mRNA is one the first molecules of life. Although it was identified six decades ago as the blueprint for proteins in living cells, its pharmaceutical potential has long been underestimated. mRNA turned out to be unpromising – too unstable, too weak 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. This success has been based on repeated progress over decades, thanks to the independent contributions of scientists from all over the world.
We fell in love with mRNA in the 90s for its versatility, its ability to stimulate the immune system, and its safety profile—after completing its biological task, the molecule is completely degraded, leaving no trace in the body. We have discovered ways to exponentially improve the properties of mRNA, increasing its stability and efficiency, as well as the ability to deliver it to the right immune cells in the body. This progress has enabled us to create effective mRNA vaccines that, when administered in small amounts to humans, elicit powerful immune responses. In addition, we have implemented fast, scalable processes to produce new clinical vaccine candidates within weeks. The result was an mRNA breakthrough in the fight against Covid-19.
The potential of mRNA vaccines goes beyond the coronavirus. Now we want to use this technology to fight two of the world’s oldest and deadliest pathogens: malaria and tuberculosis. About 10 million new cases of tuberculosis are registered annually in the world. For malaria, the health needs are even greater, with about 230 million cases of malaria in the WHO African Region in 2020, with most deaths occurring in children under 5 years of age.
The convergence of medical advances, from next-generation sequencing to technologies for characterizing immune responses on large datasets, is increasing our ability to find ideal vaccine targets. Science has also made progress in understanding how the causative agents of malaria and tuberculosis hide and elude the immune system, helping to understand how to fight them.
The ongoing revolution in computational protein structure prediction enables the modeling of three-dimensional protein structures. This helps us decipher the regions of these proteins that are optimal targets for vaccine development.
One of the benefits of mRNA technology is that it allows us to rapidly test hundreds of vaccine targets. What’s more, we can combine multiple mRNAs, each encoding a different pathogen antigen, in a single vaccine. For the first time, it has been possible for an mRNA-based vaccine to train the human immune system to fight multiple vulnerable pathogen targets. In 2023, we plan to start clinical trials of the first mRNA vaccines against malaria and tuberculosis, combining known and new targets. If successful, this effort could change the way these diseases are prevented and could contribute to their eradication.
Medical innovations can only matter to 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-intensive and time-consuming, as well as error-prone. To overcome this bottleneck, we developed a high-tech solution called BioNTainer, a ready-to-ship modular mRNA production facility. This innovation could support decentralized and scalable vaccine production worldwide by shifting to automated, digitized and scalable mRNA manufacturing capabilities. We expect the first facility to be launched in Rwanda in 2023.
We expect 2023 to bring us these and other important milestones that can help shape a healthier future, a future that can build on the potential of mRNA and its promise to democratize access to innovative medicines. The time has come to make this change.