Oligo Insights | Global and China Oligonucleotide Industry Update – Issue 12, 2025

Jan 16, 2026 12:18 CST Updated 12:18

SANEGENEBIO

Small Nucleic Acid Drug Developer

Rona Therapeutics

Nucleic Acid Drug Developer

Innovation in China

01 Pristine Derivatives

Rona Therapeutics' LDR2515 Injection Clinical Trial Application Accepted by CDE

On January 4, 2026, the Investigational New Drug (IND) application for LDR2515 injection, an siRNA drug for the treatment of obesity developed by Chengdu Xianyan Biotechnology Co., Ltd., was accepted by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China.

The INHBE gene is primarily expressed in the liver, and its encoded protein, Activin E, promotes lipid accumulation. Human genomic studies have shown that loss-of-function mutations in the INHBE gene improve waist-to-hip ratio, reduce abdominal fat, enhance metabolic health, and lower the risk of cardiovascular diseases and type 2 diabetes. Consistent with human genomic research, INHBE siRNA in obese mouse models achieves sustained weight loss without affecting food intake. Moreover, when combined with a GLP-1 receptor agonist, it enhances weight loss, mitigates muscle loss caused by the GLP-1 receptor agonist, and suppresses weight rebound after discontinuation of the GLP-1 receptor agonist. Preclinical studies indicate that LDR2515 is safe, effectively and persistently inhibiting INHBE expression in the liver through subcutaneous injection. It is expected to allow for dosing once every six months to one year in clinical settings, offering a new therapeutic option for high-quality weight loss and metabolic health.

02 SANEGENEBIO

SANEGENEBIO's SGB-7342 Approved for Clinical Trials in China to Treat Obesity

On December 16, 2025, SANEGENEBIO announced that its self-developed small interfering RNA (siRNA) candidate drug SGB-7342, targeting inhibin βE subunit (INHBE), has received the implied permission for clinical trial application from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) for the treatment of obesity.

SGB-7342 is an siRNA candidate drug targeting INHBE for the treatment of obesity, utilizing SANEGENEBIO's next-generation GalNAc conjugation technology. Its mechanism of action involves precisely silencing the expression of INHBE mRNA in the liver through RNAi technology, reducing the levels of its encoded protein, Activin E, thereby promoting fat breakdown without inducing muscle breakdown, ultimately improving metabolic abnormalities and insulin resistance. Preclinical trial data show that a single subcutaneous administration of SGB-7342 can achieve potent and sustained knockdown of liver INHBE mRNA, with significant weight loss, improved body composition, and the expected effect of maintaining muscle observed in animal models, while demonstrating good safety and tolerability.

03 Rona Therapeutics

Rona Therapeutics' Dual-Target siRNA Drug Enters Clinical Development

On December 17, 2025, Rona Therapeutics announced that the company has successfully submitted a clinical trial application for RN5681 to the Australian Human Research Ethics Committee (HREC), advancing the company’s first dual-target siRNA into the clinical development stage. This Phase I clinical trial is expected to begin dosing in the first quarter of 2026.

RN5681 is a GalNAc-conjugated dual-targeting siRNA that can simultaneously silence PCSK9 and LPA, two genetically validated complementary drivers of atherosclerotic cardiovascular disease. This drug, with a single molecule, can synchronously reduce low-density lipoprotein cholesterol and lipoprotein (a), achieving potent and long-lasting lipid regulation, thereby addressing persistent residual risks that current therapies struggle to eliminate.

04 YooYoung Pharmaceuticals

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

YKYY032 Injection from Yoku Pharma Receives Clinical Trial Approvals from China NMPA and U.S. FDA

On December 19, 2025, Beijing Yookon Sci-Tech Innovation Pharmaceutical Technology Co., Ltd. and Hangzhou Tianlong Pharmaceutical Co., Ltd., subsidiaries of Yookon Pharmaceutical Group Co., Ltd., received the "Drug Clinical Trial Approval Notice" issued by the NMPA for the use of YKYY032 injection in treating hyperlipoproteinemia(a). Yookon Sci-Tech Innovation recently received a Study May Proceed Letter from the FDA approving the clinical trial of YKYY032 injection for treating hyperlipoproteinemia(a) (IND Number: 178774).

