mRNA Revolution: A new generation of medicine
We are experiencing what has been described as an “RNAaissance” since the COVID-19 pandemic in 2020. RNA therapeutics rose to prominence with mRNA/LNP COVID-19 Vaccines from Moderna and Pfizer-BioNTech. However, there is more to RNA therapies than just prophylactic vaccines; RNA-based therapeutics can target diverse cellular molecules, including those deemed “undruggable” pathways or molecules that are not amenable to targeting by conventional drugs. Here, we are focussing on mRNA-based therapeutics.
We are experiencing what has been described as an “RNAaissance” since the COVID-19 pandemic in 2020. RNA therapeutics rose to prominence with mRNA/LNP COVID-19 Vaccines from Moderna and Pfizer-BioNTech. However, there is more to RNA therapies than just prophylactic vaccines; RNA-based therapeutics can target diverse cellular molecules, including those deemed “undruggable” pathways or molecules that are not amenable to targeting by conventional drugs. Here, we are focussing on mRNA-based therapeutics.
Why RNA?
There are several advantages to using mRNA as a therapeutic – mRNA is simpler to synthesise than conventional therapies, making it both faster and cheaper to produce. It is also more widely adaptable than conventional therapies and can be used to treat a variety of diseases and conditions, ranging from infectious diseases to cancer and rare genetic disorders. mRNA is also an alternative to protein therapy and is able to overcome some of the limitations and challenges of delivering proteins directly into the body, such as stability, immunogenicity, and bioavailability. Finally, mRNA is transient and non-integrating, hence it does not alter the genome of the cells and only expresses the desired protein for a limited time, reducing the risk of unwanted side effects or long-term consequences.
However, there are also some challenges to using mRNA as a therapeutic. mRNA is susceptible to degradation and unable to cross the cell membrane, therefore it needs to be delivered in a way that protects it from the body’s natural defences and helps deliver it into cells. mRNA can also trigger an immune response, which can limit its therapeutic potential. Despite these challenges, mRNA is a promising new drug platform with the potential to revolutionize the way we treat diseases. This explainer will define mRNA, discuss its role in medicines manufacturing, and explore its potential as a next-generation treatment.
mRNA Revolution: A new generation of medicine
mRNA is a promising new drug platform with the potential to revolutionize the way we treat diseases. Our explainer will define mRNA, discuss its role in medicines manufacturing, and explore its potential as a next-generation treatment.
What is mRNA?
Simply, mRNA is a molecule that carries instructions from DNA to the ribosomes, where proteins are made. Proteins are the building blocks of life and are responsible for a wide range of functions, including cell structure, metabolism, and signalling.
Deoxyribonucleic acid (DNA) is a molecule that contains our genetic code, the blueprint of life. This essential molecule is the foundation for the “central dogma of biology”, or the sequence of events necessary for life to function. DNA is a long, double-stranded molecule made up of bases, located in the cell’s nucleus. The order of these bases determines the genetic blueprint. To ‘read’ these blueprints, the double-helical DNA is unzipped to expose the individual strands and an enzyme translates them into a mobile, intermediate message, called ribonucleic acid (RNA). This intermediate message is called messenger RNA (mRNA), and it carries the instructions for making proteins.