Global Antisense oligonucleotides (ASO) Drug and siRNA Drug Market Growth (Status and Outlook) 2025-2031
Description
According to this study, the global Antisense oligonucleotides (ASO) Drug and siRNA Drug market size will reach US$ 11559 million by 2031.
Small nucleic acid drugs are short in length and have a base of less than 30 nt. They mainly act on mRNA in cells through the principle of complementary base pairing to regulate protein expression, thereby achieving therapeutic effects. It can be further divided into single-stranded antisense oligonucleotide ASO, and double-stranded nucleotide siRNA.
The main market drivers of antisense oligonucleotide (ASO) drugs and siRNA drugs include the following:
1. Technological breakthrough: the leap from laboratory to clinic
Technological evolution of ASO drugs
Chemical modification and delivery system optimization: The first generation of ASO drugs were limited by poor stability and high immunogenicity. The third generation of ASO significantly improved the resistance to nucleases and pharmacokinetic properties through thiophosphate backbone modification, glycosylation modification and other technologies. For example, Ionis' Eplontersen uses ligand coupling technology to achieve subcutaneous administration once a month, which greatly improves patient compliance.
Indication expansion: ASO drugs extend from genetic diseases (such as Duchenne muscular dystrophy) to tumors and metabolic diseases. Geron's Imetelstat was approved as the first telomerase inhibitor, providing a new treatment for myelodysplastic syndrome.
Technological revolution of siRNA drugs
Delivery system breakthrough: In 2014, the GalNAc ligand technology came into being, solving the problem of liver targeting and promoting Alnylam's Onpattro (approved in 2018) to become the first siRNA drug. Current research focuses on multi-organ delivery, such as Alnylam's mivelsiran (Phase II for cerebral amyloid angiopathy) and Arrowhead's ARO-RAGE (Phase I for asthma), which achieve cross-blood-brain barrier targeting through lipid nanoparticles (LNP) or antibody coupling.
Advantages of long-term efficacy and reversibility: siRNA drugs such as Inclisiran (lipid-lowering) can be administered once every six months, and the effect is reversible, avoiding the permanent risk of gene editing.
2. Policy support: Catalyst for the global innovative drug ecosystem
Regulatory approval acceleration
The FDA accelerates the approval of small nucleic acid drugs through mechanisms such as "fast track" and "breakthrough therapy". For example, Eplontersen took only 3 years from clinical to approval, which is far lower than the 10-year cycle of traditional drugs.
China's "14th Five-Year Plan for the Development of the Pharmaceutical Industry" clearly supports the development of new delivery systems, optimizes the review process, and promotes ASO/siRNA drugs into clinical trials.
Medical insurance coverage and payment innovation
European and American medical insurance explores the "pay-by-efficacy" model for high-priced small nucleic acid drugs (such as Onpattro, which costs about $450,000 per year) to reduce the burden on patients.
China is incorporating drugs for rare diseases into medical insurance negotiations. The ASO drug Spinraza (nusinersen) has been negotiated to reduce its price, and its accessibility has been improved.
3. Market demand: a blue ocean with unmet clinical needs
Genetic diseases and rare diseases
ASO drugs dominate the field of spinal muscular atrophy (SMA), with global sales of Spinraza exceeding US$2 billion. siRNA drugs such as Amvuttra (transthyretin amyloidosis) fill the gap in the treatment of genetic diseases.
There are more than 300 million patients with rare diseases worldwide, but approved drugs only cover 5%, and the market potential is huge.
Expansion of chronic and metabolic diseases
The siRNA drug Inclisiran has expanded its indications from familial hypercholesterolemia to atherosclerotic cardiovascular disease, and the patient population has expanded 10 times.
In the field of metabolic-associated fatty hepatitis (MASH), Alnylam's Lundquist and other siRNA drugs have entered Phase III clinical trials, and the market size is expected to exceed US$35 billion in 2030.
IV. Capital investment: the "fuel" for innovative drug research and development
Venture capital and IPO are active
In 2023, there will be 31 transactions in the global small nucleic acid field, with a total amount of US$8.496 billion (up 371% year-on-year). China Jingyin Pharmaceutical completed nearly US$50 million in B2 round financing to accelerate the development of cardiovascular siRNA pipeline.
In 2024, Boehringer Ingelheim and Ribo Bio reached a US$2 billion cooperation to develop siRNA drugs for MASH.
The layout of multinational pharmaceutical companies is accelerating
AstraZeneca introduced Ionis's Eplontersen for US$3.585 billion to expand the field of heart failure; Novartis consolidated the cardiovascular market through Leqvio (lipid-lowering siRNA).
In 2025, GSK acquired Boston Pharmaceuticals' FGF21 siRNA project for US$1.2 billion, betting on metabolic diseases.
