A novel gateway to the brain

Nanomedicine can make the difference: While currently available treatments to diseases such as Alzheimer’s or Spinal and bulbar muscular atrophy (SBMA) only supress the symptoms or delay further deterioration, the B-SMART approach targets the actual cause of the disease.

A novel gateway to the human brain will enable RNA-based nanomedicines to reach the brain cells affected by the disease. Once this has been achieved, the RNA delivery system can be used to treat other incurable diseases such as Parkinson’s by simply adapting the RNA sequence.

The modular delivery system

The RNA delivery system is based on three essential components which can only be successful if combined.

RNA therapeutics
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The "payload" targeting the direct cause of the disease

(Established, emerging or exploratory) nanocarriers
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The "carrier" protecting the RNA payload against enzymatic degradation

Nanobody-based targeting ligands
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The "key" enabling the crossing of the blood-cerebrospinal fluid barrier

Microfluidic Technology

The B-SMART consortium selected the Precision NanoSystems NanoAssemblr™ platform for the controlled manufacture of nanomedicines. Using this easy to operate microfluidic assembly system will ensure quality-by-design: uniform nanocarriers across research sites, excellent control over the physico-chemical parameters, linear scalability, and compatibility with GMP production.

The NanoAssemblr platform simplifies the formulation of nanoparticles to accelerate nanoparticle research from drug target discovery to therapeutic nanoparticle manufacturing for the clinic. Having a NanoAssemblr Benchtop instrument in each of the eight laboratories involved in the B-SMART project will allow us to manufacture the same particles at each site in a reliable and reproducible manner, helping to accelerate our research and transferring results from a scientific to a clinical setting.

Find out more about the NanoAssemblr technology in the video by Precision Nanosystems.

Quality-by-design

The project consortium aims to improve nanomedicine manufacturability by choosing an operator-independent and scalable production method based on microfluidics. The modular nanocarriers will be validated for safety and efficacy using in vitro and in vivo assays.

Collaboration and open access

The multinational B-SMART consortium strives to cooperate with related scientific projects and international networks and guarantees open access to scientific publications and research outcomes at the earliest possible stage. Moreover, the project will provide training opportunities and career perspectives for young European scientists.

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