Funding led by Guozhong Capital will accelerate Phase 2 development for saRNA candidate RAG-01 and CNS asset RAG-17, advance systemic saRNA program RAG-18 toward IND, and expand extrahepatic RNA delivery technologies.

由国中资本领投的融资将加速自扩增RNA（saRNA）候选药物RAG-01和中枢神经系统资产RAG-17的第二阶段开发，推动系统性saRNA项目RAG-18向新药临床试验申请（IND）迈进，并拓展肝外RNA递送技术。

NANTONG, China

中国南通

,

，

July 29, 2026

2026年7月29日

/PRNewswire/ -- Ractigen Therapeutics, a clinical-stage biotechnology company pioneering small activating RNA (saRNA) therapeutics and advanced extrahepatic delivery systems, today announced the successful closing of a new financing round exceeding $31 million (over RMB 200 million).

/美通社/ -- 临床阶段生物技术公司Ractigen Therapeutics致力于开创小激活RNA（saRNA）疗法及先进的肝外递送系统，今日宣布成功完成新一轮融资，金额超过3,100万美元（逾2亿元人民币）。

The round was led by

本轮由……领投

Guozhong Capital

国中资本

, with participation from

，并有以下参与方

IDG Capital

IDG资本

,

，

China Everbright Limited

中国光大有限公司

,

，

Jolmo Capital

乔尔莫资本

,

，

Win-Win Capital

双赢资本

, and

，和

SND Financial Holdings

SND金融控股

. Existing shareholder

现有股东

Longmen Capital

龙门资本

participated for its third consecutive round.

连续第三轮参赛。

Proceeds will primarily accelerate the clinical development of Ractigen's saRNA assets and validate its proprietary extrahepatic delivery platforms. Key priorities include:

所得资金将主要用于加速 Ractigen 公司 saRNA 资产的临床开发，并验证其专有的肝外递送平台。关键优先事项包括：

Advancing lead saRNA oncology program (RAG-01)

推进领先的saRNA肿瘤学项目（RAG-01）

: Accelerating Phase 2 clinical trials in non-muscle-invasive bladder cancer (NMIBC) following positive clinical proof-of-concept and U.S. FDA Fast Track Designation.

：在获得积极的临床概念验证及美国食品药品监督管理局（FDA）快速通道认定后，加速推进非肌层浸润性膀胱癌（NMIBC）的二期临床试验。

Driving systemic saRNA asset toward IND (RAG-18)

推动全身性saRNA资产RAG-18迈向新药临床试验申请（IND）

: Advancing ongoing investigator-initiated trials (IIT) and preparing for IND filing for Duchenne muscular dystrophy (DMD).

：推进正在进行的由研究者发起的临床试验（IIT），并为杜氏肌营养不良症（DMD）的新药临床试验申请（IND）做准备。

Progressing CNS clinical asset (RAG-17)

推进中枢神经系统临床资产（RAG-17）

: Moving forward with Phase 2 clinical trials in amyotrophic lateral sclerosis (ALS) following Phase 1 safety and biomarker validation published in

在发布第一阶段安全性和生物标志物验证结果后，推进肌萎缩侧索硬化症（ALS）的第二阶段临床试验

Nature Medicine

《自然医学》

.

。

Expanding extrahepatic delivery platforms

拓展肝外递送平台

: Continuously enhancing the company's proprietary

持续提升公司的专有技术

SCAD™

SCAD™

(CNS) and

（中枢神经系统）和

LiCO™

LiCO™

(systemic multi-tissue) delivery technologies to unlock new therapeutic targets outside the liver.

（系统性多组织）递送技术，以解锁肝脏之外的新治疗靶点。

Pioneering saRNA Technology: Unlocking Gene Activation for Unmet Needs

开创性saRNA技术：释放基因激活潜力，满足未竟之需

Oligonucleotide therapeutics represent the third major wave of biopharmaceutical innovation following small molecules and monoclonal antibodies. However, traditional RNA therapeutics have been largely limited to gene-silencing approaches (siRNA and ASO).

寡核苷酸疗法代表了继小分子和单克隆抗体之后生物制药创新的第三大浪潮。然而，传统RNA疗法主要局限于基因沉默方法（siRNA和ASO）。

As the global pioneer in RNA activation (RNAa), Ractigen was founded to redefine genetic medicine by enabling precise

作为RNA激活（RNAa）领域的全球先驱，Ractigen的成立旨在通过实现精准调控，重新定义基因医学。

gene activation

基因激活

. Discovered by Ractigen's founder, RNAa utilizes saRNAs targeting gene promoter regions to upregulate endogenous protein expression at the transcriptional level without altering the genome. This unique mechanism unlocks previously undruggable therapeutic targets, expanding treatment possibilities across genetic disorders, haploinsufficiencies, cancer, and metabolic diseases..

