B-SMARTProject ID: 721058
Brain-Specific, Modular and Active RNA Therapeutics
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.
EU contribution: EUR 861 080
3584 CX UTRECHT
EU contribution: EUR 551 375
COLEXIO DE SAN XEROME PRAZA DO OBRADOIRO S/N
15782 SANTIAGO DE COMPOSTELA
EU contribution: EUR 398 125
JAN KROEZESTRAAT 8
7552 GS HENGELO
EU contribution: EUR 862 812,50
9052 ZWIJNAARDE - GENT
EU contribution: EUR 889 151,25
University Offices, Wellington Square
OX1 2JD OXFORD
EU contribution: EUR 691 250
VIA FOSSIGNANO 2
EU contribution: EUR 669 510
EU contribution: EUR 571 250
WR14 1XZ MALVERN
EU contribution: EUR 503 750