RNA-based therapies

RNA-based therapeutics hold the promise of tremendously expanding the number of ‘druggable’ targets by overcoming the major limitation of existing medicines, which are able to target only a limited number of proteins involved in disease pathways. Over the past 20 years, scientific and technical breakthroughs have significantly advanced the field of RNA-based therapeutics. Several classes of molecules and approaches have been investigated as RNA therapeutics, including antisense RNA, ribozymes, RNA decoys, aptamers, small interfering RNA (siRNA) and microRNA (miRNA)1 (TABLE 1). However, potential applications of RNA drugs in the treatment of human diseases remain in their infancy (TABLE 2). Multiple challenges, such as optimization of selectivity, stability, delivery and longterm safety, have to be addressed in order for RNA drugs to become a successful therapeutic category. Nevertheless, this area represents a major value creation opportunity in the industry, as evidenced by the increasing number of high-profile partnerships and merger and acquisition deals between pioneering biotechnology companies and traditional large pharmaceutical companies (FIG. 1).