中美瑞康saRNA 疗法 RAG-01 获国家药监局批准开展非肌层浸润性膀胱癌 II 期临床试验

中美瑞康

03/19

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非肌层浸润性膀胱癌非肌

中国南通，2026 年 3 月 19 日—— 专注于创新小激活RNA（saRNA）疗法研发的临床阶段生物技术公司中美瑞康今日宣布，其自主研发的RAG-01 注射液已获得中国国家药品监督管理局药审中心（NMPA CDE）批准开展针对非肌层浸润性膀胱癌（NMIBC）的 II 期临床试验。

这一重要里程碑标志着RAG-01成为国内第二款获批临床的saRNA疗法（两款均由中美瑞康开发）。此次获批不仅巩固了中美瑞康在全球RNA激活（RNAa）技术领域的绝对领先地位，也为该平台技术的巨大临床转化潜力提供了强有力的背书。

RAG-01是一款创新型saRNA 疗法，通过 RNA 激活机制靶向肿瘤抑制基因 p21 并激活其表达。p21 作为调控细胞周期阻滞与细胞衰老的关键因子，能够有效抑制膀胱癌细胞的异常增殖。

RAG-01在中国顺利推进至II期临床，主要得益于其在澳洲I期临床试验中取得的优异数据。中期数据展示了明确的靶点结合证据（p21显著上调）、高度良好的安全性以及令人鼓舞的完全缓解（CR）信号。

中美瑞康创始人兼首席执行官李龙承博士表示：“我们非常高兴 RAG-01 获得国家药监局药审中心批准开展II 期临床试验。这一进展不仅是对RAG-01 项目的重要认可，也进一步验证了saRNA 疗法的临床开发潜力。RAG-01体现了公司的核心创新理念——通过激活内源性疾病相关基因，去应对长期以来难以成药的靶点。我们相信，这一差异化机制结合局部膀胱递送方式，有望为NMIBC 患者带来新的治疗选择。我们将继续与监管机构、研究者及临床中心紧密合作，积极推动项目开发。”

本次获批的 II 期临床试验为一项随机、对照、多中心研究，旨在评估 RAG-01 单药以及联合化疗在中高危 NMIBC 患者中的有效性与安全性。该研究的开展建立在澳大利亚 I 期临床试验所取得的积极初步数据基础之上；现有数据显示，RAG-01 已表现出良好的安全性、明确的靶点作用证据以及抗肿瘤活性。

关于RAG-01

RAG-01是一款具有开创性的saRNA 候选药物，通过RNA 激活机制靶向并上调抑癌基因 p21 的表达。该药物采用中美瑞康专有的 LiCO™ 递送技术，通过膀胱灌注给药。在澳大利亚开展的 I 期临床试验中，RAG-01 已展现出积极的安全性、靶点激活及初步疗效数据。2024 年，RAG-01 获得美国食品药品监督管理局（FDA）IND 批准，并被授予快速通道资格（Fast Track Designation）。

关于NMIBC

非肌层浸润性膀胱癌（NMIBC）是一种常见的恶性肿瘤，病变局限于膀胱内壁。中 / 高危NMIBC患者标准一线治疗是经尿道膀胱肿瘤切除术（TURBT），随后进行膀胱内BCG或化疗药物灌注治疗。然而，相当比例的患者会出现治疗无效或反复复发，因此亟需开发新型有效的治疗方法。

关于RNA激活

RNA激活是中美瑞康创始人李龙承博士及其团队在国际上开创，并已经过临床验证的平台技术。该技术利用靶向基因启动子区域的双链RNA来激活基因表达，以恢复治疗性蛋白的水平。RNA激活技术是制药领域十分稀缺的平台技术，在药物开发上具有广阔的应用前景，包括遗传性疾病、慢性疾病、代谢性疾病、心脑血管疾病、肿瘤等。

中美瑞康

Ractigen Therapeutics

中美瑞康是一家临床阶段的生物制药公司，致力于开发突破性小核酸药物与疾病治疗方法。中美瑞康是全球少数同时掌握有肝内与肝外递送的小核酸药企之一，开发出了具有独立自主知识产权的SCAD™、LiCO™及GLORY™等多个具有国际领先水平的小核酸药物递送平台技术。基于RNA激活技术和自主开发的Smart-TTC saRNA药物开发平台，公司建立了具有高度差异化的小核酸药物管线，适应症涵盖神经退行性疾病、神经肌肉疾病、肿瘤、代谢与血液系统疾病等，为诸多疾病领域中无法成药的靶点、无法治愈的疾病提供创新型治疗方案。详情请访问官网www.ractigen.com。