5. Competition landscape: Evolution from "unipolar" to "multipolar"
Monopoly and breakthroughs of international giants
Ionis (ASO) and Alnylam (siRNA) occupy the main global market share, but newcomers such as Arrowhead and Dicerna challenge the leading position through differentiated technologies (such as multi-organ delivery).
Eli Lilly cooperates with Alnylam to develop siRNA drugs for Alzheimer's disease, using its delivery technology to break through the blood-brain barrier.
The rise of Chinese companies
Domestic ASO field: Ruibo Bio and Yuekang Pharmaceuticals advance clinical pipelines through cooperative development, but no self-developed products have been launched.
siRNA field: Many drugs of Shengyin Bio and Bowang Pharmaceutical have entered Phase I, targeting common diseases such as hypertension and hyperlipidemia, filling the domestic gap.
6. Challenges and future trends
Technical bottlenecks to be broken through
The off-target effects and immunogenicity of ASO drugs still need to be optimized; the kidney and lung delivery efficiency of siRNA is low, which limits the expansion of indications.
Competitive pressure from gene editing technologies (such as CRISPR): Verve Therapeutics' PCSK9 gene editing therapy has entered clinical trials and may replace part of the siRNA market.
Driven by policy support and capital, the growth rate of the Chinese market will be higher than the global average, and ASO/siRNA drugs are expected to become the core track of the "new infrastructure" of biomedicine.
The market explosion of ASO and siRNA drugs is the result of the resonance of technological breakthroughs, policy dividends, capital catalysis and unmet needs. In the future, with the innovation of delivery systems and the expansion of multiple indications, small nucleic acid drugs will move from "niche genetic diseases" to "mass chronic diseases", reshaping the global pharmaceutical landscape.
LPI (LP Information)' newest research report, the “Antisense oligonucleotides (ASO) Drug and siRNA Drug Industry Forecast” looks at past sales and reviews total world Antisense oligonucleotides (ASO) Drug and siRNA Drug sales in 2024, providing a comprehensive analysis by region and market sector of projected Antisense oligonucleotides (ASO) Drug and siRNA Drug sales for 2025 through 2031. With Antisense oligonucleotides (ASO) Drug and siRNA Drug sales broken down by region, market sector and sub-sector, this report provides a detailed analysis in US$ millions of the world Antisense oligonucleotides (ASO) Drug and siRNA Drug industry.
This Insight Report provides a comprehensive analysis of the global Antisense oligonucleotides (ASO) Drug and siRNA Drug landscape and highlights key trends related to product segmentation, company formation, revenue, and market share, latest development, and M&A activity. This report also analyses the strategies of leading global companies with a focus on Antisense oligonucleotides (ASO) Drug and siRNA Drug portfolios and capabilities, market entry strategies, market positions, and geographic footprints, to better understand these firms’ unique position in an accelerating global Antisense oligonucleotides (ASO) Drug and siRNA Drug market.
This Insight Report evaluates the key market trends, drivers, and affecting factors shaping the global outlook for Antisense oligonucleotides (ASO) Drug and siRNA Drug and breaks down the forecast by Type, by Application, geography, and market size to highlight emerging pockets of opportunity. With a transparent methodology based on hundreds of bottom-up qualitative and quantitative market inputs, this study forecast offers a highly nuanced view of the current state and future trajectory in the global Antisense oligonucleotides (ASO) Drug and siRNA Drug.
This report presents a comprehensive overview, market shares, and growth opportunities of Antisense oligonucleotides (ASO) Drug and siRNA Drug market by product type, application, key players and key regions and countries.
Segmentation by Type:
Antisense Oligonucleotides (ASO) Drug
siRNA Drug
Segmentation by Application:
Single Gene Rare Disease
Protein Deposition Disease
Chronic Liver Disease
Others
This report also splits the market by region:
Americas
United States
Canada
Mexico
Brazil
APAC
China
Japan
Korea
Southeast Asia
India
Australia
Europe
Germany
France
UK
Italy
Russia
Middle East & Africa
Egypt
South Africa
Israel
Turkey
GCC Countries
The below companies that are profiled have been selected based on inputs gathered from primary experts and analyzing the company's coverage, product portfolio, its market penetration.
Alnylam
Ionis
Biogen
Novartis
Sarepta Therapeutics
Therapeutics
Akcea
WAVElife
ProQR
Exicure
Secarna
AstraZeneca
The WhiteOak Group, Inc.
Dicerna
Silence
Arrowhead
Miragen Therapeutics
Genzyme
Quark Pharmaceuticals
Sylentis
Avidity Biosciences
Novo Nordisk
Sirnaomics
Please note: The report will take approximately 2 business days to prepare and deliver.
Small nucleic acid drugs are short in length and have a base of less than 30 nt. They mainly act on mRNA in cells through the principle of complementary base pairing to regulate protein expression, thereby achieving therapeutic effects. It can be further divided into single-stranded antisense oligonucleotide ASO, and double-stranded nucleotide siRNA.
