RNA Targeting Small Molecule Drug Discovery Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases, Others), By End Users (Pharmaceutical and Biopharmaceutical Companies, Academic and Research Institutes, Others), By Region and Competition, 2021-2031F

Forecast Period	2027-2031
Market Size (2025)	USD 1.95 Billion
CAGR (2026-2031)	22.13%
Fastest Growing Segment	Cancer
Largest Market	North America
Market Size (2031)	USD 6.47 Billion

Market Overview

The Global RNA Targeting Small Molecule Drug Discovery Market is projected to grow from USD 1.95 Billion in 2025 to USD 6.47 Billion by 2031 at a 22.13% CAGR. The Global RNA Targeting Small Molecule Drug Discovery Market centers on the identification and development of low molecular weight compounds designed to bind directly to RNA tertiary structures, such as bulges, loops, and pseudoknots, to modulate their function. Unlike antisense oligonucleotides or siRNAs, these small molecules offer the distinct advantages of oral bioavailability, superior tissue distribution, and the potential to cross the blood-brain barrier. The market is primarily driven by the urgent need to address "undruggable" protein targets—such as MYC or KRAS—by intercepting their pathogenesis at the transcript level, alongside advancements in fragment-based screening and computerized structure prediction that enable the rational design of ligands for dynamic RNA pockets.

Despite this progress, the field faces a significant challenge in achieving sufficient selectivity, as the high structural plasticity and electrostatic nature of RNA make it difficult to develop compounds that avoid off-target interactions with abundant cellular RNAs like ribosomes. However, the broader RNA therapeutic sector continues to expand rapidly, fostering a robust environment for investment and innovation. According to the American Society of Gene & Cell Therapy (ASGCT), in 2025, the global pipeline for gene, cell, and RNA-based therapies comprised more than 3,200 active clinical trials, underscoring the immense clinical momentum and industrial focus propelling this diverse therapeutic modality forward.

Key Market Drivers

The surge in strategic pharmaceutical partnerships and venture capital investment is a primary force accelerating the Global RNA Targeting Small Molecule Drug Discovery Market, as major entities seek to diversify therapeutic pipelines beyond traditional protein targets. Large pharmaceutical companies are increasingly validating this modality by securing access to proprietary platforms capable of modulating RNA function in neurodegenerative and oncological conditions. For instance, according to Wayfinder Biosciences, April 2025, in the "Wayfinder Biosciences and Daiichi Sankyo Collaborate to Develop Novel RNA-Targeting Therapies for Neurodegenerative Disease" announcement, the company entered a strategic partnership with Daiichi Sankyo to leverage its RNA-targeting small molecule platform for identifying treatments against complex neurodegeneration targets. This trend highlights the industry's shift towards external innovation to overcome the limitations of conventional drug discovery.

Concurrently, the rising global prevalence of cancer and rare genetic disorders is necessitating the development of novel therapeutic agents for previously "undruggable" drivers of disease. As conventional inhibitors often fail to address non-enzymatic protein targets, the ability to intercept pathogenesis at the transcript level has become critical for meeting this escalating medical demand. According to the American Cancer Society, January 2025, in the "Cancer Statistics, 2025" report, it was projected that 2,041,910 new cancer cases would be diagnosed in the United States alone for that year, underscoring the urgent need for advanced therapeutic modalities. Furthermore, the commitment to addressing rare conditions is evident in recent funding initiatives; according to ReviR Therapeutics, November 2025, in the "ReviR Therapeutics Receives $4.6 Million CIRM Grant" press release, the company was awarded $4.6 million by the California Institute for Regenerative Medicine to advance its RNA-targeting small molecule program for Huntington’s disease.

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Key Market Challenges

The difficulty in achieving sufficient selectivity due to the structural plasticity and electrostatic nature of RNA stands as a critical barrier hampering the growth of the Global RNA Targeting Small Molecule Drug Discovery Market. Because these small molecules often interact promiscuously with abundant cellular RNAs like ribosomes, developers face heightened risks of off-target toxicity, which significantly increases the attrition rate during preclinical development. This technical complexity forces companies to invest heavily in extensive validation studies, prolonging development timelines and elevating the financial threshold for entry, which in turn deters risk-averse capital allocation.

This elevated risk profile has a direct, negative impact on the investment climate for early-stage companies attempting to translate discovery platforms into clinical assets. The uncertainty surrounding safety profiles leads to a tightening of the necessary funding for these novel modalities. According to the American Society of Gene & Cell Therapy (ASGCT), in the second quarter of 2025, start-up financings across the gene, cell, and RNA therapy sector raised only $197 million, continuing a decline in deal volume and value observed over the previous three quarters. This contraction in capital availability, driven by the high technical stakes of ensuring selectivity, directly restricts the number of viable candidates advancing into the market.

Key Market Trends

The advancement of small molecule splicing modulators represents a pivotal shift in the market, enabling the correction of pre-mRNA processing defects in genetic diseases previously deemed untreatable. By selectively binding to splice sites, these compounds can promote the inclusion or exclusion of specific exons to restore functional protein production, offering a potent alternative to complex gene therapies. This therapeutic potential was validated recently; according to Skyhawk Therapeutics, September 2025, in the 'Skyhawk Therapeutics Announces Positive First Interim Results in Patients from its Phase 1 Clinical Trial of SKY-0515' press release, the company reported that its lead splicing modulator achieved a 62% reduction of mutant huntingtin protein in patients with Huntington’s disease, demonstrating the modality's ability to engage difficult central nervous system targets.

