The RNA treatment revolution
By Rob Clancy, staff writer
Today, Associate Professor Michael Gantier is leading research into RNA therapeutics, a rapidly expanding category of drugs with the potential to revolutionise treatments for many diseases, including those driven by inflammation like autoinflammatory diseases.
When he was 11 years old, A/Prof Gantier had a close shave with death. Failing to recover after having his appendix removed, he was rushed to emergency surgery where doctors discovered he had sepsis. He would have died within hours if they hadn’t acted so quickly.
“The experience cemented my interest in science and how inflammation could be controlled to help patients,” says A/Prof Gantier.
RNA treatment – potential realised
When A/Prof Gantier was doing his PhD 20 years ago, RNA therapeutics was an emerging field. But it was clear to him that its potential would revolutionise medicine.
But science time is slow – it takes a mountain of patience, global collaboration, and an Everest of funding to make progress. Over the past two decades, global RNA therapeutics researchers have made solid headway, including scooping a couple of Nobel Prizes. Then came the real game-changer – the pandemic, and that elusive ‘X’ factor, funding, which enabled laboratory potential to be transformed into patient treatments.
“Thanks to the funds invested in vaccines for COVID-19, the mainstream use of RNA therapeutics is around 5-10 years ahead. It is now clear that RNA therapeutics, beyond vaccines, will grow to benefit millions of people including those with autoinflammatory disease,” A/Prof Gantier says.
RNA treatments for auto-inflammatory disease
A focus of A/Prof Gantier’s research is understanding how RNA molecules interact with the immune system to help create RNA-based treatments for autoinflammatory diseases such as lupus, psoriasis, Parkinson’s and fatty liver diseases.
“Our vision is to create long-lasting therapies for autoinflammatory diseases, including those currently considered untreatable.” Professor Michael Gantier
By examining how the immune system detects RNA molecules, A/Prof Gantier is bringing the incredible potential of RNA therapies closer to patient with autoinflammatory disease. His unique body of work is the largest in this field and is being used globally to define how some RNA therapies can block key immune system sensors that normally alert our body to infections.
In 2021, he published the most comprehensive description of how RNA therapeutics interact with the immune system in Nucleic Acid Research. This important work has the potential to unlock the secrets of RNA’s inflammatory effects, and to improve patient treatments in three areas
- Vaccines | While mRNA vaccines are life-saving, they require multiple doses to be effective and come with side effects including fatigue and muscle pain. Long-lasting vaccines with fewer side effects could be possible.
- Autoimmune disease | There could be treatments for many previously undruggable conditions.
- Fewer treatments | Patients’ quality of life could be improved greatly by replacing a daily cycle of drugs or weekly injections with six-monthly injections with RNA therapeutics.
A/Prof Gantier’s vision also took a step closer to being realised with the signing of a commercial licencing agreement to develop RNA therapies for patients with autoinflammatory diseases. See story below.
RNA technology licensing deal
Cutting-edge ribonucleic acid (RNA) technology, with the potential to reduce inflammatory side effects of drugs and make them easier to manufacture, has been developed by Hudson Institute and secured through an exclusive global licensing deal.
RNA technology has come to the fore during the COVID-19 pandemic with the approval and use of mRNA-based vaccines – it is seen as a key contributor to future drug development for not only vaccines, but other diseases as well.
The licence is for RNA drug discovery and mRNA vaccine manufacture with Australian clinical-stage drug development company Noxopharm Ltd, through its wholly owned subsidiary, Pharmorage Pty Ltd.
Leading RNA researcher Associate Professor Michael Gantier’s RNA technology was developed over 15 years and targets key immune sensors at the root of unwanted inflammatory responses.
“We discovered a new class of inhibitors that can outcompete immune sensing of therapeutic RNAs like those used in mRNA vaccines. That means fewer inflammatory side effects, optimum therapeutic potential without reducing manufacturing efficiency,” says A/Prof Gantier.
Pharmorage will use the technology to pursue the development of drugs to treat inflammatory and autoimmune diseases and improve the safety and efficiency of mRNA vaccines.
What is RNA?
DNA and RNA are a class of molecules called nucleic acids (the ‘NA’ in DNA and RNA). They contain and access the genetic information that controls which cells do what in our bodies. They are present in all forms of life, including bacteria and viruses, and are essential for our immune system to detect infections. In a cell, the main job of RNA is to convert information stored in DNA – our genetic blueprint or instruction – into proteins. This task is carried out by a specific type of RNA called ‘messenger’ RNA, or mRNA.
What are RNA therapeutics?
RNA therapeutics are a rapidly expanding category of drugs that have the potential to revolutionise treatments for many diseases, including autoinflammatory diseases. The RNA (ribonucleic acid) molecule is essential in gene coding, decoding, regulation and expression. RNA therapeutics aim to modulate the immune system’s response by tapping into a short RNA sequence to prevent the production of rogue proteins that can cause problems. The best-known examples of RNA therapeutics are mRNA COVID-19 vaccines.
Funders NHMRC/ARC; Québec Fonds de Recherche du Québec, Canada
Collaborators Pharmorage Pty Ltd; Integrated DNA Technologies
Global experts guiding Victoria’s mRNA future
Leading RNA researcher Dr Minni (Minna-Liisa) Änkö was enlisted to join an esteemed global panel of RNA biology and virology vaccine experts who will advise on Victoria’s mRNA research and manufacturing capability.
Established in June 2021, mRNA Victoria’s Scientific Advisory Group (SAG) comprises 10 global and local leaders in the fields of immunology, virology and infectious diseases. SAG Chair and Victoria’s Lead Scientist, Dr Amanda Caples, will harness the group’s significant knowledge to guide Victoria’s mRNA future.
“The benefits of SAG will be widespread, from preparing for emerging threats which can be tackled with RNA-based treatments, to the creation of jobs in research and production. This is a crucial time for Australia’s growing biotech industry and Victoria is at the centre, but there is a lot be done,” Dr Änkö says.
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