RNA-targeted drug discovery and development is beginning to deliver important and broad therapeutic benefits. The field also continues to progress at a rapid pace with advances in the technology being converted into enhanced performance of new medicines in the clinic. The advances are so broad and occurring so fast that it is difficult even for experts in the field to stay abreast of the progress.
In a review published in Cell Metabolism, my colleagues and I provided a summary of the current state of the field and the tremendous progress in RNA-targeted therapeutics that is brief enough to be accessible to a broad audience yet detailed enough to be useful. A key objective was to describe a framework that can support a proper focus and approach to understanding the technology. We described the basic properties of different types of RNAs and the RNA-targeted medicines in development and provided a quick look at the characteristics of RNA-targeting medicines and the potential molecular mechanisms for which the drugs may be designed. We conclude by focusing on the performance of these medicines in the clinic, including newly approved therapies like SPINRAZA® (nusinersen)* and TEGSEDI™ (inotersen)**.
*SPINRAZA is marketed by Biogen
**TEGSEDI is marketed by Ionis’ commercial affiliate, Akcea Therapeutics.