mRNA is one of the first molecules of life. Although identified six decades ago as the carrier of the blueprint for proteins in living cells, its pharmaceutical potential has long been underestimated. mRNA did not seem promising – too unstable, too weak in potency and too incendiary.
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 based on iterative progress over decades, driven by the independent contributions of scientists around the world.
In the 1990s, we fell in love with mRNA for its versatility, its ability to boost the immune system, and its safety profile. After fulfilling its biological task, the molecule is completely broken down and leaves no trace in the body. We discovered ways to exponentially improve the properties of mRNA, increasing its stability and efficacy, as well as its ability to deliver it to the right immune cells in the body. Those advancements enabled us to create effective mRNA vaccines that elicit powerful immune responses when administered to humans in small amounts. In addition, we have established fast, scalable processes to produce new candidate vaccines for clinical application within weeks. The result was the breakthrough of mRNA 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 about 10 million new cases of tuberculosis each year. For malaria, the medical need is even greater: in 2020, approximately 230 million cases of malaria have been reported in the WHO Africa region, with most deaths occurring in children under 5 years of age.
The convergence of medical advances – from next-generation sequencing to technologies to characterize immune responses on large data sets – is increasing our ability to discover ideal vaccine targets. Science has also made strides in understanding how malaria and tuberculosis pathogens hide and evade the immune system, giving insight into how to fight them.
The ongoing revolution in computational protein structure prediction enables the modeling of three-dimensional structures of proteins. This helps us decipher regions in these proteins that are optimal targets for vaccine development.
One of the beauties of mRNA technology is that it allows us to quickly test hundreds of vaccine targets. In addition, we can combine multiple mRNAs – each encoding a different pathogen antigen – into a single vaccine. For the first time, it has become feasible for an mRNA-based vaccine to train the human immune system to fight against multiple vulnerable targets of a pathogen. In 2023, we plan to initiate clinical trials for the first mRNA vaccine candidates against malaria and tuberculosis that combine known and novel targets. If successful, this could change the way we prevent these diseases and contribute to their eradication.
Medical innovations can only make a difference to people around the world if they are available worldwide. The production of mRNA is complex and involves tens of thousands of steps, making technology transfer laborious, time-intensive and error-prone. To overcome this bottleneck, we have developed a high-tech solution called BioNTainer, a shippable, modular mRNA production facility. This innovation could support decentralized and scalable vaccine production globally by moving to automated, digitized and scalable mRNA production capabilities. We expect the first facility to be operational in Rwanda in 2023.
We expect 2023 to bring us these and other key milestones that can help shape a healthier future, one 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.
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