Oligo Insights | Global and China Oligonucleotide Industry Update – Issue 9, 2025

Oct 17, 2025 09:59 CST Updated 09:59

China Resources Double-Crane

Chemical Pharmaceutical Preparations Manufacturer

Mabwell

Innovative Biopharmaceutical Company

Aditum Bio

Developer and Investor in Biopharmaceutical R&D Technology

Argo

RNAi Drug Developer

Innovation in China

01 Xuanjing Bio

Argo's First siRNA Drug Completes First Patient Dosing in Phase II Clinical Trial

On September 10, 2025, Xuanjing Bio announced that the Phase II clinical trial of its self-developed innovative drug RG002C0106 injection for the treatment of complement-mediated diseases has recently completed the enrollment and dosing of the first patient.

RG002C0106 is an siRNA drug independently developed by Xuanjing Biotechnology based on its self-established small nucleic acid drug technology R&D platforms such as RIHOST®, RICMO®, and LICOD®. It is one of the world's first siRNA drugs targeting complement factor C3 to enter clinical trials, demonstrating good safety and tolerability in Phase I clinical trials.

In addition, Xuanjing Biotech and China Resources Double-Crane have officially signed a cooperation agreement to jointly develop the innovative small nucleic acid drug RG008, focusing on the field of chronic metabolic diseases, and are committed to providing patients with more breakthrough treatment options.

02 Mabwell

Shenji Changhua Collaborates with Mr. Cai Lei to Build a Joint Laboratory, Aiding ALS Drug Development

Mabwell Dual-Target siRNA Innovative Drug NewCo Goes Overseas

On September 17, 2025, Mabwell and Aditum Bio Fund 3, L.P. announced the establishment of Kalexo Bio, Inc., and entered into an exclusive global licensing agreement for 2MW7141, a dual-target siRNA innovative drug in the cardiovascular field. Aditum Bio will provide funding to Kalexo Bio, which will collaborate with Mabwell to advance the development of 2MW7141.

According to the agreement, Mabwell grants Kalexo Bio exclusive global rights for the development, manufacturing, and commercialization of 2MW7141. Mabwell is entitled to receive up to $1 billion in upfront and milestone payments, as well as tiered royalties. This includes a one-time, non-refundable upfront payment and a near-term cash payment totaling $12 million. As part of the consideration, Mabwell will also receive a certain equity stake in Kalexo Bio under agreed conditions.

2MW7141 is a dual-target small nucleic acid drug independently developed by Mabwell, currently in the preclinical stage. It mainly focuses on lipid regulation for populations with dyslipidemia and the prevention of high-risk cardiovascular events. 2MW7141 demonstrates a clear synergistic regulatory effect, showing potent and long-lasting inhibition of target genes in preclinical studies, with a low risk of off-target effects.

03 Argo

Argo Biopharma's siRNA New Drug Receives NMPA Approval for Phase II Clinical Trial

On September 15, 2025, Argo Biopharma ("Argo," "the Company") announced that its investigational small interfering RNA (siRNA) drug BW-40202 has received clinical trial approval from the National Medical Products Administration (NMPA) of China to initiate a Phase II clinical study for paroxysmal nocturnal hemoglobinuria (PNH). This study is expected to commence in January 2026. BW-40202 is a drug targeting complement factor B (CFB) for the treatment of PNH and other complement-mediated diseases. BW-40202 is also being evaluated in clinical trials for IgA nephropathy (IgAN), with the company having received NMPA approval in June 2025 to conduct a Phase I/IIa clinical trial for IgAN.

BW-40202 is an siRNA therapy targeting CFB mRNA. It inhibits the expression of CFB mRNA through the RNA interference (RNAi) mechanism, thereby reducing serum CFB protein levels and suppressing the activity of the complement alternative pathway (CAP). Preclinical studies have shown that BW-40202 exhibits excellent pharmacological activity, significantly and persistently lowering serum CFB protein levels, effectively inhibiting CAP activity over the long term, and demonstrating favorable safety characteristics.

