The oligonucleotides clinical trial analysis report delivers important insights into ongoing research on 600+ oligonucleotides in the pipeline, clinical strategies, upcoming therapeutics, and commercial analysis.

New York, USA, May 14, 2026 (GLOBE NEWSWIRE) -- Oligonucleotides Clinical Trial Drug Development Pipeline Expands with Contributions from 200+ Key Companies | DelveInsight

DelveInsight’s Oligonucleotides Competitive Landscape 2026 report provides comprehensive global coverage of pipeline therapies for oligonucleotides across various stages of clinical development. The report offers an in-depth analysis of key trends, emerging therapies, and competitive landscape dynamics, highlighting the strategies of major pharmaceutical companies to advance the pipeline and capitalize on future growth opportunities. In addition, it includes critical insights into clinical trial benchmarking, partnering and licensing activities, and regulatory pathways involving the FDA and EMA, enabling stakeholders to make informed decisions and optimize development strategies within the oligonucleotides domain.

Oligonucleotides Clinical Trial Analysis Summary

DelveInsight’s oligonucleotides pipeline report depicts a robust space with 200+ active players working to develop 600+ pipeline oligonucleotides.
Key oligonucleotide companies, such as Novartis Pharmaceuticals, Sylentis, Amgen, Alnylam Pharmaceuticals, Kardigan, SanegeneBio, WaVe life Sciences, Rigerna Therapeutics, Eli Lily and Company, PYC Therapeutics, ADARx Pharmaceuticals, Denali Therapeutics and others, are evaluating new oligonucleotides to improve the treatment landscape.
Promising pipeline oligonucleotides, such as Pelacarsen, Tivanisiran, Olpasiran, Zilebesiran, Tonlamarsen, SGB-9768, WVE-N531, RG002C0106, LY-3954068, PYC-003, ADX-626, DNL422 and others, are under different phases of oligonucleotide clinical trials.
Approximately 20+ oligonucleotides are in the late stage of development, whereas 150+ drugs are in the mid and early stages of development.
Notable MoAs in oligonucleotide clinical trials include ASO targeting lipoprotein, Angiotensinogen expression inhibitor, RNA interference, Complement C3 expression inhibitors, PKD1 modulator, and others.

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What are Oligonucleotides?

Oligonucleotides are short, synthetic strands of nucleic acids, composed of DNA or RNA building blocks, typically ranging from a few to a few dozen nucleotides in length. They are designed to bind selectively to complementary sequences of genetic material, enabling precise control over gene expression and function. In biomedical research and therapeutics, oligonucleotides are widely used in applications such as gene silencing, molecular diagnostics, and PCR amplification. Their ability to modulate specific genetic targets has made them a cornerstone of modern biotechnology, particularly in the development of targeted therapies for genetic disorders, cancers, and rare diseases.

Find out more about oligonucleotide drugs @ Oligonucleotide Analysis

A snapshot of the Pipeline Oligonucleotides mentioned in the report:

Drugs	Company	Phase	MoA	RoA
Pelacarsen	Novartis Pharmaceuticals	III	ASO targeting lipoprotein	Subcutaneous
Tivanisiran	Sylentis	III	TRPV1 receptor antagonists	Ophthalmic
Olpasiran	Amgen	III	Lipoprotein A expression inhibitors; RNA interference	Subcutaneous
Zilebesiran	Alnylam Pharmaceuticals	III	Angiotensinogen expression inhibitor	Subcutaneous
WVE-N531	WaVe life Sciences	II	Dystrophin expression stimulants	Intravenous
SGB-9768	SanegeneBio	II	Complement C3 expression inhibitors	Subcutaneous
RG002C0106	Rigerna Therapeutics	II	Complement C3 expression inhibitors	Subcutaneous
PYC-003	PYC Therapeutics	I	PKD1 modulator	Intravenous infusion
LY-3954068	Eli Lily and Company	Phase I	Tau protein inhibitor	Intrathecal
ADX-626	ADARx Pharmaceuticals	Phase I	Factor XI expression inhibitors	Subcutaneous
DNL422	Denali Therapeutics	Preclinical	Alpha-synuclein expression inhibitors	NA

Learn more about the emerging oligonucleotides @ Oligonucleotides Clinical Trials

As per Stuti Mahajan, consulting manager at DelveInsight, the oligonucleotide therapeutics landscape is driven by a small group of established leaders and a large pool of emerging innovators. Companies like Ionis Pharmaceuticals and Alnylam Pharmaceuticals lead the market with proven antisense and RNAi platforms, supported by strong pipelines and commercial success, which creates high entry barriers. At the same time, big pharma players such as Novartis and Biogen are expanding their presence mainly through partnerships and licensing deals rather than building capabilities from scratch. Emerging biotechs, including Arrowhead Pharmaceuticals and Silence Therapeutics, are competing by advancing novel delivery technologies and targeting approaches. Overall, competition is less about individual drugs and more about the strength of underlying platforms and delivery systems, with future leadership likely to depend on innovation in targeting, scalability, and cost-effective commercialization.

