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ENDOSCAPE, a clinically applicable non-viral gene delivery technology

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.
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Coordinator

CHARITE - UNIVERSITAETSMEDIZIN BERLIN

Address

Chariteplatz 1
10117 Berlin

Germany

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 1 381 303,75

Participants (11)

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SAPREME TECHNOLOGIES BV

Netherlands

EU Contribution

€ 799 160

MAX-PLANCK-GESELLSCHAFT ZUR FORDERUNG DER WISSENSCHAFTEN EV

Germany

EU Contribution

€ 671 878,75

VIB

Belgium

EU Contribution

€ 584 087,50

FREIE UNIVERSITAET BERLIN

Germany

EU Contribution

€ 429 000

UNIVERSIDAD DE SANTIAGO DE COMPOSTELA

Spain

EU Contribution

€ 444 237,50

UNIVERSITA DEGLI STUDI DI ROMA TOR VERGATA

Italy

EU Contribution

€ 407 500

EXTRASYNTHESE SAS

France

EU Contribution

€ 472 500

FRAUNHOFER GESELLSCHAFT ZUR FOERDERUNG DER ANGEWANDTEN FORSCHUNG E.V.

Germany

EU Contribution

€ 540 476,25

UNIVERSITA DEGLI STUDI DI FERRARA

Italy

EU Contribution

€ 488 750

UNIVERSITAT BASEL

Switzerland

EU Contribution

€ 261 750

TP21 GMBH

Germany

EU Contribution

€ 370 000

Project information

Grant agreement ID: 825730

Status

Ongoing project

  • Start date

    1 January 2019

  • End date

    31 December 2022

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 6 850 643,75

  • EU contribution

    € 6 850 643,75

Coordinated by:

CHARITE - UNIVERSITAETSMEDIZIN BERLIN

Germany