Dr Knott came from the lab of Nobel prize winning American biochemist Jennifer Doudna (pioneer of CRISPR, the RNA-based tool revolutionising gene-editing) to head his own lab at Monash University, which is quickly assembling a pool of RNA talent. There’s Chen Davidovich, also out of a Nobel Prize-winning RNA lab, and Traude Beilharz – one of the true believers, who’s been working on RNA long before anyone thought it would change the world. Across campus Professor Colin Pouton has millions in funding to trial Australia’s first mRNA vaccine.
“All of a sudden they are on the cutting edge of where therapeutics are,” says Professor John Carroll, who hired them to Monash’s Biomedicine Discovery Institute.
At government level, Victoria and NSW have ploughed millions into duelling mRNA research funds, both competing for the big prize: a federally-funded mRNA vaccine manufacturing facility.
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We are at the “beginning of the race”, says Professor William Charman, who sits on mRNA Victoria’s Scientific Advisory Group. The barriers to entry remain low enough for Australia to become a major player, he believes.
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The University of Queensland’s Professor Trent Munro, one of Australia’s leading experts on vaccine development, is more skeptical. We’re getting in right at the top of the “hype cycle”, he fears.
“What worries me, a little bit, is there is this expectation all science is translational, all science leads to short-term financial gains. The reality is very different.”
Drugs change the chemistry within our bodies. RNA essentially allows scientists to issue a set of new genetic instructions: Body, change thyself. Unlike gene editing, RNA is temporary – the code is discarded within days, no permanent changes are made.
Pfizer and Moderna’s vaccines contain the RNA code for COVID-19’s spike protein; when injected, our cells read the code and make copies of the spike, in turn causing the immune system to think it is under attack.
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This sounds cutting edge. To Associate Professor Beilharz, it looks more like the crude steam engines built at the dawn of the industrial revolution. The RNA inside the vaccines is inefficient and therefore expensive. “We can’t afford to have vaccines that cost $10 if we’re going to vaccinate the whole world,” says Professor Beilharz.
RNA can do far more than vaccinate.
Other drugs use RNA to cut a misshapen human protein into its correct shape; two have been approved for spinal muscular atrophy and muscular dystrophy. Another uses RNA to block the action of a mutated gene, and already have been approved in Europe for the treatment of Familial amyloid polyneuropathy.
RNA can also bind directly to proteins and block their activities, another trick DNA lacks. One already-approved therapy uses this technique to block the growth of blood vessels in the eye, staving off vision loss.
In the future, drugs might even make use of RNA’s dual roles, turning off a faulty gene and replacing it with the correct set of instructions.
But the largest focus worldwide so far is cancer – a disease often caused by gene mutations. Send in new instructions and you could silence, say, the gene that confers immune-system invisibility to a tumour – allowing it to be attacked.
The discovery of insulin in 1921, and then penicillin in 1942, heralded the dawn of the chemical drug age. Next came proteins – the building blocks of the cell, and then DNA treatments. “And now,” says Professor Beilharz, “the next wave is RNA.”
Source: SMH
