New horizon 2020 B-SMART project begins developing therapies for degenerative pathologies of the Nervous System that relies on RNA

06 February 2018

  • The new European B-SMART research project uses nanopharmaceuticals containing RNA for the treatment of Alzheimer's and spinal and bulbar muscular atrophy (SBMA)
  • Innovative RNA transport systems based on nanotechnology aimed at removing the primary etiology of the pathology
  • B-SMART brings together renowned international experts, from 9 academic institutions and industrial partners, coordinated by the Medical Center of the University of Utrecht
  • The European Commission invests € 6 million over the next 5 years

Aprilia, 6 February 2017 - Neurodegenerative diseases such as Alzheimer's affect more than 7 million people in Europe - a figure that will duplicate every 20 years as the population ages and put a strain on European health care systems. In spite of this, few treatments are available for this group of diseases. The drugs available today only suppress the symptoms or delay the progression of the disease but do not remove the causes. Furthermore, no drug has been approved for Alzheimer's in the last 5 years.

Nanomedicine can make a difference by making new therapeutic options available that allow drugs to reach the site of action in the brain. This is why the B-SMART multinational research project aims to develop a therapy based on RNA with the aim of directly affecting the cause of the disease instead of its symptoms.

The project is funded for the next 5 years by the European Commission under the Horizon 2020 framework program with a total budget of € 6 million.

"Transferring therapeutic RNA to the brain by crossing the blood-brain fluid barrier is an original research approach," said Prof. Raymond Schiffelers at the University of Utrecht Medical Center and coordinator of B-SMART. "Identifying a new window for the transport of RNA to the brain will allow the development of therapies aimed at the etiology not only for Alzheimer's, but also for other diseases".

The consortium will work to develop specific nanocarriers to protect RNA from enzymatic degradation and to transport it to the human brain. Crossing the blood-brain barrier requires specific ligands for transport, which will be made up of "nanocorps", i.e. antibodies made up of only the heavy and therefore smaller and more stable chains of conventional antibodies. The modular transport system will be tested for safety and efficacy both in vitro and in vivo. To improve the production of nanomedicine, B-SMART has chosen a method based on the use of a microfluidic system, independent of the operator and easily scalable.

Within this ambitious project, the Process Development Group of the Italian Biochemical Institute G. Lorenzini, with consolidated experience in aseptic dosage and in the study of the production processes of injectable organic medicinal products, is strongly motivated to provide innovative solutions for the industrial scaling-up of the production process, in order to meet cGMP quality standards and regulatory authorities' requests.

B-SMART brings together an international team of renowned experts from 9 institutions with consolidated experience in their respective fields of specialization. B-SMART partners are involved in numerous national and international research programs in close cooperation with industry at all stages of the project to bridge the gap between research and industry and enable the timely exploitation of search results.

B-SMART officially started the activity with the first meeting held in Utrecht on February 1st and 2nd 2017.


University Medical Center Utrecht 20 Med Therapeutics BV

University of Santiago de Compostela


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

Italian Biochemical Institute Giovanni Lorenzini SpA

Stiftelsen SINTEF

EURICE - European Research and Project Office GmbH


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 - Phone: +31 88 7556512            


Coordinator: University Medical Center Utrecht - Prof. Raymond Schiffelers - Email: - Phone: +31 88 7556512
Project Management: EURICE - European Research and Project Office GmbH - Corinna Hahn - Email: - Phone: +49 681 95 923362

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