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The dawn of an era in the biotech industry

It was back in 1953 when the elucidation of the DNA structure by Dr. James D. Watson and Dr. Francis Crick, widely regarded as the starting point of modern molecular biology, ignited the fuse that led to unraveling the central role of RNA in life. From that point on, numerous molecular mechanisms such as RNA interference (RNAi) have been described, and an unprecedented number of non-coding RNAs – RNA molecules that are not translated into protein – such as small interfering RNAs (siRNA), micro RNAs (miRNA) or ribozymes have been reported and characterized.

All these discoveries led to the apparition of a plethora of companies pioneering therapeutics based on synthetic RNAs – RNA molecules that are synthesized in the lab – designed to regulate abnormal gene expression caused by certain diseases, thus targeting their root causes rather than just the symptoms. Decades of paramount difficulties in the development of RNA-based therapeutics culminated in 2018, when approval to commercialize Patisiran was granted in the US and Europe, making it the first ever RNAi therapy available in the market.

More recently, just a month ago, Dr. Jennifer A. Doudna and Dr. Emmanuelle Charpentier were awarded the Nobel prize in Chemistry for the development of the CRISPR/Cas9 method for genome editing. Again, this is a method that uses synthetic RNA molecules: the so-called guide RNAs, which target the region of DNA to be modified and direct the editing molecular machinery there. Not only a number of therapies based on CRISPR/Cas9 are today under clinical development, but variants of this genome editing mechanism have been also used to develop tests for the diagnosis of COVID-19.

Even more recently, two names have been sweeping the board of media headlines during the last two weeks: Pfizer and Moderna. Both companies are racing to bring the first COVID-19 vaccine to the market and both companies claim that their vaccines have achieved an outstanding 95% efficacy. On top of that, and what is central to this article, is that both companies are using synthetic RNA molecules to induce immunity. In this case, those synthetic RNAs are messenger RNAs (mRNA) that code for the disease specific antigen which, once produced by the body, is recognized by the immune system, preparing it to fight the actual virus.

We strongly believe that synthetic RNA has sparked a revolution in the biotech industry, a revolution that started many years ago and that has gained a lot of momentum during the last couple of years. A revolution that, without a doubt, will completely reshape the industry in the coming years.

And we are proud to be part of it.

Amadís

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