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RNA靶向小分子藥物研發市場－全球產業規模、佔有率、趨勢、機會、預測：按適應症、最終用戶、地區和競爭格局分類，2021-2031年

RNA Targeting Small Molecule Drug Discovery Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Indication, By End Users, By Region & Competition, 2021-2031F
出版日期: | 出版商: TechSci Research | 英文 188 Pages | 商品交期: 2-3個工作天內

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簡介目錄

全球 RNA 標靶小分子藥物發現市場預計將從 2025 年的 19.5 億美元成長到 2031 年的 64.7 億美元，複合年成長率為 22.13%。

該市場主要專注於鑑定和開發能夠直接與複雜RNA結構（例如凸起、環狀結構和假結）結合並影響其生物學功能的小分子化合物。與反義寡核苷酸和siRNA等其他基於RNA的治療方法相比，這些小分子化合物具有明顯的優勢，包括可透過口服給藥來提高生物利用度、改善組織內分佈以及能夠穿過血腦障壁。推動這一市場成長的主要因素包括…

市場概覽
預測期 2027-2031
市場規模：2025年 19.5億美元
市場規模：2031年 64.7億美元
複合年成長率：2026-2031年 22.13%
成長最快的細分市場 癌症
最大的市場 北美洲

市場促進因素

推動全球RNA標靶小分子藥物研發市場發展的主要因素是策略性醫藥夥伴關係和創投的激增。領先企業正將其治療產品線拓展至傳統蛋白質標靶之外，並透過獲取能夠調控RNA功能的專有平台，不斷提升該療法在神經退化性疾病和腫瘤等疾病治療中的有效性。 Wayfinder Biosciences與第一三共株式會社於2025年4月簽署的合作協議便是這一趨勢的鮮明例證。該合作旨在利用Wayfinder的RNA靶向小分子平台治療神經退化性疾病，凸顯了該產業對外部創新的依賴。

同時，全球癌症和罕見遺傳疾病的日益普遍，推動了對能夠治療這些疾病的新療法的需求。

市場挑戰

全球RNA標靶小分子藥物研發市場成長面臨的主要障礙之一是難以實現足夠的標靶選擇性，這主要是由於RNA卓越的結構可塑性和靜電特性所致。這種複雜性使得RNA更容易與細胞內豐富的RNA（例如核醣體）發生特異性相互作用，導致更高的脫靶毒性風險。因此，研發人員在臨床前開發階段面臨較高的失敗率，需要投入大量資金進行廣泛的檢驗研究。這延長了研發週期，增加了市場准入的財務門檻，並最終阻礙了風險規避型資本的配置。

這些日益成長的技術風險正直接對尋求將藥物發現平台轉化為臨床資產的新興企業的投資環境產生負面影響。安全性方面的普遍不確定性阻礙了這些創新治療方法所需的關鍵資金籌措。例如，美國基因與細胞治療學會 (ASGCT) 報告稱，到 2025 年第二季度，基因、細胞和 RNA 療法領域的新創公司資金籌措僅為 1.97 億美元，延續了過去三個季度交易數量和金額的下滑趨勢。這種資金籌措緊張的環境源自於確保選擇性的高技術要求，直接限制了有前景的候選藥物進入市場。

市場趨勢

小分子剪接調變器的進步代表著重要的市場趨勢，使得糾正以往無法治療的遺傳疾病中前驅mRNA加工缺陷成為可能。這些化合物特異性結合剪接位點，精確地促進特定外顯子的插入或排除，從而恢復功能性蛋白質的生成。這種方法為更為複雜的基因療法提供了強而有力的替代方案。 Skyhawk Therapeutics公司近期展現了此治療方法的潛力。該公司於2025年9月公佈了SKY-0515的I期臨床試驗的積極中期結果，顯示亨廷頓病患者體內突變亨廷頓蛋白減少了62%，並證實了其對中樞神經系統靶點的治療方法。

