RNA Targeted Therapeutics Market Size - Analysis
The RNA targeted therapeutics market is estimated to be valued at USD 1.34 Bn in 2024 and is expected to reach USD 11.2 Bn by 2031, growing at a compound annual growth rate (CAGR) of 35.2% from 2024 to 2031.
Market Size in USD Bn
CAGR35.2%
| Study Period | 2024 - 2031 |
| Base Year of Estimation | 2023 |
| CAGR | 35.2% |
| Market Concentration | High |
| Major Players | Abivax, AC Immune, Arrakis Therapeutics, eFFECTOR Therapeutics, Eloxx Pharmaceuticals and Among Others |
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RNA Targeted Therapeutics Market Trends
The pharmaceutical industry has recognized the immense potential of RNA targeted therapeutics and has been steadily increasing investments in this promising area of drug development. Many big pharma companies are now dedicating large portions of their research & development budgets to further our understanding of RNA biology and develop novel RNA targeting molecules.
The strategic vision behind this investment trend is clear. Large pharmaceutical firms realize that RNA offers unprecedented potential for developing truly transformative therapies across disease areas. By modulating gene expression and protein levels, RNA targeted drugs have the ability to treat conditions previously believed to be "undruggable". This promises to open up whole new treatment paradigms and multi-billion dollar markets. The first wave of promising clinical RNA candidates validates this perception, with RNA interference and antisense oligonucleotide based medicines already demonstrating efficacy against cancer, neurological disorders as well as rare genetic diseases. With more investment pouring in, rapid scientific progress is expected to make RNA drugs a mainstay of treatment in the near future. This investment momentum is thus a major driver propelling the RNA targeted therapeutics market to new heights.
Market Driver - Rising clinical trials demonstrating the efficacy of RNA targeting small molecules
Clinical validation of these small molecule therapies has been rising steadily. For example, recent late phase studies showed that inclisiran, a small interfering RNA targeting PCSK9, can safely and effectively lower cholesterol and reduce cardiovascular events when dosed just twice a year. Patisiran, an interfering RNA targeting transthyretin achieved overwhelmingly positive results for treating hereditary transthyretin-mediated amyloidosis in phase 3 trials. Eplontersen, an antisense oligonucleotide is demonstrating efficacy in phase 3 for treating spinal muscular atrophy. Even in oncology, clinical studies indicate that mRNA encoded vaccines can generate robust antitumor immune responses.
Market Challenge - High cost of RNA targeting drug development
One of the key opportunities for growth in the RNA targeted therapeutics market is the expanding market potential resulting from the untapped class of non-coding RNA targets. While developments so far have majorly focused on targeting messenger RNA (mRNA) to block the translation of disease-causing proteins, recent research has revealed that non-coding RNAs like microRNAs (miRNAs), long non-coding RNAs (lncRNAs), and circular RNAs (circRNAs) also play vital roles in various disease pathways. Non-coding RNAs have emerged as crucial regulators of gene expression and several diseases have been linked to their dysregulation. However, compared to mRNA targeting, relatively few therapies aiming to modulate non-coding RNAs are under development. As research progresses in understanding the roles and functions of non-coding RNAs, several novel targets for conditions like cancer, neurological disorders, and metabolic diseases are expected to be identified. This opens up a huge opportunity for companies to develop innovative non-coding RNA targeting therapies. Targeting these untapped spaces has the potential to expand the current applications and market size of RNA drugs.
Key winning strategies adopted by key players of RNA Targeted Therapeutics Market
In 2020, Alnylam Pharmaceuticals partnered with Regeneron Pharmaceuticals to develop RNAi therapeutics for eye diseases. This allows both companies to leverage their expertise and resources to accelerate the development of RNAi therapies for ocular conditions.
In 2021, Arrowhead Pharmaceuticals acquired Takeda's pipeline of RNAi therapeutics to enhance its portfolio of RNA-based drugs. This added multiple clinical stage programs to Arrowhead's pipeline.
Segmental Analysis of RNA Targeted Therapeutics Market
The cap binding complex sub-segment dominated the RNA targeted therapeutics market with a share of 25.3% in terms of target molecule type. This is due to the central role the Cap Binding Complex plays in initiating mRNA translation. The cap binding complex recognizes the 5' cap structure found in eukaryotic messenger RNAs and recruits the large ribosomal subunit to begin protein synthesis. By disrupting the interaction between the Cap Binding Complex and the mRNA cap, translation can be halted for genes of interest. Many diseases are caused by aberrant expression of certain genes. Targeting the cap binding complex allows for precise control over translation and offers a promising approach for developing therapies.
