Victorian researchers unlock RNA treatment for rare childhood diseases

We’re proud to have supported Victorian researchers to discover a new RNA-based treatment for young people battling rare diseases.

Associate Professor Andrew Deans from the St Vincent’s Institute of Medical Research developed the new therapy, using Prime Editing technology. This technology is currently progressing to clinical trials. His project was funded by the mRNA Victoria Research Acceleration Fund in 2023.

This cutting-edge treatment corrects the genetic defect that causes Fanconi anaemia and other life-threatening blood disorders. It offers a safer, more effective alternative to risky bone marrow transplants.

Fanconi anaemia is a rare, inherited disorder that causes bone marrow failure syndrome. It mainly affects young people aged 17 to 40. About 190 young Australians are diagnosed with the syndrome every year. Sadly, more than half will die from the disease.

Amelia Hawkshaw, diagnosed with Fanconi anaemia at 23 will benefit from this breakthrough. Amelia works as a youth worker, bringing patient voices and advocacy to Fanconi Anemia Support Australasia.

Through its grant programs, mRNA Victoria has provided $30.6 million to support 63 research projects since 2021 – unlocking medicines and vaccines and establishing a world-leading RNA industry in Victoria.