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市場調查報告書
商品編碼
1874264
siRNA藥物:全球市場佔有率和排名、總收入和需求預測(2025-2031年)siRNA Drugs - Global Market Share and Ranking, Overall Sales and Demand Forecast 2025-2031 |
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2024 年全球 siRNA 藥物市場規模估計為 24.43 億美元,預計到 2031 年將達到 162.38 億美元,在預測期(2025-2031 年)內複合年成長率為 28.9%。
小干擾RNA(siRNA),也稱為短干擾RNA或定序RNA,是一種雙股RNA,最初被認為是非編碼RNA分子。其長度通常為20-24個鹼基對(常見為21個鹼基對),在RNA干擾(RNAi)路徑中發揮與微型RNA (miRNA)相似的作用。 siRNA在基因靜默和基因表現調控中扮演重要角色。近年來,基於siRNA的療法因其能夠靶向特定基因治療癌症、遺傳性疾病、病毒感染疾病和神經退化性疾病等疾病而備受關注。 siRNA藥物產品的研究範圍涵蓋多個方面,包括其設計、開發、測試和監管核准。
siRNA藥物的主要生產商包括Alnylam、諾華、諾和諾德、健贊和Arrowhead。從消費地區來看,美國是最大的消費市場,約佔全球市場佔有率的57%,其次是歐洲。按產品類型分類,皮下注射是最大的細分市場,市佔率超過77%。依產品應用領域分類,神經系統治療是最大的應用市場,市佔率約佔52%。
對siRNA藥物市場促進因素的深入分析
一、技術突破:從實驗室到臨床
交付系統創新
脂質奈米顆粒(LNP)技術的最佳化:早期siRNA藥物由於遞送效率低、標靶化差,應用範圍僅限於罕見疾病領域。近年來,諸如改進的可電離脂質成分和PEG修飾等技術突破,使得LNP能夠遞送至肝臟(例如Alnylam公司的Onpattro)並穿過血腦障壁(例如Denali公司的RVT-1201),顯著拓展了其適應症範圍。
GalNAc偶聯技術的廣泛應用:該技術透過與去唾液酸糖蛋白受體結合,實現高效的肝臟標靶遞送,減少給藥頻率(例如,諾華的Leqvio皮下注射每六個月一次),並促進siRNA滲透到心血管和代謝疾病等慢性疾病中。
成熟的化學改質技術
增強穩定性:2'-O-甲基修飾和硫代磷酸酯骨架等技術可將siRNA的半衰期延長至數週,並降低免疫抗原性風險。
減少脫靶效應:人工智慧輔助序列設計(例如 Sirnaomics 的人工智慧演算法)結合生物資訊篩檢可提高 siRNA 特異性,從而顯著提高臨床安全性。
2. 市場需求:未滿足的臨床需求的出現
適應症範圍從罕見疾病擴大到常見疾病。
慢性病治療領域的突破:全球約有10億人患有心血管疾病,高膽固醇症蘊藏著巨大的市場潛力。
腫瘤治療的新範式:將 siRNA 與 PD-1 抑制劑(例如 Arrowhead 的 ARO-HIF2)結合使用,在腎癌治療中顯示出協同效應,引領腫瘤免疫療法進入基因調控時代。
個人化醫療的興起:基於基因型的siRNA療法(例如,針對攜帶APOE4突變患者的阿茲海默症治療)滿足了精準醫療的需求,並提高了患者的支付意願。
3. 政策支持:完善全球法規結構
中國的政策紅利
《「十四五」醫藥產業發展計畫》明確支持新型遞送系統和核酸藥物的研發,提供稅收優惠和研發補助。
優先審查通道:中國國家藥品監督管理局(CDE)正在對被認定為突破性治療方法的創新siRNA藥物實施快速核准,例如Sirnaomics公司的STP705(用於治療鱗狀細胞皮膚癌)。
國際監管合作
FDA加速核准:Onpattro(2018年)成為首個核准監管部門批准的siRNA藥物。
EMA 適應性核准途徑:允許基於替代終點(例如生物標記變化)進行有條件核准,以縮短研發週期。
4. 資本流入:產業生態系的催化作用
活性化投資和資金籌措活動
全球資本部署:2022 年,小分子核酸藥物領域共有 33 筆資金籌措,總額約 60 億美元(例如,Sirnaomics 的 IPO 籌集了 1.5 億美元)。
大型跨國企業紛紛加入競爭:羅氏以 28 億美元收購 Dicerna,諾華和 Alnylam 以 35 億美元合作加速產品線開發。
CDMO服務生態系的成熟
銳博生物、無錫艾普泰克:提供從序列設計到 cGMP 生產的「一站式」服務,降低研發成本 50% 以上。
大規模生產取得突破:連續流合成技術已將 siRNA 的年產量從克級擴大到千噸,並將每批的成本降低到 100 美元以下。
5. 競爭格局:從技術壁壘到生態學壁壘
專利佈局改進
遞送系統核心專利:Alnylam 擁有叢集關鍵技術專利,包括 LNP 和 GalNAc,使其擁有技術壟斷地位。
化學修飾專利網路:Ionis 的 2'-MOE 修飾和 Sirnaomics 的 GalNAc-siRNA 偶聯技術形成專利護城河。
跨境合作加速
製藥和生物技術合作:例如,Astra Zeneca和 Silence Therapeutics 正在合作開發心血管 siRNA 藥物,共用研發的風險和效益。
透過產學研合作實現協同創新:哈佛大學和 Moderna 正在合作開發 RNA 編輯技術,加速基礎研究向臨床應用的轉化。
6. 挑戰與解決方案
克服技術挑戰
滲透性:透過細胞滲透性肽 (CPP) 修飾和外泌體遞送技術,克服了 siRNA 在肺部和腦部等器官中遞送的挑戰。
長期安全性評估:將建立即時定量PCR來監測脫靶效應,並結合單細胞定序技術最佳化序列設計。
成本效益平衡
分級定價策略:在低收入國家,將採用「專利聯盟」模式(如藥品價格協議)來改善藥品的可及性。
未來,siRNA藥物有望成為繼小分子藥物和抗體醫藥品之後的第三大類藥物,重塑生物製藥產業的模式。企業需要專注於核心技術的突破、差異化適應症的開發以及全球合作,以掌握市場機會。
本報告旨在按地區/國家、類型和應用對全球siRNA藥物市場進行全面分析,重點關注總銷售額、市場佔有率和主要企業的排名。
本報告以銷售收入為指標,對siRNA藥物市場規模、估算和預測進行了呈現,以2024年為基準年,並涵蓋了2020年至2031年的歷史數據和預測數據。透過定量和定性分析,我們旨在幫助讀者制定業務/成長策略,評估競爭格局,分析公司在當前市場中的地位,並就siRNA藥物做出明智的商業決策。
市場區隔
公司
按類型分類的細分市場
應用領域
按地區
The global market for siRNA Drugs was estimated to be worth US$ 2443 million in 2024 and is forecast to a readjusted size of US$ 16238 million by 2031 with a CAGR of 28.9% during the forecast period 2025-2031.