04 Daring Bio

Shenji Changhua Collaborates with Mr. Cai Lei to Establish a Joint Laboratory, Aiding ALS Drug Development

Daru Biopharma's siRNA New Drug RN3161 Australia Clinical Trial Application Accepted

On September 15, 2025, Rona Therapeutics announced that the company had successfully submitted a clinical trial application for its self-developed RN3161 to the Australian Human Research Ethics Committee (HREC). RN3161 is a GalNAc-siRNA targeting INHBE, developed on the self-developed GAIA™ platform, aiming to provide a replicable and sustainable high-quality weight loss solution for overweight and obese populations.。

RN3161, developed based on Argo's self-developed GAIA™ platform, can reduce fat while maintaining lean body mass by precisely silencing INHBE, thereby differentiating itself from existing treatment methods. Its optimized GalNAc delivery design combined with chemical modifications reduces off-target risks. In monkey models, a single low-dose administration resulted in over 90% downregulation of INHBE mRNA expression, demonstrating leading performance and durability. Preclinical studies show that RN3161 achieves gene silencing effects lasting more than six months, potentially reducing dosing frequency to just 1–2 times per year, significantly improving patient compliance and long-term management value.

05 Yuhang Pharmaceutical

Shenji Changhua Collaborates with Mr. Cai Lei to Build a Joint Laboratory, Aiding in ALS Drug Development

YKYY013 Injection from Yuekang Pharmaceutical Receives FDA Approval for Clinical Trials in the United States

On September 22, 2025, Beijing Yooking Biotechnology Co., Ltd. (hereinafter referred to as "Yooking Biotech"), a subsidiary of Yooking Pharmaceutical Group Co., Ltd., received a Study May Proceed Letter from the U.S. Food and Drug Administration (hereinafter referred to as "FDA") approving the clinical trial of YKYY013 Injection for the treatment of chronic hepatitis B virus infection (IND Number: 177486).

YKYY013 Injection is developed by Yuekang Innovation andYuekang PharmaceuticalA chemically synthesized double-stranded siRNA drug conjugated with an N-acetylgalactosamine ligand, independently developed by Hangzhou Tianlong Pharmaceutical Co., Ltd., a wholly-owned subsidiary. The drug effectively silences messenger RNA (mRNA) transcribed from the hepatitis B virus (HBV) genome through RNA interference, thereby inhibiting the production of HBV antigen proteins, suppressing HBV replication, and creating conditions for host immune reconstitution, ultimately achieving functional cure of hepatitis B. It is clinically intended for the treatment of chronic hepatitis B virus infection.

Overseas Hotspots

01 Avidity

Del-zota Announces Data from Two Key Clinical Trials

On September 10, 2025, Avidity Biosciences announced positive new data from its investigational therapy delpacibart zotadirsen (del-zota) in the EXPLORE44 and EXPLORE44-OLE trials.These data indicate that DMD patients who have been continuously receiving del-zota treatment for one year have shown a reversal of disease progression across multiple functional indicators, and demonstrated unprecedented improvements compared to baseline and the natural course of the disease. Avidity plans to submit a Biologics License Application (BLA) for del-zota to the U.S. FDA by the end of 2025.

Del-zota, composed of a monoclonal antibody targeting Type 1 Transferrin Receptor (TfR1) and an oligonucleotide conjugate that promotes exon 44 skipping in the mRNA precursor encoding dystrophin, is highly expressed in muscle tissue. This therapy works by altering the mRNA splicing process to produce a protein with functionality nearly identical to normal dystrophin. The monoclonal antibody targeting TfR1 enhances muscle-specific delivery of the oligonucleotide by binding to receptors on the surface of muscle cells. In July 2025, this therapy received Breakthrough Therapy Designation from the U.S. FDA for treating DMD patients suitable for exon 44 skipping therapy.

