The magic of mRNA will drive medical progress for everyone

The magic of mRNA will drive medical progress for everyone

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mRNA is one of the first molecules of life. While it was identified six decades ago as the blueprint carrier for proteins in living cells, its pharmaceutical potential was long underestimated. mRNA seemed unpromising—too unstable, too weak in potency, and too inflammatory.

The successful one development of the first mRNA vaccines against Covid-19 in 2020 was an unprecedented achievement in the history of medicine. This success was built on iterative progress over decades, driven by the independent input of scientists around the world.

We fell in love with mRNA in the 1990s because of its versatility, its ability to stimulate the immune system and its safety profile – once it has completed its biological task, the molecule is completely degraded without leaving a trace in the body. We have discovered ways to exponentially improve the properties of mRNA, increasing its stability and efficacy, and the ability to deliver it to the correct immune cells in the body. These advances have allowed us to create effective mRNA vaccines that, when administered in small amounts to humans, elicit a powerful immune response. In addition, we have established rapid, scalable processes to produce new vaccine candidates for clinical use within weeks. The result was the 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 tackle two of the world’s oldest and deadliest pathogens: malaria and tuberculosis. There are about 10 million new cases of tuberculosis worldwide each year. For malaria, medical needs are even higher: an estimated 230 million cases of malaria were reported in the WHO African Region in 2020, with most deaths among 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 progress in understanding how malaria and tuberculosis pathogens hide and evade the immune system, providing insight 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 regions in these proteins that are optimal targets for vaccine development.

One of the advantages 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 against multiple vulnerable pathogen targets. 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 endeavor could change the way we prevent these diseases and could contribute to their eradication.

Medical innovations can only make a difference to the people of 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-consuming, as well as error-prone. To overcome this obstacle, we developed a high-tech solution called the BioNTainer—a deliverable, modular mRNA production facility. This innovation could support decentralized and scalable vaccine production worldwide by moving to automated, digitized and scalable mRNA production capabilities. We expect the first facility to be operational in Rwanda in 2023.

We expect that 2023 will bring us these and other milestones that could help shape 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.

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