RNA therapeutics — a new class of treatments for human disease

RNA is often seen as a molecule with a supporting role in human physiology– a go-between for DNA and proteins. In fact, RNA is a macromolecule with immense versatility, which can be harnessed to tackle the most complex and difficult to treat diseases.

Promise of RNA

The promise of RNA has been demonstrated through the success of vaccines to treat the global SARS-CoV-2 pandemic. While vaccine development has historically taken an average of 10 to 15 years, the highly effective SARS-CoV-2 mRNA vaccines, with efficacy rates greater than 90%, were developed, manufactured and rolled out to the public in less than 12 months.

Now, with its efficacy and safety having been proven, the versatility of RNA can be applied across a wide range of therapeutic areas, such as cancer, infectious disease, and rare disease.



The power of RNA to address the burden of disease is clear. From mRNA to RNAi to siRNA and beyond, RNA therapeutics have been proven effective in diverse indications, including SARS-CoV-2 and hereditary ATTR amyloidosis.

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RNA is a well-characterized natural molecule that is abundant throughout the human body. RNA has a very short cytoplasmic half-life that enables an outstanding safety profile rarely seen with other therapeutic solutions.

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RNA therapeutics are able to go from sequence to clinic in a fraction of the time of other drug modalities, with dramatically lower manufacturing footprints and costs.


RNA has tremendous adaptability and functional versatility, with the ability to encode for antibodies, antigens, cytokines and a multitude of other therapeutic proteins.

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