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Smart Nanogel for Intranasal Brain Delivery of RNA

Macquarie University in partnership with the Woolcock Institute of Medical Research

  • RNA Future Leaders Program
Date Funded:
  • 3 August, 2021
Chief Investigator/s:
  • Professor Daniela Traini

Project Summary

Development of a temperature-responsive nanogel nasal formulation to deliver RNA to the brain for the treatment of Alzheimer’s Disease.

The main researcher for this project is Professor Daniela Traini.

What is the issue for NSW?

Alzheimer’s disease (AD) is the most common age-related neurodegenerative disorder and is estimated to cost the global healthcare system $1 trillion annually, which will continue to increase every year due to the persistent lack of advancement in current treatments and an aging population. Intranasal brain delivery is a promising non-invasive treatment alternative for many diseases of the central nervous system, allowing therapeutics to reach a target effectively with minimal side effects.  However, there is still a very poor understanding of how drugs may be formulated to specifically target the appropriate region in the nasal cavity and also about how drugs may be effectively transported through the nasal tissue to reach the brain. As such, there is an unmet clinical for smart vehicle systems to enable effective nasal delivery of targeted therapeutics. Additionally, advancement of knowledge in this field is mostly hindered by the lack of multidisciplinary approaches in NSW and globally to design novel delivery vehicles and investigational tools.

What does the research aim to do and how?

Together with a multidisciplinary research team and Tetratherix, an Australian medtech company, the current PhD program aims to address this complex problem by:

  • Developing a nasal Alzheimer’s disease treatment using silencing RNA incorporated into a smart nanogel to facilitate brain penetration that is more effective than currently available treatments.
  • Developing nose-to-brain models for preclinical testing of efficacy and toxicity screening of novel intranasal RNA treatment for AD.
  • Understanding the mechanism of interactions between the aerosol RNA nanogel formulation and biological barriers present in the upper nose.