由 Ractigen 创始人发现的 RNAa 技术，利用靶向基因启动子区域的小激活 RNA（saRNA），在不改变基因组的前提下，于转录水平上调内源性蛋白表达。这一独特机制解锁了此前无法成药的治疗靶点，从而拓展了遗传性疾病、单倍体剂量不足、癌症及代谢性疾病等领域的治疗可能性。

Breaching Extrahepatic Barriers: Proprietary Delivery Systems

突破肝外屏障：专有递送系统

To deliver saRNA and targeted oligonucleotide payloads beyond the liver, Ractigen engineered two complementary, carrier-free delivery platforms:

为了将saRNA和靶向寡核苷酸有效载荷递送至肝脏以外的组织，Ractigen开发了两种互补的无载体递送平台：

SCAD™ (CNS delivery platform)

SCAD™（中枢神经系统递送平台）

: Utilizes an accessory oligonucleotide (ACO)-enabled self-delivery mechanism to cross central nervous system barriers, achieving clinical proof-of-concept for safety and target engagement via RAG-17.

利用辅助寡核苷酸（ACO）赋能的自递送机制穿越中枢神经系统屏障，通过RAG-17实现了安全性和靶点结合的临床概念验证。

LiCO™ (Systemic multi-tissue platform)

LiCO™（全身多组织平台）

: Conjugates specialized lipids to oligonucleotides via proprietary SDL™ linkers. LiCO™ enables durable, carrier-free delivery to muscle, heart, bladder, and eye tissues, sustaining therapeutic activity for up to nine months per single administration with simplified, cost-effective manufacturing.

通过专有的SDL™连接子将特化脂质与寡核苷酸偶联。LiCO™技术能够实现向肌肉、心脏、膀胱和眼部组织的持久、无载体递送，单次给药即可维持长达九个月的治疗活性，且制造工艺简化、成本效益高。

Clinical & Commercial Momentum

临床与商业势头

Ractigen has successfully translated its saRNA and extrahepatic delivery technologies into three differentiated clinical-stage assets:

Ractigen 已成功将其 saRNA 和肝外递送技术转化为三种具有差异化的临床阶段资产：

RAG-01

RAG-01

: The world's first conjugate-delivered saRNA therapeutic to demonstrate clinical proof-of-concept in oncology. In Phase 1 trials, RAG-01 achieved a

：全球首款在肿瘤学中展示临床概念验证的结合递送自扩增RNA（saRNA）疗法。在1期临床试验中，RAG-01实现了

preliminary any-time 67% complete response (CR) rate

初步的随时67%完全缓解（CR）率

in BCG-unresponsive high-risk NMIBC patients.

在BCG无反应的高危非肌层浸润性膀胱癌（NMIBC）患者中。

RAG-18

RAG-18

: The world's first saRNA program targeting Duchenne muscular dystrophy (DMD) and a major milestone in breaching extrahepatic muscle delivery barriers. Currently in investigator-initiated trials (IIT), RAG-18 delivers saRNA systemically to upregulate Utrophin expression, demonstrating

：全球首个靶向杜氏肌营养不良症（DMD）的自扩增RNA（saRNA）项目，也是突破肝外肌肉递送屏障的一项重大里程碑。RAG-18目前正处于研究者发起的临床试验（IIT）阶段，通过全身给药方式上调Utrophin蛋白的表达，展现出

clear target engagement, marked biomarker reductions, and histopathological improvements in muscle tissue

明确的靶点参与、显著的生物标志物降低以及肌肉组织的组织病理学改善

in DMD patients.

在杜氏肌营养不良症患者中。

RAG-17

RAG-17

: A clinical-stage, CNS-targeted oligonucleotide therapeutic for amyotrophic lateral sclerosis (ALS). Phase 1 clinical data demonstrated robust SOD1 protein knockdown (~60% CSF SOD1 reduction) and a favorable safety profile, with Phase 2 patient enrollment now fully completed.

：一种处于临床阶段、靶向中枢神经系统的寡核苷酸疗法，用于治疗肌萎缩侧索硬化症（ALS）。I期临床数据显示出显著的SOD1蛋白敲低效果（脑脊液中SOD1降低约60%）以及良好的安全性特征，目前II期患者入组已全面完成。

Reflecting the global commercial value of its platform, Ractigen entered into a strategic drug discovery and platform licensing collaboration with a publicly listed overseas pharmaceutical company in late 2025, valued at over

为反映其平台的全球商业价值，Ractigen 于 2025 年下半年与一家海外上市制药公司达成了战略药物发现及平台授权合作，交易价值超过

$3 billion

30亿美元

.

