mRNA Advances Allow Drug Development Focus on Rare Diseases (Part One)

Chris Spivey, editorial director for Pharmaceutical Technology®, discussed messenger RNA (mRNA) advances at the leading scientific conference for the field, the 11th International mRNA Health Conference in Berlin, Germany, which was held Oct. 29– Nov. 2, 2023, with Thomas Langenickel, MD, the chief medical officer at Ethris, a Germany-based biotechnology company specializing in next-generation RNA therapeutics and vaccines.

Langenickel has more than 15 years of experience in clinical development and translational medicine in cardiovascular and respiratory disease areas. He previously held the position of executive director and head of Respiratory Profiling at Novartis and served as medical director at Bristol-Myers Squibb. Ethris worked to create a new path from genes to therapeutic proteins through non-immunogenic mRNA (SNIM mRNA) and lipidoid nanoparticle (SNaP LNP) technology platforms. These can produce drug candidates with superior thermostability and resistance to mechanical manipulation and that are nebulizable at high throughput, without change in biophysical properties or potency.

During the conversation, Langenickel discusses the focus on rare lung diseases and advances made over the last few years with an approach not available via previous efforts. This obviates some of the biodistribution challenges. Also, various pharmacokinetics become more tractable alongside certain posttranslational modification issues, in particular during the manufacturing process of these therapies. For these respiratory viral infection applications, the host immune system is modulated. Clinical trial applications have been submitted and were awaiting approval at time of discussion. Regulatory challenges also came up as a common barrier for mRNA medicines, up to and including being considered a biologic in one geographic region versus a gene therapy in others. A call for more harmonization across geographies to build a clearer path for these products is considered an important step forward for increased commercial success.