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Cellular production of targeted RNA therapies

The University of Newcastle

  • RNA Future Leaders Program
Date Funded:
  • 3 August, 2021
Chief Investigator/s:
  • Dr. Gerard Kaiko

Project Summary

A cellular system for the manufacture of exosome-based targeted RNA therapies to treat cystic fibrosis.

The main researcher for this project is Dr Gerard Kaiko.

What is the issue for NSW?

  1. Clinical and healthcare issue: Development of targeted RNA therapies and a delivery system using cellular production. Specifically, this will be used to treat cystic fibrosis patients with an unmet clinical need in NSW and improve life expectancy, while also reducing frequent in-patient hospitalisation and large associated costs of healthcare.
  2. Commercialisation issue: Boosting the development and delivery of targeted RNA therapies in NSW and contribute to the ecosystem through the NSW RNA Production and Research Network.

What does the research aim to do and how?

Improve the treatment of cystic fibrosis through development and late-stage preclinical testing of a targeted RNA therapy. Develop a scalable and commercialisable platform to produce and deliver RNA therapies for cystic fibrosis (also applicable to other diseases), through the use of cellular engineering.