DelveInsight’s, “Antisense Oligonucleotide Therapeutics Pipeline Insight 2025,” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Antisense Oligonucleotide Therapeutics pipeline landscape. It covers the Antisense Oligonucleotide Therapeutics pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Antisense Oligonucleotide Therapeutics Pipeline Report

• In June 2025, GlaxoSmithKline announced a study will assess the safety, efficacy and immune response following the sequential treatment of GlaxoSmithKline’s (GSK) ASO compound (GSK3228836) and CHB-TI (GSK3528869A) in participants 18 to 65 years stable on NA treatment for CHB. The aim is to quantify the efficacy of sequential therapy as well as to determine an added value of sequential therapy over GSK3228836 therapy in CHB patients treated with NAs. In addition, the study will assess the effect of different treatment durations of GSK3228836 (12 or 24 weeks) prior to initiating GSK3528869A treatment.
• In June 2025, Laboratoires Thea conducted a study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290.
• DelveInsight’s Antisense Oligonucleotide Therapeutics pipeline report depicts a robust space with 15+ active players working to develop 15+ pipeline therapies for Antisense Oligonucleotide Therapeutics treatment.
• The leading Antisense Oligonucleotide Therapeutics Companies such as Ionis Pharmaceuticals, Secarna Pharmaceuticals, Aro Biotherapeutics, NeuBase Therapeutics, Bio-Path Holdings, Inc., Scopus Biopharma, Dyne Therapeutics, CAMP4 Therapeutics, Pulmotect, GeneTx Biotherapeutics, Aligos Therapeutics, WaVe Life Sciences and others.
• Promising Antisense Oligonucleotide Therapeutics Pipeline Therapies such as Bepirovirsen, QR-421a, SB012, Sepofarsen, QR-110, PGN-EDO51, GSK3228836 and others.

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Antisense Oligonucleotide Therapeutics Emerging Drugs Profile

• Cobitolimod: InDex Pharmaceuticals

Cobitolimod is part of InDex Pharmaceuticals’ DNA-based ImmunoModulatory Sequence (DIMS) platform. It is administered locally in the large intestine, where the substance binds to Toll-like receptor 9 (TLR9) present inside immune cells as well as on the surface of epithelial cells. The binding triggers the cells to product anti-inflammatory cytokines, agents that play an important role in reducing the inflammation and healing the wounds in the large intestine caused by UC. It is currently being evaluated in Phase III stage of development of Ulcerative colitis.

• Prexigebersen: Bio-Path Holdings

Prexigebersen (previously BP 100-1-01) is a neutral-charge, liposomal-delivered antisense cancer drug. It is designed to inhibit protein synthesis of Grb2 (growth factor receptor bound protein 2). Bio-Path is currently evaluating prexigebersen in Phase II program for the treatment of acute myeloid leukemia (AML). Prexigebersen has received orphan drug designation from the FDA and from the European Medicines Agency for AML.

The Antisense Oligonucleotide Therapeutics Pipeline Report provides insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Antisense Oligonucleotide Therapeutics with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Antisense Oligonucleotide Therapeutics Treatment.
• Antisense Oligonucleotide Therapeutics Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Antisense Oligonucleotide Therapeutics Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Antisense Oligonucleotide Therapeutics market.

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Antisense Oligonucleotide Therapeutics Companies

Ionis Pharmaceuticals, Secarna Pharmaceuticals, Aro Biotherapeutics, NeuBase Therapeutics, Bio-Path Holdings, Inc., Scopus Biopharma, Dyne Therapeutics, CAMP4 Therapeutics, Pulmotect, GeneTx Biotherapeutics, Aligos Therapeutics, WaVe Life Sciences and others.

Antisense Oligonucleotide Therapeutics pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical.
• Molecule Type

Antisense Oligonucleotide Therapeutics Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy
• Product Type

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Scope of the Antisense Oligonucleotide Therapeutics Pipeline Report

• Coverage- Global
• Antisense Oligonucleotide Therapeutics Companies- Ionis Pharmaceuticals, Secarna Pharmaceuticals, Aro Biotherapeutics, NeuBase Therapeutics, Bio-Path Holdings, Inc., Scopus Biopharma, Dyne Therapeutics, CAMP4 Therapeutics, Pulmotect, GeneTx Biotherapeutics, Aligos Therapeutics, WaVe Life Sciences and others.
• Antisense Oligonucleotide Therapeutics Pipeline Therapies- Bepirovirsen, QR-421a, SB012, Sepofarsen, QR-110, PGN-EDO51, GSK3228836 and others.
• Antisense Oligonucleotide Therapeutics Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Antisense Oligonucleotide Therapeutics Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Contents

1. Introduction
2. Executive Summary
3. Antisense Oligonucleotide Therapeutics: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Late Stage Products (Phase III)
7. Cobitolimod: InDex Pharmaceuticals
8. Mid Stage Products (Phase II)
9. Prexigebersen: Bio-Path Holdings
10. Early stage products (Phase I/II)
11. ALG-020572: Aligos Therapeutics
12. Inactive Products
13. Antisense Oligonucleotide Therapeutics Key Companies
14. Antisense Oligonucleotide Therapeutics Key Products
15. Antisense Oligonucleotide Therapeutics- Unmet Needs
16. Antisense Oligonucleotide Therapeutics- Market Drivers and Barriers
17. Antisense Oligonucleotide Therapeutics- Future Perspectives and Conclusion
18. Antisense Oligonucleotide Therapeutics Analyst Views
19. Antisense Oligonucleotide Therapeutics Key Companies
20. Appendix

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