The main market drivers of antisense oligonucleotide (ASO) drugs and siRNA drugs include the following:
1. Technological breakthrough: the leap from laboratory to clinic
Technological evolution of ASO drugs
Chemical modification and delivery system optimization: The first generation of ASO drugs were limited by poor stability and high immunogenicity. The third generation of ASO significantly improved the resistance to nucleases and pharmacokinetic properties through thiophosphate backbone modification, glycosylation modification and other technologies. For example, Ionis' Eplontersen uses ligand coupling technology to achieve subcutaneous administration once a month, which greatly improves patient compliance.
Indication expansion: ASO drugs extend from genetic diseases (such as Duchenne muscular dystrophy) to tumors and metabolic diseases. Geron's Imetelstat was approved as the first telomerase inhibitor, providing a new treatment for myelodysplastic syndrome.
Technological revolution of siRNA drugs
Delivery system breakthrough: In 2014, the GalNAc ligand technology came into being, solving the problem of liver targeting and promoting Alnylam's Onpattro (approved in 2018) to become the first siRNA drug. Current research focuses on multi-organ delivery, such as Alnylam's mivelsiran (Phase II for cerebral amyloid angiopathy) and Arrowhead's ARO-RAGE (Phase I for asthma), which achieve cross-blood-brain barrier targeting through lipid nanoparticles (LNP) or antibody coupling.
Advantages of long-term efficacy and reversibility: siRNA drugs such as Inclisiran (lipid-lowering) can be administered once every six months, and the effect is reversible, avoiding the permanent risk of gene editing.
2. Policy support: Catalyst for the global innovative drug ecosystem
Regulatory approval acceleration
The FDA accelerates the approval of small nucleic acid drugs through mechanisms such as "fast track" and "breakthrough therapy". For example, Eplontersen took only 3 years from clinical to approval, which is far lower than the 10-year cycle of traditional drugs.
China's "14th Five-Year Plan for the Development of the Pharmaceutical Industry" clearly supports the development of new delivery systems, optimizes the review process, and promotes ASO/siRNA drugs into clinical trials.
Medical insurance coverage and payment innovation
European and American medical insurance explores the "pay-by-efficacy" model for high-priced small nucleic acid drugs (such as Onpattro, which costs about $450,000 per year) to reduce the burden on patients.
China is incorporating drugs for rare diseases into medical insurance negotiations. The ASO drug Spinraza (nusinersen) has been negotiated to reduce its price, and its accessibility has been improved.
3. Market demand: a blue ocean with unmet clinical needs
Genetic diseases and rare diseases
ASO drugs dominate the field of spinal muscular atrophy (SMA), with global sales of Spinraza exceeding US$2 billion. siRNA drugs such as Amvuttra (transthyretin amyloidosis) fill the gap in the treatment of genetic diseases.
There are more than 300 million patients with rare diseases worldwide, but approved drugs only cover 5%, and the market potential is huge.
Expansion of chronic and metabolic diseases
The siRNA drug Inclisiran has expanded its indications from familial hypercholesterolemia to atherosclerotic cardiovascular disease, and the patient population has expanded 10 times.
In the field of metabolic-associated fatty hepatitis (MASH), Alnylam's Lundquist and other siRNA drugs have entered Phase III clinical trials, and the market size is expected to exceed US$35 billion in 2030.
IV. Capital investment: the "fuel" for innovative drug research and development
Venture capital and IPO are active
In 2023, there will be 31 transactions in the global small nucleic acid field, with a total amount of US$8.496 billion (up 371% year-on-year). China Jingyin Pharmaceutical completed nearly US$50 million in B2 round financing to accelerate the development of cardiovascular siRNA pipeline.
In 2024, Boehringer Ingelheim and Ribo Bio reached a US$2 billion cooperation to develop siRNA drugs for MASH.
The layout of multinational pharmaceutical companies is accelerating
AstraZeneca introduced Ionis's Eplontersen for US$3.585 billion to expand the field of heart failure; Novartis consolidated the cardiovascular market through Leqvio (lipid-lowering siRNA).
In 2025, GSK acquired Boston Pharmaceuticals' FGF21 siRNA project for US$1.2 billion, betting on metabolic diseases.
5. Competition landscape: Evolution from "unipolar" to "multipolar"
Monopoly and breakthroughs of international giants
Ionis (ASO) and Alnylam (siRNA) occupy the main global market share, but newcomers such as Arrowhead and Dicerna challenge the leading position through differentiated technologies (such as multi-organ delivery).
Eli Lilly cooperates with Alnylam to develop siRNA drugs for Alzheimer's disease, using its delivery technology to break through the blood-brain barrier.
