Antisense Oligonucleotide Therapeutics - Pipeline Insight, 2025

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 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.

Geography Covered

• Global coverage

Antisense Oligonucleotide Therapeutics Understanding

Antisense Oligonucleotide Therapeutics: Overview

Antisense oligonucleotide (ASOs) are small-sized single-stranded nucleic acids that offer advantages over siRNAs in terms of targeting both nuclear and cytoplasmic located lncRNAs. Antisense oligonucleotide-based therapeutics involves downregulation of gene expression. Antisense oligonucleotides (AOs) are a class of synthetic nucleic acid molecules that are capable of regulating gene expression through binding to premature or mature messenger RNA (mRNA) targets. Antisense oligonucleotides (ASOs), known as genetic materials, are promising agents used in the regulation of unsuitable expression of genes in pathological situations by the specific inhibition of expression of their mRNA targets. By capitalizing “antisense” DNA approach, the overexpressed proteins can be blocked in several diseases such as cancer, neurological diseases, cardiovascular diseases, inflammation and autoimmune diseases, infectious diseases, etc. ASOs are emerging as a class of therapeutics with high potential to treat diseases affecting the CNS.

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 for 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 III, 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

• Cobitolimod: InDex Pharmaceuticals

Cobitolimod is a Toll-like receptor 9 (TLR9) agonist that can provide an anti-inflammatory effect locally in the large intestine, which may induce mucosal healing and relief of the clinical symptoms in ulcerative colitis. Cobitolimod is a DNA-based ImmunoModulatory Sequence (DIMS) that is administered locally inside the large intestine, where it binds to Toll-like receptor 9 (TLR9) present inside immune cells as well as on the surface of epithelial cells. Cobitolimod is a first in class treatment that can provide a local anti-inflammatory effect, which can lead to healing of the mucosa and improvement of clinical symptoms. Cobitolimod is has a very limited systemic absorption. US Food and Drug Administration (FDA) has granted orphan-drug designation for the drug candidate cobitolimod for treatment of ulcerative colitis in pediatric patients.

• 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.

Further product details are provided in the report……..

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, such as:

• Major Players working on Antisense Oligonucleotide Therapeutics

There are approx. 15+ key companies which are developing the Antisense Oligonucleotide Therapeutics. The companies which have their Antisense Oligonucleotide Therapeutics drug candidates in the most advanced stage, i.e. Phase III include, InDex Pharmaceuticals.

• Phases

DelveInsight’s report covers around 15+ products under different phases of clinical development like

• Late-stage products (Phase III and
• Mid-stage products (Phase II and
• Early-stage products (Phase I/II and 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

• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravaginal
• Oral
• Parenteral
• Subcutaneous
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Vaccines
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Product Type

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

Antisense Oligonucleotide Therapeutics: Pipeline Development Activities

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

Pipeline 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 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 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

• InDex Pharmaceuticals
• Bio-Path Holdings
• Akcea Therapeutics
• Ionis Pharmaceuticals
• AstraZeneca
• Gene Signal
• Chamishi Therapeutics
• NeuBase Therapeutics

Key Products

• Cobitolimod
• Prexigebersen
• Inotersen
• AKCEA APOCIII LRx
• ION 736
• NS-089/NCNP-02
• Aganirsen
• Research programme : antisense oligonucleotide therapeutics
• NT0100