Recent Developments in the Oligonucleotide Treatment Space

In December 2025, SanegeneBio announced the closing of Series B financing round, raising over USD 110 million. The financing will advance its clinical stage assets towards registrational studies and accelerate the development of programs across several therapeutic areas, powered by its industry-leading RNAi technologies including the LEAD™ (Ligand and Enhancer Assisted Delivery) delivery platform.
In December 2025, Praxis Precision Medicines, Inc., announced the completion of a Type C meeting with the U.S. Food and Drug Administration (FDA) and agreement to immediately convert the EMBRAVE3 registrational study of elsunersen in early-onset SCN2A developmental and epileptic encephalopathy (DEE) into a single-arm study where all patients will receive elsunersen for 24 weeks, followed by an open-label extension.
In December 2025, Sunhawk Vision received the approval of Australia patent for the company’s novel eye drops, SHJ002, in treating ocular surface diseases (including dry eye disease).
In November 2025, PepGen Inc. announced that the United States Patent and Trademark Office (USPTO) has issued the Company a new composition of matter patent covering PGN-EDODM1, which leverages PepGen’s proprietary Enhanced Delivery Oligonucleotide (EDO) platform, including its unique peptide and linker chemistry. This newly issued composition of matter patent is expected to provide exclusivity for PGN-EDODM1 in the United States into second half of 2042, with the possibility of patent term extension following FDA approval of PGN-EDODM1.
In October 2025, Nippon Shinyaku Co., Ltd. announced the efficacy and safety data of 3.5 years of administration based on an open-label extension study, including an investigator-initiated clinical trial of brogidirsen (NS-089/NCNP-02).
In October 2025, SanegeneBio announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SGB-9768 for the treatment of C3 Glomerulopathy (C3G).
In October 2025, ADARx Pharmaceuticals, Inc. announced that the first patient has been dosed in its Phase III STOP-HAE clinical trial evaluating ADX-324 in patients with hereditary angioedema (HAE). Additionally, ADARx announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to ADX-324 for the treatment of patients with HAE.
In October 2025, Suzhou Ribo Life Science Co., Ltd. and Ribocure Pharmaceuticals AB (Ribo), announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to RBD1016 for the treatment of Hepatitis D Virus (HDV) infection.
In September 2025, Argo Biopharmaceutical Co., Ltd. announced that the first patient dosing has been completed for the Phase II clinical trial of its innovative siRNA-based therapy in chronic hepatitis B (CHB) patients. The Phase II study will test both BW-20507 mono-therapy and combination therapy of BW-20507 and PEG-IFNα.
In July 2025, Argo Biopharmaceutical Co., Ltd. announced that National Medical Products Administration of China (NMPA) has granted Investigational New Drug (IND) approval for Phase II clinical trial of its innovative siRNA-based therapy, BW-20507 to be combined with standard-of-care PEG-IFNα for the treatment of chronic hepatitis B (CHB, chronic HBV).
In June 2025, Argo Biopharmaceutical Co., Ltd. announced that its investigational drug BW-20507 has been granted Breakthrough Therapy Designation (BTD) by China Center for Drug Evaluation (CDE), National Medical Products Administration (NMPA) for the treatment of chronic hepatitis B (CHB, chronic HBV) infection. This recognition is based on the molecule’s demonstrated potent antiviral activity and favorable safety profile in early-stage clinical trials, positioning it as a potential transformative therapy for HBV patients.
Sapablursen was granted Fast Track designation in January 2024 and orphan drug designation in August 2024 by the US Food and Drug Administration (FDA), along with Breakthrough Therapy designation in May 2025.
In April 2025, Biogen Inc. announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to BIIB080, an investigational antisense oligonucleotide (ASO) therapy targeting tau, for the treatment of Alzheimer’s disease.
In April 2025, Oak Hill Bio entered into an exclusive license agreement with Roche to obtain global rights for rugonersen (RO7248824). Rugonersen is an ASO designed as a potential best-in- class treatment for individuals with Angelman syndrome, a rare, genetic neurodevelopmental condition for which there is no approved disease-modifying treatment. Oak Hill plans to initiate a Phase III study in early 2026.
In March 2025, Wave also announced that the company met with the U.S. Food and Drug Administration (FDA) on WVE-N531 to discuss its interim 24-week data and initial plans for the confirmatory trial, where the Agency confirmed that the accelerated approval pathway using dystrophin expression as a surrogate endpoint remains open. Based on the FDA feedback and the 48-week data, Wave intends to file a New Drug Application (NDA) in 2026 for accelerated approval of WVE-N531. The NDA filing will be based on all FORWARD-53 data, which will include additional data to support monthly dosing. Furthermore, Wave will continue to engage the Agency with the new 48-week data, including functional outcomes, and its planned global confirmatory trial of WVE-N531.
In March 2025, Alys Pharmaceuticals, Inc. ("Alys"), an immune-dermatology focused company, announced that the first patient has been dosed in its Phase IIa trial of ALY-101 in patients suffering from Alopecia Areata (AA), a chronic autoimmune disease with significant unmet medical need that often leads to significant psychological distress and a reduced quality of life.
In March 2025, Rona Therapeutics Inc. announced the clearance of an Investigational New Drug (IND) by the US Food and Drug Administration (FDA) for RN0361-an APOC3 targeted siRNA (small interfering RNA) therapeutic for severe hypertriglyceridemia (SHTG), mixed dyslipidemia, and familial chylomicronemia syndrome (FCS) treatment. The company plans to advance RN0361 development in HTG patients based on a favorable safety profile and sustained triglyceride (TG) reduction in a 6-month single dose Phase I clinical trial.