The rise of Chinese companies
Domestic ASO field: Ruibo Bio and Yuekang Pharmaceuticals advance clinical pipelines through cooperative development, but no self-developed products have been launched.
siRNA field: Many drugs of Shengyin Bio and Bowang Pharmaceutical have entered Phase I, targeting common diseases such as hypertension and hyperlipidemia, filling the domestic gap.
6. Challenges and future trends
Technical bottlenecks to be broken through
The off-target effects and immunogenicity of ASO drugs still need to be optimized; the kidney and lung delivery efficiency of siRNA is low, which limits the expansion of indications.
Competitive pressure from gene editing technologies (such as CRISPR): Verve Therapeutics' PCSK9 gene editing therapy has entered clinical trials and may replace part of the siRNA market.
Driven by policy support and capital, the growth rate of the Chinese market will be higher than the global average, and ASO/siRNA drugs are expected to become the core track of the "new infrastructure" of biomedicine.
The market explosion of ASO and siRNA drugs is the result of the resonance of technological breakthroughs, policy dividends, capital catalysis and unmet needs. In the future, with the innovation of delivery systems and the expansion of multiple indications, small nucleic acid drugs will move from "niche genetic diseases" to "mass chronic diseases", reshaping the global pharmaceutical landscape.
LPI (LP Information)' newest research report, the “Antisense oligonucleotides (ASO) Drug and siRNA Drug Industry Forecast” looks at past sales and reviews total world Antisense oligonucleotides (ASO) Drug and siRNA Drug sales in 2024, providing a comprehensive analysis by region and market sector of projected Antisense oligonucleotides (ASO) Drug and siRNA Drug sales for 2025 through 2031. With Antisense oligonucleotides (ASO) Drug and siRNA Drug sales broken down by region, market sector and sub-sector, this report provides a detailed analysis in US$ millions of the world Antisense oligonucleotides (ASO) Drug and siRNA Drug industry.
This Insight Report provides a comprehensive analysis of the global Antisense oligonucleotides (ASO) Drug and siRNA Drug landscape and highlights key trends related to product segmentation, company formation, revenue, and market share, latest development, and M&A activity. This report also analyses the strategies of leading global companies with a focus on Antisense oligonucleotides (ASO) Drug and siRNA Drug portfolios and capabilities, market entry strategies, market positions, and geographic footprints, to better understand these firms’ unique position in an accelerating global Antisense oligonucleotides (ASO) Drug and siRNA Drug market.
This Insight Report evaluates the key market trends, drivers, and affecting factors shaping the global outlook for Antisense oligonucleotides (ASO) Drug and siRNA Drug and breaks down the forecast by Type, by Application, geography, and market size to highlight emerging pockets of opportunity. With a transparent methodology based on hundreds of bottom-up qualitative and quantitative market inputs, this study forecast offers a highly nuanced view of the current state and future trajectory in the global Antisense oligonucleotides (ASO) Drug and siRNA Drug.
This report presents a comprehensive overview, market shares, and growth opportunities of Antisense oligonucleotides (ASO) Drug and siRNA Drug market by product type, application, key players and key regions and countries.
Segmentation by Type:
Antisense Oligonucleotides (ASO) Drug
siRNA Drug
Segmentation by Application:
Single Gene Rare Disease
Protein Deposition Disease
Chronic Liver Disease
Others
This report also splits the market by region:
Americas
United States
Canada
Mexico
Brazil
APAC
China
Japan
Korea
Southeast Asia
India
Australia
Europe
Germany
France
UK
Italy
Russia
Middle East & Africa
Egypt
South Africa
Israel
Turkey
GCC Countries
The below companies that are profiled have been selected based on inputs gathered from primary experts and analyzing the company's coverage, product portfolio, its market penetration.
Alnylam
Ionis
Biogen
Novartis
Sarepta Therapeutics
Therapeutics
Akcea
WAVElife
ProQR
Exicure
Secarna
AstraZeneca
The WhiteOak Group, Inc.
Dicerna
Silence
Arrowhead
Miragen Therapeutics
Genzyme
Quark Pharmaceuticals
Sylentis
Avidity Biosciences
Novo Nordisk
Sirnaomics
Please note: The report will take approximately 2 business days to prepare and deliver.
Table of Contents
148 Pages
- *This is a tentative TOC and the final deliverable is subject to change.*
- 1 Scope of the Report
- 2 Executive Summary
- 3 Antisense oligonucleotides (ASO) Drug and siRNA Drug Market Size by Player
- 4 Antisense oligonucleotides (ASO) Drug and siRNA Drug by Region
- 5 Americas
- 6 APAC
- 7 Europe
- 8 Middle East & Africa
- 9 Market Drivers, Challenges and Trends
- 10 Global Antisense oligonucleotides (ASO) Drug and siRNA Drug Market Forecast
- 11 Key Players Analysis
- 12 Research Findings and Conclusion
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