New European research project B-SMART targets RNA-based nanomedicines against Alzheimers and spinal and bulbar muscular atrophy (SBMA). The project aims to develop 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 will invest 6 million over the next 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 Commissions 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 Alzheimers, 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 brain-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.

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 Belgium VIB

Germany EURICE European Research and Project Office GmbH

Italy Istituto Biochimico Italiano Giovanni Lorenzini SpA

Netherlands University Medical Center Utrecht (Coordinator) 20 Med Therapeutics B.V.

Norway Stiftelsen SINTEF

Spain University of Santiago de Compostela

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

Project Details Name: B-SMART Brain-specific, Modular and Active RNA Therapeutics Start date: 2017-01-01 Duration: 60 months Budget: 6 million Coordination: University Medical Center Utrecht

Contact Coordinator University Medical Center Utrecht Prof. Raymond Schiffelers Email: R.Schiffelers@umcutrecht.nl Phone: +31 88 7556512

Project Management EURICE European Research and Project Office GmbH Corinna Hahn Email: c.hahn@eurice.eu Phone: +49 681 95 923362

This project has received funding from the European Unions Horizon 2020 research and innovation programme under grant agreement No 721058.

Original post:
New Horizon 2020 Project B-SMART Develops RNA-based Therapy for Neurodegenerative Diseases - Cordis News