RNA therapeutics from rare to common diseases

Abstract

RNA therapeutics have taken a center stage during the pandemic, due to the successful development and launch of two mRNA COVID-19 vaccines (by Moderna and Pfizer/BioNTech). Although there are already multiple commercially successful RNA drugs for the treatment of orphan indications, this is the first time that RNA therapies unlock their commercial value in mass population. With the backdrop of massive capital and interest flooding into the field of RNA therapeutics, many companies began to expand their orphan-indication-centered RNA therapeutic portfolio into common diseases. In this article, we calculated the success rate of publicly available RNA therapeutic pipelines at each development stage as well as their likelihood of approval (LOA).  We found those targeting common diseases have a much lower LOA rate when compared with pipelines targeting rare diseases (5.8% vs. 23.8%). Consequently, we discussed the underlying challenges and potential opportunities for RNA therapeutics moving from rare to common diseases.