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Brain-Specific, Modular and Active RNA Therapeutics

Objective

The overall objective of B-SMART is: 1. to design modular nanoparticles, 2. to manufacture them via a quality-by-design protocol, 3. to achieve delivery of therapeutic RNAs to the brain and treat neurodegenerative diseases.

I. To design modular nanoparticles consisting of
o an active RNA payload
o established (lipid-based), emerging (trigger-responsive polymer-based) or exploratory (extracellular vesicle-based) nanoparticles
o a targeting ligand consisting of the variable domain of heavy chain only antibodies (also known as VHHs or nanobodies), which are coupled to the carrier platform

II. To manufacture the modular nanoparticles using a microfluidic assembly system that will ensure quality-by-design: uniform nanoparticles across research sites and excellent control over the physico-chemical parameters.

III. To test pre-clinical activity of formulations with promising in vitro activity with good cell/blood compatibility and to select the best RNA-formulation for clinical translation to treat neurodegenerative diseases.
Pre-clinical efficacy is tested after
o local injection
o nasal administration
o systemic administration
The neurodegenerative diseases carry a high burden for patients since they are without exception progressive. But they also carry a substantial socio-economic burden with estimated costs of 130 billion euro. per year (2008).

IV. The technical work in B-SMART will be supported by project management. It ensures that the project is coordinated in a clear, unambiguous and mutually acceptable manner and that the project achieves its objectives, within the given financial and time constraints.

in B-SMART we expect to arrive at a scale-able nanoparticle formulation with uniform characteristics that shows strong pre-clinical evidence of therapeutic efficacy and is ready for clinical translation.

Coordinator

UNIVERSITAIR MEDISCH CENTRUM UTRECHT

Address

Heidelberglaan 100
3584 Cx Utrecht

Netherlands

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 861 080

Participants (9)

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UNIVERSIDAD DE SANTIAGO DE COMPOSTELA

Spain

EU Contribution

€ 551 375

20MED THERAPEUTICS BV

Netherlands

EU Contribution

€ 398 125

VIB

Belgium

EU Contribution

€ 862 812,50

THE CHANCELLOR, MASTERS AND SCHOLARS OF THE UNIVERSITY OF OXFORD

United Kingdom

EU Contribution

€ 889 151,25

ISTITUTO BIOCHIMICO ITALIANO GIOVANNI LORENZINI SPA

Italy

EU Contribution

€ 691 250

STIFTELSEN SINTEF

Norway

EU Contribution

€ 88 736,25

MALVERN INSTRUMENTS LTD

United Kingdom

EU Contribution

€ 571 250

EURICE EUROPEAN RESEARCH AND PROJECT OFFICE GMBH

Germany

EU Contribution

€ 503 750

SINTEF AS

Norway

EU Contribution

€ 580 773,75

Project information

Grant agreement ID: 721058

Status

Ongoing project

  • Start date

    1 January 2017

  • End date

    31 December 2021

Funded under:

H2020-EU.2.1.2.

  • Overall budget:

    € 5 998 303,75

  • EU contribution

    € 5 998 303,75

Coordinated by:

UNIVERSITAIR MEDISCH CENTRUM UTRECHT

Netherlands