The magic of mRNA will drive medical progress for all

The magic of mRNA will drive medical progress for all

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 seemed unpromising—too unstable, too weak in potency, and too inflammatory.

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 built on decades of iterative progress, fueled by independent contributions from 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 is completely degraded, leaving no trace in the body. We 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. These advances have allowed us to create effective mRNA vaccines that, when administered in small amounts to humans, elicit strong immune responses. Moreover, we have established rapid, scalable processes to produce new vaccine candidates for clinical use 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. Worldwide, there are approximately 10 million new cases of tuberculosis each year. For malaria, the medical needs are even greater: an estimated 230 million cases of malaria were reported in the WHO African region in 2020, and most deaths occurred among 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 discover ideal vaccine targets. Science has also advanced in understanding how malaria and tuberculosis agents hide and evade the immune system, providing insights into 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 in these proteins that are optimal targets for vaccine development.

One of the beauties of mRNA technology is that it allows 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 possible for an mRNA-based vaccine to teach the human immune system to fight multiple vulnerable pathogen targets. In 2023, we plan to start clinical trials for the first mRNA vaccine candidates against malaria and tuberculosis that combine known and novel targets. If successful, this endeavor could change the way we prevent these diseases and could contribute to their eradication.

Medical innovations can only make a difference to people around the world if they are available globally. The production of mRNA is complex and involves tens of thousands of steps, making technology transfer resource- and time-consuming and error-prone. To overcome this bottleneck, we developed a high-tech solution called BioNTainer—a modular, shippable mRNA production facility. This innovation could support decentralized and scalable vaccine production worldwide by moving towards automated, digitized and scalable mRNA production. We expect the first plant to be operational in Rwanda in 2023.

We predict that 2023 will bring these and other important milestones that could help shape a healthier future, a future that can be built on the potential of mRNA and its promise to democratize access to innovative medicines. Now is the time to initiate that change.

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