December 8th, 2025Groundbreaking RNA treatment targeting rare childhood diseases

The state government is supporting Melbourne researchers who have discovered a groundbreaking RNA treatment targeting rare childhood diseases, including bone marrow failure syndrome.

Minister for Economic Growth and Jobs Danny Pearson has visited St Vincent’s Institute of Medical Research to meet Associate Professor Andrew Deans, the local researcher behind an innovative RNA therapy that could transform the lives of children with rare blood disorders.

In a major breakthrough, the cutting-edge RNA Prime Editing technology corrects the genetic defect that causes life-threatening blood disorders such as Fanconi anemia, potentially eliminating the need for risky and costly bone marrow transplants.

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

The new treatment is progressing through safety studies, in partnership with Maddie Riewoldt’s Vision – an organisation dedicated to finding a cure for bone marrow failure syndrome, inspired by Maddie’s tragic death in 2015 – with the aim of launching a world-first clinical trial for patients.

Minister for Economic Growth and Jobs Danny Pearson said it was exciting to see this research rapidly advancing towards clinical trials, offering real hope for children and families battling these devastating diseases.

“Our medical research and health technology sector is the largest sector in Australia – supporting more than 30,000 jobs and enabling Victorian scientists to change lives around the world.”