Australia’s mRNA Revolution is Accelerating Drug Development

When there’s a breakthrough in mRNA-based medicines, molecular biologist and geneticist Seth Cheetham is often caught up in the media whirlwind. From his beginnings in basic research studying how RNA contributed to brain development in fruit flies, he has become one of Australia’s leading mRNA experts. Now at the helm of the National Biologics Facility (NBF) and leading a research team at the Australian Institute for Bioengineering and Nanotechnology, Cheetham focuses on building up a pipeline of mRNA therapies and ensuring the manufacturing infrastructure to support their development exists.

“Having the manufacturing capability paired with [our clinical trials system] really enables Australia to emerge as a real center for this kind of translational mRNA science,” Cheetham said. “I view it as being completely essential.”

Targeted Delivery of mRNAs Beyond the Liver

Out of necessity, several of Cheetham’s key research projects focus on improving the delivery of mRNA medicines beyond the liver, which has been a major challenge for the field broadly.¹ “When I started working with mRNA, essentially you had two choices: either work on something that affects the liver or work on targeting,” said Bettina Dietmair, a PhD student in Cheetham’s laboratory, “because you couldn't really develop a medicine and then sit around and wait for someone to solve the targeting issue for you.”

Dietmair’s PhD research uses bispecific antibodies (BsAbs) to deliver mRNAs packaged inside lipid nanoparticles (LNPs). Using patented platform technology co-invented by Dietmair’s co-supervisor Chris Howard of the University of Queensland, the BsAbs are designed to target nanomaterials, allowing them to bind both to the LNP and the surface markers on the target cell type for delivery of the mRNA cargo. After some early challenges working on the technology during her master’s research, Dietmair was tying up some loose ends when she identified ideal conditions for BsAbs. “These conditions worked well for the cells that we tested them on, and then essentially every other cell since,” she explained.

What began as a side-quest became the focus of Dietmair’s doctoral research. “It was just that these [BsAbs] happen to work so well, and you kind of end up following the data, following what works,” Cheetham remarked. “This was just such a runaway success that she ended up devoting all her time to that.”

The research was published in Molecular Therapy Nucleic Acids in 2025 and also won the Best of Molecular Therapy Nucleic Acids Award for that year.² “The nice thing about this technology is that it's very flexible,” Dietmair explained. “So, you can use any mRNA LNP with any of the approved formulation recipes, and then also you can change the part that binds to the target cell.”

The team has since explored a range of possible targets, including cancer, autoimmune disorders, and genetic diseases, and is currently homing in on some lead indications. “[The approach] is generalizable to all kinds of cell types,” Cheetham added. “So, it really allows you to go after any kind of indication that you can really think of because this is essentially a way to manipulate the function of cells in a programmable way.”

A Unique Clinical Trials System Brings Australia to the Forefront of Translational Research

According to Cheetham, there are two key factors driving the rapid evolution of Australia’s mRNA ecosystem. The country saw massive investments in mRNA technology following the COVID-19 pandemic, including the launch of a commercial-scale Moderna manufacturing facility, and preparing for ‘virus X’ is still on the agenda. Australia also has an ideal translational ecosystem to support early-stage clinical trials, making it highly attractive for sponsors of novel therapies. At the NBF, Cheetham focuses on developing experimental medicines for Phase I trials, which do not require GMP-grade materials.

“We take advantage of the unique Australian regulatory landscape that allows you to get into a Phase I much more easily than in other places,” he said. In fact, this was a major draw for students like Dietmair, who said she would otherwise have had to stay in basic research. “A major reason for me to move to Australia in the first place was the translational research that's going on here,” Dietmair added.

With the manufacturing capacity and translational infrastructure in place, there seems to be no limit to the possibilities for mRNA technology in Australia. Outside of their own research, Cheetham’s team is busy enabling the translation of novel therapies by collaborating with sponsors to deliver mRNA molecules for early-phase trials. They are also leading the push to develop personalized cancer vaccines, launching an mRNA cancer vaccine hub that aims to collaborate with researchers and clinicians from around Australia to design these vaccines and rapidly advance them into the clinic; one of their collaborators has already progressed their vaccine for pediatric brain cancer to clinical trials. “We really need to keep on climbing that pipeline of innovation to find ways to make [personalized cancer vaccines] even more effective, apply them against cancers that haven't yet been studied, and also work on better models for studying these things and better algorithms for designing them,” Cheetham concluded. “It's a very exciting position to be in.”