UNSW RNA Institute Files Prospectus to Advance RNA Therapeutics in Oncology, Antiviral, and Rare Diseases

“The RNA revolution has only just begun.”Pall Thordarson, Director of the UNSW RNA InstituteThe professor has publicly stated.

In recent years, Pfizer and Moderna have reaped substantial profits from their mRNA COVID-19 vaccines, significantly elevating their standing in the pharmaceutical industry. Reportedly, in 2021, Pfizer supplied 2.2 billion doses of its mRNA COVID-19 vaccine globally, while Moderna delivered 807 million doses worldwide in the same year. The development of mRNA COVID-19 vaccines has become a highly competitive and sought-after sector.

In fact,The Future of RNA: Beyond COVID-19 Vaccines,The success of Pfizer and Moderna in mRNA vaccines is merely the tip of the iceberg in the RNA medical technology revolution. Beyond addressing current and future infectious diseases, RNA therapies are also poised to treat cancer, genetic disorders, and autoimmune diseases.

To keep pace with this revolution, Australia, as a major biotechnology R&D hub in the Asia-Pacific region,The University of New South Wales also invested A$25 million in 2021 to establish the UNSW RNA Institute.(hereinafter referred to as the RNA Institute), dedicated to the development and manufacturing of RNA therapies.

It is reported that the institute will use RNA therapies produced in New South Wales for preclinical trials to treat COVID-19 and cancer, and in June this year, commence pilot-scale production of siRNA-based therapeutic drugs.

So, how was the UNSW RNA Institute established? And how will it translate scientific research achievements into practical applications in the future? With these questions in mind, VCBeat’s Chengguo Bureau interviewed Professor Pall Thordarson, Director of the UNSW RNA Institute, to gain a deeper understanding of the institute.

Professor Pall Thordarson, Director of the UNSW RNA Institute

To facilitate reading, VCBeat’s Orange Bureau has edited the interview transcript without altering its original meaning:

VCBeat: Could you please share the story behind the establishment of the RNA Institute, and what roles did you and the University of New South Wales play?

Professor Pall Thordarson:The story dates back more than a decade, when the Centre for Nanomedicine was established in New South Wales. The centre has placed significant emphasis on delivery systems, including siRNA delivery for cancer therapy. Here, numerous polymer and chemistry scientists have collaborated with a focus on pediatric cancers. Consequently, we have maintained collaborative relationships with these scientists over the years, and through this research, I have come to realizeRNA Therapies Will Become a Future Trend.

In 2019, I spoke with representatives from the Office of the Chief Scientist of the New South Wales Government and presented this proposal to them, marking its inception. The outbreak of the pandemic and the significant international success of RNA therapies further propelled the initiative forward. In 2021, the university approached me, deciding to take action. More importantly, the state government and the Premier shared the same vision and established a task force,All universities in New South Wales and other representative institutions have jointly established an RNA ecosystem.

Over the next decade, in addition to UNSW’s own investments,The state government will also invest $200 million., for the manufacturing, research, and development of RNA therapeutics.

At both the university and state levels, RNA research institutes possess sufficient capacity to lead global applications of RNA gene therapy and genetic medicine. This is driven by concurrent co-investment in viral vector technologies by the national government, Sydney’s long-standing historical advantages in rare diseases and epigenetics, and the further integration of nanomedicine technology research.

In RNA therapeutics, whether involving mRNA or other types of RNA, the primary challenge lies in delivery. The UNSW RNA Institute boasts expertise and knowledge in biology, medicine, chemistry, and related fields, which also form the foundation of the aforementioned national initiative and the RNA Institute established last year.

VCBeat: What are the criteria for recruiting researchers and students (specialization, research capabilities)? What aspects are prioritized?

Professor Pall Thordarson：Academic capability remains the key metric.Recently, the RNA Institute has added new members to ensure team diversity and complementarity.

At present, the team comprises both internationally renowned scientists and rising stars, including experts in epigenetics, basic biology, and RNA fundamental chemistry. Additionally, the RNA Institute is conducting research on new polymers and delivery systems, microfluidics, novel sensors, and the application of RNA therapeutics in areas such as cancer, HIV, COVID-19, and rare diseases.

Therefore, we focus onRNA Biology, RNA Medicine, and RNA ChemistryConducting research on these three themes is both an advantage and a challenge.

Specifically, the core challenges fall into four areas: first, manufacturing; second, analytics in the RNA field; third, sensing, with a focus on liquid biopsy—novel approaches dedicated to directly measuring RNA in blood samples; and fourth, RNA–protein interactions.

In the health sector, we are currently focusing on three key areas:

The first key area isDevelopment of siRNA Antiviral Drugs, and conducts research on populations with impaired immune function following HIV, COVID-19, and pediatric viral infections; the second key area isOncology or Cancer, particularly in the two key areas of pediatric cancer and pancreatic cancer; the third priority area isRare Genetic Diseases, including pediatric spinal muscular atrophy.

VCBeat: Many issues in the healthcare sector remain unresolved. Why did you choose to focus on these areas first?

Professor Pall Thordarson:In fact, we mainlySelect the appropriate domain based on existing competitive advantages.

Currently, we have established a robust research institute dedicated to the development of antiviral drugs for COVID-19 and HIV, and we are collaborating with another research institution on pediatric cancer research. Meanwhile, we have strengthened our clinical research partnerships with the University of New South Wales and have been conducting international clinical trials in collaboration with global companies in the field of rare genetic diseases.

VCBeat: Could you share some of the key research projects you are currently working on or are most excited about? How are they progressing?

Professor Pall Thordarson:The first isDevelopment of Antiviral Drugs Using siRNA, currently in the animal testing phase; the second isApplications of siRNA in Oncology, and is also about to enter animal testing; the third isCollaboration with Other States, launched the development of mRNA vaccines and initiated animal studies.

