RNA Therapeutics on the Rise
NEWS & ANALYSIS FROM THE ANALYST’s COUCH RNA therapeutics on the rise Feng Wang, Travis Zuroske and Jonathan K. Watts Digifoto Bronze/ Alamy Stock Photo The broad spectrum of options for increased 94.2% from 2015 to 2020. from Informa Pharma Intelligence’s therapeutic targeting of RNA has attracted Three representative mRNA therapeutic Biomedtracker. Of these drug candidates, substantial interest from both academic companies (Moderna Therapeutics, 63% are in the pre-IND stage, 32% are in research institutes and pharmaceutical BioNtech, and CureVac) attracted early- stage clinical trials (phase I or II), companies. With a growing number of US$2.8 billion of private investment since 3% are in phase III and 5 drugs are awaiting approved RNA therapeutics now generating 2015. Notably, Moderna set a record for regulatory decisions. significant profits, the level of investment the biggest biotech IPO with its value at The largest focus for all three modalities in the field has grown. In this article, we roughly $7.6 billion in 2018. Since 2017, is oncology, encompassing 22% of oligo- analyse investment data for companies RNA- related small molecule companies nucleotide candidates and 45% of mRNA developing RNA therapeutics and their have raised significant investment, including candidates. Beyond oncology, oligonucleo- pipelines. those targeting RNA directly ($262 million tide biodistribution has shaped pipeline to Arrakis, Expansion, Skyhawk and priorities. N-acetylgalactosamine (GalNAc) Investment focus for RNA therapeutics Ribometrix) and those targeting conjugation and lipid nano particle To understand where this investment is epitranscriptomics- related proteins approaches enable robust delivery of focused, we categorized RNA therapeutics ($194 million to Accent, Storm, Gotham oligonucleotides into hepatocytes, which into three groups (oligonucleotides, mRNA, and Twentyeight- Seven Therapeutics).
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