What is the issue for NSW?
The development of targeted gene therapies and a delivery system using cellular production will, specifically, be used to treat autosomal dominant polycystic kidney disease patients with an unmet clinical need in NSW. The targeted therapies are aimed at improving life expectancy, reducing renal failure and also minimising the frequent hospitalisations and large associated costs of healthcare including dialysis.
The development of targeted gene therapies will boost the development and delivery of targeted therapies in NSW and contribute to the ecosystem of local biotech development.
What does the research aim to do and how?
This project aims to improve the treatment of autosomal dominant polycystic kidney disease through development of one of the first therapies targeting the actual cause of the disease. This research also aims to develop a scalable and commercially viable platform to produce and deliver nanovesicle encapsulated gene therapies (also applicable to other diseases), using cellular engineering.