New Horizon 2020 Project B-SMART Develops RNA-based Therapy for Neurodegenerative Diseases

1 February 2017
  • ​New European research project B-SMART targets RNA-based nanomedicines against Alzheimer’s and spinal and bulbar muscular atrophy (SBMA)
  • Innovative nanotechnological RNA delivery systems to provide first causal therapy approach
  • B-SMART unites internationally renowned experts from 9 academic institutions and industrial partners coordinated by the University Medical Center Utrecht
  • The European Commission invests €6 million over the next five years

Neurodegenerative diseases such as Alzheimer’s affect over 7 million people in Europe – a figure which is expected to double every 20 years as the population ages and thus poses a significant healthcare burden to European society. Despite this fact, few treatments for this group of diseases are available so far. Existing drugs, however, only suppress symptoms or delay further deterioration but do not address the cause of the disease. For Alzheimer’s disease, no drug has been approved in the past five years.

Nanomedical approaches can make a difference, providing new therapeutic options by helping drugs to enter the brain. Therefore, the multinational research project B-SMART sets out to provide an RNA-based therapy perspective for neurodegenerative diseases targeting the direct cause of the disease instead of its symptoms.
The project is funded with a total budget of €6 million over the next five years by the European Commission’s current Research Framework Programme Horizon 2020.

“Transferring RNA therapeutics to the brain crossing the blood-cerebrospinal fluid barrier is a unique research endeavor,” said Prof. Raymond Schiffelers from the University Medical Center Utrecht and coordinator of B-SMART. “Finding a novel gateway to transport RNA to the brain will enable the development of causal therapies not only for Alzheimer’s, but for other diseases as well.”

The consortium will work on specific nanocarriers protecting the RNA against enzymatic degradation while delivering it to the human brain. Crossing the blood-cerebrospinal fluid barrier requires specific targeting ligands, which will be based on heavy chain-only nanobodies that are smaller and more stable than conventional antibodies. The modular delivery system will be tested for safety and efficacy using in vitro and in vivo assays. To improve the nanomedicine manufacturability, B-SMART chose an operator-independent and scalable production method based on microfluidics.

Prof. Roos Vandenbroucke (VIB-UGent): “Our input in the project is the innovative idea to develop Nanobodies that specifically target the blood-cerebrospinal fluid (CSF) barrier, one of the barriers that separates the periphery from the brain. Our aim is to identify Nanobodies that bind the choroid plexus epithelial cells and are subsequently delivered into the CSF via transcytosis. Promising Nanobodies will be attached to different nucleic acid delivery systems developed by the other partners and safety and efficacy of the resulting nanopartikels will be tested in vitro and in vivo. After determining which nanoparticle(s) has therapeutic potential, we will team up with the research group of Mathew Wood (Oxford, UK) and Bart De Strooper (VIB - KU Leuven, Belgium) and study their therapeutic value in pre-clinical models of spinal and bulbar muscular atrophy and Alzheimer's disease.”

B-SMART assembles a multi-national team of leading experts from 9 partnering institutions with long-standing experience in their respective fields. The B-SMART partners are involved in numerous exceptional national and international research programmes targeting close communication with the industry throughout all project stages in order to bridge the research-industry gap and enable timely exploitation of the project results.

B-SMART officially kicks off its activities with a first project meeting held in Utrecht on 1 and 2 February 2017.

B-SMART Partners
- University Medical Center Utrecht
- 20 Med Therapeutics B.V.

- University of Santiago de Compostela


United Kingdom
- The Chancellor, Masters and Scholars of the University of Oxford
- Malvern Instruments Ltd

- Istituto Biochimico Italiano Giovanni Lorenzini SpA

- Stiftelsen SINTEF

- EURICE – European Research and Project Office GmbH

Project Details
Name: B-SMART Brain-specific, Modular and Active RNA Therapeutics
Start date: 2017-01-01
Duration: 60 months
Budget: €6 million
Coordination: Universitair Medisch Centrum Utrecht

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