecules specifically at low endosomal pH, thereby inducing a 40-fold enhanced gene delivery efficacy in vivo. However, endosomal escape enhancers and gene therapeutic product must be applied as two components, thus complicating therapeutic approval and clinical applicability.

The ENDOSCAPE technology platform will develop and collect proof of concept for a non-viral gene delivery technology where endosomal escape enhancers, gene therapeutic product and targeting ligand are all bound to one molecular scaffold. Proof of concept of the ENDOSCAPE technology has a major impact on the therapeutic opportunities for current and future macromolecule drugs for a broad range of diseases and large patient groups.

All this induces new biotech-based businesses, new research projects and creates new technology platforms for development of new macromolecule therapeutics for a broad range of disease indications. The non-viral based ENDOSCAPE technology will enhance therapeutic efficacy with lower therapeutic dose thereby reducing costs of healthcare, improving the health of patients worldwide, and strengthening the competitive landscape of the EU in the worldwide quest for such an advanced technology.

Call for proposal

H2020-SC1-BHC-2018-2020

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Sub call

H2020-SC1-2018-Single-Stage-RTD

Coordinator

CHARITE - UNIVERSITAETSMEDIZIN BERLIN
Net EU contribution
€ 1 381 303,75
Address
Chariteplatz 1
10117 Berlin
Germany

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Region
Berlin Berlin Berlin
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00

Participants (11)

SAPREME TECHNOLOGIES BV
Netherlands
Net EU contribution
€ 799 160,00
Address
Professor Bronkhorstlaan 10 G 92
3723 MB Bilthoven

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
West-Nederland Utrecht Utrecht
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
MAX-PLANCK-GESELLSCHAFT ZUR FORDERUNG DER WISSENSCHAFTEN EV
Germany
Net EU contribution
€ 671 878,75
Address
Hofgartenstrasse 8
80539 Munchen

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Region
Bayern Oberbayern München, Kreisfreie Stadt
Activity type
Research Organisations
Non-EU contribution
€ 0,00
VIB VZW
Belgium
Net EU contribution
€ 584 087,50
Address
Rijvisschestraat 120
9052 Zwijnaarde - Gent

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Region
Vlaams Gewest Prov. Oost-Vlaanderen Arr. Gent
Activity type
Research Organisations
Non-EU contribution
€ 0,00
FREIE UNIVERSITAET BERLIN
Germany
Net EU contribution
€ 429 000,00
Address
Kaiserswerther Strasse 16-18
14195 Berlin

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Region
Berlin Berlin Berlin
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
UNIVERSIDAD DE SANTIAGO DE COMPOSTELA
Spain
Net EU contribution
€ 444 237,50
Address
Colexio De San Xerome Praza Do Obradoiro S/n
15782 Santiago De Compostela

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Region
Galicia A Coruña
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
UNIVERSITA DEGLI STUDI DI ROMA TOR VERGATA
Italy
Net EU contribution
€ 407 500,00
Address
Via Cracovia 50
00133 Roma

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Region
Centro (IT) Lazio Roma
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
EXTRASYNTHESE SAS
France
Net EU contribution
€ 472 500,00
Address
Impasse Jacquard Zi Lyon Nord
69730 Genay

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Auvergne-Rhône-Alpes Rhône-Alpes Rhône
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
FRAUNHOFER GESELLSCHAFT ZUR FORDERUNG DER ANGEWANDTEN FORSCHUNG EV
Germany
Net EU contribution
€ 540 476,25
Address
Hansastrasse 27c
80686 Munchen

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Region
Bayern Oberbayern München, Kreisfreie Stadt
Activity type
Research Organisations
Non-EU contribution
€ 0,00
UNIVERSITA DEGLI STUDI DI FERRARA
Italy
Net EU contribution
€ 488 750,00
Address
Via Ariosto 35
44121 Ferrara

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Region
Nord-Est Emilia-Romagna Ferrara
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
UNIVERSITAT BASEL
Switzerland
Net EU contribution
€ 261 750,00
Address
Petersplatz 1
4051 Basel

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Region
Schweiz/Suisse/Svizzera Nordwestschweiz Basel-Stadt
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
TP21 GMBH
Germany
Net EU contribution
€ 370 000,00
Address
Luisenstrasse 14
10117 Berlin

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Berlin Berlin Berlin
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00