Oligonucleotide Industry Dynamics Brief — November 2025 Issue

Dec 11, 2025 17:16 CST Updated 17:16

Visirna

siRNA Drug Developer

SANEGENEBIO

Small Nucleic Acid Drug Developer

Innovation in China

01 Visirna

Visirna VSA012 Injection New Indication Approved for Clinical Trial

On November 27, 2025, according to the announcement on the CDE official website, the IND application for Visirna's VSA012 injection was approved for the treatment of complement-related kidney diseases, including but not limited to lupus nephritis, IgA nephropathy, C3 glomerulopathy, immune complex-mediated membranoproliferative glomerulonephritis, and atypical hemolytic uremic syndrome (aHUS). Previously, the VSA012 injection had been approved in China for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

VSA012 is Visirna's first double-stranded small interfering RNA (siRNA) developed based on its proprietary intellectual property siRNA technology platform. Conjugated with N-acetylgalactosamine (GalNAc), it specifically targets complement factor B (CFB) mRNA in the liver, inhibiting the expression of CFB protein in the bloodstream. Preclinical data show that VSA012 can achieve long-term inhibition of hepatic CFB mRNA expression, significantly reducing CFB protein levels and alternative complement pathway activity in the blood, while demonstrating good safety.

02 Dongyang Light Medicine

HEC Pharm's HECN30227 Approved for Clinical Trials

On November 1, 2025, according to the latest disclosure on the CDE's official website, HECN30227, a Class 1 new drug applied for by Dongyangguang Pharmaceutical, has been approved for clinical trials with the proposed indication being chronic hepatitis B. As per Dongyangguang Pharmaceutical's public data, this is its self-developed siRNA therapy.

HECN30227 is the first siRNA drug developed by Dongyang Light Pharmaceutical based on its small nucleic acid technology platform. It can simultaneously eliminate hepatitis B surface antigen (HBsAg) derived from cccDNA and intDNA. Preclinical data show that this product has pan-genotypic activity, can efficiently reduce HBsAg levels, and maintains significant efficacy against nucleoside drug-resistant strains. The drug adopts Dongyang Light Pharmaceutical's uniquely designed HEC-GalNova (N-acetylgalactosamine) liver-targeted delivery system, which achieves precise and efficient liver delivery while significantly reducing off-target risks. Additionally, Dongyang Light Pharmaceutical is concurrently developing a triple combination therapy of “siRNA+ASO+immune modulator,” which comprehensively inhibits HBV and HBsAg through multi-target synergy and initiates a new era of “functional cure” for hepatitis B by rebuilding immunity, bringing new hope to patients.

03 SANEGENEBIO

SANEGENEBIO Partners with Eli Lilly for RNAi Drug Development Strategic Collaboration

On November 8, 2025, SANEGENEBIO announced a global research and development collaboration and licensing agreement with Eli Lilly and Company ("Lilly"). The two parties will jointly advance the development of RNAi drug candidates targeting metabolic diseases based on SANEGENEBIO's proprietary LEAD™ platform.

LEAD™ (Ligand and Enhancer Assisted Delivery, a novel ligand and enhancer co-delivery) platform is a tissue-selective delivery technology independently developed by SANEGENEBIO. It enables efficient and specific delivery of RNAi drugs to extrahepatic tissues and cells. Based on this platform, there is potential to develop a breakthrough therapy for metabolic diseases that requires only two subcutaneous injections per year. SANEGENEBIO will be responsible for screening and identifying the optimal RNAi active molecules for the collaborative projects between both parties using the LEAD™ platform, while Eli Lilly and Company will oversee subsequent IND-enabling studies, clinical development, and commercialization of the drug.

04 CSPC Pharmaceutical Group

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

CSPC Group's SYH2061 Injection Approved for Clinical Trials in the United States

On November 24, 2025, CSPC Pharmaceutical Group (1093.HK) announced that its self-developed chemical Class 1 new drug, a double-stranded small interfering RNA (siRNA) drug (SYH2061 Injection) (hereinafter referred to as "the product"), has been approved by the U.S. Food and Drug Administration (FDA) for clinical trials in the United States. The product was also approved by the National Medical Products Administration of the People's Republic of China in October 2025 to conduct clinical trials in China.

SYH2061 is a siRNA drug that achieves liver-targeted delivery through conjugation with N-acetylgalactosamine (GalNAc). Administered subcutaneously, it targets complement protein C5 (C5) and effectively reduces C5 levels. By optimizing sequence design and chemical modifications, the product achieves more durable gene silencing effects. It is China's first domestically developed ultra-long-acting siRNA drug to lower C5 levels that has entered clinical trials, applicable for treating IgA nephropathy and other complement-mediated related diseases.

Overseas Hotspots

01 Arrowhead

Plozasiran Approved for Marketing by U.S. FDA

On November 18, 2025, Arrowhead announced that the U.S. FDA had approved the new drug application for its RNAi therapy, Plozasiran Sodium Injection (Plozasiran), which can be used as an adjunct to dietary therapy to reduce triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS). This is the first and currently the only siRNA drug approved by the FDA for the treatment of FCS, allowing patients to self-administer at home with a simple subcutaneous injection once every three months.

The sodium injection of volanesorsen is a "first-in-class" RNAi therapy targeting the APOC3 gene. It is developed based on Arrowhead's proprietary and differentiated Targeted RNAi Molecule (TRiM) platform, aiming to reduce the synthesis of apolipoprotein C-III (APOC3). The therapeutic goal of volanesorsen sodium injection is to effectively control triglycerides and restore blood lipid levels to a more normal range by lowering APOC3 levels. Previously, volanesorsen sodium injection for the treatment of FCS has been granted Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation by the U.S. FDA, as well as Orphan Drug Product Designation by the European Medicines Agency.

02 Ionis

Zilganersen Receives Breakthrough Therapy Designation

On December 3, 2025, Ionis Pharmaceuticals announced that the U.S. FDA had granted Breakthrough Therapy Designation to its investigational antisense oligonucleotide (ASO) therapy, zilganersen (ION373), for the treatment of Alexander Disease (AxD). Ionis plans to submit a New Drug Application (NDA) in the first quarter of 2026.

ION373 is an ASO drug candidate developed by Ionis, designed to inhibit the excessive expression of GFAP protein caused by pathogenic variants in the GFAP gene, thereby slowing or stabilizing disease progression in patients with AxD. Previously, ION373 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the FDA, as well as Orphan Drug Designation by the European Medicines Agency (EMA).

03 Tangram

TGM-312 Initiates Phase 1/2 Clinical Trial Application in the UK

On December 3, 2025, Tangram Therapeutics announced that it had submitted a Clinical Trial Application (CTA) to the UK Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial of its lead candidate drug, TGM-312. Subject to regulatory approval, the study is expected to commence in the UK in early 2026, with preliminary data anticipated to be released in the second half of 2026.

TGM-312 is a GalNAc-conjugated small interfering RNA (GalNAc-siRNA) developed based on Tangram Therapeutics' proprietary RNAi chemistry platform, GalOmic. It aims to specifically silence a novel target gene in hepatocytes for the treatment of Metabolic Dysfunction-Associated Steatohepatitis (MASH). This drug is expected to enable once-quarterly subcutaneous administration, enhancing patient convenience.

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: Information compiled from official websites and public sources of various companies)

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