Oligo Insights: Domestic and Global Oligonucleotide Industry Updates – Issue 5, 2025

Jun 09, 2025 11:18 CST Updated 11:18

Ribo Life Science

Small Nucleic Acid Drug Developer

Qilu Pharmaceutical

Specialty Formulations and Active Pharmaceutical Ingredients (API) Developer

Synerk

Small Nucleic Acid Drug Developer

SANEGENEBIO

Small Nucleic Acid Drug Developer

Innovation in China

01 Ribo Life Science

Qilu PharmaceuticalQilu Pharmaceutical and Ribo Life Science Jointly Announce: PCSK9-Targeted Innovative Drug RBD7022 Completes Enrollment for Phase II Clinical Trial

May 7, 2025, China — Qilu Pharmaceutical and Ribo Life Science jointly announced that the Phase II clinical trial (registration number: CTR20244751) of their collaboratively developed PCSK9-targeted small nucleic acid drug RBD7022/QLC7401 has successfully completed the enrollment of all participants. The trial included a total of 204 patients with hyperlipidemia, aiming to further evaluate the drug’s safety, efficacy, and long-term lipid-lowering potential, laying the groundwork for subsequent pivotal clinical studies.。

RBD7022 is a GalNAc-siRNA drug developed by Ribo Life Science based on its proprietary intellectual property platform, RiboGalSTARTM. It is the second siRNA drug globally, after Leqvio, to enter clinical trials targeting PCSK9. The drug effectively and precisely inhibits the expression of the PCSK9 protein in the liver, providing long-term reduction of low-density lipoprotein cholesterol (LDL-C) levels. Currently, RBD7022 has completed Phase I clinical studies, demonstrating good safety, with clinical efficacy data meeting expectations. On December 15, 2023, Ribo Life Science licensed the rights to RBD7022 in mainland China, Hong Kong, and Macao to Qilu Pharmaceutical.。

02 Synerk

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

Synerk's siRNA Drug Completes First Dosing in Phase I Clinical Trial in China

On May 14, 2025, Synerk announced that SNK-2726, the first siRNA drug developed using its proprietary new nucleic acid drug delivery technology platform GalNexus, has received approval for clinical trial applications (IND) from both the Center for Drug Evaluation (CDE) of the National Medical Products Administration in China and the U.S. FDA. The drug has also successfully completed the enrollment and dosing of the first subject in a Phase I clinical trial in China.

SNK-2726 Targets Angiotensinogen (AGT), Achieving Long-Term Blood Pressure Control by Precisely Silencing the Expression of AGT mRNA in the Liver, Blocking the Activation of the Renin-Angiotensin-Aldosterone System (RAAS) at the Genetic Level, Offering a Promising New Treatment Option for Hypertensive Patients. Huadong Medicine has Reached a Strategic Cooperation with Synerk, and Huadong Medicine Holds Exclusive Rights for the Development, Registration, Manufacturing, and Commercialization of SNK-2726 in Greater China.

03 SANEGENEBIO

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

SANEGENEBIO's Another siRNA Drug for Complement Diseases Approved for Clinical Trials in China

On May 20, 2025, SANEGENEBIO announced that the clinical trial application for SGB-3383 Injection, a small nucleic acid (siRNA) drug independently developed by the company targeting CFB, has recently received tacit approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) (Acceptance No.: CXHL2500238). The drug is intended for the treatment of complement-mediated kidney diseases (including IgA nephropathy, C3 glomerulopathy, immune complex-mediated membranoproliferative glomerulonephritis, atypical hemolytic uremic syndrome, etc.).

SGB-3383 is an RNAi therapy targeting CFB, delivered to hepatocytes using SANEGENEBIO's uniquely innovative next-generation GalNAc liver delivery platform. It treats complement-mediated kidney diseases by inhibiting the expression of CFB in the liver through RNAi. Currently, there are no siRNA drugs targeting the complement system approved globally, and SGB-3383 is expected to become the first such drug in China and a global leader in targeting CFB.。

04 Synerk Pharmaceutical

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

Guowei Pharmaceutical Licenses Hypertension siRNA New Drug to Salubris

2On May 27, 2025, Chengdu Guowei Biopharmaceutical Co., Ltd. ("Guowei Pharmaceutical" for short) and Shenzhen Salubris Pharmaceuticals Co., Ltd. ("Salubris" for short) jointly signed a new drug research and development cooperation agreement. Guowei Pharmaceutical exclusively granted Salubris the rights related to the API and formulations of the in-house developed AGT-siRNA small interfering nucleic acid drug GW906 in the Chinese market, including but not limited to R&D, registration, production, and commercialization. According to the agreement, Guowei Pharmaceutical will receive an upfront payment and research and development milestone payments totaling up to 180 million yuan. After the product is approved and launched in the future, the cumulative sales milestones could reach up to 370 million yuan. Meanwhile, within the agreed period of the agreement, Guowei Pharmaceutical will receive a certain percentage of annual net sales as royalties.