Small interfering RNA (siRNA), sometimes known as short interfering RNA or silencing RNA, is a class of double-stranded RNA at first non-coding RNA molecules, typically 20-24 (normally 21) base pairs in length, similar to miRNA, and operating within the RNA interference (RNAi) pathway. siRNA plays a crucial role in gene silencing and regulation of gene expression. siRNA-based therapeutics have gained significant attention in recent years due to their potential to target specific genes involved in diseases such as cancer, genetic disorders, viral infections, and neurodegenerative diseases. The scope of siRNA drug products encompasses various aspects, including their design, development, testing, and regulatory approval.
Core manufacturers of siRNA Drugs include Alnylam, Novartis, Novo Nordisk, Genzyme and Arrowhead. USA is the largest consumption region, accounting for approximately 57% of the global market, followed by Europe. In terms of product type, subcutaneous injection are the largest market segment, with a share of more than 77%. In terms of product application, the largest application market is nervous system therapy, with a share of approximately 52%.
In-depth analysis of siRNA drug market driving factors
I. Technological breakthroughs: the leap from laboratory to clinic
Delivery system innovation
Lipid nanoparticle (LNP) technology optimization: Early siRNA drugs were limited to the field of rare diseases due to low delivery efficiency and insufficient targeting. In recent years, through technological breakthroughs such as ionized lipid component improvement and PEG modification, LNP can achieve liver-targeted delivery (such as Alnylam's Onpattro) or cross the blood-brain barrier (such as Denali's RVT-1201), significantly broadening the scope of indications.
Popularization of GalNAc coupling technology: This technology achieves efficient liver-targeted delivery by binding to asialoglycoprotein receptors, reducing the frequency of administration (such as Novartis' Leqvio subcutaneous injection once every six months), and promoting siRNA to penetrate into chronic diseases such as cardiovascular and metabolic diseases.
Mature chemical modification technology
Improved stability: Through 2'-O-methyl modification, thiophosphate backbone and other technologies, the half-life of siRNA is extended to several weeks, reducing the risk of immunogenicity.
Reduced off-target effects: AI-assisted sequence design (such as Sirnaomics' AI algorithm) combined with bioinformatics screening improves siRNA specificity and significantly improves clinical safety.
2. Market demand: The outbreak of unmet clinical needs
Indications expand from rare diseases to common diseases
Breakthrough in chronic diseases: Among the approximately 1 billion cardiovascular disease patients worldwide, the market potential for hypercholesterolemia is huge.
New paradigm for tumor treatment: siRNA combined with PD-1 inhibitors (such as Arrowhead's ARO-HIF2) shows synergistic effects in the treatment of renal cancer, pushing tumor immunotherapy into the era of gene regulation.
