Oligonucleotides Competitive Landscape 2025: 320+ Therapies Shaping the Future of Precision Medicine | DelveInsight

DelveInsight's "Oligonucleotides - Competitive Landscape, 2025" report offers a deep dive into the global therapeutic race driven by oligonucleotide-based innovations. With over 280+ companies and 320+ products in various stages of development, the report provides a detailed snapshot of the competitive efforts to harness oligonucleotides for treating rare diseases, genetic disorders, and cancers.

The landscape features a broad mix of antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), microRNA modulators, aptamers, and splice-switching oligonucleotides. These modalities are being explored for diverse indications such as Duchenne muscular dystrophy, spinal muscular atrophy, Huntington's disease, amyloidosis, and various cancers.

The report segments oligonucleotide therapies by product type, stage of development, route of administration (intravenous, subcutaneous, intrathecal, etc.), and molecule class. It also tracks inactive and discontinued programs to help identify evolving trends and therapeutic gaps.

Key players, including Ionis Pharmaceuticals, Alnylam Pharmaceuticals, Sarepta Therapeutics, Wave Life Sciences, Silence Therapeutics, and Dyne Therapeutics, are leading the charge with next-generation chemistry, improved delivery platforms, and tissue-targeted innovations to boost efficacy and reduce off-target effects.

With increasing FDA approvals, strategic partnerships, and regulatory support for orphan drugs and RNA-based technologies, oligonucleotides are emerging as a transformative force in modern medicine. DelveInsight's report captures these dynamics, offering valuable competitive intelligence for stakeholders aiming to navigate the future of nucleic acid therapeutics.

Interested in learning more about the current treatment landscape and the key drivers shaping the oligonucleotides pipeline? Click here: https://www.delveinsight.com/report-store/oligonucleotides-competitive-landscape?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the Oligonucleotides Competitive Landscape Report
• DelveInsight's oligonucleotides competitive analysis depicts a strong space with 280+ active players working to develop 320+ drugs for oligonucleotides treatment.
• The leading oligonucleotides companies include Novartis, Astellas, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, 4D Molecular Therapeutics, Avidity Biosciences, Suzhou Ribo Life Science, Amgen, GSK, ProQR Therapeutics, Stoke Therapeutics, MiNA Therapeutics, Sylentis, GSK, Silexion Therapeutics, Novo Nordisk A/S, Bio-Path Holdings, Sunhawk Vision Biotech, Isarna Therapeutics, Sirnaomics, Laboratoire Thea, Dyne Therapeutics, Vertex Pharmaceuticals, Korro Bio, Praxis-Precision-Medicines, Vico Therapeutics, BioMarin Pharmaceutical, TransCode Therapeutics, TME therapeutics, ARTHEx Biotech, aptaTargets, CSPC Zhongnuo Pharmaceutical, ExoRNA Bioscience, Visirna Therapeutics, AiCuris, Comanche Biopharma, Tallac Therapeutics, and others are evaluating their lead assets to improve the oligonucleotides treatment landscape.
• Key oligonucleotides pipeline therapies in various stages of development include Pelacarsen, Trabedersen, 4D-150, Delpacibart Etedesiran, GSK3228836, WVE-N531, ATX-01, TAC001, ATB 301, and others.
• In June 2025, Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) announced that the FDA granted Fast Track designation to AMX0114, an investigational antisense oligonucleotide targeting calpain-2 for treating amyotrophic lateral sclerosis.
• In April 2025, Biogen Inc. (Nasdaq: BIIB) announced that the FDA granted Fast Track designation to BIIB080, an antisense oligonucleotide therapy targeting tau, for Alzheimer's disease treatment, aiming to speed up its development and review.
• In March 2025, Korro Bio, Inc. a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, announced that the FDA has granted orphan drug designation to the investigational medicine KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD).
• In January 2025, the FDA granted fast-track designation to DYNE-101, an investigational antisense oligonucleotide for treating myotonic dystrophy type 1 (DM1). Dyne Therapeutics Inc. announced that DYNE-101 targets muscle to reduce harmful RNA in the cell nucleus, to slow or halt disease progression. It is currently being evaluated in the ongoing Phase I/II ACHIEVE global clinical trial.
• In January 2025, Arrowhead Pharmaceuticals, Inc. announced that the FDA had accepted the New Drug Application (NDA) for investigational plozasiran for the treatment of familial chylomicronemia syndrome (FCS), a severe and rare genetic disease.
• In February 2025, AusperBio Therapeutics, Inc. and Ausper Biopharma Co., Ltd. (together AusperBio), a clinical-stage biotechnology company, announced recent progress in the ongoing clinical development of its lead candidate AHB-137, an antisense oligonucleotide (ASO) therapeutic for functional cure of chronic Hepatitis B (CHB).

