Messenger RNA (mRNA) technology, which continues to play a key role in the ongoing fight against Covid-19, represents one of the most important scientific breakthroughs of our time. The widespread effectiveness of mRNA-based vaccines has drawn much attention to the prospect of mRNA technology serving as a platform that can be used to develop a range of preventive and therapeutic drugs, including vaccines for infectious diseases and cancer treatments.
Such a technology architecture is quite 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 gave rise to ecosystems of innovation: a group of companies that share core technologies and develop new products and services around this common foundation. We believe that mRNA technology platforms can create similar ecosystems, which will have implications for innovation in the pharmaceutical industry.
Advantages of Platforms
Platforms have deservedly garnered a lot of attention in recent years, thanks, for example, to Bob Langer’s lab at MIT’s Koch Institute and Flagship Pioneering’s venture creation approach in the life sciences.
A Flagship product, Moderna, Pfizier, and BioNTech have used their mRNA technology platform to develop an important product – a safe and highly effective Covid-19 vaccine – at unprecedented speed. also modern claims have a team of several hundred scientists dedicated to improving the firm’s platform. This includes the development of delivery systems for mRNA-based drugs that are more biodegradable, less toxic, and therefore more tolerated at high doses than current therapies.
For example, mRNA therapies for cancer can offer highly effective treatments without the extreme side effects that patients experience with many conventional chemotherapy. Stéphane Bancel, CEO of Moderna, announced: “We think there are 10 or 20 different cell types that we can deliver mRNA to. … You can make a lot of different drugs after you make the delivery, because RNA is information.” In this sense, mRNA is “software” that can be rewritten.
Historically, drug development has been one-off – focused on identifying a molecule that modulates a disease target and seeking the protection and regulatory approval of that molecule, and their use based on evidence from clinical trials. While these efforts involve lengthy, complex, and increasingly costly processes, an approach to addressing one disease target often does not work for another. As a result, R&D projects had to be highly customized.
However, using a platform will make it possible to achieve economies of scope, thereby significantly increasing the productivity of the drug development process – for example, by allowing manufacturers to skip multiple steps of developing a new mRNA-based therapy. In addition, drugs and vaccines based on the same platform can gain regulatory approval more quickly as the platform has already been validated and its safety has already been established by regulators and clinicians.
How Platforms Can Reveal Ecosystems
Equally important, the way pharmaceutical companies innovate will likely change more profoundly as mRNA platforms more broadly share their interfaces and create innovation ecosystems. Platform owners will play a regulatory role in an ecosystem of external adopters and complements. The owners will control the key infrastructure and do so by publishing, sharing the interfaces with external parties such as Apple and Google. numerous application programming interfaces (APIs) motivate software developers to create innovations that complement the platform.
both BioNTech and modern They’ve taken the first steps to open mRNA platforms to other major pharmaceutical and biotech companies, including immuno-oncology, viral vaccines, and treatments for rare diseases. These steps are effectively turning internal mRNA platforms into startups of industry platforms and potentially innovation ecosystems.
Outside parties can design their own mRNA therapies based on platform technology to take advantage of more innovation opportunities (both preventive and therapeutic) than the platform owner can pursue alone. External parties can also contribute to platform development by improving the core infrastructure of the mRNA platform, such as the delivery system that safely transports mRNA to cells, thereby creating additional product development opportunities for themselves and other companies.
All of the above will increase the value of a platform and provide more incentive for others to join it. A virtuous cycle will enable some platforms to attract outside innovators and enable them to contribute to their development, enabling them to become dominant innovation ecosystems over time.
In general, three factors will determine whether platforms are successful in attracting external partners:
Applicability width: Whether the platform technology offers more opportunities for drug development than the platform owner alone could exploit – for example, because the owner has limited capacity or wants to draw attention to certain therapeutic areas.
The criticality of technology: to the extent that platform technology is the key activation technology for a new drug that cannot be created without it.
provability: whether the platform technology has been scientifically proven and whether the initial applications have been verified 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 to have broad applicability, it could be a powerful platform in the pharmaceutical industry whatever Apple’s iOS and Google’s Android have become in the digital sector: open to adopters and complements, but a technological core tightly controlled by the platform owner. The effects of pharmaceutical industry innovation will be significant. Several dominant mRNA innovation ecosystems may emerge that determine the pace and direction of innovation based on this technology.
In the coming months and years, mRNA platform owners such as Moderna, Pfizer, and BioNTech will decide to what extent they open their platforms to outsiders and become regulators of an innovation ecosystem. They will have to weigh the benefits and drawbacks of allowing or not allowing wide access.
Allowing access to a platform will be attractive to the owner if it can derive value from the new product development efforts of others – 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 the risks of being accountable to some extent for the drugs others develop with their platform. Another consideration for the owner is whether outside parties will be allowed to use his platform to develop drugs for the same goal, which would reduce incentives to innovate in that space.
Pharmaceutical companies that don’t have their own mRNA platform will have to weigh the pros and cons of joining or building someone else’s existing platform. On the one hand, adopting another platform would significantly reduce development costs, allowing the firm to focus on specific disease targets, using an approved technology and potentially avoiding it. controversial intellectual property fights. On the other hand, building on someone else’s platforms and developing applications and components for it means sharing the value of them with the platform owner, and using a particular platform can lock a firm into using it in the future because as often happens with digital. world, it may or may not be possible to switch to another platform.
Another issue for companies, policy makers and regulators will be whether the dominance of one or more platforms and their ecosystems will hinder innovation by limiting the diversity of approaches to developing new drugs. Policymakers and regulators will have to figure out how to ensure fair and sustainable access to platforms and adequate competition in individual therapeutic areas. This is important for patients and broader public health who may benefit from access to alternative treatments. For example, it could be that a mixture 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 next-generation therapeutics and produce enormous social benefits. Businesses, policy makers and regulators have the opportunity to help them reach this potential. The choices platform owners make about whether or how to open their platforms to others, pharmaceutical companies to join a platform, and the choices policymakers and regulators make about how they manage platforms will greatly affect the ecosystems that emerge and the benefits they deliver. produce for society.
The authors thank Arti Rai for valuable input on previous drafts of this manuscript. The research by Timo Minssen and Nicholson Price for this article was supported by a Novo Nordisk Foundation grant for a scientifically independent collaborative research program in the law of biomedical innovation.