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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.

Field of science

  • /natural sciences/biological sciences/genetics and heredity/rna

Call for proposal

H2020-NMBP-2016-two-stage
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

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)

UNIVERSIDAD DE SANTIAGO DE COMPOSTELA
Spain
EU contribution
€ 551 375
Address
Colexio De San Xerome Praza Do Obradoiro S/n
15782 Santiago De Compostela
Activity type
Higher or Secondary Education Establishments
20MED THERAPEUTICS BV
Netherlands
EU contribution
€ 398 125
Address
Drienerlolaan 5 Kamer Zh252
7522 NB Enschede
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
VIB VZW
Belgium
EU contribution
€ 862 812,50
Address
Rijvisschestraat 120
9052 Zwijnaarde - Gent
Activity type
Research Organisations
THE CHANCELLOR, MASTERS AND SCHOLARS OF THE UNIVERSITY OF OXFORD
United Kingdom
EU contribution
€ 889 151,25
Address
Wellington Square University Offices
OX1 2JD Oxford
Activity type
Higher or Secondary Education Establishments
ISTITUTO BIOCHIMICO ITALIANO GIOVANNI LORENZINI SPA
Italy
EU contribution
€ 691 250
Address
Via Fossignano 2
04011 Aprilia
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
STIFTELSEN SINTEF

Participation ended

Norway
EU contribution
€ 88 736,25
Address
Strindveien 4
7034 Trondheim
Activity type
Research Organisations
MALVERN PANALYTICAL LIMITED
United Kingdom
EU contribution
€ 571 250
Address
Grovewood Road Enigma Business Park
WR14 1XZ Malvern
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
EURICE EUROPEAN RESEARCH AND PROJECT OFFICE GMBH
Germany
EU contribution
€ 503 750
Address
Heinrich-hertz-allee 1
66386 St Ingbert
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
SINTEF AS
Norway
EU contribution
€ 580 773,75
Address
Strindvegen 4
7034 Trondheim
Activity type
Research Organisations