同時，隨著研發重點擴展到非編碼RNA（ncRNA）靶點，傳統蛋白質編碼基因以外的轉錄組領域正被逐步揭示。研發人員擴大利用標靶長鏈非編碼RNA（lncRNA）的精準療法，已知lncRNA在多種細胞狀態和疾病進展中發揮關鍵的調控作用，尤其是在纖維化和腫瘤學領域。投資者對非編碼RNA領域的信心正在顯著增強。例如，2025年5月，HAYA Therapeutics公司獲得6,500萬美元A輪資金籌措，用於推進其旗艦lncRNA標靶候選藥物HTX-001在心臟衰竭治療的臨床試驗。這凸顯了業界對標靶調控基因組藥物研發的重視。

目錄

第1章概述

第2章：調查方法

第3章執行摘要

第4章：客戶心聲

第5章：RNA標靶小分子藥物研發的全球市場展望

  • 市場規模及預測
    • 按金額
  • 市佔率及預測
    • 適應症（癌症、感染疾病、代謝性疾病、神經系統疾病、其他）
    • 按最終用戶（製藥和生物製藥公司、學術和研究機構、其他）分類
    • 按地區
    • 按公司（2025 年）
  • 市場地圖

第6章：北美RNA標靶小分子藥物研發市場展望

  • 市場規模及預測
  • 市佔率及預測
  • 北美洲：國別分析
    • 美國
    • 加拿大
    • 墨西哥

第7章：歐洲RNA標靶小分子藥物研發市場展望

  • 市場規模及預測
  • 市佔率及預測
  • 歐洲：國別分析
    • 德國
    • 法國
    • 英國
    • 義大利
    • 西班牙

第8章：亞太地區RNA標靶小分子藥物研發市場展望

  • 市場規模及預測
  • 市佔率及預測
  • 亞太地區：國別分析
    • 中國
    • 印度
    • 日本
    • 韓國
    • 澳洲

第9章：中東和非洲RNA標靶小分子藥物研發市場展望

  • 市場規模及預測
  • 市佔率及預測
  • 中東與非洲：國別分析
    • 沙烏地阿拉伯
    • 阿拉伯聯合大公國
    • 南非

第10章：南美RNA標靶小分子藥物發現市場展望

  • 市場規模及預測
  • 市佔率及預測
  • 南美洲：國別分析
    • 巴西
    • 哥倫比亞
    • 阿根廷

第11章 市場動態

  • 促進因素
  • 任務

第12章 市場趨勢與發展

  • 併購
  • 產品發布
  • 近期趨勢

第13章 全球RNA標靶小分子藥物研發市場：SWOT分析

第14章：波特五力分析

  • 產業競爭
  • 新進入者的潛力
  • 供應商的議價能力
  • 顧客權力
  • 替代品的威脅

第15章 競爭格局

  • 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.

第16章 策略建議

第17章：關於研究公司及免責聲明

簡介目錄
Product Code: 15457

The Global RNA Targeting Small Molecule Drug Discovery Market is projected to expand significantly, growing from USD 1.95 Billion in 2025 to USD 6.47 Billion by 2031, at a Compound Annual Growth Rate (CAGR) of 22.13%. This market primarily focuses on identifying and developing small molecular weight compounds that directly bind to complex RNA structures like bulges, loops, and pseudoknots to influence their biological functions. These small molecules offer distinct advantages over alternative RNA-based therapies, such as antisense oligonucleotides or siRNAs, including the potential for oral bioavailability, enhanced tissue distribution, and the ability to penetrate the blood-brain barrier. Key drivers for this growth include the urgent need to address previously

Market Overview
Forecast Period2027-2031
Market Size 2025USD 1.95 Billion
Market Size 2031USD 6.47 Billion
CAGR 2026-203122.13%
Fastest Growing SegmentCancer
Largest MarketNorth America

Market Driver

A primary force accelerating the Global RNA Targeting Small Molecule Drug Discovery Market is the sharp increase in strategic pharmaceutical partnerships and venture capital investments. Major companies are seeking to diversify their therapeutic pipelines beyond traditional protein targets, increasingly validating this modality by securing access to proprietary platforms capable of modulating RNA function in conditions such as neurodegenerative diseases and oncology. An illustrative example of this trend is Wayfinder Biosciences' collaboration with Daiichi Sankyo in April 2025, which aims to leverage Wayfinder's RNA-targeting small molecule platform for neurodegeneration, underscoring the industry's reliance on external innovation.

