Antisense Oligonucleotide Therapeutics Clinical Trial Pipeline Appears Robust With 70+ Key Pharma Companies Actively Working in the Domain | DelveInsight

DelveInsight's, "Antisense Oligonucleotide Therapeutics Pipeline Insights 2026" report provides comprehensive insights about 70+ companies and 90+ 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.

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Key Takeaways from the Antisense Oligonucleotide Therapeutics Pipeline Report

* On January 08=9, 2026- Laboratoires Thea a paired-eye 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 70+ active players working to develop 90+ 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 VCA-894A, BP1001, GSK3228836, QR-010, Sepofarsen, SB012 and others.

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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 (ASO) Therapeutics represent a rapidly advancing class of precision medicines designed to modulate gene expression at the RNA level. These short, synthetic strands of nucleic acids are engineered to bind selectively to target messenger RNA (mRNA), enabling the inhibition, correction, or modification of disease-causing gene products. By acting upstream of protein synthesis, ASO therapeutics offer a highly targeted approach to treating diseases with well-defined genetic or molecular drivers. One of the key strengths of ASO therapeutics lies in their mechanistic versatility. Depending on their design, ASOs can induce RNA degradation via RNase H, alter RNA splicing, block translation, or modulate microRNA activity. This flexibility has positioned ASOs as a promising modality across a wide range of indications, including neurological disorders, rare genetic diseases, cardiovascular conditions, metabolic disorders, and oncology.

Antisense Oligonucleotide Therapeutics Emerging Drugs Profile

* Pelacarsen: Novartis Pharmaceuticals

Pelacarsen (TQJ230), also known as IONIS-APO(a)-LRx, AKCEA-APO(a)-LRx, and TQJ230, is an investigational antisense medicine designed to reduce apolipoprotein(a) in the liver to offer a direct approach for reducing lipoprotein(a), or Lp (a) - a very atherogenic and thrombogenic form of LDL. Elevated Lp (a) is recognized as an independent genetic cause of coronary artery disease, heart attack, stroke, and peripheral arterial disease. Pelacarsen, an investigational antisense medicine designed to lower Lp(a), was discovered by Ionis and licensed to Novartis in 2019. Currently the drug is in Phase III for the treatment of Hyperlipoproteinaemia.

* WVE-N531: Wave Life Sciences

WVE-N531 is an exon skipping oligonucleotide being developed as a disease modifying treatment for boys with Duchenne muscular dystrophy amenable to exon 53 skipping. WVE-N531 was designed using Wave's best-in-class oligonucleotide chemistry modifications, including PN backbone chemistry. WVE-N531 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food & Drug Administration. Currently the drug is in Phase III for the treatment of Duchenne Muscular Dystrophy.

* BP1002: Bio-Path Holdings

BP1002 (Liposomal Bcl-2) is a neutral-charge, liposome-incorporated antisense drug designed to inhibit protein synthesis of Bcl-2, a protein involved in blocking programmed cell death. Bcl-2 is overexpressed in a wide variety of tumors, including NHL and chronic lymphocytic leukemia. Overexpression of Bcl-2 stops affected cells from being killed by chemotherapy. We believe that BP1002 will inhibit Bcl-2 protein expression without inherent toxicity. The introduction of a new, non-toxic, and specific Bcl-2 inhibitor could be a major advance in cancer therapeutics. A Phase I clinical trial for BP1002 in patients with relapsed or refractory NHL or chronic lymphocytic leukemia is currently underway.

* NS-051/NCNP-04: NS Pharma

NS051/NCNP04 is an antisense oligonucleotide codeveloped by NS Pharma (a subsidiary of Nippon Shinyaku) and Japan's National Center of Neurology and Psychiatry to treat Duchenne muscular dystrophy by targeting exon 51 of the dystrophin gene. It promotes skipping of exon 51, enabling production of a shorter yet functional dystrophin protein to preserve or improve muscle function in patients with mutations amenable to this approach. The compound is currently in preclinical development and was granted FDA Rare Pediatric Disease. NS051/NCNP04 exemplifies the company's strategic focus on exonskipping therapies for neuromuscular disorders. The drug is currently in preclinical stage of its 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

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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

* Intra-articular
* Intraocular
* Intrathecal
* Intravenous
* Oral
* Parenteral
* Subcutaneous
* Topical
* Transdermal

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

* Oligonucleotide
* Peptide
* Small molecule

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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- VCA-894A, BP1001, GSK3228836, QR-010, Sepofarsen, SB012 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 [https://www.delveinsight.com/sample-request/antisense-oligonucleotide-therapeutics-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Table of Contents

* Introduction
* Executive Summary
* Antisense Oligonucleotide Therapeutics: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Antisense Oligonucleotide Therapeutics- DelveInsight's Analytical Perspective
* Late Stage Products (Phase III)
* Pelacarsen: Novartis Pharmaceuticals
* Mid Stage Products (Phase II)
* WVE-N531: Wave Life Sciences
* Early Stage Products (Phase I)
* BP1002: Bio-Path Holdings
* Preclinical Stage Products
* NS-051/NCNP-04: NS Pharma
* Inactive Products
* Antisense Oligonucleotide Therapeutics - Collaborations Assessment- Licensing / Partnering / Funding
* Antisense Oligonucleotide Therapeutics - Unmet Needs
* Antisense Oligonucleotide Therapeutics - Market Drivers and Barriers
* Appendix

About Us

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