GW906 is a small interfering ribonucleic acid (siRNA) drug independently developed by Guowei Pharmaceutical, targeting angiotensinogen (AGT). Its proposed indication is primary hypertension and it is currently undergoing Phase I clinical trials.

Overseas Hotspots

01 Arbutus

Arbutus Announces Phase 2a Combination Therapy Trial Data for Imdusiran

On May 7, 2025, Arbutus Biopharma Corporation presented the data from the Phase 2a clinical trial IM-PROVE II conducted in patients with chronic HBV infection at the 2025 European Association for the Study of the Liver (EASL) conference. The results showed that the combination of Imdusiran, VTP-300, and low-dose nivolumab can achieve functional cure in patients with chronic HBV infection. In terms of safety, the combination of Imdusiran, VTP-300, and low-dose nivolumab was well-tolerated, with no serious adverse events, deaths, or discontinuations of treatment reported. Additionally, no immune-related adverse events, including thyroid abnormalities, were observed.

Imdusiran is an RNA interference (RNAi) therapy specifically designed to reduce all HBV viral proteins and antigens, including the hepatitis B surface antigen, which is considered a key prerequisite for reawakening the patient's immune system to respond to the virus. Imdusiran uses Arbutus' novel covalently conjugated N-acetylgalactosamine (GalNAc) delivery technology to target liver cells, enabling subcutaneous delivery.

02 Ionis

IonIS Announces Positive Phase 3 Results for Antisense Oligonucleotide Therapy Olezarsen

On May 19, 2025, Ionis Pharmaceuticals announced positive topline results from the Phase 3 Essence clinical study of its "first-in-class" antisense oligonucleotide therapy Tryngolza (olezarsen). The study targeted patients with moderate hypertriglyceridemia (TG) with or without atherosclerotic cardiovascular disease (ASCVD) risk. Results showed that olezarsen significantly reduced patients' triglyceride levels after six months of treatment, meeting the primary endpoint. At six months, the 80 mg and 50 mg dose groups achieved TG reductions of 61% and 58%, respectively, compared to placebo (p<0.0001). Statistically significant improvements were also observed across all key secondary endpoints. Ionis plans to present the full data from this study at an upcoming scientific conference.

OLezarsen is an antisense oligonucleotide therapy designed to inhibit the body's production of apolipoprotein C-III (apoC-III, a protein produced in the liver that regulates triglyceride metabolism in the blood). In February 2024, the U.S. FDA granted this therapy Orphan Drug Designation and Breakthrough Therapy Designation (BTD), and in December approved it as an adjunct to dietary control to reduce triglycerides in adult patients with familial chylomicronemia syndrome.

03 Alnylam

Alnylam Announces Latest Phase 3 Clinical Trial Results for siRNA Therapy Vutrisiran

Alnylam Pharmaceuticals Announces Latest Analysis Results from Phase 3 HELIOS-B Trial Evaluating Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)

Vutrisiran is a subcutaneously administered siRNA therapy that blocks the production of both wild-type and mutant transthyretin by targeting and silencing specific mRNA. Designed based on Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate delivery platform, Vutrisiran increases potency and metabolic stability, allowing for infrequent subcutaneous injections. Previously, Vutrisiran received approval from the U.S. FDA for treating patients with wild-type or hereditary transthyretin-mediated ATTR-CM.

Selected Articles from Previous Issues

1. Small Nucleic Acid Drugs Overseas Market 2025 Outlook

For more industry information, please refer to:

OligoView Industry Dynamics

Clinical Progress of Small Nucleic Acid Drugs in China

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

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About Ouli Bio

Suzhou Oli Biopharmaceutical Technology 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 drug registration. Oli Bio'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: solid-phase nucleic acid synthesis, chemical modification and conjugation, process development and analysis, and CMC pharmaceutical research. It has also initiated 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 clients address critical challenges 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 clients 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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