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Welcome to the mRNA revolution

The story of messenger RNA (mRNA) begins with one of the most classic moves in scientific history.

In early 1961, three scientists – Sydney Brenner, François Jacob and Matt Meselson – submitted an article to the journal Naturedetailing their groundbreaking discovery of a molecule they dubbed “messenger” RNA – or mRNA for short.

Aluminum templates representing the structure of DNA discovered by Francis Crick and James Watson. Photo: Getty Images

But they weren’t the only ones to discover mRNA; The British-American research group of Professor James Watson – of Watson and Crick’s DNA double helix fame – had them too. He asked the trio to delay publication until his band had also submitted their own article.

Scientific breakthroughs usually result in a frantic race to publish first, but Professor Brenner and his colleagues generously agreed, and the two papers appeared back-to-back in a May 1961 edition of Nature.

We have come a long way since then.

After decades of being dismissed as scientific backwater, mRNA now represents arguably our best hope for fighting the COVID-19 pandemic – thanks to the success of Moderna and Pfizer/BioNTech vaccines in protecting many of us against the virus.

Much of the appeal of mRNA lies in its versatility and programmability, which have the potential to compress regulatory timelines and reduce development costs.

And it goes far beyond COVID-19 – and even beyond the realm of infectious diseases.

UNDERSTANDING mRNA

Basically, an mRNA molecule is simply a written instruction for a cell to make a specific protein. And by stringing together each of the four different chemical bases – adenine, guanine, cytosine and uracil – in different arrangements, it can be programmed to code for virtually any protein imaginable.

Overview of the rationale for mRNA-based treatment of NP-C1 disease. Graphic: provided

My research, in collaboration with other scientists, aims to apply this technology to the treatment of monogenic diseases, which are caused by harmful mutations within a single gene – for example, Niemann-Pick disease type C1 ( NP-C1) is one of these .

Patients with NP-C1 have an impaired ability to process cholesterol in their cells due to malfunctioning of the gateway protein, often resulting in severe clinical symptoms affecting the brain, liver, spleen, and in some cases , lungs.

Our theory was that by providing cells with mRNA coding for a functional version of this protein, we could potentially restore healthy protein levels, normalize cholesterol processing, and reverse the downstream effects of disease.

Indeed, our first experiments using patient cells confirmed our hypothesis. We are now taking the next steps to develop a delivery system that can transport mRNA to disease-affected organs, such as the brain.

If successful, the same technology could be applied to a whole range of other monogenic diseases such as Friedreich’s ataxia and Rett syndrome – and there are thousands more.

A MOLECULE OF INTEREST

mRNA is rapidly becoming a molecule of interest to scientists in the biomedical research community.

But it goes further.

The Australian state and federal governments have negotiated the establishment of a national mRNA manufacturing capability here, but an investment like this requires a steady and rich stream of mRNA candidates entering the pipeline to ensure continued commercial feasibility. .

mRNA now represents arguably our best hope for fighting the COVID-19 pandemic. Photo: Getty Images

Realizing that our research findings could be generalized to benefit others, I recently teamed up with Professor Frank Caruso, head of the Caruso Nanoengineering group at the University, to launch a new startup called Messenger Bio.

The startup’s core mission is to democratize access to cutting-edge technologies, starting with mRNA, so that researchers around the world can catch up on new projects faster and cheaper than ever before.

We currently do this by providing personalized mRNA synthesis and lipid nanoparticle formulation.

Previously, only highly specialized mRNA experts could work in this field. But by building an infrastructure that supports the end-to-end design, production, purification, and formulation of mRNA, this technology can now be made available to anyone who wants to use it.

Our longer-term vision is to build a platform that can serve scientists at all stages of their research journey – from bench to bedside.

Research like ours has only just begun to scratch the surface of mRNA’s potential.

Soon, the technology could be used clinically in applications ranging from cancer immunotherapy and allergy research to protein replacement, genome and epigenome editing – and even stem cell reprogramming. , which could see human cells tasked with repairing damaged tissue in conditions like Parkinson’s disease.

The opportunity to shape the future of genetic medicine is well and truly upon us – and now is the time to be part of the mRNA revolution.

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