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市场调查报告书
商品编码
1874264
siRNA药物:全球市场份额和排名、总收入和需求预测(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 药物市场规模估计为 24.43 亿美元,预计到 2031 年将达到 162.38 亿美元,在预测期(2025-2031 年)内复合年增长率为 28.9%。
小干扰RNA(siRNA),也称为短干扰RNA或定序RNA,是一种双股RNA,最初被认为是非编码RNA分子。其长度通常为20-24个碱基对(常见为21个碱基对),在RNA干扰(RNAi)路径中发挥与微型RNA (miRNA)相似的作用。 siRNA在基因静默和基因表现调控中扮演重要角色。近年来,基于siRNA的疗法因其能够靶向特定基因治疗癌症、遗传性疾病、病毒感染疾病和神经退化性疾病等疾病而备受关注。 siRNA药物产品的研究范围涵盖多个方面,包括其设计、开发、测试和监管核准。
siRNA药物的主要生产商包括Alnylam、诺华、诺和诺德、健赞和Arrowhead。从消费地区来看,美国是最大的消费市场,约占全球市场份额的57%,其次是欧洲。按产品类型划分,皮下注射是最大的细分市场,市占率超过77%。依产品应用领域划分,神经系统治疗是最大的应用市场,市占率约占52%。
对siRNA药物市场驱动因素的深入分析
一、技术突破:从实验室到临床
交付系统创新
脂质奈米颗粒(LNP)技术的最佳化:早期siRNA药物由于递送效率低、标靶化差,应用范围仅限于罕见疾病领域。近年来,诸如改进的可电离脂质成分和PEG修饰等技术突破,使得LNP能够递送至肝臟(例如Alnylam公司的Onpattro)并穿过血脑障壁(例如Denali公司的RVT-1201),显着拓展了其适应症范围。
GalNAc偶联技术的广泛应用:该技术透过与去唾液酸糖蛋白受体结合,实现高效的肝臟标靶递送,减少给药频率(例如,诺华的Leqvio皮下注射每六个月一次),并促进siRNA渗透到心血管和代谢疾病等慢性疾病中。
成熟的化学改质技术
增强稳定性:2'-O-甲基修饰和硫代磷酸酯骨架等技术可将siRNA的半衰期延长至数週,并降低免疫抗原性风险。
减少脱靶效应:人工智慧辅助序列设计(例如 Sirnaomics 的人工智慧演算法)结合生物资讯筛检可提高 siRNA 特异性,从而显着提高临床安全性。
2. 市场需求:未满足的临床需求的出现
适应症范围从罕见疾病扩大到常见疾病。
慢性病治疗领域的突破:全球约有10亿人患有心血管疾病,高胆固醇症蕴藏着巨大的市场潜力。
肿瘤治疗的新范式:将 siRNA 与 PD-1 抑制剂(例如 Arrowhead 的 ARO-HIF2)结合使用,在肾癌治疗中显示出协同效应,引领肿瘤免疫疗法进入基因调控时代。
个人化医疗的兴起:基于基因型的siRNA疗法(例如,针对携带APOE4突变患者的阿兹海默症治疗)满足了精准医疗的需求,并提高了患者的支付意愿。
3. 政策支持:完善全球法规结构
中国的政策红利
《「十四五」医药产业发展计画》明确支持新型递送系统和核酸药物的研发,提供税收优惠和研发补助。
优先审查通道:中国国家药品监督管理局(CDE)正在对被认定为突破性治疗方法的创新siRNA药物实施快速核准,例如Sirnaomics公司的STP705(用于治疗鳞状细胞皮肤癌)。
国际监管合作
FDA加速核准:Onpattro(2018年)成为首个核准监管部门批准的siRNA药物。
EMA 适应性核准途径:允许基于替代终点(例如生物标记变化)进行有条件核准,以缩短研发週期。
4. 资本流入:产业生态系的催化作用
活性化投资和资金筹措活动
全球资本部署:2022 年,小分子核酸药物领域共有 33 笔资金筹措,总额约 60 亿美元(例如,Sirnaomics 的 IPO 筹集了 1.5 亿美元)。
大型跨国企业纷纷加入竞争:罗氏以 28 亿美元收购 Dicerna,诺华和 Alnylam 以 35 亿美元合作加速产品线开发。
CDMO服务生态系的成熟
锐博生物、无锡艾普泰克:提供从序列设计到 cGMP 生产的「一站式」服务,降低研发成本 50% 以上。
大规模生产取得突破:连续流合成技术已将 siRNA 的年产量从克级扩大到公斤级,并将每批的成本降低到 100 美元以下。
5. 竞争格局:从技术壁垒到生态学壁垒
专利布局改进
递送系统核心专利:Alnylam 拥有丛集关键技术专利,包括 LNP 和 GalNAc,使其拥有技术垄断地位。
化学修饰专利网络:Ionis 的 2'-MOE 修饰和 Sirnaomics 的 GalNAc-siRNA 偶联技术形成专利护城河。
跨境合作加速
製药和生物技术合作:例如,Astra Zeneca和 Silence Therapeutics 正在合作开发心血管 siRNA 药物,共用研发的风险和效益。
透过产学研合作实现协同创新:哈佛大学和 Moderna 正在合作开发 RNA 编辑技术,加速基础研究向临床应用的转化。
6. 挑战与解决方案
克服技术挑战
渗透性:透过细胞渗透性肽 (CPP) 修饰和外泌体递送技术,克服了 siRNA 在肺部和脑部等器官中递送的挑战。
长期安全性评估:将建立即时定量PCR来监测脱靶效应,并结合单细胞定序技术优化序列设计。
成本效益平衡
分级定价策略:在低收入国家,将采用「专利联盟」模式(如药品价格协议)来改善药品的可及性。
未来,siRNA药物有望成为继小分子药物和抗体医药品之后的第三大类药物,重塑生物製药产业的模式。企业需要专注于核心技术的突破、差异化适应症的开发以及全球合作,以掌握市场机会。
本报告旨在按地区/国家、类型和应用对全球siRNA药物市场进行全面分析,重点关注总销售额、市场份额和主要企业的排名。
本报告以销售收入为指标,对siRNA药物市场规模、估算和预测进行了呈现,以2024年为基准年,并涵盖了2020年至2031年的历史数据和预测数据。透过定量和定性分析,我们旨在帮助读者制定业务/成长策略,评估竞争格局,分析公司在当前市场中的地位,并就siRNA药物做出明智的商业决策。
市场区隔
公司
按类型分類的细分市场
应用领域
按地区
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.