Oligo Insight | Global and China Oligonucleotide Industry Dynamics – Q3 2025

Apr 09, 2025 14:07 CST Updated 14:07

Visirna

siRNA Drug Developer

SANEGENEBIO

Small Nucleic Acid Drug Developer

Sirius Therapeutics

Oligonucleotide Drug Developer

Innovation in China

01 Visirna

Visirna Announces Positive Results from Phase 3 Clinical Trial of VSA001 Injection in Chinese Patients with Familial Chylomicronemia Syndrome

On March 17, 2025, Visirna Biotechnology (Suzhou) Co., Ltd. ("Visirna") announced that the Phase 3 clinical trial (CTR20231418/NCT05902598) conducted by Visirna for Chinese patients with Familial Chylomicronemia Syndrome (FCS) achieved positive topline data, successfully meeting the primary efficacy endpoint and all key secondary endpoints.

VSA001 (Plozasiran), previously also known as ARO-APOC3, is a first-in-class RNA interference (RNAi) therapeutic drug designed to reduce the production of apolipoprotein C-III (APOC3). APOC3 is a component of triglyceride-rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. APOC3 increases triglyceride levels in the blood by inhibiting the breakdown of TRLs by lipoprotein lipase and the uptake of TRL remnants by liver receptors. VSA001 reduces triglycerides and normalizes lipid levels by lowering APOC3 levels.

VSA001 is conducting a series of studies in the SUMMIT clinical program, including the PALISADE Phase 3 study for FCS patients, the SHASTA study for SHTG patients, and the MUIR and CAPITAN studies for patients with mixed hyperlipidemia.

02 SANEGENEBIO

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

SANEGENEBIO's C3-Targeted siRNA Drug SGB-9768 Initiates Phase II Clinical Trial

On March 7, 2025, SANEGENEBIO announced that the company's self-developed small nucleic acid (siRNA) drug SGB-9768 injection targeting complement C3 has officially launched Phase II clinical trials in China. This study is led by Peking University First Hospital and conducted across multiple centers in China.

SGB-9768 is a siRNA drug independently developed by SANEGENEBIO that targets the Complement 3 (C3) protein. It utilizes the company's uniquely innovative next-generation LEAD™ GalNAc technology for delivery to liver cells, inhibiting the synthesis of C3 protein via the RNAi mechanism, thereby suppressing complement activation. It is intended for treating complement-mediated kidney diseases, including adult IgA nephropathy, C3 glomerulopathy, and immune complex-mediated membranoproliferative glomerulonephritis.

03 Sirius Therapeutics

Sirius Therapeutics' siRNA Drug SRSD216 Approved for New Drug Clinical Trial Application

2On March 20, 2025, Sirius Therapeutics announced that the National Medical Products Administration (NMPA) had approved its Investigational New Drug (IND) application to initiate the first-in-human Phase I clinical trial of SRSD216. SRSD216 is an innovative small interfering nucleotide (siRNA) therapy designed to treat hyperlipoproteinemia(a).

SRSD216 Injection is a novel double-stranded small interfering ribonucleic acid (siRNA). It specifically modulates the LPA gene, reducing hepatic Apo(a) production and lowering circulating Lp(a) levels. Preclinical in vivo studies have shown that a single dose resulted in an almost 100% reduction in Lp(a) levels, with effects lasting over six months, and no significant safety events were observed.

Overseas Hotspots

01 Ionis

Ionis and Ono Reach Global Licensing Agreement for Sapablursen to Treat Polycythemia Vera

March 11, 2025 — Ionis and Ono Pharmaceutical Co., Ltd. announced that they have entered into a licensing agreement granting Ono exclusive global development and commercialization rights for sapablursen.

Sapablursen is an investigational RNA-targeted therapeutic agent for the treatment of polycythemia vera (PV), a rare and potentially life-threatening blood disorder. Sapablursen is currently being evaluated for its efficacy in adult patients with PV in the fully enrolled Phase 2 IMPRESSION study. In 2024, sapablursen received Fast Track designation and Orphan Drug designation from the U.S. Food and Drug Administration (FDA).

Currently, sapablursen has completed patient enrollment for the Phase II IMPRESSION study and received Fast Track designation and Orphan Drug status granted by the FDA in 2024.

02 Alnylam

FDA Approves AlnylamvutrisiranFor ATTR-CM

On March 20, 2025, Alnylam announced that the U.S. FDA had approved the supplemental New Drug Application (sNDA) for its RNAi therapeutic vutrisiran (brand name AMVUTTRA®) for the treatment of transthyretin amyloidosis cardiomyopathy (ATTR-CM).

AMVUTTRA® is an RNAi therapy for the treatment of adults with wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) cardiomyopathy, and it is currently the first and only FDA-approved treatment for adult ATTR-CM and hereditary transthyretin-mediated amyloidosis (hATTR-PN) polyneuropathy.

PreviouslyAMVUTTRA®It was approved in 2022 for the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR-PN) and has been approved and launched in more than 15 countries/regions. This approval further expands its indications to include ATTR-CM.

03 Sanofi

Sanofi's siRNA Therapy Receives FDA Approval for Market Launch, Treating Hemophilia

On March 28, 2025, Sanofi announced that its siRNA therapy Qfitlia (Fitusiran) had been approved by the U.S. FDA for marketing. It is used to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A or B.

Qfitlia (fitusiran) is a small interfering RNA (siRNA) therapy that works by reducing antithrombin, thereby promoting thrombin generation to achieve hemostatic balance. The drug is administered via subcutaneous injection (50mg pre-filled injection pen) and only requires six injections per year.

Qfitlia was discovered by Alnylam Pharmaceuticals and licensed to Sanofi for global development and commercialization in 2014. Regulatory submissions for Qfitlia have been completed in China and Brazil, with a regulatory decision expected in China in the second half of 2025.

04 Lilly

Lilly's Long-Acting siRNA Therapylepodisiran Phase II Study Results Released: Lp(a) Levels Reduced by 94%!

On March 30, 2025, Eli Lilly announced positive results from the Phase II ALPACA study of its small interfering RNA (siRNA) therapy, lepodisiran. The therapy aims to reduce the production of lipoprotein(a) (Lp(a)), a genetic risk factor for cardiovascular disease. In the ALPACA study, lepodisiran at the highest dose (400mg) reduced Lp(a) levels by an average of 93.9% during the 60 to 180 days post-treatment period, meeting the primary endpoint, with some patients experiencing reductions lasting nearly 1.5 years. Participants receiving lepodisiran doses of 16mg and 96mg saw their Lp(a) levels decrease by 40.8% and 75.2%, respectively, during the same period.

Selected Articles from Previous Issues

1. Small Nucleic Acid Drugs Overseas Market 2025 Outlook

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Oli Biopharmaceuticals (Suzhou) Co., Ltd. is a professional medicinal 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 pharmaceutical registration. The technical team of Oli Bio comes from one of the earliest teams in China that started the process development and CMC research for nucleic acid drugs, possessing extensive experience in project development and product registration. Currently, the company has established four industry-leading core technology platforms: nucleic acid solid-phase synthesis, chemical modification and conjugation, process development and analysis, as well as CMC pharmaceutical research. It has also engaged in in-depth collaborations with multiple pharmaceutical companies and biotechnology firms both domestically and internationally.

In 2023, the company's 3,000-square-meter GMP-standard pilot platform officially began offering services, focusing on helping clients 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 compliant, high-quality, reliable, and efficient services to help clients enhance R&D efficiency, expedite product registration and market entry, and jointly support the entire process of small nucleic acid drugs from preclinical to commercial production.

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