Request a sample and discover the recent breakthroughs happening in the oligonucleotides competitive landscape at https://www.delveinsight.com/report-store/oligonucleotides-competitive-landscape?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Oligonucleotides Overview
Oligonucleotides is a progressive eye disease characterized by damage to the optic nerve, often caused by elevated intraocular pressure (IOP). It is the leading cause of irreversible blindness worldwide. The two main types are open-angle Oligonucleotides (OAG)-the most common form-and angle-closure (narrow-angle) Oligonucleotides. Both refer to how fluid drains from the eye, affecting pressure levels and optic nerve health.

In OAG, the drainage angle remains open but is partially blocked, causing gradual pressure buildup and unnoticed vision loss. Angle-closure Oligonucleotides involve a restricted or blocked drainage angle, leading to a sudden rise in eye pressure and an urgent risk of vision loss.

Oligonucleotides often develop without early symptoms. Vision loss, especially in peripheral or central vision, may not be noticed until significant optic nerve damage has occurred. Studies now show that both central and peripheral vision can be affected earlier than previously believed.

Risk factors include family history, thin corneas, diabetes, obesity, and extreme myopia or hyperopia. Although there is no cure, early diagnosis and treatment-via eye drops, laser therapy, or surgery-can help preserve vision and slow disease progression.

Find out more about oligonucleotides medication at https://www.delveinsight.com/report-store/oligonucleotides-competitive-landscape?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Oligonucleotides: Company and Product Profiles (Marketed Therapies)
1. Company Overview: Novartis
Novartis International AG, headquartered in Switzerland, is one of the world's largest pharmaceutical companies. Formed in 1996 through the merger of Ciba-Geigy and Sandoz, Novartis is a leader in innovative medicines, generics, and biosimilars. The company focuses on sustainable healthcare, with initiatives aimed at improving access to affordable medications in low- and middle-income countries while minimizing environmental impact.

Product Spotlight: LEQVIO (inclisiran)
LEQVIO is a first-in-class siRNA therapy targeting PCSK9 mRNA to reduce LDL-C levels. By inhibiting PCSK9 production in the liver, LEQVIO enhances LDL-C clearance from the bloodstream. Approved in 2023 by Japan's MHLW for both familial and non-familial hypercholesterolemia, it is administered initially, again at three months, and then every six months. Clinical studies show it lowers LDL-C by ~50% when used with statins.

2. Company Overview: Astellas Pharma
Astellas Pharma Inc., based in Tokyo, Japan, was created in 2005 from the merger of Yamanouchi and Fujisawa Pharmaceutical. The company focuses on oncology, urology, immunology, and neuroscience. Astellas emphasizes R&D and global collaboration to deliver innovative therapies addressing critical unmet needs across North America, Europe, and Asia.

Product Spotlight: IZERVAY (avacincaptad pegol)
IZERVAY is an RNA aptamer targeting complement factor C5, approved by the FDA in August 2023 for geographic atrophy (GA) secondary to AMD. By blocking C5 activation, it reduces inflammation and slows GA progression. It is also under EMA review and is being explored for Stargardt disease.

Oligonucleotides: Company and Product Profiles (Pipeline Therapies)
1. Company Overview: Novartis Pharmaceuticals
Novartis Pharmaceuticals, headquartered in Switzerland, is a global leader in healthcare innovation, committed to transforming medicine and improving lives. The company focuses on key therapeutic areas including oncology, immunology, neuroscience, and cardiovascular disease. Leveraging advanced science, digital technologies, and a robust R&D pipeline, Novartis operates in over 150 countries, delivering sustainable, patient-centric healthcare solutions.

