Key Considerations When Selecting an mRNA CDMO Partner

Complex macromolecules are becoming a mainstay of pharmaceutical strategy, and the ability to successfully develop these biologics into viable commercial drugs is accelerating at a significant rate.

Gene therapy has made tremendous strides over the past decade or so. The first approved drugs are on the market, and literally hundreds of trials are underway offering unprecedented hope to patients with rare, debilitating genetic disorders. Nevertheless, safety issues continue to swirl around the field as adverse events are reported in trials involving both lentivirus and AAV vectors. Pricing and manufacturing pose additional hurdles that could significantly challenge the field.

Understanding the underlying biological processes in human health and disease requires studies integrating data from genomic, epigenomic, transcriptomic, proteomic, and metabolomic experiments.

Discovery of RNA therapies, such as antisense oligonucleotides, small interfering RNA, and, somewhat topically, mRNA vaccines, is becoming an increasingly important component of drug discovery research. This applies to both smaller companies focused on specific therapeutic types and to large biopharma, who often apply a multi-modal (i.e., “whatever works”) approach to solving disease problems. A feature of this research is that it typically involves cross-functional teams of chemists and biologists, working together to drive innovations for the types of chemical modifications of RNAs that are required to optimize efficacy and drug metabolism and pharmacokinetic property profiles. In order for these teams to work effectively and productively, it is critical that data capture, management, and analysis systems are able to handle these novel entity types and present data in ways that suit the respective needs of the team. This means that informatics systems must be equally adept at storing and representing the same entity as both a chemical structure and biological sequence.