TIA-supported facility helps drive global effort to develop new therapies for mitochondrial disease

7 August 2026

The TIA-supported National Biologics Facility will play a key role in the only non-European node of a major international collaboration developing new therapies for mitochondrial disease.

New genetic medicines have the potential to transform the lives of people living with devastating mitochondrial diseases, rare genetic disorders that currently have few treatment options. Researchers at The University of Queensland will play a key role in making that future possible as part of a major international collaboration developing next-generation mitochondrial genome therapeutics.

Associate Professor Seth Cheetham, Director of the National Biologics Facility (NBF), will lead the only non-European node of the new £50 million Medical Research Council Centre of Research Excellence for Mitochondrial Genome Therapeutics. NBF is supported by Therapeutic Innovation Australia (TIA) through the Australian Government’s National Collaborative Research Infrastructure Strategy (NCRIS), providing the advanced biomanufacturing capability and expertise that help Australian researchers translate promising discoveries towards new therapies.

The UQ team, based at the Australian Institute for Bioengineering and Nanotechnology (AIBN), will work alongside leading researchers in the UK, Germany and France to develop technologies that deliver genetic medicines and accelerate the development of treatments for mitochondrial disease. The consortium aims to advance credible therapeutic candidates into clinical trials within seven years, bringing new hope to people living with these devastating genetic disorders.

The collaboration highlights how Australia’s national research infrastructure enables local researchers to contribute to world-leading international initiatives. Through TIA-supported facilities such as NBF, Australian scientists have access to the specialist capabilities needed to help translate innovative research into future therapies that improve human health.

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"Drug development traditionally takes decades, so this represents a completely new paradigm – making precise corrections to DNA in a living patient."

Researcher Hannah Tompkins and Associate Professor Seth Cheetham (Photo credit: The University of Queensland )