The rise of personalized medicine: siRNA therapy based on genotyping (such as Alzheimer's disease drugs for patients with APOE4 mutations) meets the needs of precision treatment and increases patients' willingness to pay.
3. Policy support: Improvement of the global regulatory framework
China's policy dividends
"The 14th Five-Year Plan for the Development of the Pharmaceutical Industry": Clearly support the development of new delivery systems and nucleic acid drugs, and provide tax incentives and research and development subsidies.
Priority review channel: CDE implements fast approval for innovative siRNA drugs, such as Sirnaomics' STP705 (treatment of squamous cell skin cancer) included in the breakthrough therapy.
International regulatory coordination
FDA accelerated approval: Onpattro (2018) was approved as the first siRNA drug, turning on the regulatory green light.
EMA adaptive path: Allows conditional approval based on alternative endpoints (such as biomarker changes) to shorten the R&D cycle.
4. Capital influx: catalytic effect of industrial ecology
Increased investment and financing activity
Global capital layout: 33 financings in the field of small nucleic acid drugs in 2022, with an amount of nearly US$6 billion (such as Sirnaomics IPO raising US$150 million).
MNC giants enter the market: Roche acquires Dicerna for US$2.8 billion, and Novartis and Alnylam reach a US$3.5 billion cooperation to accelerate pipeline development.
CDMO service ecosystem matures
RiboBio, WuXi AppTec: Provide "one-stop" services from sequence design to cGMP production, reducing R&D costs by more than 50%.
Breakthrough in large-scale production: Through continuous flow synthesis technology, the annual production capacity of siRNA has been increased from grams to kilograms, and the cost per dose has been reduced to less than $100.
5. Competitive landscape: From technical barriers to ecological barriers
Strengthening of patent layout
Core patents for delivery systems: Alnylam owns key technology patent clusters such as LNP and GalNAc, forming a technological monopoly.
Chemical modification patent network: Ionis' 2'-MOE modification and Sirnaomics' GalNAc-siRNA coupling technology build a patent moat.
Cross-border cooperation is accelerating
Pharmaceutical company-biotechnology company alliance: For example, AstraZeneca and Silence Therapeutics cooperate to develop cardiovascular siRNA drugs, sharing R&D risks and benefits.
Collaborative innovation between industry, academia and research: Harvard University and Moderna cooperate to develop RNA editing technology to promote the transformation of basic research to clinical practice.
6. Challenges and coping strategies
Breakthrough in technical bottlenecks
Improved tissue penetration: Through cell penetrating peptide (CPP) modification or exosome delivery, the problem of siRNA delivery in organs such as the lungs and brain is solved.
Long-term safety assessment: Establish real-time quantitative PCR to monitor off-target effects, and optimize sequence design in combination with single-cell sequencing technology.
Balance between cost and accessibility
Tiered pricing strategy: Adopt the "patent pool" model (such as MPP) in low-income countries to improve drug accessibility.
In the future, siRNA drugs are expected to become the third largest drug type after small molecules and antibodies, reshaping the biopharmaceutical industry landscape. Companies need to focus on core technology breakthroughs, differentiated indication layout and global cooperation to seize market opportunities.
This report aims to provide a comprehensive presentation of the global market for siRNA Drugs, focusing on the total sales revenue, key companies market share and ranking, together with an analysis of siRNA Drugs by region & country, by Type, and by Application.
The siRNA Drugs market size, estimations, and forecasts are provided in terms of sales revenue ($ millions), considering 2024 as the base year, with history and forecast data for the period from 2020 to 2031. With both quantitative and qualitative analysis, to help readers develop business/growth strategies, assess the market competitive situation, analyze their position in the current marketplace, and make informed business decisions regarding siRNA Drugs.
Market Segmentation
By Company
Segment by Type
Segment by Application
By Region
Chapter Outline
Chapter 1: Introduces the report scope of the report, global total market size. This chapter also provides the market dynamics, latest developments of the market, the driving factors and restrictive factors of the market, the challenges and risks faced by manufacturers in the industry, and the analysis of relevant policies in the industry.
Chapter 2: Detailed analysis of siRNA Drugs company competitive landscape, revenue market share, latest development plan, merger, and acquisition information, etc.
Chapter 3: Provides the analysis of various market segments by Type, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different market segments.
Chapter 4: Provides the analysis of various market segments by Application, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different downstream markets.
Chapter 5: Revenue of siRNA Drugs in regional level. It provides a quantitative analysis of the market size and development potential of each region and introduces the market development, future development prospects, market space, and market size of each country in the world.
Chapter 6: Revenue of siRNA Drugs in country level. It provides sigmate data by Type, and by Application for each country/region.
Chapter 7: Provides profiles of key players, introducing the basic situation of the main companies in the market in detail, including product revenue, gross margin, product introduction, recent development, etc.
Chapter 8: Analysis of industrial chain, including the upstream and downstream of the industry.
Chapter 9: Conclusion.