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RNA Therapies: From Pandemic Vaccines to Tomorrow’s Medicine

RNA Therapies: From Pandemic Vaccines to Tomorrow’s Medicine
While mRNA vaccines dominated headlines, the real promise of RNA lies in its ability to treat a wide range of diseases, from cancer to rare genetic disorders. -iStock)

Since the COVID-19 pandemic, RNA-based therapies have experienced an unprecedented surge, showcasing their potential to revolutionize medicine. While mRNA vaccines dominated headlines, the real promise of RNA lies in its ability to treat a wide range of diseases, from cancer to rare genetic disorders. In Québec, a region known for its strength in life sciences, the newly launched AReNA (ARN Québec) initiative is paving the way to make RNA therapeutics a cornerstone of the province’s biotech future.

The Rise of RNA: From Obscurity to Global Spotlight

The pandemic catapulted RNA science from academic obscurity to global prominence.

“If you go back six years ago, nobody wanted to talk to an mRNA scientist. Nobody even knew what mRNA was. Then suddenly, it was in the news every day,” recalled Germain Morin, a seasoned pharmaceutical executive with over 35 years of experience, who served as Pfizer’s Global VP of Supply Chain for Vaccines and Rare Diseases during the height of the COVID-19 pandemic. 

He spoke during a panel discussion at the Effervescence Forum in Montreal last April, where we had the opportunity to attend as invited media.

With the world racing to develop vaccines in record time, platforms like those developed by Moderna and Pfizer-BioNTech demonstrated that RNA was not just a theoretical technology—it was a viable, life-saving solution.

But the impact of RNA technologies extends far beyond COVID-19 vaccines. Researchers and biotech leaders now see RNA as a flexible, modular platform capable of producing customized therapies that target specific genetic mutations, generate antibodies directly in the body, or modulate the immune response in real time.

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Québec’s Ambition: Building a Hub for RNA Innovation

And Québec is positioning itself as a global player in this emerging field. The province is home to a rich scientific ecosystem, anchored by world-class institutions such as McGill University and the Université de Sherbrooke. Nearly half of Canada’s 300 RNA-focused research teams are based in Québec, a testament to the region’s expertise and momentum.

“We saw genuine excitement post-COVID,” explained Véronique Dugas, CEO of pharma-led biopharma research consortium CDQM. “People began to understand just how far RNA technologies could go—not just for vaccines, but for cancer, rare diseases, and more.”

In response, Québec launched the AReNA initiative in 2023 with CA$20.3 million in funding as part of its Life Sciences Strategy.

 “Our ambition is twofold,” said Xavier Linker, Director of Strategic Initiatives at the CDQM. “We aim to strengthen the local RNA ecosystem while positioning Québec on the global stage.”

AReNA supports nearly 30 academic and industrial projects and is building a province-wide RNA infrastructure—including biomanufacturing platforms across Sherbrooke, McGill, and the Innovation Santé Sherbrooke hub, and even a training ‘factory-school’ to develop skilled RNA manufacturing talent.

More Than mRNA: The Full Spectrum of RNA Therapeutics

Though the general public is most familiar with messenger RNA (mRNA) due to COVID-19 vaccines, therapeutic RNA comes in various forms, each with distinct applications:

  • mRNA: Delivers instructions to cells to produce therapeutic proteins or antigens.
  • Antisense oligonucleotides (ASO): Bind to specific mRNA sequences to modulate gene expression.
  • Small interfering RNAs (siRNA): Trigger degradation of disease-related mRNAs.
  • Self-amplifying RNAs (saRNA): Enhance protein production for stronger or longer-lasting effects.
  • Circular RNA: Offer improved stability for prolonged therapeutic expression.

This expanding toolkit makes RNA one of the most versatile platforms in modern drug development.

Agility and Accessibility: RNA as a Disruptive Technology

One of RNA’s biggest advantages is how quickly and easily it can be designed and manufactured. Unlike traditional small molecule drugs, which require complex chemical synthesis, RNA can be conceptualized on a computer and ordered for lab testing within weeks.

“As someone who worked on RNA during my studies, I know how easy it is to design a new RNA molecule,” said Dr Yassine El Bakkouri, Project Manager at Montréal InVivo in charge of the AReNA project. “We can draw the sequence on a computer, send it out, and receive it in the lab two weeks later. That doesn’t mean it’s ready to inject, but this speed is revolutionary for therapeutic development.”

Indeed, strategic partnerships—like the one formed between Pfizer and BioNTech back in 2012—helped companies respond quickly to the pandemic. Now, the same flexibility is being used to tackle cancers, neurodegenerative diseases, and rare conditions previously considered untreatable.

“With ASO technologies, for example, we can theoretically target almost any disease,” said Linker.

A New Frontier for Rare Diseases and Personalized Medicine

Rare diseases, though often well-understood scientifically, usually affect very small patient populations—too small to justify traditional drug development. RNA changes the equation.

“Rare diseases often affect only a handful of patients,” explained Dr. David Ferland-McCollogh, a senior RNA scientist at RNA Technologies and Therapeutics.“Despite robust research, they fall through the cracks because traditional drug pipelines can’t afford to screen millions of molecules for five patients. RNA therapeutics are more agile and modular, making them ideal for these cases.”

Moreover, RNA platforms are opening the door to personalized medicine—treatments tailored to an individual’s unique genetic profile. 

“We can now imagine RNA therapies designed for a specific person,” said Ferland-McCollogh. “That level of customization was science fiction just a few years ago.”

The Université de Sherbrooke is already exploring RNA molecules that alter mRNA splicing, a promising strategy for genetic diseases. In Montréal, clinical trials are already underway testing mRNA-based therapies for lung cancer and other tumors.

Cost, Scalability, and the Road to Democratization

Compared to gene and cell therapies—which are highly personalized, expensive, and difficult to manufacture—RNA therapies offer a path to greater accessibility. Their design and production are inherently faster and more scalable.

“Compared to gene and cell therapies, RNA could be more democratized,” said Dugas. “Right now, the costs are still high because we haven’t fully optimized the production process. But we’re seeing significant progress in scaling.”

Xavier Linker agrees, noting that while some production steps are still costly, others are more efficient than traditional drug development. He highlighted emerging compact bioreactor systems—some no larger than a household fridge—that allow RNA to be produced in smaller, cell-free environments.

“If these technologies take off,” said Linker, “we could see decentralized, hospital-based RNA production. Imagine university hospitals manufacturing personalized RNA therapies locally. That could drastically lower costs.”

The Future: Patients as Factories?

One of the most compelling—and futuristic—ideas in RNA therapeutics is to use the patient’s body as the manufacturing site. Instead of producing monoclonal antibodies in large bioreactors, what if the patient produced the therapy themselves?

“Monoclonal antibodies are expensive to manufacture,” explained El Bakkouri. “What if, instead of purifying them in a lab, we encoded them in mRNA and injected the RNA directly? The patient would become the factory, producing the antibody inside their own cells. It could target a specific molecule in cancer, for example. That’s the future we’re heading toward.”

With a clear strategy, targeted investments, and a collaborative scientific ecosystem, Québec is well-positioned to become a global leader in RNA therapeutics with AReNA aiming to create a true bridge from academic research all the way to the patient’s bedside.

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