RNA therapeutics is a rapidly expanding area of next-generation biological drugs. RNA is a versatile molecule and naturally regulates gene expression, either by upregulation or downregulation. This function is employed to design novel RNA therapeutics. The fundamentals of RNA therapeutics consists of delivering functional RNA at a target; however, this is restricted due to its fragile nature and lack of appropriate delivery systems.
Advances in RNA stability, chemical modification, and delivery systems have led to the commercial translation of RNA therapeutics during the past two decades. Moreover, RNAs are relatively easy to manufacture, are cost-effective, and can act on undruggable targets. Conventionally, only academic labs and small biotech companies focus on RNA therapeutics research.
Previously, RNA therapeutics R&D was focused on rare diseases and cancer. However, recent advances in chemical modification, improved stability, formulation, and nucleic acid delivery systems have improved the prospects of RNA therapeutics, and pharmaceutical and biotechnology interest are on the rise.
mRNA-based expression of CRISPR/Cas 9 gene editing tool and monoclonal antibodies, self-amplifying RNA, and small activating RNA are some of the new concepts in RNA therapeutic research.
Initial commercialization of antisense oligonucleotides (ASOs) and short interfering RNA (siRNA)-based therapeutics shows great promise. mRNA applications in the expression of missing proteins or mRNA encoding antigens is the next area to be explored for applications into genetic diseases and expression of immunomodulatory molecules in cancer and vaccinology.
RNA therapeutics is widely applicable to tumors, neurological diseases, cardiovascular diseases, rare genetic diseases, and infectious diseases. Despite having high potential for treating disease through altering gene regulation, RNAs are fragile in nature, large in size, and negative charge needs modification and formulation to improve stability, targeting, and cell uptake. Therefore, efficient delivery of therapeutic RNA molecules to targeted cells, activation of RNA molecules, and specificity without stimulating immune response are of paramount importance for the success of RNA therapeutics.
RNA therapeutics consist of several types of drugs, some of which are commercially available, while most are in the development phase. Despite a wide variety, messenger RNA therapeutics and RNA interference are the most widely studied approaches among RNA therapeutics. The following figure shows a variety of RNA therapeutics divided into 4 major types, and then it further classifies RNA interference and messenger RNAs.
The research is intended to answer the following questions:
• What are the driving factors for RNA therapeutics?
• What challenges and impediments remain to the adoption of RNA therapeutics?
• What initiatives are industry participants undertaking to accelerate adoption?
• What are the RNA therapeutics industry-desired business outcomes compared to traditional drugs?