Messenger RNA (mRNA) technology, which continues to play a key role in the ongoing fight against Covid-19, represents one of the most significant scientific breakthroughs of our time. The widespread effectiveness of mRNA-based vaccines has attracted considerable attention to the prospect of mRNA technology serving as a platform that can be used to develop an array of preventative and therapeutic medicines, includings for infectious diseases and cancer vaccine treatments.
Such a technology architecture is remarkably similar not only to the architecture of other health and life science technology platforms, such as CAR-T cell therapies or CRISPR-Cas technologies, but also to some in the digital sector. There, many platforms have given rise to innovation ecosystems: a group of companies that share core technologies and develop new products and services around that common base. We believe that mRNA technology platforms could spawn similar ecosystems, which would have implications for innovation in the pharmaceutical industry.
The Advantages of Platforms
Platforms have rightly garnered tremendous attention in recent years thanks to the work, for example, of Bob Langer’s laboratory at MIT’s Koch Institute and Flagship Pioneering’s approach to venture creation in the life sciences.
One Flagship offspring, Moderna, and Pfizier and BioNTech have used their mRNA technology platforms to develop an important product — a safe and highly effective Covid-19 vaccine — at first speed. Moderna also claims to have a team of several hundred scientists dedicated to advancing the firm’s platform. This involves developing delivery systems for mRNA-based medicines that may be more biodegradable, less toxic, and thus more tolerable at high doses than existing therapies.
For example, mRNA therapies for cancer may be able to deliver highly effective treatments without the extreme side effects that patients experience with many traditional chemotherapies. Stéphane Bancel, Moderna’s CEO, has explained: “We think there are 10 or 20 different cell types to which we can deliver mRNA. … Once you have the delivery, then you can do lots of different drugs, because RNA is information.” In that sense, mRNA is the “software” that can be rewritten.
Historically, drug development has been one-off — focused on identifying a molecule that modulates a disease target and seeking protection and regulatory approval of that molecule and its uses based on evidence from clinical studies. While these efforts involve long, complex, and increasingly costly processes, an approach to addressing one disease target often does not work for another target. As a result, R&D projects have had to be highly customized.
Using a platform, however, would make it possible to achieve growth of scope, thereby significantly increasing the productivity of the drug development process — for instance, by allowing manufacturers to leapfrog multiple steps of developing a new mRNA-based therapy. Moreover, drugs and vaccines based on the same platform may gain faster regulatory approval because the platform has already been validated and its safety already established with regulators and clinicians.
How Platforms Could Spawn Ecosystems
Just as important, the way pharmaceutical companies innovate will likely change more profoundly when mRNA platforms share their interfaces more broadly, building innovation ecosystems. Platform owners would play an orchestrating role in an ecosystem of external adopters and complementors. The owners would control the key infrastructure and share the interfaces with external parties, like Apple and Google, for example, have done by publishing numerous application programming interfaces (APIs) to motivate software developers to create platform-complementary innovations.
Both BioNTech and Moderna have taken first steps to open up their mRNA platforms to other large pharmaceutical and biotech companies in areas including immuno-oncology, viral vaccines, and therapies for rare diseases. These steps are effectively converting their internal mRNA platforms into the beginnings of industry platforms and potentially innovation ecosystems.
External parties could design their own mRNA therapies that build on the platform technology to exploit more innovation opportunities — both preventative and therapeutic — than the platform owner could pursue alone. External parties could also contribute to platform development, improving the mRNA platform’s core infrastructure, such as the delivery system that transports the mRNA safely to the cells, thereby creating additional product-development opportunities for themselves and other firms.
All of the above would increase the value of a platform and provide further incentives for others to join it. A virtuous cycle would ensue, enabling some platforms that excel in attracting outside innovators and getting them to contribute to their improvement to become dominant innovation ecosystems over time.
Overall, three factors will determine whether platforms succeed in attracting external partners:
Breadth of applicability: Whether the platform technology offers more opportunities for drug development than the platform owner could exploit alone — for example, because the owner is capacity constrained or wants to focus attention on certain therapeutic areas.
Criticality of the technology: the extent to which the platform technology is the key enabling technology for a new drug that could have not been created without it.
Demonstrability: Whether and the extent to which the platform technology has been scientifically proven and early applications have been validated by regulators.
How to Navigate the New World
While the extent to which mRNA platforms can be used to develop other drugs remains to be seen, if it turns out that there’s broad applicability, a strong platform could become in the pharmaceutical industry what Apple’s iOS and Google’s Android have become in the digital sector: A technological core is open to adopters and complementors but tightly controlled by the platform owner. The implications for pharmaceutical industry innovation would be substantial. A few dominant mRNA innovation ecosystems could emerge that determine the rate and direction of innovation based on that technology.
In the months and years ahead, mRNA platform owners such as Moderna and Pfizer and BioNTech will decide to what degree to open up their platforms to external parties and become orchestrators of an innovation ecosystem. They will have to weigh the benefits and drawbacks of allowing broad access or not.
Allowing access to a platform will be attractive for its owner if it can capture value from others’ new product development efforts — for example through licensing or revenue-sharing models. (In the digital sector, innovation ecosystems have been hugely profitable for platform owners.) But an owner will also have to worry about risks of becoming liable, to an extent, for the drugs that others develop with its platform. Another consideration for the owner is whether to allow external parties to use its platform to develop drugs for the same target, which would reduce the incentives to innovate in that area.
For their part, pharmaceutical companies that lack their own mRNA platform will have to weigh the benefits and drawbacks of joining another’s existing platform or building one themselves. On one hand, adopting another platform would reduce development costs, allow the firm to focus on certain disease targets, use a validated technology, and potentially avoid contentious intellectual property fights. On the other hand, building on another’s platforms and developing applications and components for it means sharing their value with the platform owner, and using a given platform may lock a firm into using it in the future because, as is often the case in the digital world, switching to another platform may not be feasible or possible.
Another issue for companies, policymakers, and regulators will be whether the dominance of one or a few platforms and their ecosystems will stymie innovation by limiting the diversity of approaches to develop new drugs. Policymakers and regulators will have to figure out how to ensure that there is fair and sustainable access to the platforms and sufficient competition in individual therapeutic areas. This matters for patients who may benefit from having access to alternative treatments and, more broadly, for public health. For instance, it may be that a mix of different mRNA-based vaccines provides better protection against a disease than only one type of mRNA-derived vaccine.
mRNA technology has the potential to streamline the development of a new generation of therapeutics and generate enormous social benefits. Businesses and policymakers and regulators have an opportunity to help it achieve that potential. The choices that platform owners make about whether or how to open up their platforms to others, that pharmaceutical companies make about joining a platform, and that policymakers and regulators make about how to govern platforms will heavily influence the benefits ecosystems that take form and the regulators that they produce for society.
The authors thank Arti Rai for valuable input on earlier drafts of this manuscript. Timo Minssen’s and Nicholson Price’s research for this article was supported by a Novo Nordisk Foundation grant for a scientifically independent collaborative research program in biomedical innovation law.