This fills me with great pride, as we only began manufacturing RNA this April, and we are now already producing RNA materials that people use for animal research.

VBInsight: How to Connect with Clinical Practice? What Are the Outcomes?

Professor Pall Thordarson:Our institution itself is more of aFocus on Production Scale, thus maintaining close ties with other research institutions within the university, including large-scale clinical trial units.

In Sydney, the state government also has a highly effective system to help people establish clinical trials. Furthermore, we have built international partnerships and are currently in discussions with companies from around the world that are interested in collaborating with us.

VCBeat Orange Bureau: What is the plan for advancing the project to clinical stages? Will there be follow-up collaborations? How will they be implemented specifically?

Professor Pall Thordarson:In fact, thisRelated to Government Plans。

The government is currently investing in a 10-year research program, in which we may also participate. Meanwhile, the government is also funding a manufacturing initiative to be operated by a private company, with the operator currently under selection. This means that projects from the RNA Institute can leverage large-scale GMP facilities for material production and clinical trials. If overseas partners seeking to conduct clinical trials approach us, they can also produce materials here and carry out clinical trials in Sydney.

In addition, we areStriving to Construct the Entire Value Chain。

The RNA Institute has completed its basic scientific research and is equipped with the necessary facilities to help scale up operations. Meanwhile, the government is supporting these efforts and the translation of clinical trials, aiming to pave the way for the new company’s operations.

VCBeat Orange Bureau: What is the primary mission of the RNA accelerator, and how do we achieve it?

Professor Pall Thordarson:It has three main tasks: the first task isFor Our Researchers, not only researchers from the University of New South Wales, but also others in Sydney, providing large-scale preclinical materials.

Because academic researchers are typically confined to bench-scale operations and can only conduct preliminary testing, there is a significant gap between their work and large-scale trials conducted in manufacturing facilities. Therefore, accelerators primarily focus on preclinical pilot production, providing materials at scale with more consistent purity.

The second task isTalent Training. This is a brand-new technology. Training can be conducted at intermediate stages, or leveraging the facilities used during factory trials. Such training opportunities are not only available to the Sydney plant but also extended to other plants worldwide.

Third, we hopeCollaborating with centers and companies around the world, to help them rapidly translate scientific research achievements from the laboratory to clinical practice.

VCBeat: Based on the work of RNA Accelerator, could you share some specific cases?

Professor Pall Thordarson:Now, we have beenProduced several batches of product raw materials scheduled for upcoming animal testing.For example, we have developed siRNA therapies targeting COVID-19 or cancer and are also producing the raw materials required for animal studies.

Currently, we have also begun collaborating with the Ministry of Primary Industries in a similar manner to develop animal vaccines for clinical services. In fact, we have not yet advanced significantly in this area; however, we are considering establishing graduate programs and micro-credential short courses. If companies send personnel to the RNA Institute for short-term training, we will provide them with coursework and certificates.

In terms of collaboration with various centers,We have collaborated with several key teams in Queensland and Melbourne, Australia., and is also gradually establishing more formal relationships with certain companies and centers around the world.

VCBeat: Earlier, you discussed the training of a new generation of technical experts. Do you encourage researchers to start their own businesses, or to transfer these technologies to companies?

Professor Pall Thordarson:UNSW (The University of New South Wales) is currentlyThere is growing encouragement for scholars to establish their own companies, and we have robust mechanisms in place to facilitate the commercialization of their research achievements.The RNA Institute has also partnered with SynBio 10x, an organization established to support startups in the field of synthetic biology by more effectively teaching entrepreneurs how to launch their own companies.

VCBeat Orange Fruit Bureau: What specific assistance and guidance did the RNA Institute provide in the translation of scientific research achievements?

Professor Pall Thordarson:"In fact, funding programs from universities and the government help researchers translate their findings into practical applications."

Therefore, weIt does not replicate everything that universities are already capable of doing,Researchers can seek expert advice at universities. Recently, we also appointed a development officer with extensive experience in the U.S. investment community to assist researchers who have limited knowledge of fundraising.

More importantly, in the early stages, many startups may encounter the challenge of acquiring materials or specific instruments; therefore, in the first phase, we will strive to help people address this issue.

VBInsight Orange Fruit Bureau: From your perspective, what advice do you have for researchers seeking to commercialize their scientific achievements?

Professor Pall Thordarson:Obviously, many people are directly engaged in developing new mRNA for vaccines or protein replacement therapies, among other applications. However, it is important to emphasize that many others fail to recognize that they possess truly critical key technologies, even if they are not directly involved in RNA-related work. Examples include polymer systems suitable for delivery, microfluidic technologies applicable to sensing, or even simply improvements in material biocompatibility.

I come from the field of chemistry and have extensive work in analytical chemistry. While some may consider this unrelated to RNA, these techniques could actually provide solutions to challenging problems in the RNA field.

Therefore, I believe that if you are interested, thenKeep an open mind and dare to take a few risks.

Artery Orange Bureau: What is the long-term strategic plan of the RNA Institute? What are its hopes and expectations?

Professor Pall Thordarson:Given the significant investment by the state government, we hope to establish a thriving presence in Sydney within 10 years.RNA Ecosystem.

At the institute level, we hope to evolve from our current state to having our own building, perhaps with 100 researchers. We hope that by then,Several successful RNA companies will emerge from the UNSW RNA Institute.

We currently have 20 academic researchers, a number that may increase to around 50. However, we do not all work at the same location. We share facilities, butThe next step is to establish an independent office building, where our core research teams will be co-located, enabling us to also begin supporting nearby spin-off companies.

"Within the next decade, we hope to see truly significant international scientific breakthroughs in RNA research."