Novel RNA Therapeutic Modalities

Given the success of the COVID-19 vaccines and the progress that has been made in understanding the role of RNA in health and disease, it is not surprising that RNA-based therapeutics have gained significant attention and are showing the potential to grow considerably. This growth is also being fueled by tools that enable the development and evaluation of RNA modalities. For example, Revvity’s Dharmacon portfolio has been developing and manufacturing custom RNA for more than two decades. These include siRNA, shRNA, miRNA, CRISPR mRNAs, and guide RNAs and Pin-point™ base editing reagents. Furthermore, the ongoing improved understanding of the mechanism of action of a range of RNA types, such as siRNA, shRNA, and miRNA, will continue to uncover novel causes of disease and offer emerging and potentially transformative therapeutic modalities. In addition to the fundamental science and the multidisciplinary nature of future therapeutic modalities, collaboration at every level should be encouraged. Bio-technology, pharmaceutical, academic, and government organizations must consider how science, process development, informatics, supply chain, manufacturing, and regulatory interact. Hence, development teams ought to follow an integrated approach. © 2024 Mary Ann Liebert Inc.. All rights reserved.