Scope of the Oligonucleotides Pipeline Report

Coverage: Global
Oligonucleotides Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
Oligonucleotides Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
Oligonucleotides Therapeutics Assessment By Route of Administration: Implant, Inhalation, Intradermal, Intrahepatic, Intramuscular, Intrarectal, Intrathecal, Intratumoral, Intravenous, Intravesical, Intravitreal, Ophthalmic, Oral, Subcutaneous, Topical
Oligonucleotides Therapeutics Assessment By Mechanism of Action: ASO targeting lipoprotein, Angiotensinogen expression inhibitor, RNA interference, Complement C3 expression inhibitors, PKD1 modulator, and others
Key Oligonucleotides Companies: Novartis Pharmaceuticals, Sylentis, Amgen, Alnylam Pharmaceuticals, Kardigan, SanegeneBio, WaVe life Sciences, Rigerna Therapeutics, Eli Lily and Company, PYC Therapeutics, ADARx Pharmaceuticals, Denali Therapeutics, and others
Key Pipeline Oligonucleotides: Pelacarsen, Tivanisiran, Olpasiran, Zilebesiran, Tonlamarsen, SGB-9768, WVE-N531, RG002C0106, LY-3954068, PYC-003, ADX-626, DNL422 and others

Dive deep into rich insights for new oligonucleotides, visit @ Oligonucleotides Drugs

Table of Contents

1.	Oligonucleotides Pipeline Report Introduction
2.	Oligonucleotides Pipeline Report Executive Summary
3.	Oligonucleotides Pipeline: Overview
4.	Oligonucleotides Marketed Drugs
4.1.	IZERVAY: Astellas Pharma Inc.
4.2.	AMVUTTRA: Alnylam Pharmaceuticals
4.1.	Inmazeb: Regeneron Pharmaceuticals
5.	Oligonucleotides Clinical Trial Therapeutics
6.	Oligonucleotides Pipeline: Late-Stage Products (Pre-registration)
7.	Oligonucleotides Pipeline: Late-Stage Products (Phase III)
7.1.	Pelacarsen: Novartis Pharmaceuticals
8.	Oligonucleotides Pipeline: Mid-Stage Products (Phase II)
8.1.	WVE-N531: WaVe life Sciences
9.	Oligonucleotides Pipeline: Early-Stage Products (Phase I)
9.1.	PYC-003: PYC Therapeutics
10.	Oligonucleotides Pipeline: Preclinical and Discovery Stage Products
10.1.	DNL422: Denali Therapeutics
11.	Oligonucleotides Pipeline Therapeutics Assessment
12.	Inactive Products in the Oligonucleotides Pipeline
13.	Company-University Collaborations (Licensing/Partnering) Analysis
14.	Unmet Needs
15.	Oligonucleotides Market Drivers and Barriers
16.	Appendix

For further information on the oligonucleotides pipeline therapeutics, reach out @ Oligonucleotides Therapeutics

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