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Viral vectors for gene therapy applications targeting the central nervous system.

Children's Medical Research Institute

  • Early-Mid Career Fellowship
Date Funded:
  • 17 May, 2021
Chief Investigator/s:
  • Dr. Matthieu Drouyer

Research intent

Development of next-generation liver-detargeted AAV vectors for gene therapy applications in the central-nervous system.

What is the issue?

The current generation of gene therapy AAV vectors is not efficient at therapeutic cargo delivery to the intended target tissue, with high percentage of vectors entering unintended off-target tissues. This drives the need for high vector dose, which increases the cost of therapies and increasing the risk of toxic side effects.

Despite access to globally recognised expertise in AAV vectorology, bioengineering and manufacturing (A/Prof. Lisowski and Prof. Alexander), NSW is not leading the development of new therapies but rather accepts/adopts clinical tools developed off-shore (Europe and the U.S.).

The goal of this project is to develop the next generation superior AAV vectors for CNS directed gene therapy and position NSW as the globally recognised leader in the AAV research, development and translation. This will facilitate growth of local start-up companies, lead to job and wealth creation, while having a profound positive effect on the patient population.

What does the research aim to do and how?

This project aims to develop novel viral vector-based gene delivery vehicles (AAV) that are specifically bioengineered for CNS applications with improved efficiency and safety.

We will take advantage of proprietary cutting-edge technologies, access to most clinically-predictive preclinical models (xenograft mice and NHP) and the unique complementary skillsets of the assembled team to develop bespoke safer and highly functional AAV vectors.

In addition to direct translational potential the tools developed will also be powerful resources in support of fundamental research projects, which will support development of additional CNS-targeted gene therapies, thus increasing the overall impact on healthcare and society.