Oligo Insights | Global and Domestic Oligonucleotide Industry Updates – Issue 6, 2025

Jul 10, 2025 10:23 CST Updated 10:23

Argo

RNAi Drug Developer

SANEGENEBIO

Small Nucleic Acid Drug Developer

Innovation in China

01 Argo

BW-00163 Enters Phase II Clinical Trial and Receives Milestone Payment

On June 5, 2025, Argo Biopharma announced that its hypertension treatment drug BW-00163 has entered Phase II clinical trials. This drug is a key component of the exclusive licensing agreement between Argo and Novartis. With this asset advancing to Phase II clinical development, Argo has received a milestone payment.

BW-00163 is the fourth drug developed based on Argo's new generation siRNA platform RADS to enter mid-stage clinical research. It efficiently degrades AGT gene mRNA through siRNA, reducing the production of angiotensin at its source and exerting a long-acting blood pressure lowering effect.

02 Livzon Pharmaceutical

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

Livzon Pharmaceutical's Long-Acting siRNA Drug for Gout Treatment Approved for Clinical Trials

On June 24, 2025, the official website of the Center for Drug Evaluation (CDE) under China's National Medical Products Administration (NMPA) announced that the investigational new drug YJH-012 injection, submitted by Livzon Pharmaceutical, has been approved for clinical trials. This drug, developed based on small interfering RNA (siRNA) technology, is a potential First-in-class small nucleic acid drug in the global gout treatment field, offering the possibility of a more effective and safer long-acting treatment option for patients with gout.

YJH-012 is a novel siRNA drug jointly developed by Livzon Pharmaceutical and Youjia Biotech. It utilizes a specific liver-targeted delivery system, GalNAc, to precisely deliver siRNA to the liver, the primary site of uric acid synthesis. A single dose of YJH-012 can achieve continuous uric acid reduction for 3-6 months. Preclinical validation: In cynomolgus monkey models, the uric acid-lowering effect lasted up to 180 days after a single dose (10 mg/Kg). Additionally, uric acid suppression increased steadily and was maintained long-term, avoiding the rapid drop in uric acid at the beginning of treatment that could lead to "crystal dissolution reactions" and trigger secondary gout. Compared with existing treatments, it demonstrates superior therapeutic potential.

03 SANEGENEBIO

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

SANEGENEBIO's siRNA Drug Targeting CFB for Complement-Related Diseases Completes First Dosing in Subjects

On June 30, 2025, SANEGENEBIO announced that the Phase I clinical study in China of its self-developed small nucleic acid (siRNA) drug SGB-3383 injection, targeting CFB, has recently completed the first subject dosing at Peking University Third Hospital. SGB-3383 is an siRNA drug targeting Complement Factor B (CFB), intended for treating complement-mediated kidney-related diseases.

SGB-3383 is a siRNA drug independently developed by SANEGENEBIO that targets CFB. It is delivered to hepatocytes using the company's uniquely innovative next-generation LEAD™ GalNAc liver delivery platform and inhibits the expression of CFB in the liver through RNAi, thereby treating complement-mediated kidney diseases. Preclinical trial data show that SGB-3383 can significantly and durably reduce the levels of CFB in the liver and blood, with good safety and tolerability. Currently, no siRNA drugs targeting the complement system have been approved globally, and SGB-3383 is expected to become the best-in-class, world-leading siRNA drug targeting CFB.

Overseas Hotspots

01 Arrowhead

ARO-ALK7 Initiates Phase 1/2a Clinical Trial

On June 2, 2025, Arrowhead announced that the phase 1/2a clinical trial of its investigational small interfering RNA (siRNA) therapy ARO-ALK7 for the treatment of obesity has dosed the first cohort of participants. According to Arrowhead, ARO-ALK7 is the first RNAi investigational therapy targeting fat tissue-expressed genes to enter clinical research.

ARO-ALK7 aims to silence the expression of the ACVR1C gene in adipocytes to reduce the production of Activin Receptor-Like Kinase 7 (ALK7), which acts as a receptor in pathways regulating energy homeostasis in adipose tissue. In preclinical studies, ARO-ALK7 inhibited ALK7 expression in adipose tissue, thereby reducing body weight and fat mass while preserving lean muscle mass.

02 Alnylam

Amvuttra EU Approved for Treating Wild-Type or Hereditary Transthyretin-Mediated Amyloidosis Cardiomyopathy (ATTR-CM)

On June 9, 2025, Alnylam Pharmaceuticals announced that the European Commission (EC) has approved its RNAi therapy Amvuttra (vutrisiran) for the treatment of patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). According to the press release, this approval makes Amvuttra the first RNAi therapy to receive EC approval for treating both ATTR-CM and hereditary transthyretin-mediated (hATTR) amyloidosis polyneuropathy.

Amvuttra (vutrisiran) is an RNAi therapy that rapidly knocks down both mutant and wild-type transthyretin (TTR), addressing the root cause of transthyretin amyloidosis (ATTR). Vutrisiran is administered quarterly via subcutaneous injection and has been approved in more than 15 countries/regions for the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. The drug is also approved in the United States and Brazil for the treatment of wild-type or hereditary ATTR amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

03 Ionis

ION582 Phase III Clinical Trial Completes First Dosing

On June 11, 2025, Ionis Pharmaceuticals announced that the first participant in the global Phase 3 REVEAL study has been dosed. This study aims to evaluate the efficacy and safety of ION582, an investigational drug for the treatment of Angelman syndrome (AS), a severe rare neurodevelopmental disorder that can cause significant physical and cognitive impairments.。

ION582 is an investigational ASO therapy administered via intrathecal injection through lumbar puncture, designed to suppress the expression of the UBE3A antisense transcript (UBE3A-ATS) and activate the paternal UBE3A allele, thereby increasing UBE3A protein production in the brain. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted ION582 orphan drug designation. Additionally, the FDA has awarded ION582 Fast Track and Rare Pediatric Disease designations.

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 publicly available information of various companies)

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In 2023, the company's 3,000 square meter GMP-compliant pilot platform officially began offering services, focusing on helping customers address key challenges such as the scale-up of small nucleic acid drug manufacturing processes 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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