China Greenlights Trial for Novel RNA Drug Targeting Bladder Cancer

NANTONG, China – March 19, 2026 – Ractigen Therapeutics has received a pivotal Investigational New Drug (IND) approval from China's National Medical Products Administration (NMPA), allowing the company to proceed with a Phase II clinical trial for RAG-01, a novel therapy for Non-Muscle Invasive Bladder Cancer (NMIBC). The announcement marks a significant validation for the company's pioneering RNA activation technology and solidifies its leadership in a competitive and rapidly advancing field.

This is the second saRNA (small activating RNA) therapeutic developed by Ractigen to gain IND approval in China, a milestone that underscores the country's accelerating embrace of innovative drug platforms. The upcoming trial will evaluate RAG-01's potential to address a significant unmet need for patients battling a common and often recurrent malignancy.

A New Frontier: Activating the 'Undruggable'

At the heart of Ractigen's strategy is a groundbreaking technology known as RNA activation (RNAa). Unlike more common RNA interference (RNAi) techniques that silence or 'turn off' genes, RNAa uses small activating RNA molecules to selectively 'turn on' the expression of specific genes. This approach holds the potential to restore the function of protective proteins that are deficient in certain diseases.

RAG-01 is designed to target and upregulate p21, a critical tumor suppressor gene. The p21 protein acts as a brake on the cell cycle, halting abnormal cell proliferation and promoting cellular senescence, or aging. In many cancers, this protective mechanism is silenced. For decades, activating genes like p21 has been considered a formidable challenge, placing them in the category of 'difficult to drug' targets for traditional small molecule drugs or antibodies.

Ractigen's saRNA therapeutic bypasses these challenges by directly targeting the gene's regulatory machinery to boost the production of p21 mRNA and, subsequently, the p21 protein. The therapy is administered directly into the bladder via intravesical instillation, a method enabled by the company's proprietary LiCO™ delivery technology. This localized delivery is designed to maximize therapeutic effect within the bladder tissue while minimizing systemic exposure and potential side effects.

Promising Early Signs Offer New Hope

The NMPA's decision to approve the Phase II trial was heavily supported by highly encouraging data from a Phase I first-in-human study conducted in Australia. The study, which enrolled patients with NMIBC who had previously failed standard BCG therapy, demonstrated a favorable safety profile, with no dose-limiting toxicities or serious adverse events reported in the initial cohorts.

More importantly, the trial provided clear evidence of target engagement, with dose-dependent increases in p21 protein expression observed in bladder tissue samples. This confirmed that RAG-01 was performing its intended biological function.

The preliminary efficacy signals were particularly striking. In the two lowest dose cohorts, a remarkable 66.7% complete response rate was seen in patients with carcinoma in situ (CIS), a high-risk form of NMIBC. Furthermore, two-thirds of patients with papillary tumors remained disease-free at their three-month assessment. Researchers involved in the Australian trial considered these early results highly encouraging, especially for a patient population with few effective treatment options left.

"This approval is an important validation of both the RAG-01 program and the broader potential of saRNA therapeutics," said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, in a statement. "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."

Strategic Victory in a Critical Market

Securing IND approval in China is a significant strategic victory for Ractigen. Bladder cancer is a major health issue in the country, with over 100,000 new cases diagnosed annually and a high prevalence of NMIBC. The standard of care has long been hampered by high recurrence rates, with a substantial portion of patients eventually failing treatment, creating a large and growing unmet medical need.

The Chinese NMIBC market is poised for significant growth, making it a critical geography for any novel oncology therapeutic. Ractigen's progress is facilitated by reforms within the NMPA that have created accelerated review pathways for innovative drugs addressing urgent clinical needs. This regulatory environment allows companies like Ractigen to potentially bring new therapies to patients faster.

The approval in China builds on global regulatory momentum for RAG-01. In 2024, the therapy received IND clearance from the U.S. Food and Drug Administration (FDA) and was granted Fast Track designation, a program designed to expedite the development and review of drugs that treat serious conditions and fill an unmet medical need. This dual validation from two of the world's largest regulatory bodies positions RAG-01 as a globally significant clinical asset.

Leading the Charge in RNA Therapeutics

While the RNA therapeutics field has been dominated by mRNA vaccines and gene-silencing siRNA drugs, Ractigen is carving out a leadership position in the emerging space of gene activation. The company's progress has not gone unnoticed by the investment community. Ractigen has successfully raised over $54 million from a syndicate of top-tier investors, including Hillhouse Venture Capital, SDIC Venture Capital, and the corporate venture arm of pharmaceutical giant Eisai Co., Ltd. This strong financial backing provides the stability needed to advance its ambitious pipeline through resource-intensive clinical development.

With two saRNA therapies now cleared for clinical trials in China and a lead asset progressing in the U.S., Ractigen has substantiated its claim as a pioneer in the field. The company's versatile RNAa platform is also being applied to a diverse pipeline targeting unmet needs in neurological diseases and genetic disorders, demonstrating the broad potential of its technology beyond oncology. As RAG-01 enters this next crucial phase of testing, it carries the potential to not only transform care for bladder cancer patients but also to further validate the power of activating genes to fight disease.