Simultaneously, the expansion of R&D focus to Non-Coding RNA (ncRNA) targets is unlocking a vast, unexplored segment of the transcriptome beyond protein-coding genes. Developers are increasingly leveraging precision therapies to target long non-coding RNAs (lncRNAs), which play critical regulatory roles in cell states and disease progression, particularly in fibrosis and oncology. This growing investor confidence in the non-coding sector is evident; according to HAYA Therapeutics, May 2025, in the 'HAYA Therapeutics Raises $65 Million in Series A Funding' announcement, the company secured significant capital to advance its lead lncRNA-targeting candidate, HTX-001, into clinical trials for heart failure, underscoring the industry's commitment to drugging the regulatory genome.

Segmental Insights

The Cancer segment stands as the fastest-growing vertical in the Global RNA Targeting Small Molecule Drug Discovery Market, driven by the urgent need to treat malignancies with historically inaccessible targets. This expansion is fueled by the ability of these small molecules to bind RNA and prevent the formation of disease-causing proteins which conventional medicines often fail to address. Moreover, the US Food and Drug Administration has facilitated this progress through supportive regulatory pathways, thereby encouraging pharmaceutical companies to prioritize these innovative mechanisms for effective oncology management.

Regional Insights

North America maintains a dominant position in the Global RNA Targeting Small Molecule Drug Discovery Market, driven primarily by a robust biotechnology ecosystem in the United States. Market intelligence indicates that this leadership stems from the dense concentration of pioneering industry players and aggressive research and development investment within the region. Furthermore, the U.S. Food and Drug Administration (FDA) supports this innovation through progressive regulatory pathways, including orphan drug designations, which encourage the development of novel therapeutics for previously undruggable targets. This conducive environment, coupled with strong academic partnerships, solidifies North America’s status as the central hub for advancing RNA-targeted modalities.

Recent Developments

• In March 2025, Arrakis Therapeutics presented new preclinical data for its RNA-targeted small molecule program designed to treat myotonic dystrophy type 1 (DM1) at the Muscular Dystrophy Association Clinical and Scientific Conference. The presentation detailed the discovery of small molecules that bind to the toxic CUG repeat expansions in RNA, thereby displacing muscleblind proteins and correcting the splicing defects associated with the disease. This advancement demonstrates the expanding capabilities within the Global RNA Targeting Small Molecule Drug Discovery Market to develop oral, disease-modifying treatments for severe genetic disorders by directly targeting toxic RNA structures.
• In April 2024, Ribometrix presented preclinical data at the American Association for Cancer Research (AACR) Annual Meeting, validating its ability to design small molecules that target functional 3D RNA structures. The company disclosed data demonstrating the identification of potent small molecules that directly bind to the c-MYC mRNA, a driver of cancer growth that has historically been considered undruggable. These molecules were shown to reduce c-MYC protein levels in a dose-dependent manner. This research represents a technical breakthrough in the Global RNA Targeting Small Molecule Drug Discovery Market, proving that direct RNA-binding small molecules can effectively modulate difficult-to-drug oncogenes.
• In January 2024, Remix Therapeutics entered into a collaboration and license agreement with Roche to discover and develop small molecule therapeutics that modulate RNA processing. This strategic partnership utilizes the company's proprietary REMaster drug discovery platform to identify and target specific RNA processing mechanisms. Under the terms of the agreement, the company received an upfront payment of $30 million and is eligible to receive up to $1 billion in preclinical, clinical, and commercial milestones, as well as tiered royalties. This deal highlights the significant pharmaceutical interest in the Global RNA Targeting Small Molecule Drug Discovery Market for addressing disease drivers at the RNA level.
• In January 2024, Accent Therapeutics announced the completion of a $75 million Series C financing round to advance its pipeline of small molecule precision cancer therapies. The financing was supported by new and existing investors, including the venture arms of major pharmaceutical corporations such as Bristol Myers Squibb and Johnson & Johnson. The proceeds were allocated to support the advancement of the company's lead programs targeting RNA-modifying proteins, specifically DHX9 and KIF18A. This investment underscores the growing momentum and capital influx within the Global RNA Targeting Small Molecule Drug Discovery Market aimed at novel oncology targets.

Key Market Players

• Accent Therapeutics, Inc.
• Anima Biotech Inc.
• Arrakis Therapeutics, Inc.
• AstraZeneca Plc
• Epics Therapeutics, Inc.
• Expansion Therapeutics, Inc.
• F. Hoffmann-La Roche AG
• H3 Biomedicine Inc.
• PTC Therapeutics, Inc.
• Ribometrix, Inc.

By Indication	By End Users	By Region
Cancer Infectious Diseases Metabolic Diseases Neurological Diseases Others	Pharmaceutical and Biopharmaceutical Companies Academic and Research Institutes Others	North America Europe Asia Pacific South America Middle East & Africa

Report Scope:

In this report, the Global RNA Targeting Small Molecule Drug Discovery Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

• RNA Targeting Small Molecule Drug Discovery Market, By Indication:

• Cancer
• Infectious Diseases
• Metabolic Diseases
• Neurological Diseases
• Others

• RNA Targeting Small Molecule Drug Discovery Market, By End Users:

• Pharmaceutical and Biopharmaceutical Companies
• Academic and Research Institutes
• Others

• RNA Targeting Small Molecule Drug Discovery Market, By Region:

• North America
• United States
• Canada
• Mexico
• Europe
• France
• United Kingdom
• Italy
• Germany
• Spain
• Asia Pacific
• China
• India
• Japan
• Australia
• South Korea
• South America
• Brazil
• Argentina
• Colombia
• Middle East & Africa
• South Africa
• Saudi Arabia
• UAE