Scientists from CMRI have partnered with RNA researchers from the Monash Institute of Pharmaceutical Sciences (MIPS) to combine mRNA delivery with viral gene delivery to treat metabolic liver disease in infants and children.

The partnership will be funded by a $99k grant from the mRNA Victoria Research Acceleration Fund along with grants from Monash University and CMRI, and will be led by Professor Colin Pouton from MIPS alongside Dr Samantha Ginn and Professor Ian Alexander from CMRI’s Gene Therapy Research Unit.

The MIPS team is also made up of mRNA researchers, Dr Harry Al-Wassiti, Dr Joan Ho and Dr Stewart Fabb.

For several years, CMRI’s Dr Ginn and Professor Alexander have been working on gene editing using CRISPR-Cas9 (a family of DNA sequences) with the aim to directly repair defective genes in infants and children with ornithine transcarbamylase (OTC) deficiency, a genetic disorder that can lead to progressive liver damage.

“We are thrilled to be working with Professor Colin Pouton, Dr Harry Al-Wassiti and the team from Monash to develop gene therapy treatments for children with genetic liver disease,’’ Dr Ginn said. This is made possible by our combined expertise in gene delivery and genome editing technology and this grant will enable us to continue our efforts in this space.”

Professor Pouton’s team at MIPS has partnered with the CMRI team to make CRISPR-Cas9 gene correction safer by using mRNA technology, with preliminary experiments producing some very exciting data.

Professor Pouton, who is also leading the development of Australia’s first mRNA COVID-19 vaccine, said it’s an exciting time for mRNA technology and its potential to transcend a broad range of medical applications.

“It’s very encouraging to see Australia continue to build its capabilities in mRNA research, development and manufacturing, which all contribute towards building a sustainable end-to-end ecosystem for producing world-leading RNA-based medical products,” Professor Pouton said.

“This grant will enable the two groups to combine expertise in mRNA and gene editing to develop a technological platform for gene correction with broad therapeutic applicability and a universal therapeutic approach to the treatment of infants and children with OTC deficiency.”