Ractigen Therapeutics Announces China NMPA IND Approval for Phase II Clinical Trial of saRNA Therapy RAG-01 in Non-Muscle Invasive Bladder Cancer

Nantong, China – March 19, 2026 – Ractigen Therapeutics, a pioneering clinical-stage biotechnology company developing innovative small activating RNA (saRNA) therapeutics, today announced that China’s National Medical Products Administration (NMPA) Center for Drug Evaluation (CDE) has approved its Investigational New Drug (IND) application to initiate a Phase II clinical trial of RAG-01 for the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC).

This significant milestone makes RAG-01 the second saRNA therapeutic to receive IND approval in China, both of which were developed by Ractigen. This approval not only solidifies Ractigen’s global leadership in saRNA technology but also serves as a critical clinical validator for the company's broader RNA activation (RNAa) platform and its LiCO^TMdelivery technology.

RAG-01 is a novel saRNA therapeutic designed to upregulate p21, a key regulator of cell cycle arrest and cellular senescence, to inhibit the abnormal proliferation of bladder cancer cells. This innovative saRNA therapeutic aims to treat NMIBC by increasing p21 mRNA and protein levels.

The progression of RAG-01 into Phase II clinical development follows highly encouraging Phase I data from Australia, which demonstrated clear target engagement (p21 protein upregulation), a favorable safety profile, and promising complete response (CR) signals.

“We are very pleased to receive IND approval from the China NMPA CDE for the Phase II trial of RAG-01,” said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. “This approval is an important validation of both the RAG-01 program and the broader potential of saRNA therapeutics. RAG-01 reflects our innovation strategy of activating endogenous disease-relevant genes that have long been considered difficult to drug. We believe this differentiated mechanism, together with local bladder delivery, may offer a meaningful new treatment option for patients with NMIBC. We look forward to working closely with investigators and regulatory authorities to advance this program.”

The Phase II study is a randomized, controlled, multi-center trial designed to evaluate the efficacy and safety of RAG-01 as monotherapy and in combination with chemotherapy in patients with intermediate- and high-risk NMIBC. The China study builds on encouraging preliminary data from the ongoing Phase I clinical trial in Australia, where RAG-01 has demonstrated favorable safety, clear target engagement, and anti-tumor activity.

About RAG-01

RAG-01 is a pioneering saRNA therapeutic candidate designed to activate the tumor suppressor gene p21 via RNA activation (RNAa). The product is administered through intravesical instillation using Ractigen’s proprietary LiCO™ delivery technology. In the Phase I clinical trial conducted in Australia, RAG-01 has shown encouraging preliminary safety, target engagement, and efficacy data. In 2024, RAG-01 received IND clearance from the U.S. Food and Drug Administration (FDA) and was granted Fast Track designation.

About NMIBC

Non-muscle invasive bladder cancer (NMIBC) is a common malignancy confined to the lining of the bladder. The standard first-line treatment is transurethral resection of the bladder tumor (TURBT) followed by intravesical BCG or chemotherapy. However, a significant proportion of patients experience treatment failure or multiple recurrences, highlighting the need for new and effective treatment options.

About RNAa
RNA activation is a clinically validated platform technology developed by Dr. Long-Cheng Li and his team. It utilizes saRNAs to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This innovative technology holds vast potential for developing therapeutic drugs across various diseases, particularly where traditional methods fall short.

About Ractigen Therapeutics
Ractigen Therapeutics is a clinical-stage biopharmaceutical company innovating next-generation RNA therapeutics, with a primary focus on small activating RNAs (saRNAs) developed through its clinically validated RNA activation (RNAa) technology. Leveraging proprietary delivery platforms such as SCAD™, LiCO™, and GLORY™, Ractigen is advancing a robust pipeline addressing unmet medical needs in oncology, neurological diseases, and genetic disorders. Its versatile technologies also enable the rapid development of RNA-based solutions, including siRNAs, where applicable, to target life-threatening, fast-progressing conditions such as those in the CNS. Committed to scientific excellence and patient-centered innovation, Ractigen strives to transform healthcare through the power of RNA therapeutics. For more information, visit www.ractigen.com.