What if we could genetically “program” a drug to treat cancers, prevent malaria, contain HIV, cure certain rare diseases? This is the promise of the big pharmaceutical companies that are developing “platforms” of RNA therapies. The potential is immense, but raises fears of the concentration of power in the hands of a few giants, such as Apple, Microsoft or Google in the digital universe.
Nevertheless: enthusiasm reigns among researchers, who see new outlets appear for the fruit of their work. The stunning proof of concept of messenger RNA vaccines (mRNA) against COVID-19 paves the way for a small revolution in medicine, they believe.
“It’s the dream of all those who do research, particularly in cancer: to tell themselves that the mechanism they have discovered will live, 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 will not be limited to vaccines, but will extend to all RNA therapies. The major breakthrough concerns the delivery of these very fragile molecules inside 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, that is to say to the desired cells.
Once there, the RNA molecules do their job. They can give instructions to our cells to produce 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 linked to a rare disease, etc
Traditionally, drugs mimic enzymes, which are proteins. Their action in the body is sometimes difficult to predict and circumscribe. Genetic therapies, on the other hand, hit the desired target very precisely. “That’s magic as a curse!” says biochemist Jean-Pierre Perreault, an RNA specialist at the University of Sherbrooke.
At the end of April, Moderna announced with great fanfare the installation in the Montreal region of a factory capable of producing 100 million doses of vaccine. per year. His arrival in the city also includes a research partnership with McGill University.
Concretely, this means that academics will be able to send the genetic code of the therapy they are developing to Moderna, and that it will send them back vials of their experimental serum. Researchers will then be able to test these RNA therapies in mice and, possibly, humans. If the clinical trials are conclusive, the company will launch into production. Industry players call this “ready-to-use” service “a platform”.
“You really need optimization, a platform, to produce RNA on a larger scale,” says Anne Gatignol, researcher at the Lady Davis Institute and professor of experimental medicine at McGill University. This HIV specialist hopes to be able to create an RNA vaccine against the structural proteins of this virus responsible for AIDS thanks to the platform that will be made available to her from 2024.
The future is in platforms
“Platforms, I think that’s probably the future of biopharmaceuticals,” said Patricia Gauthier, CEO of Moderna Canada, in an interview with the To have to. Certainly, there are financial and technological risks inherent in the development of the platforms, “but once it works, it actually reduces the risks during the research. Afterwards, we can build an ecosystem with partnerships, for example”.
Moderna’s business model revolves precisely around the outsourcing of research and development (R&D) that the platform allows. With 3,200 employees around the world, Moderna “really doesn’t claim to be experts in all the therapeutic fields we want to go into,” says Ms.me Gauthier, specifying that the outsourcing of research makes it possible to “work with the best in their field, no matter where they are”.
Result: no more traditional R&D laboratories. What’s more, RNA vaccine production techniques make it possible to drastically reduce the size of manufacturing plants. “It’s like science fiction. It’s very computerized, ”said the lawyer, who has been the head of the vaccines division at GSK Canada for the past few years. A few dozen people are enough for the plant’s activities “in peacetime”, that is to say when there is no pandemic.
“The beauty of messenger RNA is that you can make a flu vaccine one week and ten days later make a COVID-19 vaccine…because it’s the same chemistry and the same ingredients,” she adds.
This business model has prompted Moderna in recent months to multiply “collaboration agreements” with universities here and elsewhere: the University of Queensland in Australia, the Sanofi Pasteur Institute in France and Tunisia, Vanderbilt University in Tennessee, the American Institutes of Health (NIH) and Harvard University.
In Canada, shortly after the announcement of the partnership with McGill University, the American biotech confirmed a partnership with the University of Toronto in which it will make its technologies available to researchers working in molecular genetics, biomedical engineering and biochemistry.
And this is only the beginning, says Patricia Gauthier. “Moderna 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 have our seats. »
Concentration risks
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 transform 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 says.
A new era that could benefit the owners of these platforms, who already “play an orchestration role” in research. While several biotechs are developing their own platforms, the pandemic has propelled a few companies to the fore: Moderna and the alliance formed by BioNtech and Pfizer.
Mr. Minssen does not hesitate to compare these “platform ecosystems” to those developed since 2000 by the digital giants thanks to their operating systems. “Owners control key infrastructures whose interfaces they share with external parties. As with Apple and Google, sharing these interfaces leads those who collaborate to create innovations that become complementary to their platform,” he says.
Are these fears of concentration justified? “That’s a very good question,” concedes M.me Gauthier, from Moderna. “It creates a certain interdependence. But we are not the only ones, there are other companies who are interested in this new trend. »
And indeed, since the start of the pandemic, things have been moving in the industry. In 2020, the British giant GSK entered into a partnership with CureVac, owner of an mRNA technology platform. Ditto for the French giant Sanofi, which acquired Translate Bio for 3.2 billion. This biotech has created an mRNA platform to develop drugs to treat or prevent debilitating and potentially fatal diseases.
Quebec strength
Despite the risks of dependency on the new platform, the Quebec scientists interviewed by The duty welcome the arrival of the US$59 billion company. Already, they are overflowing with ideas, to be tested with the tools made available to them.
The deal with pharma comet Moderna will ensure that academics no longer need to “spend a career fighting to develop a drug from an idea they had 20 or 30 years ago”, believes Mr. Duchaine. A closer association between academia and industry “could be extremely beneficial,” adds Ms.me Catignol.
Especially since Quebec has a “scientific force” unique in 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 a whole generation of researchers.
Industry players now expect Moderna’s presence to 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 protection of the intellectual property of discoveries that will be made in Quebec universities? No clause in the Moderna-McGill agreement establishes the sharing of rights between the researcher, the University and the company, according to information obtained by Professor Duchaine. Negotiations will take place on a “case by case” basis.
If these details will certainly interest the lawyers of the parties concerned, Mr. Duchaine, who knows that many cancer patients rely on researchers like him, thinks of something else. “We have a pipeline that starts from training and arrives at therapy. And that, for me, is more important than knowing that a researcher has money in his pocket. »