RNA therapeutics from rare to common diseases

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 firs...

Full description

Saved in:
Bibliographic Details
Published in:Journal of commercial biotechnology 2022, Vol.27 (3), p.112-117
Main Authors: Tsai, Yueh-Lin, Chen, Yaqiong
Format: Article
Language:English
Subjects:
Biotechnology
COVID-19 vaccines
Drugs
Lipids
Marketing
Monetary incentives
mRNA
Pharmaceutical industry
Rare diseases
Registration
Statistical significance
Success
Toxicity
Trends
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary: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.
ISSN:1462-8732
1478-565X
DOI:10.5912/jcb1019