。

Executive & Investor Commentary

高管与投资者评论

Dr. Long-Cheng Li, Founder and Chief Executive Officer of Ractigen Therapeutics:

李龙成博士，Ractigen Therapeutics 创始人兼首席执行官：

'Closing this financing round reflects strong capital market validation of Ractigen's critical leap from pioneering scientific discovery to human clinical proof-of-concept. Over the past decade, the oligonucleotide field achieved tremendous commercial success in liver-targeted silencing, yet extrahepatic delivery and gene activation remained unaddressed global frontiers.

“完成本轮融资，体现了资本市场对Ractigen从开创性科学发现迈向人体临床概念验证这一关键跨越的强力认可。在过去十年中，寡核苷酸领域在肝脏靶向沉默方面取得了巨大的商业成功，但肝外递送和基因激活仍是全球范围内尚未攻克的 frontier。”

Over nearly two decades, we progressed from discovering RNA activation to solving extrahepatic delivery bottlenecks and translating multiple assets into positive human clinical data. Moving forward, Ractigen will continue expanding the boundaries of RNA technology, accelerating clinical translation to deliver transformative, First-in-Class therapeutics to patients worldwide.'.

在近二十年的时间里，我们从发现RNA激活机制起步，逐步攻克肝外递送瓶颈，并将多个候选药物成功转化为积极的临床人体数据。展望未来，Ractigen将继续拓展RNA技术的边界，加速临床转化进程，为全球患者带来具有变革意义的首创疗法。

Investment Team, Guozhong Capital:

国中资本投资团队：

'Ractigen Therapeutics is dedicated to pioneering next-generation RNA therapeutics globally. Its proprietary extrahepatic delivery platforms breach core bottlenecks across the field. RAG-17 is a pioneering CNS-targeting oligonucleotide therapy with Phase 1 results featured in top-tier peer-reviewed journals, while RAG-01 marks a historic clinical application of saRNA technology in oncology.

Ractigen Therapeutics 致力于在全球范围内开创下一代 RNA 疗法。其专有的肝外递送平台突破了该领域的核心瓶颈。RAG-17 是一款开创性的靶向中枢神经系统的寡核苷酸疗法，其一期临床试验结果已刊登于顶级同行评审期刊；而 RAG-01 则标志着自扩增 RNA（saRNA）技术在肿瘤学领域的首次历史性临床应用。

We look forward to supporting Ractigen as its saRNA platform expands further into genetic, oncologic, and metabolic diseases.'.

我们期待在Ractigen的saRNA平台进一步拓展至遗传病、肿瘤病和代谢性疾病领域之际，为其提供支持。

Haining Wang, Founding Partner of Longmen Capital:

龙门资本创始合伙人王海宁：

'We have been a long-term believer in Ractigen since our initial investment in 2021, and this marks our third consecutive round of support. Their clinically validated extrahepatic delivery systems and highly differentiated saRNA pipelines create an unrivaled competitive moat. We will continue leveraging our industry and capital market resources to help Ractigen bring saRNA therapies to patients globally.'.

“自2021年首次投资以来，我们一直是Ractigen的长期坚定支持者，此次已是连续第三轮为其提供支持。其经临床验证的肝外递送系统以及高度差异化的saRNA管线，构筑了无可匹敌的竞争护城河。我们将继续依托我们的产业与资本市场资源，助力Ractigen将saRNA疗法带给全球患者。”

About Ractigen Therapeutics

关于 Ractigen Therapeutics

Ractigen Therapeutics is a clinical-stage biopharmaceutical company innovating next-generation RNA therapeutics, with a primary focus on saRNAs developed through its clinically validated RNA activation (RNAa) technology. Leveraging proprietary extrahepatic delivery platforms such as SCAD™, LiCO™, and GLORY™, Ractigen is advancing a robust pipeline addressing severe unmet medical needs in oncology, neurological diseases, and genetic disorders.

Ractigen Therapeutics 是一家处于临床阶段的生物制药公司，致力于创新下一代 RNA 疗法，主要聚焦于通过其经临床验证的 RNA 激活（RNAa）技术开发的小激活 RNA（saRNA）。凭借 SCAD™、LiCO™ 和 GLORY™ 等专有肝外递送平台，Ractigen 正在推进一个强大的产品管线，以满足肿瘤学、神经系统疾病和遗传性疾病领域尚未被满足的重大医疗需求。

Committed to scientific excellence and patient-centered innovation, Ractigen strives to transform healthcare through the power of targeted RNA activation and delivery. For more information, visit .

Ractigen 致力于科学卓越与以患者为中心的创新，力求通过靶向 RNA 激活和递送技术变革医疗健康。欲了解更多信息，请访问 。

www.ractigen.com

www.ractigen.com

.

。

SOURCE Ractigen Therapeutics

来源：Ractigen Therapeutics

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