Domestic Oligonucleotide Therapeutics Clinical Pipeline Update – OliBio Industry Insights

May 13, 2025 12:18 CST Updated 12:18

Sirius Therapeutics

Oligonucleotide Drug Developer

Rona Therapeutics

Nucleic Acid Drug Developer

CSPC

Innovative Drug Research and Development, Manufacturer

Innovation in China

01 Sirius Therapeutics

Sirius Therapeutics' Third siRNA Therapy IND Application Approved by FDAAccurate

On April 9, 2025, Sirius Therapeutics announced,U.S. Food and Drug Administration (FDA) has approved itssiRNANew Therapy DrugsSRSD216Clinical Trial Application (IND), allowing clinical trials to be conducted. Notably, the product's ChinaIThe phase clinical trial has been conducted at4Month8The first subject was dosed on [date].

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, after a single dose, Lp(a) levels were reduced by nearly 100%, with effects lasting over six months, and no significant safety events were observed.。

02 Rona Therapeutics

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

Rona Therapeutics' siRNA Drug Targeting AGT Receives NMPA Clinical Trial Implied Permission

2025Year4Month21Day, Rona Therapeutics (RonaTherapeutics) announced that its self-developed angiotensinogen-targeted (Angiotensinogen,AGT) Small interferingRNA（siRNA) DrugRN1871New Drug Clinical Trial Application (IND), has officially been approved by the National Medical Products Administration of China.

RN1871By silencing in the liverAGTThemRNAExpression, precise inhibition of renin-Angiotensin-Aldosterone System (RAAS) The core precursor protein generation.RAASThe system is a key pathway in blood pressure regulation, and its overactivation is closely related to the progression of hypertension. Preclinical studies have shown,RN1871Can effectively lower blood pressure over the long term, with the potential to benefit more than15Provide better treatment options for hundreds of millions of hypertension patients.

03 CSPC

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

CSPC's Dual-Chain siRNA Approved for Clinical Trials to Treat Hyperlipoproteinemia

On April 25, 2025, CSPC announced that its self-developed chemical Class 1 new drug SYH2068 Injection (double-stranded siRNA) has been approved by the National Medical Products Administration of the People's Republic of China to conduct clinical trials in China, intended for the treatment of hyperlipoproteinemia(a).

SYH2068 is an investigational siRNA drug that achieves liver-targeted delivery through conjugation with N-acetylgalactosamine (GalNAc). Administered subcutaneously, it targets lipoprotein(a) (Lp(a)) and effectively reduces Lp(a) levels.This therapy is applicable for the treatment of hyperlipoproteinemia (a), achieving more durable gene silencing effects through strategies of sequence optimization and chemical modification. It is expected to become an ultra-long-acting siRNA drug for reducing Lp(a) and has the potential to prevent atherosclerotic cardiovascular disease (ASCVD).

04 Hengrui Medicine

Hengrui Medicine's HBV siRNA New Drug Approved for Clinical Trials

On April 29, 2025, Hengrui Medicine announced that its subsidiary, Fujian Shengdi Medicine, received the "Drug Clinical Trial Approval Notice" issued by the National Medical Products Administration for the HRS-5635 injection. The approval allows the product to proceed with clinical trials intended to be combined with Pegylated Interferon Alpha (Peg-IFNα) for the treatment of chronic hepatitis B.

HRS-5635 Injection is a new generation of liver-targeting siRNA drug for hepatitis B virus (HBV) independently developed by Hengrui Medicine. Non-clinical efficacy studies show that HRS-5635 exhibits excellent antiviral activity against all HBV genotypes and can exert efficient and long-lasting antiviral effects in vivo. Non-clinical safety evaluation studies indicate that HRS-5635 has no off-target activity and is highly safe. According to available information, no similar drugs have been approved for marketing in or outside China.

Overseas Hotspots

01 Alnylam

AlnylamLatest Phase 2 Clinical Results of Long-Acting Hypertension Therapy Announced

Alnylam Pharmaceuticals announced positive results from the Phase 2 clinical trial, KARDIA-2, of zilebesiran, an RNAi therapy co-developed with Roche for treating hypertension patients. Data analysis showed that adding zilebesiran to standard treatment regimens significantly reduced patients' average 24-hour systolic blood pressure (SBP) by the third month. Notably, some patients who received a single dose of zilebesiran still maintained sustained blood pressure reduction at the six-month follow-up.

Zilebesiran is a subcutaneously administered investigational RNAi therapeutic drug targeting angiotensinogen (AGT). AGT is the most upstream target in the renin-angiotensin-aldosterone system (RAAS), a cascade that has been shown to play a role in blood pressure regulation, and its inhibition has well-established antihypertensive effects. Zilebesiran inhibits the synthesis of AGT in the liver, potentially leading to a sustained reduction in AGT protein and ultimately resulting in decreased angiotensin levels.

02 Creyon Bio

Creyon Bio Collaborates with Eli Lilly to Develop Oligonucleotide Therapies, Exceeding $1 Billion!

On April 30, 2025, Creyon Bio (hereinafter referred to as "Creyon") announced a global licensing and multi-target research collaboration with Eli Lilly and Company (hereinafter referred to as "Lilly"). This collaboration focuses on the discovery, development, and commercialization of innovative RNA-targeted oligonucleotide therapies for various diseases. Creyon will leverage its unique AI-driven oligonucleotide engineering engine to design and optimize candidate compounds for targets specified by Lilly.

According to the terms of the agreement, Creyon will receive an upfront payment of $13 million and is eligible to receive over $1 billion in development and commercialization payments upon achieving specific milestones. Lilly obtains exclusive licenses for lead drug candidates based on each target and, after achieving relevant milestones, will be responsible for subsequent research, development, and commercialization.

Creyon Bio is dedicated to developing tissue-specific RNA-targeting oligonucleotide therapies by combining proprietary computational methods with aptamer-based tissue-specific delivery strategies. Creyon has built an AI-driven oligonucleotide engineering engine, which makes the design process for safe and highly active oligonucleotides more systematic and cost-effective.

Selected Highlights

1. Small Nucleic Acid Drugs Overseas Market 2025 Outlook

For more industry information, please refer to:

Olyview Industry Dynamics

Clinical Progress of Small Nucleic Acid Drugs in China

(Source: Compiled from the official websites and public information of various companies)

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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 drug registration. The technical team of Oli Biopharmaceuticals 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, 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 key challenges such as scaling up 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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