What if we could genetically “program” a drug to treat cancers, prevent malaria, get HIV, cure some rare diseases? This is the promise of large pharmaceutical companies that are developing “platforms” of RNA therapies. The potential is enormous, but raises concerns over the concentration of power in the hands of a few giants, such as Apple, Microsoft or Google in the digital universe.
Still: enthusiasm reigns among researchers, who are seeing new outlets emerge for the fruit of their work. The stunning proof of concept of messenger RNA vaccines (mRNA) against COVID-19 paving the way for a small medical revolution, they believe.
“It’s everyone’s dream, particularly in cancer: to tell themselves that the mechanism they’ve discovered will survive, that it will have a real impact”, says Thomas Duchaine, professor of biochemistry with enthusiasm. at McGill University and Associate Director of the Rosalind and Morris Goodman Cancer Institute.
The new possibilities are not limited to vaccines, but extend to all RNA therapies. The main success has to do with the delivery of fragile molecules within the human body. Thanks to innovative lipid envelopes – whose recipe Moderna and BioNTech have perfected – it is now possible to send them to their destination, i.e., to the desired cells.
Once there, RNA molecules do their job. They can give instructions to our cells to make proteins that trigger an immune reaction against a virus, to stimulate the expression of a protective gene against cancer, to block the expression of a harmful gene associated with a rare disease, etc.
Traditionally, drugs mimic enzymes, which are proteins. Their bodily action is sometimes difficult to predict and circumscribe. Genetic therapies, on the other hand, hit the desired target accurately. “That’s magic as a curse!” says biochemist Jean-Pierre Perreault, an RNA specialist at Sherbrooke University.
In late April, Moderna announced the installation in the Montreal region of a factory capable of producing 100 million doses of the vaccine. Every year. His arrival in the city also included a research partnership with McGill University.
Basically, this means that academics can send the genetic code of the therapy they do to Moderna, and it will send them vials of their experimental serum. Researchers will be able to test these RNA therapies in rats and, possibly, in humans. If clinical trials are specific, the company will launch production. Industry players call this “ready to use” service “one platform”.
“You really need optimization, a platform, to produce RNA on a larger scale,” said Anne Gatignol, researcher at the Lady Davis Institute and professor of experimental medicine at McGill University. This HIV specialist hopes to be able to develop an RNA vaccine against the protein structures of this virus responsible for AIDS thanks to the platform that will be available to him from 2024.
The future is in the platforms
“Platforms, I think maybe that’s the future of biopharmaceuticals,” Patricia Gauthier, CEO of Moderna Canada, said in an interview with Have. Sure, there are financial and technological risks inherent in developing platforms, “but when it does work, it really reduces the risks during research. Then, we can build an ecosystem with partnerships, for example” .
Moderna’s business model certainly revolves around the outsourcing of research and development (R&D) allowed by the platform. With 3,200 employees worldwide, Moderna “really doesn’t claim to be experts in all the therapeutic fields we want to enter,” Ms.I Gauthier, who states that outsourcing research makes it possible to “work with the best in their field, wherever they are”.
Result: no more traditional R&D laboratories. Furthermore, RNA vaccine manufacturing techniques make it possible to greatly reduce the size of manufacturing plants. “It’s like science fiction. It’s very computerized, ”said the lawyer, who has been head of the vaccines division at GSK Canada for the past few years. A few dozen people is enough for plant activities “in peacetime”, i.e. when there is no pandemic.
“The beauty of messenger RNA is you can make a flu vaccine a week and ten days later you can make a vaccine for COVID-19 … because it’s the same chemistry and the same ingredients,” he added.
This business model has prompted Moderna in recent months to proliferate “partnership agreements” with universities here and elsewhere: the University of Queensland in Australia, the Sanofi Pasteur Institute in France and Tunisia, Vanderbilt University of Tennessee, the American Institutes of Health (NIH) and Harvard University.
In Canada, shortly after the announcement of the partnership with McGill University, American biotech confirmed the partnership with the University of Toronto where it will make its technologies available to researchers working in molecular genetics, biomedical engineering and biochemistry.
And this is just the beginning, says Patricia Gauthier. “Modern Canada is only 18 months old. These agreements were established by fighting the pandemic, providing doses of vaccines and signing an agreement with the federal government. This is just the beginning; we already have a seat. »
Professor of law at the University of Copenhagen and founding director of the Center for Advanced Studies in Biomedical Innovation Law, Timo Minssen believes that platform-based research will dramatically change research in the life sciences. “The changes will be even more marked [lorsque les propriétaires des plateformes] share their interfaces more with researchers, creating innovation ecosystems, ”he said.
A new era that could benefit the owners of these platforms, who “perform orchestration” on research. While some biotechs are making their own platforms, the pandemic has pushed several companies forward: Moderna and the alliance formed by BioNtech and Pfizer.
Mr. did not hesitate. Minssen to compare these “platform ecosystems” to those developed since 2000 by digital giants thanks to their operating systems. “Owners control the core infrastructure whose interfaces they share with external parties. Like Apple and Google, sharing these interfaces leads to those working together to create innovations that are complementary to their platform, ”he said.
Are these fears of concentration justified? “That’s a very good question,” M agrees.I Gauthier, from Moderna. “It creates a certain partnership. But not only us, there are other companies interested in this new trend.»
And in fact, since the pandemic began, things have been moving in the industry. In 2020, British giant GSK partnered with CureVac, owner of an mRNA technology platform. Ditto for French giant Sanofi, which acquired Translate Bio worth 3.2 billion. This biotech has created an mRNA platform to develop drugs to treat or prevent debilitating and potentially deadly diseases.
Strength of Quebec
Despite the dangers of depending on the new platform, Quebec scientists have interviewed The duty welcome the arrival of the US $ 59 billion company. Already, they are overflowing with ideas, to be tested with the tools available to them.
Dealing with pharma comet Moderna will ensure that academics no longer have to “spend a career fighting to develop a drug from an idea they had 20 or 30 years ago”, believes Mr. Duchaine. A closer relationship between academia and industry “could be very beneficial,” Ms. added.I Catignol.
Especially since Quebec has a “scientific force” that is unique to Canada in terms of RNA research. This excellence can be explained in particular by the historic presence in Montreal of two heavyweights in the field, Robert Cedergren (University of Montreal, who died in 1998) and Nahum Sonenberg (McGill University, in office since 1979), who trained of a whole generation of researchers.
Industry players now expect that Moderna’s presence will foster the creation of an entrepreneurial ecosystem around this scientific community. To meet the expected demand for manpower, McGill University is even launching a master’s degree in pharmaceutical biomanufacturing next fall.
And what about the intellectual property protection of discoveries to be made at Quebec universities? No clause in the Moderna-McGill agreement establishes sharing rights between the researcher, University and company, according to information obtained by Professor Duchaine. Negotiations will take place on a “case by case” basis.
If these details are certainly going to be of interest to the lawyers of the parties concerned, Mr. Duchaine, who knows that many cancer patients rely on researchers like him, thinks otherwise. “We have a pipeline that starts with training and comes to therapy. And that, for me, is more important than knowing that a researcher has money in his pocket.»