Concurrently, the escalating global prevalence of cancer and rare genetic disorders is driving demand for novel therapeutic agents that can address

Market Challenge

A critical barrier impeding the growth of the Global RNA Targeting Small Molecule Drug Discovery Market is the inherent difficulty in achieving sufficient selectivity, largely due to RNA's significant structural plasticity and electrostatic nature. This complexity often leads to promiscuous interactions with abundant cellular RNAs, such as ribosomes, thereby elevating the risk of off-target toxicity. Consequently, developers face increased attrition rates during preclinical development, necessitating substantial investment in extensive validation studies. This prolongs development timelines and raises the financial threshold for market entry, ultimately deterring risk-averse capital allocation.

This elevated technical risk profile has a direct, negative impact on the investment climate for nascent companies endeavoring to translate discovery platforms into clinical assets. The pervasive uncertainty regarding safety profiles curtails the crucial funding required for these innovative modalities. For instance, in the second quarter of 2025, start-up financings across the gene, cell, and RNA therapy sector only garnered $197 million, continuing a trend of declining deal volume and value over the preceding three quarters, as reported by the American Society of Gene & Cell Therapy (ASGCT). This capital constriction, driven by the high technical demands of ensuring selectivity, directly limits the progression of viable candidates into the market.

Market Trends

The advancement of small molecule splicing modulators represents a significant market trend, facilitating the correction of pre-mRNA processing defects in genetic diseases that were previously considered untreatable. By specifically binding to splice sites, these compounds can precisely promote the inclusion or exclusion of particular exons, thereby restoring the production of functional proteins. This approach offers a powerful alternative to more complex gene therapies. This therapeutic promise was recently validated by Skyhawk Therapeutics, which reported in September 2025 positive interim results from its Phase 1 clinical trial of SKY-0515, demonstrating a 62% reduction of mutant huntingtin protein in Huntington's disease patients and proving the modality's ability to engage challenging central nervous system targets.

Simultaneously, the expanding research and development focus on Non-Coding RNA (ncRNA) targets is unlocking a vast and previously unexplored segment of the transcriptome, extending beyond traditional protein-coding genes. Developers are increasingly leveraging precision therapies to target long non-coding RNAs (lncRNAs), which are known to play crucial regulatory roles in various cellular states and disease progression, particularly within fibrosis and oncology. Growing investor confidence in this non-coding sector is evident; HAYA Therapeutics, for example, secured $65 million in Series A funding in May 2025 to advance its lead lncRNA-targeting candidate, HTX-001, into clinical trials for heart failure, underscoring the industry's commitment to developing drugs for the regulatory genome.

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.

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

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global RNA Targeting Small Molecule Drug Discovery Market.

Available Customizations:

Global RNA Targeting Small Molecule Drug Discovery Market report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report:

Company Information

  • Detailed analysis and profiling of additional market players (up to five).

Table of Contents

1. Product Overview

  • 1.1. Market Definition
  • 1.2. Scope of the Market
    • 1.2.1. Markets Covered
    • 1.2.2. Years Considered for Study
    • 1.2.3. Key Market Segmentations

2. Research Methodology

  • 2.1. Objective of the Study
  • 2.2. Baseline Methodology
  • 2.3. Key Industry Partners
  • 2.4. Major Association and Secondary Sources
  • 2.5. Forecasting Methodology
  • 2.6. Data Triangulation & Validation
  • 2.7. Assumptions and Limitations

3. Executive Summary

  • 3.1. Overview of the Market
  • 3.2. Overview of Key Market Segmentations
  • 3.3. Overview of Key Market Players
  • 3.4. Overview of Key Regions/Countries
  • 3.5. Overview of Market Drivers, Challenges, Trends

4. Voice of Customer

5. Global RNA Targeting Small Molecule Drug Discovery Market Outlook

  • 5.1. Market Size & Forecast
    • 5.1.1. By Value
  • 5.2. Market Share & Forecast
    • 5.2.1. By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases, Others)
    • 5.2.2. By End Users (Pharmaceutical and Biopharmaceutical Companies, Academic and Research Institutes, Others)
    • 5.2.3. By Region
    • 5.2.4. By Company (2025)
  • 5.3. Market Map

