Oligo Insights | Global and China Oligonucleotide Industry Update – Q2 2025
AusperBio
Biological Vaccine and Nucleic Acid Drug Developer
BRILLIANT PHARMACEUTICALS
A High-Quality Developer, Manufacturer, and Distributor of Pharmaceuticals
Ribo Life Science
Small Nucleic Acid Drug Developer
Innovation in China
01 Emma Life Science
AusperBio's Novel siRNA Drug ER2001 Successfully Enrolls First Patient in Phase 1 Clinical Trial in China
On the morning of February 11, 2025, the first dose of ER2001 injection, a novel nucleic acid drug developed by Aimax Biotechnology (Nanjing) Co., Ltd., was successfully administered at the Department of Neurology, First Affiliated Hospital of Sun Yat-sen University, with good clinical conditions.The study adopts a two-phase design with dose escalation and dose expansion, planning to enroll a total of 15-27 participants, including approximately 10 subjects from the neurology department of the hospital, to evaluate the safety and preliminary efficacy of ER2001 injection.。
The Phase I clinical study of ER2001 injection was granted the drug clinical trial approval notice by the National Medical Products Administration (NMPA) in early June 2024, and has successfully passed the review and approval of the Ethics Committee of the First Affiliated Hospital of Sun Yat-sen University to proceed with the clinical research.
02 AusperBio
AusperBio's Hepatitis B Curative Drug AHB-137 Completes Dosing in Phase IIb Clinical Trial and Officially Unblinded
February 12, 2025 - AusperBio announced: The core research product, antisense oligonucleotide (ASO) candidate drug AHB-137, has achieved significant clinical progress, marking another solid step towards the functional cure of chronic hepatitis B (CHB).
AHB-137, a non-conjugated antisense oligonucleotide (ASO) with the potential to become a cornerstone for functional cure of chronic hepatitis B, is the first innovative drug from AusperBio's self-developed platform Med-Oligo™ to enter clinical trials. Its excellent Phase I and Phase IIa clinical data gained widespread attention at the 2024 EASL™ and AASLD™ conferences.
Currently, AHB-137 is simultaneously conducting a global multi-center Phase I clinical trial and three Phase II clinical trials in China. Through a global strategic layout, it aims to accelerate the goal of achieving functional cure for hepatitis B.
03 BRILLIANT PHARMACEUTICALS
BRILLIANT PHARMACEUTICALS siRNA DrugBPR-30221616About to Launch Clinical Trials
On February 20, 2025, BRILLIANT PHARMACEUTICALS announced that its self-developed Class 1 siRNA new drug, BPR-30221616 Injection, has officially received approval from the Center for Drug Evaluation of the National Medical Products Administration. It will be used to treat transthyretin amyloid cardiomyopathy (ATTR-CM), with plans to initiate clinical trials in the near future.
04 Ribo Life Science
Ribo Life Science siRNA DrugRBD2080Approved for Clinical Use in Australia to Treat Autoimmune Diseases
On February 26, 2025, Suzhou Ribo Life Science Co., Ltd. announced that its self-developed siRNA drug RBD2080 for the treatment of autoimmune diseases has been approved by the Therapeutic Goods Administration (TGA) of Australia and will soon commence Phase I clinical trials in Australia.
RBD2080 is the sixth innovative small nucleic acid drug developed by the company based on the RIBO-GalSTARTM liver-targeting technology platform. It precisely targets specific genes associated with autoimmune diseases, effectively regulating gene expression and thereby fundamentally intervening in disease progression.
Overseas Hotspots
01 Alys
Alys's JAK1 siRNA-Lipid Conjugate Receives FDA Approval for Phase 2 Clinical Trial to Treat Alopecia Areata
02 Wave Life Sciences
Wave Life Strikes Strongly! siRNA Therapy Precisely Targets Obesity
On February 6, 2025, Wave Life Sciences, a U.S.-based biotechnology company, announced that the clinical trial application (CTA) for its novel long-acting GalNAc-siRNA drug WVE-007, targeting inhibin βE (INHBE) mRNA, has been approved. The company will initiate the Phase 1 INLIGHT clinical trial of WVE-007 in patients with obesity.
Preclinical data obtained in the diet-induced obesity (DIO) mouse model indicate that WVE-007 has therapeutic potential in various settings, including as a monotherapy, as an adjunct to GLP-1 therapy, and as a maintenance treatment to prevent weight rebound following the discontinuation of GLP-1 therapy.
03 Olix
Focus on MASH Treatment! OliX Collaborates with Eli Lilly
On February 11, 2025, Eli Lilly announced a global licensing agreement worth $630 million with South Korean biotechnology company OliX Pharmaceuticals to co-develop and commercialize OliX's metabolic dysfunction-associated steatohepatitis (MASH) treatment drug, OLX75016.
04 Stoke Therapeutics
Biogen Collaborates with Stoke Therapeutics to Treat Dravet Syndrome
February 18, 2025 — Biogen and Stoke Therapeutics announced a collaboration to jointly develop and commercialize zorevunersen, a potential first-in-class disease-modifying drug being developed for the treatment of Dravet syndrome, covering all regions outside the United States, Canada, and Mexico.
Zorevunersen is an investigational antisense oligonucleotide designed to treat the root cause of Dravet syndrome by increasing the production of NaV1.1 protein from non-mutated (wild-type) SCN1A gene copies in brain cells. This highly differentiated mechanism of action aims to reduce seizure frequency beyond the effects of existing anti-seizure medications and improve neurodevelopment, cognition, and behavior.
Zorevunersen has demonstrated disease-modifying potential and has received orphan drug designations from both the FDA and EMA. The FDA has also granted zorevunersen Rare Pediatric Disease designation and Breakthrough Therapy designation for the treatment of Dravet syndrome associated with non-functional or loss-of-function mutations in the SCN1A gene.
Selected Articles from Previous Issues
1. Small Nucleic Acid Drugs Overseas Market 2025 Outlook
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About Ouli Bio
Suzhou Oli Biopharmaceuticals Technology Co., Ltd. is a professional medicinal nucleic acid CRDMO.The company provides customers with "one-stop" services including nucleic acid drug discovery, laboratory R&D, process and analytical development, CMC services, API production, and drug registration. The technical team of Oulibiotech comes from one of the earliest teams in China that carried out 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: solid-phase synthesis of nucleic acids, chemical modification and conjugation, process development and analysis, and CMC pharmaceutical research. It has also engaged in in-depth cooperation with many 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 customers address key 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 customers 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.
Contact Us
Email: order@olipharma.com
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Official Website: www.olipharma.com
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