(Albany, US), September 21, 2026 – DelveInsight’s, “Antisense Oligonucleotide Therapeutics Pipeline Insights 2026” report provides comprehensive insights about 150+ companies and 200+ pipeline drugs in the Antisense Oligonucleotide Therapeutics pipeline landscape. It covers the Antisense Oligonucleotide Therapeutics pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Antisense Oligonucleotide Therapeutics pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Discover the latest drugs and treatment options in the Antisense Oligonucleotide Therapeutics Pipeline @ https://www.delveinsight.com/sample-request/antisense-oligonucleotide-therapeutics-pipeline-insight

Key Takeaways from the Antisense Oligonucleotide Therapeutics Pipeline Report

• In August 2026- Vanda Pharmaceuticals initiated a study to evaluate the safety of therapy with VCA-894A, an ASO designed to rescue and restore the activity of IGHMBP2, when administered by intrathecal injection.
• In July 2026- Laboratoires Thea conducted a Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter, double-masked, Randomized, Sham-controlled study which will enroll 81 subjects.
• In July 2026- GlaxoSmithKline highlighted a phase 3 study is intended to confirm the efficacy, safety, pharmacokinetic (PK) profile, and the durability of hepatitis B virus surface antigen (HBsAg) suppression observed with bepirovirsen for 24 weeks (with loading doses) as compared to the placebo arm. This study will have 4 stages: a) Double-blind treatment (bepirovirsen or placebo) for 24 weeks. b) Nucleos(t)ide analogue (NA) treatment for 24 weeks. c) NA cessation stage OR Continue NA for 24 weeks. d) Durability of response and follow up for further 24 weeks for participants who stopped NA treatment at Week 48.
• DelveInsight’s Antisense Oligonucleotide Therapeutics pipeline report depicts a robust space with 150+ active players working to develop 200+ pipeline therapies for Antisense Oligonucleotide Therapeutics treatment.
• The leading Antisense Oligonucleotide Therapeutics Companies such as Novartis Pharmaceuticals, GSK, Ionis Pharmaceuticals, Wave Life Sciences, Bio-Path Holdings, Autotelic Bio, NS Pharma, TransCode Therapeutics, Amylyx Pharmaceuticals Inc., and others.
• Promising Antisense Oligonucleotide Therapeutics Pipeline Therapies such as Bepirovirsen, QR-421a, SB012, Sepofarsen, QR-110, GSK3228836, VCA-894A and others.

Get insights into clinical trials, emerging therapies, and leading companies with DelveInsight @ Antisense Oligonucleotide Therapeutics Treatment Drugs

The Antisense Oligonucleotide Therapeutics Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Antisense Oligonucleotide Therapeutics Pipeline Report also highlights the unmet needs with respect to the Antisense Oligonucleotide Therapeutics.

Antisense Oligonucleotide Therapeutics Overview

Antisense Oligonucleotide Therapeutics are short, synthetic strands of nucleic acids designed to selectively bind to specific RNA sequences and modulate gene expression at the molecular level. These therapies can work through mechanisms such as mRNA degradation, splice modulation, or alteration of RNA processing, offering a targeted approach to treating genetic and acquired diseases. Advances in oligonucleotide chemistry, delivery technologies, and tissue targeting have improved their stability, specificity, and therapeutic potential across neurological, metabolic, cardiovascular, and rare diseases. The growing understanding of disease-associated genetic pathways, increasing investment in RNA-based drug development, and expansion of clinical research are further supporting the development of Antisense Oligonucleotide Therapeutics.

Antisense Oligonucleotide Therapeutics Emerging Drugs Profile

• DYNE-101: Dyne Therapeutics

DYNE-101 is Dyne’s therapeutic candidate being developed for people living with myotonic dystrophy type 1 (DM1). DYNE-101 consists of an antigen-binding fragment antibody (Fab) conjugated to an antisense oligonucleotide (ASO) to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus, releasing splicing proteins, allowing normal mRNA processing and translation of normal proteins, and potentially stopping or reversing the disease. Currently, the drug is in Phase III clinical development for myotonic dystrophy.

• Pelacarsen: Novartis

Pelacarsen (TQJ230), also known as IONIS-APO (a)-LRx and AKCEA-APO(a)-LRx, is an investigational antisense medicine designed to reduce apolipoprotein(a) in the liver to lower lipoprotein(a) levels. Elevated Lp(a) is recognized as an independent genetic cause of coronary artery disease, stroke, heart attack, and peripheral arterial disease. The drug is currently in Phase III development for Hyperlipoproteinaemia and Atherosclerosis.

• AHB-137: Ausper Bio

AHB-137 is a novel unconjugated antisense oligonucleotide developed through AusperBio’s proprietary Med-Oligo™ ASO platform for chronic hepatitis B treatment. The dual-mechanism ASO is currently undergoing Phase Ib and Phase II clinical trials and is advancing toward functional cure strategies for HBV infection. The drug is presently in Phase III stage development for Chronic Hepatitis B.

