Antisense Oligonucleotide Therapeutics - Pipeline Insight, 2025

This “Antisense Oligonucleotide Therapeutics - Pipeline Insight, 2025” report provides comprehensive insights about 70+ companies and 75+ pipeline drugs in Antisense Oligonucleotide Therapeutics pipeline landscape. It covers the 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.

Antisense Oligonucleotide Therapeutics: Understanding

Antisense Oligonucleotide Therapeutics: Overview

Antisense Oligonucleotide (ASO) Therapeutics are short, synthetic strands of nucleic acids designed to bind specifically to messenger RNA (mRNA) and modulate gene expression. They work by binding to messenger RNA (mRNA), they modify gene expression, often by inhibiting translation, altering splicing, or inducing mRNA degradation. ASOs offer a highly targeted approach to treat genetic, neuromuscular, and rare diseases. Their precision makes them especially valuable for conditions with limited treatment options.

Antisense oligonucleotides (ASOs) are short, single-stranded synthetic nucleic acids, typically 15-25 bases long, designed to bind complementary RNA sequences and modulate gene expression. Their structure includes nucleotide bases, a sugar backbone often modified for stability (e.g., 2'-O-methyl, 2'-MOE), and phosphate linkages frequently replaced by phosphorothioate bonds to resist degradation. ASOs are further enhanced with chemical modifications such as locked nucleic acids (LNA) or constrained ethyl (cEt) to improve binding affinity and specificity. Structural formats like gapmers and steric-blocking ASOs are tailored for different mechanisms - either promoting RNA degradation or blocking translation/splicing. The structure critically influences the ASO’s potency, stability, tissue distribution, and safety profile.

Depending on the design and chemical modifications, ASOs work by:

RNase H-mediated degradation: ASO binds to mRNA, forming a DNA-RNA hybrid that recruits RNase H to degrade the mRNA.

Steric blocking: ASO binds mRNA and physically blocks translation or splicing machinery.

Splice modulation: ASOs can alter splicing of pre-mRNA to exclude or include specific exons (used in genetic diseases).

Antisense oligonucleotides (ASOs) have emerged as a promising therapeutic strategy for targeting diseases at the genetic level. By binding to specific mRNA sequences, ASOs can modulate gene expression through mechanisms such as mRNA degradation, translation inhibition, or splicing correction. This high level of sequence specificity allows ASOs to target genes previously considered “undruggable,” making them particularly valuable for rare genetic, neuromuscular, and neurodegenerative disorders. Their versatility enables the development of both personalized and broad-spectrum therapies. Ongoing advancements in delivery systems and chemical modifications continue to enhance their safety, stability, and clinical potential. Several ASO therapies have already received regulatory approval, validating the clinical success of this approach. Continued investment in ASO research is expected to expand their application across more complex and widespread diseases.

“Antisense Oligonucleotide Therapeutics - Pipeline Insight, 2025" report outlays comprehensive insights of present scenario and growth prospects across the mechanism of action. A detailed picture of the Antisense Oligonucleotide Therapeutics pipeline landscape is provided which includes the disease overview and Antisense Oligonucleotide Therapeutics treatment guidelines. The assessment part of the report embraces, in depth Antisense Oligonucleotide Therapeutics commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Antisense Oligonucleotide Therapeutics collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Antisense Oligonucleotide Therapeutics R&D. The therapies under development are focused on novel approaches to treat/improve Antisense Oligonucleotide Therapeutics.

Antisense Oligonucleotide Therapeutics Emerging Drugs Chapters

This segment of the Antisense Oligonucleotide Therapeutics report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Antisense Oligonucleotide Therapeutics Emerging Drugs

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

NS-051/NCNP-04 is an antisense oligonucleotide co-developed 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. NS-051/NCNP-04 exemplifies the company’s strategic focus on exon-skipping therapies for neuromuscular disorders. The drug is currently in preclinical stage of its development.

Antisense Oligonucleotide Therapeutics: Therapeutic Assessment

This segment of the report provides insights about the different Antisense Oligonucleotide Therapeutics drugs segregated based on following parameters that define the scope of the report.

Major Players in Antisense Oligonucleotide Therapeutics
There are approx. 70+ key companies which are developing the therapies for Antisense Oligonucleotide Therapeutics. The companies which have their Antisense Oligonucleotide Therapeutics drug candidates in the most advanced stage, i.e. Phase III include, Novartis Pharmaceuticals.

Phases

The report covers around 75+ products under different phases of clinical development, like:

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of:
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration
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

Molecule Type
Products have been categorized under various Molecule types, such as:

• Oligonucleotide
• Peptide
• Small molecule

Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Antisense Oligonucleotide Therapeutics: Pipeline Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Antisense Oligonucleotide Therapeutics drugs key players involved in developing key drugs.

Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Antisense Oligonucleotide Therapeutics drugs.

Antisense Oligonucleotide Therapeutics Report Insights

• Antisense Oligonucleotide Therapeutics Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Antisense Oligonucleotide Therapeutics Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Antisense Oligonucleotide Therapeutics drugs?
• How many Antisense Oligonucleotide Therapeutics drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Antisense Oligonucleotide Therapeutics?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Antisense Oligonucleotide Therapeutics therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Antisense Oligonucleotide Therapeutics and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

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

Key Products

• Pelacarsen
• GSK3228836
• WVE-N531
• ION717
• BP1002
• ATB 301
• NS-051/NCNP-04
• TTX MC 138
• AMX0114

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