YKYY032 Injection is a chemically synthesized double-stranded siRNA drug conjugated with an N-acetylgalactosamine (GalNAc) ligand. It specifically silences the mRNA transcribed from the LPA gene through the RNA interference mechanism, blocking the production of Lp(a) at its source, and is intended for the treatment of hyperlipoproteinemia(a). Preclinical studies have shown that the drug exhibits significant pharmacological activity both in vitro and in vivo, effectively reducing LPAmRNA and Lp(a) protein levels in various animal models. In non-human primates, it also leads to a reduction in LDL-C and ApoB levels, demonstrating good and long-lasting lipid-lowering effects. Repeat-dose toxicity studies have confirmed its excellent safety and tolerability.

Overseas Hotspots

01 Wave

Wave Life Sciences Announces Positive Interim Data for siRNA Weight Loss Drug

On December 8, 2025, Wave Life Sciences announced the interim data from the Phase I INLIGHT trial of its obesity candidate drug WVE-007. The data, sourced from 32 subjects who received a single 240mg subcutaneous injection, showed a 9.4% reduction in visceral fat, a 4.5% decrease in total body fat, and a 3.2% increase in lean body mass after 12 weeks. This outcome provides significant support for WVE-007's potential dosing regimen of once or twice per year. The company is currently planning to initiate a Phase II clinical trial and expects to release additional clinical data updates in the first quarter of 2026, including six-month follow-up data from the 240 mg single-dose cohort and three-month follow-up data from the 400 mg single-dose cohort.

WVE-007 is an RNA interference drug based on GalNAc-siRNA technology that precisely silences INHBE gene mRNA in the liver, thereby reducing the expression level of its downstream protein product, Activin E. The selection of this target is based on strong human genetic evidence: individuals carrying protective inactivating variants of the INHBE gene exhibit healthier body composition and lower cardiometabolic disease risk, including reduced visceral fat and decreased incidence of type 2 diabetes and cardiovascular diseases.

02 Dyne

Dyne Therapeutics Announces Positive Results for DMD Drug

Dyne Therapeutics announced on December 9 that its investigational therapy zeleciment rostudirsen achieved positive topline results in the registrational expansion cohort (REC) of the Phase 1/2 DELIVER trial for patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Dyne stated that it is on track to submit an application for accelerated approval to the U.S. FDA in the second quarter of 2026.

Zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) is composed of a phosphorodiamidate morpholino oligomer (PMO) conjugated with an antibody fragment (Fab) targeting transferrin receptor 1 (TfR1). TfR1 is highly expressed in muscle tissue. This therapy aims to achieve targeted delivery to muscle tissue, promoting exon skipping inside and outside the nucleus, thereby enabling muscle cells to produce truncated but functional dystrophin protein, with the goal of halting or reversing disease progression. The therapy has previously been granted Fast Track designation, Orphan Drug designation, Rare Pediatric Disease designation, and Breakthrough Therapy designation by the FDA.

03 GSK

GSK's World-First Hepatitis B Drug Successfully Completes Two Phase III Studies

GSK Announces Positive Results from Two Pivotal Phase 3 Clinical Trials, B-Well 1 and B-Well 2, for Bepirovirsen in Chronic Hepatitis B Treatment

Bepirovirsen is an antisense oligonucleotide (ASO) therapy that GSK licensed from Ionis. It aims to inhibit the replication of hepatitis B virus DNA, thereby reducing the levels of hepatitis B surface antigen (HBsAg) in the blood and stimulating the immune system to produce a durable response.

Selected Articles from Previous Issues

1. Small Nucleic Acid Drugs Overseas Market 2025 Outlook

For more industry information, please refer to:

Oligene Industry Dynamics

Clinical Progress of Small Nucleic Acid Drugs in China

(According to the information compiled from the official websites and public data of various companies,As of January 15, 2026）

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About Oulibio

OliBio (Suzhou) Pharmaceutical Technology 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. OliBio's 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: nucleic acid solid-phase synthesis, chemical modification and conjugation, process development and analysis, as well as CMC pharmaceutical research. It has also fostered in-depth collaborations with multiple pharmaceutical enterprises and biotechnology companies both domestically and internationally.

In 2023, the company's 3,000-square-meter GMP-standard pilot platform officially began offering services, focusing on helping customers address "bottleneck" issues such as small nucleic acid drug process scale-up and CMC pharmaceutical research. The company will continue to provide compliant, high-quality, reliable, and efficient services to help customers enhance R&D efficiency, accelerate 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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