Product Spotlight: Pelacarsen
Pelacarsen (TQJ230), an investigational antisense therapy licensed from Ionis, targets elevated lipoprotein(a) [Lp(a)]-a genetic risk factor for heart attack, stroke, and peripheral arterial disease. It works by reducing apolipoprotein(a) production in the liver. Currently in Phase III trials, Pelacarsen holds promise for treating hyperlipoproteinaemia by addressing a major unmet cardiovascular need.

2. Company Overview: Oncotelic Therapeutics
Oncotelic Therapeutics is a US-based clinical-stage biopharma company specializing in RNA-based therapies and immuno-oncology for cancer and rare diseases. Its pipeline includes candidates for glioblastoma, melanoma, and fibrotic conditions. Oncotelic is advancing therapies targeting the TGF-ß pathway to reactivate immune responses against difficult-to-treat tumors.

Product Spotlight: Trabedersen (OT-101)
Trabedersen is an antisense oligodeoxynucleotide designed to inhibit TGF-ß2, a protein that helps tumors evade immune detection. By blocking TGF-ß2 mRNA, Trabedersen lifts the tumor's immune suppression, enabling effective immune attack. It has shown positive results in Phase II trials for glioblastoma, pancreatic cancer, and melanoma.

3. Company Overview: Wave Life Sciences
Wave Life Sciences is a biotechnology company advancing RNA-based medicines through its proprietary PRISM® platform. With expertise in RNA editing, silencing, splicing, and interference, Wave is developing therapies for both rare and common diseases. Its pipeline spans Duchenne muscular dystrophy, Huntington's disease, and alpha-1 antitrypsin deficiency.

Product Spotlight: WVE-N531
WVE-N531 is an exon-skipping therapy for Duchenne muscular dystrophy patients amenable to exon 53 skipping. Built using Wave's advanced oligonucleotide chemistry, it has received Orphan Drug and Rare Pediatric Disease designations from the FDA. Currently in Phase II, WVE-N531 aims to modify disease progression in DMD.

Discover how emerging oligonucleotides therapies stack up against current treatments in the evolving market landscape: https://www.delveinsight.com/report-store/oligonucleotides-competitive-landscape?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Oligonucleotides Therapeutics Assessment
Company Analysis
Therapeutic Assessment
Pipeline Assessment
Inactive drugs assessment
Unmet Needs

Scope of the Oligonucleotides Competitive Landscape Report
• Coverage: Global
• Key Oligonucleotides Companies: Novartis, Astellas, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, 4D Molecular Therapeutics, Avidity Biosciences, Suzhou Ribo Life Science, Amgen, GSK, ProQR Therapeutics, Stoke Therapeutics, MiNA Therapeutics, Sylentis, GSK, Silexion Therapeutics, Novo Nordisk A/S, Bio-Path Holdings, Sunhawk Vision Biotech, Isarna Therapeutics, Sirnaomics, Laboratoire Thea, Dyne Therapeutics, Vertex Pharmaceuticals, Korro Bio, Praxis-Precision-Medicines, Vico Therapeutics, BioMarin Pharmaceutical, TransCode Therapeutics, TME therapeutics, ARTHEx Biotech, aptaTargets, CSPC Zhongnuo Pharmaceutical, ExoRNA Bioscience, Visirna Therapeutics, AiCuris, Comanche Biopharma, Tallac Therapeutics, and others.
• Key Oligonucleotides Pipeline Therapies: Pelacarsen, Trabedersen, 4D-150, Delpacibart Etedesiran, GSK3228836, WVE-N531, ATX-01, TAC001, ATB 301, and others.

To dive deep into rich insights for drugs used for oligonucleotides treatment, visit: https://www.delveinsight.com/report-store/oligonucleotides-competitive-landscape?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Oligonucleotides Overview
4. Analytical & Commercial Assessment
5. Competitive Landscape
6. Marketed Therapies
7. Pipeline Therapies (by development stage)
8. Unmet Needs
9. Market Drivers & Barriers
10. Appendix

Contact Us:
Jatin Vimal
jvimal@delveinsight.com
+14699457679
Healthcare Consulting
https://www.delveinsight.com/consulting-services

About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform, PharmDelve.

This release was published on openPR.