Antisense Oligonucleotide (ASO) Therapeutics Market Size to Reach USD 7,864.9 Million in 2032

The Antisense Oligonucleotide (ASO) Therapeutics market, valued at USD 2,355.4 million in 2024, is expected to register robust revenue CAGR of 16.2%.

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June 02, 2025 - The increasing incidence of genetic and rare diseases is a major factor driving revenue growth in the Antisense Oligonucleotide (ASO) therapeutics market. For instance, the American Lung Association reports that cystic fibrosis affects approximately 40,000 individuals in the U.S. and around 100,000 globally. In the U.S., about one in every thirty people is a carrier of the condition. Children born with cystic fibrosis between 2019 and 2023 are expected to have an average life expectancy of 61 years, while half of those born in 2023 are projected to live to at least 68 years.

Advancements in antisense oligonucleotide (ASO) technology offer a promising approach for addressing a broad spectrum of genetic disorders. As researchers and innovators gain deeper insights into the complexities of genetic diseases, the role of ASOs in delivering targeted and effective therapies is becoming increasingly evident. Notably, recent progress in systemic delivery methods-especially improvements in exosome loading and nanoparticle-based technologies-is expanding the potential for novel therapeutic applications.

In February 2025, Biogen Inc. and Stoke Therapeutics, Inc. announced a partnership to jointly develop and commercialize zorevunersen-a potential first-in-class disease-modifying therapy for Dravet syndrome-outside of the United States, Canada, and Mexico. Zorevunersen is an investigational antisense oligonucleotide (ASO) designed to target the SCN1A gene, which is responsible for most cases of Dravet syndrome.

Antisense oligonucleotides (ASOs) hold significant promise as disease-modifying therapies for monogenic disorders. However, their broader application is currently constrained by challenges such as limited delivery methods to target tissues and an incomplete understanding of the precise levels of protein overexpression or suppression needed to correct disease phenotypes. While ASOs typically exhibit low immunogenicity, their interaction with the immune system can vary depending on factors like their sequence, chemical modifications, and delivery method.

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Segments market overview and growth Insights:
Based on the technology, the Antisense Oligonucleotide (ASO) Therapeutics market is segmented into First-generation ASOs, Second-generation ASOs, Third-generation ASOs, and Conjugated ASOs. The First-generation ASOs segment held the largest market share in 2024. First-generation chemically modified antisense oligonucleotides (ASOs) incorporate various chemical groups such as methyl phosphonates (MP), phosphorothioates (PS), phosphoramidates, methylene (methylimino) (MMI), 5′N carbamate, triazole, amide, thioester, thioformacetal, mercapto acetamide, boranophosphate, 5′ methylurea, and guanidinium. These modifications affect the overall charge of the phosphodiester backbone, classifying them as neutral, cationic, or anionic.

Regional market overview and growth insights:
North America held the largest market share in the Antisense Oligonucleotide (ASO) Therapeutics market in 2024, driven by the increasing prevalence of genetic and rare disorders, along with significant progress in nucleic acid chemistry and delivery technologies, the field continues to advance rapidly. According to the National Center for Biotechnology Information (NCBI), Duchenne muscular dystrophy (DMD) is an X-linked recessive condition that primarily affects males. It is estimated to occur in approximately one out of every 3,600 live-born male infants. Some research indicates that DMD affects about 2 in every 10,000 individuals in the United States, making it one of the most common and severe forms of congenital myopathy.

In January 2025, Alloy Therapeutics Inc., a biotechnology ecosystem company focused on broadening access to advanced drug discovery technologies-announced a target-specific collaboration and licensing agreement with Sanofi. The partnership will utilize Alloy's innovative AntiClastic Antisense Platform to address a central nervous system (CNS) target. This proprietary technology empowers drug developers to fully leverage antisense therapies by enabling precise targeting of intracellular disease mechanisms at the RNA level.

Competitive Landscape and Key Competitors:
The Antisense Oligonucleotide (ASO) Therapeutics market is characterized by a fragmented structure, with many competitors holding a significant share of the market. List of major players included in the Antisense Oligonucleotide (ASO) Therapeutics market report are:

o Ionis Pharmaceuticals
o Isarna Therapeutics
o Regulus Therapeutics
o Sarepta Therapeutics
o Secarna Pharmaceuticals
o Wave Life Sciences
o Alloy Therapeutics
o Insud Pharma
o Bolden Therapeutics, Inc.
o Merck & Co., Inc.
o Biogen Inc.
o Arrowhead Pharmaceuticals
o F. Hoffmann-La Roche
o Nippon Shinyaku
o Alnylam Pharmaceuticals
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Major strategic developments by leading competitors:
Secarna Pharmaceuticals GmbH & Co. KG: In May 2024, Secarna Pharmaceuticals GmbH & Co. KG, a leading independent European antisense drug discovery and development company, and Orbit Discovery Ltd. (Orbit), a leader in the discovery of therapeutic peptide hits, today announced a collaboration to discover and develop peptide-conjugated targeted antisense oligonucleotide (ASO) therapeutics. The cooperation will use Orbit's expertise and a bead-based peptide display engine to identify, screen, and select cyclic peptides specific to a variety of disease targets, which will then be combined with Secarna ASO molecules.

Alloy Therapeutics: In June 2023, Alloy Therapeutics, a biotechnology ecosystem firm, has announced the debut of their revolutionary AntiClastic Antisense Oligonucleotide platform, which uses optimally constructed spatial conformation nucleic acids to increase the drug-like effects of antisense. Sudhir Agrawal's Arnay Sciences LLC has granted Alloy exclusive license to the intellectual property underlying the AntiClastic ASO platform.

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Navistrat Analytics has segmented Antisense Oligonucleotide (ASO) Therapeutics market based on type of nucleotide, technology, route of administration, development phase, and indication:
• Type of Nucleotide Outlook (Revenue, USD Million; 2022-2032)
o DNA-based ASOs
o RNA-based ASOs
o Modified/Hybrid ASOs
• Technology Outlook (Revenue, USD Million; 2022-2032)
o First-generation ASOs
o Second-generation ASOs
o Third-generation ASOs
o Conjugated ASOs
• Route of Administration Outlook (Revenue, USD Million; 2022-2032)
o Intrathecal
o Intravenous (IV)
o Subcutaneous
o Intraocular
• Development Phase Outlook (Revenue, USD Million; 2022-2032)
o Preclinical
o Clinical
o Commercial
• Indication Outlook (Revenue, USD Million; 2022-2032)
o Genetic Disorders
o Neurological Disorders
o Oncology
o Neurological Disorders
o Infectious Diseases
o Cardiovascular Diseases
o Others
• Regional Outlook (Revenue, USD Million; 2022-2032)
o North America
a. U.S.
b. Canada
c. Mexico
o Europe
a. Germany
b. France
c. U.K.
d. Italy
e. Spain
f. Benelux
g. Nordic Countries
h. Rest of Europe
o Asia Pacific
a. China
b. India
c. Japan
d. South Korea
e. Oceania
f. ASEAN Countries
g. Rest of APAC
o Latin America
a. Brazil
b. Rest of LATAM
o Middle East & Africa
a. GCC Countries
b. South Africa
c. Israel
d. Turkey
e. Rest of MEA

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