New European research project B-SMART targets RNA-based nanomedicines against Alzheimer’s and spinal and bulbar muscular atrophy (SBMA).
The project, which is funded through Horizon 2020, aims to develop innovative nanotechnological RNA delivery systems to provide a first causal therapy approach.
B-SMART unites internationally renowned experts from nine academic institutions and industrial partners co-ordinated by the University Medical Center Utrecht, the Netherlands.
The European Commission will invest €6m into the project over the next five years.
Neurodegenerative diseases are defined as hereditary and sporadic conditions characterised by progressive nervous system dysfunction. These disorders are often associated with atrophy of the affected central or peripheral structures of the nervous system and include diseases such as Alzheimer’s and other dementias, epilepsy, Multiple Sclerosis and Huntington’s disease, amongst many others.
Professor Raymond Schiffelers from the University Medical Center Utrecht and co-ordinator of B-SMART said: “Transferring RNA therapeutics to the brain crossing the blood-cerebrospinal fluid barrier is a unique research endeavour. 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 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.
B-SMART officially begins activities with an inaugural project meeting taking place in Utrecht today (2 February 2017).