6. North America RNA Targeting Small Molecule Drug Discovery Market Outlook

  • 6.1. Market Size & Forecast
    • 6.1.1. By Value
  • 6.2. Market Share & Forecast
    • 6.2.1. By Indication
    • 6.2.2. By End Users
    • 6.2.3. By Country
  • 6.3. North America: Country Analysis
    • 6.3.1. United States RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 6.3.1.1. Market Size & Forecast
        • 6.3.1.1.1. By Value
      • 6.3.1.2. Market Share & Forecast
        • 6.3.1.2.1. By Indication
        • 6.3.1.2.2. By End Users
    • 6.3.2. Canada RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 6.3.2.1. Market Size & Forecast
        • 6.3.2.1.1. By Value
      • 6.3.2.2. Market Share & Forecast
        • 6.3.2.2.1. By Indication
        • 6.3.2.2.2. By End Users
    • 6.3.3. Mexico RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 6.3.3.1. Market Size & Forecast
        • 6.3.3.1.1. By Value
      • 6.3.3.2. Market Share & Forecast
        • 6.3.3.2.1. By Indication
        • 6.3.3.2.2. By End Users

7. Europe RNA Targeting Small Molecule Drug Discovery Market Outlook

  • 7.1. Market Size & Forecast
    • 7.1.1. By Value
  • 7.2. Market Share & Forecast
    • 7.2.1. By Indication
    • 7.2.2. By End Users
    • 7.2.3. By Country
  • 7.3. Europe: Country Analysis
    • 7.3.1. Germany RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 7.3.1.1. Market Size & Forecast
        • 7.3.1.1.1. By Value
      • 7.3.1.2. Market Share & Forecast
        • 7.3.1.2.1. By Indication
        • 7.3.1.2.2. By End Users
    • 7.3.2. France RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 7.3.2.1. Market Size & Forecast
        • 7.3.2.1.1. By Value
      • 7.3.2.2. Market Share & Forecast
        • 7.3.2.2.1. By Indication
        • 7.3.2.2.2. By End Users
    • 7.3.3. United Kingdom RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 7.3.3.1. Market Size & Forecast
        • 7.3.3.1.1. By Value
      • 7.3.3.2. Market Share & Forecast
        • 7.3.3.2.1. By Indication
        • 7.3.3.2.2. By End Users
    • 7.3.4. Italy RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 7.3.4.1. Market Size & Forecast
        • 7.3.4.1.1. By Value
      • 7.3.4.2. Market Share & Forecast
        • 7.3.4.2.1. By Indication
        • 7.3.4.2.2. By End Users
    • 7.3.5. Spain RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 7.3.5.1. Market Size & Forecast
        • 7.3.5.1.1. By Value
      • 7.3.5.2. Market Share & Forecast
        • 7.3.5.2.1. By Indication
        • 7.3.5.2.2. By End Users

8. Asia Pacific RNA Targeting Small Molecule Drug Discovery Market Outlook

  • 8.1. Market Size & Forecast
    • 8.1.1. By Value
  • 8.2. Market Share & Forecast
    • 8.2.1. By Indication
    • 8.2.2. By End Users
    • 8.2.3. By Country
  • 8.3. Asia Pacific: Country Analysis
    • 8.3.1. China RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 8.3.1.1. Market Size & Forecast
        • 8.3.1.1.1. By Value
      • 8.3.1.2. Market Share & Forecast
        • 8.3.1.2.1. By Indication
        • 8.3.1.2.2. By End Users
    • 8.3.2. India RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 8.3.2.1. Market Size & Forecast
        • 8.3.2.1.1. By Value
      • 8.3.2.2. Market Share & Forecast
        • 8.3.2.2.1. By Indication
        • 8.3.2.2.2. By End Users
    • 8.3.3. Japan RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 8.3.3.1. Market Size & Forecast
        • 8.3.3.1.1. By Value
      • 8.3.3.2. Market Share & Forecast
        • 8.3.3.2.1. By Indication
        • 8.3.3.2.2. By End Users
    • 8.3.4. South Korea RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 8.3.4.1. Market Size & Forecast
        • 8.3.4.1.1. By Value
      • 8.3.4.2. Market Share & Forecast
        • 8.3.4.2.1. By Indication
        • 8.3.4.2.2. By End Users
    • 8.3.5. Australia RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 8.3.5.1. Market Size & Forecast
        • 8.3.5.1.1. By Value
      • 8.3.5.2. Market Share & Forecast
        • 8.3.5.2.1. By Indication
        • 8.3.5.2.2. By End Users