• Tonlamarsen: Kardigan

Tonlamarsen (formerly ION904) is an investigational angiotensinogen-targeted therapy designed to disrupt the pathological cycle of acute severe hypertension. The therapy aims to lower blood pressure and reduce the risk of organ damage and cardiovascular events. The drug is in Phase II/III development for Hypertension.

• WVE-N531: Wave Life Sciences

WVE-N531 is an exon-skipping oligonucleotide being developed as a disease-modifying therapy for Duchenne muscular dystrophy patients amenable to exon 53 skipping. It utilizes Wave’s PN backbone chemistry modifications and has received Orphan Drug and Rare Pediatric Disease designations from the US FDA. The drug is currently in Phase II clinical evaluation.

• ION717: Ionis Pharmaceuticals

ION717 is an investigational ASO therapy designed to lower levels of prion protein (PrP) in patients with prion diseases. Administered intrathecally, the therapy is currently under evaluation in a Phase I/IIa clinical trial for safety, tolerability, pharmacokinetics, and pharmacodynamics.

• VCA-894A: Vanda Pharmaceuticals

VCA-894A is a novel ASO therapeutic developed for Charcot-Marie-Tooth disease Type 2S (CMT2S), an ultra-rare inherited neuromuscular disorder. It is designed to target a cryptic splice site variant in the IGHMBP2 gene to restore normal gene expression. The therapy is in Phase I/II clinical development.

• QRL-201: QurAlis Corporation

QRL-201 is an investigational RNA-targeted therapy for Amyotrophic Lateral Sclerosis (ALS). It aims to restore STATHMIN-2 (STMN2) expression, which is significantly reduced in ALS patients. The therapy has received clinical trial authorizations in Canada, the European Union, and the United Kingdom and is currently in Phase I/II development.

• AX-0810: ProQR

AX-0810 is an investigational therapy being developed for cholestatic disorders, a group of liver diseases characterized by impaired bile flow and progressive liver injury. The therapy is designed to address underlying pathological mechanisms associated with bile acid accumulation and hepatocellular damage. It is currently in Phase I clinical development.

• SECN-15: Secarna Pharmaceuticals

SECN-15 is a potential first-in-class antisense oligonucleotide therapy targeting Neuropilin-1 (NRP1) for solid tumors. The program aims to improve anti-tumor immune responses and enhance the efficacy of immune checkpoint inhibitors. Preclinical studies demonstrated strong NRP1 knockdown and improved anti-tumor activity. The therapy is currently in preclinical development.

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

Explore groundbreaking therapies and clinical trials in the Antisense Oligonucleotide Therapeutics Pipeline @ New Antisense Oligonucleotide Therapeutics Drugs

Antisense Oligonucleotide Therapeutics Companies

Novartis Pharmaceuticals, GSK, Ionis Pharmaceuticals, Wave Life Sciences, Bio-Path Holdings, Autotelic Bio, NS Pharma, TransCode Therapeutics, Amylyx Pharmaceuticals Inc., 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
• Intravenous
• Subcutaneous
• Parenteral
• Topical

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

• Recombinant fusion proteins
• Small molecules
• Monoclonal antibodies
• Peptides
• Polymers
• Gene therapies

Learn about new drugs, pipeline developments, and key companies with DelveInsight’s expert analysis @ Antisense Oligonucleotide Therapeutics Market Drivers and Barriers

Scope of the Antisense Oligonucleotide Therapeutics Pipeline Report

• Coverage- Global
• Antisense Oligonucleotide Therapeutics Companies- Novartis Pharmaceuticals, GSK, Ionis Pharmaceuticals, Wave Life Sciences, Bio-Path Holdings, Autotelic Bio, NS Pharma, TransCode Therapeutics, Amylyx Pharmaceuticals, Inc. and others.
• Antisense Oligonucleotide Therapeutics Pipeline Therapies- Bepirovirsen, QR-421a, SB012, Sepofarsen, QR-110, GSK3228836, VCA-894A 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

Download DelveInsight’s in-depth pipeline report today! @ Antisense Oligonucleotide Therapeutics Companies, Key Products and Unmet Needs

Table of Contents

1. Introduction
2. Executive Summary
3. Antisense Oligonucleotide Therapeutics: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Antisense Oligonucleotide Therapeutics– DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Pelacarsen: Novartis Pharmaceuticals
9. Mid Stage Products (Phase II)
10. WVE-N531: Wave Life Sciences
11. Early Stage Products (Phase I)
12. BP1002: Bio-Path Holdings
13. Preclinical Stage Products
14. NS-051/NCNP-04: NS Pharma
15. Inactive Products
16. Antisense Oligonucleotide Therapeutics Collaborations Assessment- Licensing / Partnering / Funding
17. Antisense Oligonucleotide Therapeutics Unmet Needs
18. Antisense Oligonucleotide Therapeutics Market Drivers and Barriers
19. Appendix

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email: Send Email
Phone: 09650213330
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/report-store/antisense-oligonucleotide-therapeutics-pipeline-insight