Unless you have had your head stuck firmly in the sand, you will have heard about RNA therapeutics by now. Put simply, RNA therapies aim to prevent the translation of ‘bad’ proteins and, ultimately, block their production. For example, as problems with protein expression may be a primary cause of cancer, cardiovascular or autoimmune disorders, therapies that act to inhibit the over-expression of disease-causing proteins could provide effective treatments.

I particularly liked the simile in a recent white paper by WuXi AppTec which said, “The surging scientific excitement surrounding RNA therapeutics is a little like realizing you can pick fruit off a tree without cutting it down, or pick a flower without uprooting the plant.” In other words, RNA-based therapies manipulate the translation of genetic instructions – such as inhibiting the overexpression of disease-causing proteins, as in our example above – rather than targeting the source of those instructions. While small-molecule and protein/antibody drugs mainly act on genome-derived proteins to exert pharmacological effects, RNA-based therapies hold the promise to expand the range of treatable targets – from proteins, to RNAs and the genome. The new approach is believed to make new targets easier to reach, and to open up more options for treating a broad range of illnesses.  

I am writing about RNA therapeutics now because many experts believe that they are about to take their place on the world stage of medicines. Combined efforts by pioneering biotechnology companies, pharmaceutical and academic institutions over the past two decades have enabled these treatments to come of age, and RNA-focused drug development is emerging as a new dominant treatment modality.

Early entrants into the market included antisense oligonucleotides like Mipomersen (approved in 2013) and Spinraza (2016), which demonstrated the therapeutic potential for reducing or boosting gene expression of proteins, and Onpattro (2017), the first RNA interference (RNAi) therapeutic. However, by 2018, Drug Discovery World reported that as many as 69 companies were developing RNA therapeutics in 315 clinical trials. The same publication predicted a global market value exceeding $10 billion by 2024.

This suggests that RNA-based therapies are likely to have a huge impact on the way in which new drugs are discovered and developed over the next few years. As summarized in an Xconomy article by the former President and CEO of Regulus Therapeutics, “In the world of pharma discovery and development – full of small molecules and larger protein drugs – typically there is one established target and numerous drug candidates competing to hit that target with the best combination of safety and effectiveness… Targeting RNA has created the ability to venture into a whole new space, essentially covering the entire genome, with new mechanisms of action that can be combined with current therapies.”

However, while the promise of these therapies is great, and research is extensive, challenges have been highlighted with respect to manufacturing processes. As highlighted in WuXi AppTec’s white paper, “To improve characteristics such as stability, potency and deliverability will require better manufacturing processes, and then the technical ability to produce therapies on a commercial scale.”

This is not unusual for new technologies, for which up-scaling is a standard challenge. However, considering the potential for this market, it would appear to offer a tremendous opportunity to companies with the expertise and experience to provide this capability. In fact, harking back to my recent article on Uniting pharma for value and innovation, I would comfortably predict some profitable relationships coming out of this opportunity, as we see multinationals ‘buying and building’ their portfolios through the acquisition of small, innovative companies that are active in RNA therapeutics, and partnerships between innovators and larger companies capable of up-scaling and commercializing their technology.

Author biography

Sarah Harding, PhD

Sarah Harding worked as a medical writer and consultant in the pharmaceutical industry for 15 years, for the last 10 years of which she owned and ran her own medical communications agency that provided a range of services to blue-chip Pharma companies. In 2016, she began a new career in publishing as Editor of Speciality Chemicals Magazine, and has more recently taken up the role of Editorial Director at Chemicals Knowledge. She continues to also provide independent writing and consultancy services to the pharmaceutical and speciality chemicals industry.

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