Melbourne researchers advance potential new RNA treatment for rare childhood Bone Marrow Failure Syndromes

15 December 2025

Maddie Riewoldt’s Vision is incredibly proud to have supported cutting-edge research at St Vincent’s Institute of Medical Research (SVI) that is advancing a new RNA-based approach to treat rare childhood diseases — including Bone Marrow Failure Syndromes.

Backed by the Maddie Riewoldt’s Vision and the Victorian Government through mRNA Victoria, the project has exciting potential for children and families living with Fanconi anaemia, a rare inherited condition that causes bone marrow failure from a very young age.

Victoria’s Minister for Economic Growth and Jobs, Danny Pearson, recently visited SVI to meet the research team behind the discovery, led by Associate Professor Andrew Deans, and to see firsthand the promise of this innovative therapy for patients and families.

A/Prof Deans’ team has shown that cutting-edge Prime Editing technology can correct the genetic defect that causes life-threatening blood disorders such as Fanconi anaemia — with the potential to reduce reliance on risky and expensive bone marrow transplants.

Fanconi anaemia is a rare, genetic disorder that causes bone marrow failure and affects children from a very young age. Around 190 young Australians are currently diagnosed with the syndrome, and tragically more than half will die from the disease and its related conditions.

As the work progresses toward a world-first clinical trial, SVI and Maddie Riewoldt’s Vision will work closely with patients and families to help build understanding of the therapy and ensure it is designed with their needs at the centre.

Through mRNA Victoria and Maddie Riewoldt’s Vision’s investment in accelerating this critical work, the research represents a meaningful step forward and is creating hope of a brighter future for Bone Marrow Failure Syndrome patients and their families.

St Vincent’s Institute of Medical Research Director, Professor Tom Kay says:

“Investment in medical research gives SVI scientists the best chance of maximising new mRNA technology to develop leading-edge therapy to combat rare childhood diseases like Fanconi anaemia.”

Maddie Riewoldt’s Vision CEO, Amy Coote says:

“Maddie Riewoldt’s Vision is thrilled to partner with SVI on this groundbreaking project. We’re incredibly grateful for A/Prof Andrew Deans and his team’s commitment to patient and family engagement, and thankful for the Victorian Government’s investment in advancing gene editing technology that offers real hope for those living with Bone Marrow Failure Syndromes.”