Melbourne researchers are advancing an RNA-based treatment that could reshape care for children living with rare and life-threatening blood disorders, with support from the Victorian Government.
Minister for Economic Growth and Jobs Danny Pearson visited St Vincent’s Institute of Medical Research to meet Associate Professor Andrew Deans, whose team is developing an RNA Prime Editing therapy aimed at correcting the genetic faults behind conditions such as Fanconi anaemia. The approach focuses on repairing the underlying mutation rather than relying on traditional bone marrow transplants, which carry heavy risks and can place enormous strain on families.
Fanconi anaemia is a rare disorder that causes bone marrow failure from early childhood. Around 190 children in Australia live with the syndrome, and many face a challenging prognosis due to complications linked to the disease. The prospect of a treatment that corrects the genetic issue directly is drawing interest across the medical research community.
The therapy is now progressing through safety studies, developed in partnership with Maddie Riewoldt’s Vision. The organisation was founded in honour of Maddie Riewoldt, who passed away from a bone marrow failure syndrome in 2015, and has since become a major supporter of research in the area. If the early work continues to show promise, the team plans to move towards a first-in-human clinical trial.
The Victorian Government provided funding of 100,000 dollars last year to support development of this RNA gene editing approach. More broadly, the state has committed more than 1 billion dollars to health and medical research, aiming to support local innovation, strengthen the workforce and help turn emerging science into real-world treatments. Since 2021, more than 30 million dollars has gone toward 63 research projects, contributing to new vaccines, therapies and the growth of a local RNA sector.
Minister Pearson said the momentum behind the project reflects the strength of Victoria’s research community and the potential impact of the treatment. He noted that advances like this bring genuine hope to children and families dealing with rare diseases.
Member for Northern Metropolitan Sheena Watt said the work highlights the value of supporting scientific leadership within local communities, particularly when it can improve outcomes for young patients.
Professor Thomas Kay, Director of St Vincent’s Institute, said investment in medical research strengthens the capacity to make meaningful use of emerging RNA technologies, enabling the development of new therapies for conditions previously considered extremely difficult to treat.
Maddie Riewoldt’s Vision Chief Executive Officer Amy Coote welcomed the progress, praising Associate Professor Deans and his team for their continued engagement with affected families. She said the partnership with St Vincent’s Institute, supported by government investment, reflects a shared commitment to advancing treatments for bone marrow failure syndromes.
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