9. Middle East & Africa RNA Targeting Small Molecule Drug Discovery Market Outlook

  • 9.1. Market Size & Forecast
    • 9.1.1. By Value
  • 9.2. Market Share & Forecast
    • 9.2.1. By Indication
    • 9.2.2. By End Users
    • 9.2.3. By Country
  • 9.3. Middle East & Africa: Country Analysis
    • 9.3.1. Saudi Arabia RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 9.3.1.1. Market Size & Forecast
        • 9.3.1.1.1. By Value
      • 9.3.1.2. Market Share & Forecast
        • 9.3.1.2.1. By Indication
        • 9.3.1.2.2. By End Users
    • 9.3.2. UAE RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 9.3.2.1. Market Size & Forecast
        • 9.3.2.1.1. By Value
      • 9.3.2.2. Market Share & Forecast
        • 9.3.2.2.1. By Indication
        • 9.3.2.2.2. By End Users
    • 9.3.3. South Africa RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 9.3.3.1. Market Size & Forecast
        • 9.3.3.1.1. By Value
      • 9.3.3.2. Market Share & Forecast
        • 9.3.3.2.1. By Indication
        • 9.3.3.2.2. By End Users

10. South America RNA Targeting Small Molecule Drug Discovery Market Outlook

  • 10.1. Market Size & Forecast
    • 10.1.1. By Value
  • 10.2. Market Share & Forecast
    • 10.2.1. By Indication
    • 10.2.2. By End Users
    • 10.2.3. By Country
  • 10.3. South America: Country Analysis
    • 10.3.1. Brazil RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 10.3.1.1. Market Size & Forecast
        • 10.3.1.1.1. By Value
      • 10.3.1.2. Market Share & Forecast
        • 10.3.1.2.1. By Indication
        • 10.3.1.2.2. By End Users
    • 10.3.2. Colombia RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 10.3.2.1. Market Size & Forecast
        • 10.3.2.1.1. By Value
      • 10.3.2.2. Market Share & Forecast
        • 10.3.2.2.1. By Indication
        • 10.3.2.2.2. By End Users
    • 10.3.3. Argentina RNA Targeting Small Molecule Drug Discovery Market Outlook
      • 10.3.3.1. Market Size & Forecast
        • 10.3.3.1.1. By Value
      • 10.3.3.2. Market Share & Forecast
        • 10.3.3.2.1. By Indication
        • 10.3.3.2.2. By End Users

11. Market Dynamics

  • 11.1. Drivers
  • 11.2. Challenges

12. Market Trends & Developments

  • 12.1. Merger & Acquisition (If Any)
  • 12.2. Product Launches (If Any)
  • 12.3. Recent Developments

13. Global RNA Targeting Small Molecule Drug Discovery Market: SWOT Analysis

14. Porter's Five Forces Analysis

  • 14.1. Competition in the Industry
  • 14.2. Potential of New Entrants
  • 14.3. Power of Suppliers
  • 14.4. Power of Customers
  • 14.5. Threat of Substitute Products

15. Competitive Landscape

  • 15.1. Accent Therapeutics, Inc.
    • 15.1.1. Business Overview
    • 15.1.2. Products & Services
    • 15.1.3. Recent Developments
    • 15.1.4. Key Personnel
    • 15.1.5. SWOT Analysis
  • 15.2. Anima Biotech Inc.
  • 15.3. Arrakis Therapeutics, Inc.
  • 15.4. AstraZeneca Plc
  • 15.5. Epics Therapeutics, Inc.
  • 15.6. Expansion Therapeutics, Inc.
  • 15.7. F. Hoffmann-La Roche AG
  • 15.8. H3 Biomedicine Inc.
  • 15.9. PTC Therapeutics, Inc.
  • 15.10. Ribometrix, Inc.

16. Strategic Recommendations

17. About Us & Disclaimer