Within the approach type segment of the RNA targeted therapeutics market, indirect targeting through epitranscriptomics held the leading share of 40.2%. Epitranscriptomics refers to reversible chemical modifications on RNA molecules, distinct from changes in the nucleic acid sequence. These modifications influence key properties of RNAs like stability, localization, and interactions with other molecules to regulate gene expression. Emerging evidence indicates RNA modifications play important roles in various biological processes and human diseases. Being able to target the enzymes responsible for installing or removing epitranscriptomic marks offers an indirect yet effective way to modulate gene expression for therapeutic benefit. Many companies are investing in developing therapies focused on epitranscriptomic enzymes given its potential for treatments across multiple disease areas.
Additional Insights of RNA Targeted Therapeutics Market
The RNA targeted therapeutics market is rapidly expanding due to its potential in treating previously untreatable conditions by focusing on non-coding RNAs. Over the last five years, significant investments have been made, and multiple small biotech companies have emerged as pioneers in this field, developing innovative RNA-targeting technologies. The market is expected to witness robust growth, with key players driving advancements in oncological and non-oncological therapeutic areas.
Competitive overview of RNA Targeted Therapeutics Market
The major players operating in the RNA targeted therapeutics market include Abivax, AC Immune, Arrakis Therapeutics, eFFECTOR Therapeutics, Eloxx Pharmaceuticals, H3 Biomedicine, PTC Therapeutics, Ribometrix, Skyhawk Therapeutics and STORM Therapeutics.
RNA Targeted Therapeutics Market Leaders
- Abivax
- AC Immune
- Arrakis Therapeutics
- eFFECTOR Therapeutics
- Eloxx Pharmaceuticals
RNA Targeted Therapeutics Market - Competitive Rivalry
RNA Targeted Therapeutics Market
(Dominated by major players)
(Highly competitive with lots of players.)
Recent Developments in RNA Targeted Therapeutics Market
- In March 2023, SiSaf partnered with the University of Leipzig to develop bio-courier targeted miRNA for treating pancreatic cancer. This collaboration aims to enhance targeted delivery methods, potentially improving therapeutic outcomes for pancreatic cancer patients.
- In February 2023, ReviR Therapeutics and Asieris initiated a research agreement to develop treatments for genitourinary tumors using RNA-targeted technologies, marking significant progress in addressing serious diseases within the genitourinary field.
RNA Targeted Therapeutics Market Segmentation
- By Type of Target Molecule
- Cap Binding Complex
- CDKL5
- Dystrophin Protein
- Erα
- G542X
- MNK1 / MNK2
- PAX6 gene
- SMN2
- By Type of Approach
- Indirect RNA Targeting: Epitranscriptomics
- mRNA Translation Modulation
- RNA Splicing Modification
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Ghanshyam Shrivastava - With over 20 years of experience in the management consulting and research, Ghanshyam Shrivastava serves as a Principal Consultant, bringing extensive expertise in biologics and biosimilars. His primary expertise lies in areas such as market entry and expansion strategy, competitive intelligence, and strategic transformation across diversified portfolio of various drugs used for different therapeutic category and APIs. He excels at identifying key challenges faced by clients and providing robust solutions to enhance their strategic decision-making capabilities. His comprehensive understanding of the market ensures valuable contributions to research reports and business decisions.
Ghanshyam is a sought-after speaker at industry conferences and contributes to various publications on pharma industry.
Frequently Asked Questions :
What are the key factors hampering the growth of the RNA targeted therapeutics market?
The high cost of RNA targeting drug development and regulatory challenges associated with novel RNA-targeting approaches are the major factors hampering the growth of the RNA targeted therapeutics market.
What are the major factors driving the RNA targeted therapeutics market growth?
The increasing investment in RNA targeted therapies by pharmaceutical giants and rising clinical trials demonstrating the efficacy of RNA targeting small molecules are the major factors driving the RNA targeted therapeutics market.
Which is the leading type of target molecule in the RNA targeted therapeutics market?
The leading type of target molecule segment is cap binding complex.
Which are the major players operating in the RNA targeted therapeutics market?
Abivax, AC Immune, Arrakis Therapeutics, eFFECTOR Therapeutics, Eloxx Pharmaceuticals, H3 Biomedicine, PTC Therapeutics, Ribometrix, Skyhawk Therapeutics, and STORM Therapeutics are the major players.
What will be the CAGR of the RNA targeted therapeutics market?
The CAGR of the RNA targeted therapeutics market is projected to be 35.2% from 2024-2031.
What are the key factors hampering the growth of the RNA targeted therapeutics market?
The high cost of RNA targeting drug development and regulatory challenges associated with novel RNA-targeting approaches are the major factors hampering the growth of the RNA targeted therapeutics market.