02 Ionis

EU Approves Tryngolza (olezarsen) for the Treatment of Familial Chylomicronemia Syndrome

On September 19, 2025, Ionis Pharmaceuticals and Sobi jointly announced that Tryngolza® (olezarsen) has been approved by the European Union as a dietary adjunct for the treatment of adult patients with genetically confirmed familial chylomicronemia syndrome (FCS).

Olezarsen is an antisense oligonucleotide-N-acetylgalactosamine 3 (ASO-GalNAc3) conjugate that binds to apolipoprotein C-III (apoC-III) mRNA, leading to mRNA degradation and thereby reducing serum apoC-III protein. The reduction of apoC-III protein can increase the clearance of plasma triglycerides and very low-density lipoproteins.

03 Alnylam

First Patient Dosed in Global Phase III Trial of Hypertension siRNA Therapy Drug Zilebesiran

On October 1, 2025, Alnylam Pharmaceuticals announcedThe pivotal global Phase III cardiovascular outcomes trial (CVOT) "ZENITH" (full name: Zilebesiran Cardiovascular Outcome Study in Hypertension) has completed the first patient dosing. This trial aims to evaluate the potential of the investigational twice-yearly subcutaneous injectable RNAi therapeutic Zilebesiran to reduce the risk of major adverse cardiovascular events in patients with uncontrolled hypertension. Alnylam and Roche jointly announced the global ZENITH CVOT trial plan.

Zilebesiran is an investigational RNAi therapy administered subcutaneously being developed for the treatment of hypertension.By targeting angiotensinogen (AGT) expressed in the liver—the most upstream precursor substance of the renin-angiotensin-aldosterone system (RAAS), intervening in blood pressure regulation mechanisms and pathways related to heart and kidney health.

Selected Articles from Previous Issues

1. Small Nucleic Acid Drugs Overseas Market 2025 Outlook

For more industry information, please refer to:

OligoView Industry News

Clinical Progress of Small Nucleic Acid Drugs in China

(Source: Compiled from official websites and public information of various companies)

Welcome to contact us at any time for high-quality and efficient oligonucleotide (conjugate) drug process development services, and customize your cost-reduction and efficiency-enhancement solutions.

About Ouli Bio

Oli Biopharmaceuticals (Suzhou) Co., Ltd. is a professional pharmaceutical nucleic acid CRDMO company that provides customers with “one-stop” services ranging from nucleic acid drug discovery, laboratory R&D, process and analytical development, CMC services, API production, to drug registration. Oli Biopharmaceuticals’ technical team originates from one of the earliest groups in China to engage in the development of nucleic acid drug manufacturing processes and CMC research, possessing extensive experience in project development and product registration. Currently, the company has established four industry-leading core technology platforms: solid-phase nucleic acid synthesis, chemical modification and conjugation, process development and analysis, as well as CMC pharmaceutical research. It has also initiated in-depth collaborations with multiple domestic and international pharmaceutical companies and biotechnology firms.

In 2023, the company's 3,000-square-meter pilot platform compliant with GMP standards officially began offering services to external clients, focusing on helping customers address critical challenges such as the scale-up of small nucleic acid drug production processes and CMC pharmaceutical research. The company will continue to provide services that are compliant, high-quality, reliable, and efficient, assisting clients in enhancing R&D efficiency, accelerating product registration and market entry, and jointly supporting the entire process of small nucleic acid drugs from preclinical to commercial production.

Contact Us

Email: order@olipharma.com

Phone: 15336788818

Official Website: www.olipharma.com

R&D Center: No. 168, Yuanfeng Road, Yushan Town, Kunshan City, Jiangsu Province

Production Base: Building C3, Phase I Factory, No. 999 Yishanhu Road, Guoxiang, Wuzhong District, Suzhou City, Jiangsu Province

Common Technology Platform: No. 202, 2nd Floor, Building 2, Yard 21, Baocan South Street, Beijing Daxing District Biomedical Industry Base