Spotlight on Australian Research: mRNA Hope for NPC at The Florey
- ANPDF
- Oct 21
- 3 min read
Australia is emerging as a hub of innovation in rare disease research, and one project to watch is the mRNA gene therapy program led by Dr. Ya Hui Hung at The Florey Institute. This initiative aims to harness mRNA technology to address Niemann-Pick Disease Type C (NPC) — a neurodegenerative condition often likened to a form of childhood dementia.
Where the research stands
Building on the success of mRNA vaccines and therapies in other areas, Dr. Hung’s work explores how this platform could deliver functional copies of genes that are impaired in NPC. The ultimate goal is to restore normal cellular function and slow or stop the progression of this devastating childhood dementia.
At the recent INPDA Biannual Meeting (held in Argentina) and the Australian Niemann-Pick Conference (held at The Florey), Dr. Hung shared updates on her team’s progress. The presentation spotlighted how Florey researchers are exploring strategies to deliver functional mRNA across the blood-brain barrier, restoring neurological function in NPC models.
Dr. Hung first proposed the mRNA approach as early as 2018 — before mRNA therapeutics gained prominence with COVID-19 — reflecting her foresight and dedication.
Her work is backed by meaningful support: seed funding came via ANPDF (spurred by Mandy Whitechurch’s advocacy), and further backing includes mRNA Victoria, Australia’s Medical Research Future Fund, and the Percy Baxter Charitable Trust.
What This Means for NPC Families
“We are moving into preclinical study of an mRNA gene therapy, working on how to get it across the blood-brain barrier into the brain to restore neurological function.” — Dr. Ya Hui Hung The Florey
For families living with NPC, this research is more than academic — it’s a beacon of possibility. While still early, every step forward helps build momentum and brings us closer to therapeutic options that could change lives.
Challenges & Next Steps
Delivering across the blood-brain barrier: This is a core technical hurdle in neurologic gene therapies, and Dr. Hung’s team is actively developing strategies to surmount it.
Preclinical validation: Before any human trial, the therapy must demonstrate safety and efficacy in relevant models.
Scaling and regulation: If preclinical work succeeds, the next phases will involve scale-up, regulatory clearance, and ultimately translation into clinical trials.
Beyond the Lab: Neuartica & A-Maze-Ing Brain
Outside of her laboratory work, Ya Hui has joined ANPDF’s General Manager, Deanna Carpino, to create NeuArtica, an initiative that blends neuroscience and art. Their first project, A-Maze-Ing Brain, uses creative expression to spark conversation about complex neurological diseases like Niemann-Pick Diseases.
Learn More & Get Involved
You can find out more about mRNA and Dr Hung’s work here:
About Niemann-Pick Disease, Type C
Niemann-Pick Disease, Type C (NPC) is a rare, progressive, and life-limiting genetic disorder. Often referred to as “childhood dementia,” NPC affects the body’s ability to transport and store cholesterol and other lipids, leading to severe neurological decline. Symptoms can include difficulties with movement, learning, memory, swallowing, and speech. NPC affects children and adults alike, with no approved cure currently available in Australia. Learn more at Information and Support.
About The Florey
The Florey Institute of Neuroscience and Mental Health is a leading Australian centre dedicated to understanding brain disease and developing therapies.
Dr. Ya Hui Hung is a Senior Research Fellow leading the mRNA NPC program, aiming to pioneer a novel therapeutic approach in the Australian rare disease landscape.
About the Australian NPC Disease Foundation (ANPDF)
The ANPDF is the national organisation supporting families impacted by Niemann-Pick disease in Australia. We work to raise awareness, drive research, advocate for access to treatments, and build community support. Learn more at About Us.
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