RNA Targeted Small Molecules Market Trends, Business Overview, Industry Growth and Forecast to 2031

The global RNA-targeted small molecules market is anticipated to grow at a significant CAGR during the forecast period (2024-2031). The conventional clinical delivery of oligonucleotide-based therapies comes with certain unaddressed challenges of genetic disorders. This unmet medical need has created promising opportunities for the global RNA-targeted small molecules market. The ability of small molecules to target the more complex RNA structures is primarily anticipated to drive market growth. As of 2020, out of the 55 US FDA-approved drugs, more than 65% of them were small molecule therapeutics; the majority of which were conventionally developed to target proteins. 

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Although just 2% of the human genome is allegedly responsible for coding proteins. This is why, therapies are now focused on evaluating previously untapped targets, such as non-coding DNA and RNA. For instance, in January 2022, HotSpot Therapeutics and Caris Life Sciences had collaborated to advance precision medicine approaches for HotSpot's emerging product portfolio. The partnership will focus initially on two therapeutic programs, the first of which is HotSpot's novel allosteric inhibitor of the E3 ubiquitin ligase CBL-B, an important target in cancer immunotherapy, spanning from pre-First in Human (FIH) trials through potential Companion Diagnostics (CDx) and launch.

Some key players operating in the market include Pfizer Inc., Amgen Inc., and Novartis International AG, among others. These players are focusing on strengthening their position in the market by adopting various strategies, such as mergers and acquisitions, partnerships, collaborations, new product launches and development in the existing product portfolio, and so on. For instance, in January 2022, Amgen and Arrakis Therapeutics announced a research collaboration focused on the discovery and development of RNA degrader therapeutics against a range of difficult-to-drug targets in multiple therapeutic areas. This new class of "targeted RNA degraders" consists of small molecule drugs that selectively destroy RNAs encoding disease-causing proteins by inducing their proximity to nucleases. Amgen will pay $75 million upfront to Arrakis for five initial programs and will have the option to nominate additional programs.

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Market Coverage

•             Segment Covered- 

• By Product Type
• By Application
• By End-User 

•             Regions covered-

• North America
• Europe
• Asia-Pacific
• Rest of the World

•             Competitive Landscape:  Pfizer Inc., Amgen Inc., and Novartis International AG, among others.  

Key questions addressed by the report

• What is the market growth rate?
• Which segment and region dominate the market in the base year?
• Which segment and region will project the fastest growth in the market?
• How COVID-19 impacted the market?
  • Deviation from the pre-COVID-19 forecast
  • Most affected region and segment
• Who is the leader in the market?
• How are players addressing challenges to sustain growth?
• Where is the investment opportunity?

Global RNA Targeted Small Molecules Market Report by Segment

By Product Type

• mRNA Translation Regulation
• RNA Splicing Modification 
• Direct RNA Targeting
• Indirect RNA Targeting-Epitranscriptomics

By Application

• Drug Discovery 
• Disease Identification 
• Oncology Research

By End-User

• Hospitals
• Research Laboratories
• Contract Research Organizations
• Pharmaceutical and Biotechnology Companies

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