The Magic of mRNA Will Push Medical Advances for Everyone
mRNA is one of the first molecules of life. While identified six decades ago as the carrier of the blueprint for proteins in living cells, its pharmaceutical potential was long underestimated. mRNA appeared unpromising—too unstable, too weak in potency, and too inflammatory.
The successful development of the first mRNA vaccines against Covid-19 in 2020 was an unprecedented achievement in the history of medicine. That success was built on iterative progress over decades, driven by the independent contributions of scientists around the world. We fell in love with mRNA in the ’90s because of its versatility, its ability to stimulate the immune system, and its safety profile—after fulfilling its biological task, the molecule completely degrades, leaving no trace in the body. We discovered ways to exponentially improve the properties of mRNA, increasing its stability and efficacy, as well as the ability to deliver it to the right immune cells in the body. That progress allowed us to create effective mRNA vaccines that, when administered in small amounts to humans, elicit powerful immune responses. Moreover, we established rapid, scalable processes to manufacture new vaccine candidates for clinical application within weeks. The result was mRNA’s breakthrough in the fight against Covid-19. The potential of mRNA vaccines goes beyond the coronavirus. We now want to use this technology to tackle two of the world’s oldest and deadliest pathogens: malaria and tuberculosis. Worldwide, there are around 10 million new cases of tuberculosis every year. For malaria, the medical need is even higher: about 230 million malaria cases have been reported in the WHO Africa region in 2020, with most deaths occurring among children under 5.The convergence of medical advances—from next-generation sequencing to technologies to characterize immune responses on large data sets—boosts our ability to discover ideal vaccine targets. Science has also made progress in understanding how malaria and tuberculosis pathogens hide and evade the immune system, providing insights into how to combat them. The ongoing revolution in computational protein structure prediction allows for the modeling of three-dimensional structures of proteins. This is helping us decipher regions in these proteins that are optimal targets for vaccine development. One of the beauties of mRNA technology is that it enables us to rapidly test hundreds of vaccine targets. Moreover, we can combine multiple mRNAs—each encoding a different pathogen antigen—within a single vaccine. For the first time, it has become feasible for an mRNA-based vaccine to teach the human immune system to fight against multiple vulnerable targets of a pathogen. In 2023, we plan to begin clinical trials for the first mRNA vaccine candidates against malaria and tuberculosis that combine known and new targets. If successful, this endeavor may change the way we prevent these diseases and may contribute to their eradication.Medical innovations can only make a difference for people around the world when they are available on a global scale. The production of mRNA is complex and involves tens of thousands of steps, making technology transfer resource- and time-intensive, and error-prone. To overcome this bottleneck, we have developed a high-tech solution called BioNTainer—a shippable, modular mRNA manufacturing facility. This innovation could support decentralized and scalable vaccine production worldwide by leapfrogging toward automated, digitized, and scalable mRNA manufacturing capability. We expect the first facility to be up and running in Rwanda in 2023. We anticipate that 2023 will bring us these and other important milestones that could contribute to shaping a healthier future, a future that can build on the potential of mRNA and its promise to democratize access to innovative medicines. Now is the time to drive that change.