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26 Pharmaceutical Technology ® The Real Message Behind Commercial mRNA Products April eBook 2023 PharmTech.com CHRISTOPH BURGSTEDT - STOCK.ADOBE.COM R NA has been likened to software; once a de- livery method is discovered, the sequence can be changed to produce any protein in the body. However, the apparent simplicity of the principle belies the complexity of the produc- tion process, and many challenges remain. Messenger RNA (mRNA) prophylactic vaccines achieved major breakthroughs during the COVID-19 pandemic, gaining approval in more than 150 coun- tries. The speed with which these vaccines were taken to market was a testament to the drive of the innovators, suppliers, and regulatory bodies involved. Now that the general principle is proven, there is much work to do to fully exploit the potential of mRNA in non-COVID in- fectious diseases, such as immuno-oncology, auto-im- mune diseases, protein replacement therapies, and gene-editing, to name but a few applications. It has been speculated, and in some cases docu- mented through early pre-clinical studies, that mRNA could be leveraged as a versatile tool for treating many severe indications that plague human health, including as a combinatorial therapy to treat cardiac fibrosis (1) and several types of cancers (2,3). Innovative and robust platform technologies will help to ensure that more products based on RNA can reach the market and deliver real benefits to support patients. The mRNA field is currently exploding, and many groups wish to explore an mRNA payload in a customized lipid nano particle (cLNP) as a new thera- peutic modality for their target disease. This requires three essential pillars: • RNA chemistry to optimize RNA construct design and production • Delivery systems such as cLNPs for RNA protection and specific immune response (immune activating or silencing) • Process technologies with the ability to optimize quality and scalability, enabling the manufacture of mRNA and LNPs from research grade through good manufacturing practice (GMP)-grade. Delivering on the Potential of mRNA Therapeutics David Ricketts is director of Business Development; Nevin Witman is a consultant for etherna; Phil Challis is VP Product Development; Senne Dillen is senior scientist Drug Substance and Drug Product Development; and Bernard Sagaert is interim CEO, all with etherna. The complexity of the RNA production process creates challenges.