Pharma Pioneer

Ractigen Commences Phase I Clinical Trial with First Patient Treated for NMIBC Using RAG-01

17 May 2024
2 min read

In a significant medical advancement, Ractigen Therapeutics has initiated its first-ever human clinical trial for RAG-01, a novel small activating RNA (saRNA) treatment for non-muscle invasive bladder cancer (NMIBC), in partnership with GenesisCare, a prominent cancer care provider in Australia. The open-label, multi-center study aims to evaluate the safety, tolerability, and efficacy of RAG-01, which targets the p21 tumor suppressor gene to inhibit tumor growth in NMIBC patients who have not responded to Bacillus Calmette-Guérin (BCG) therapy. Preclinical studies have shown promising results in suppressing tumor growth and maintaining a favorable safety profile.
Dr. Long-Cheng Li, CEO and founder of Ractigen, highlighted the milestone as a testament to the company's clinical translation capabilities and its commitment to developing innovative treatments for NMIBC using the saRNA platform. RAG-01 is administered via intravesical instillation with Ractigen's proprietary LiCOTM delivery system, which has demonstrated its ability to deliver RNA to challenging-to-reach tissues and organs, offering a versatile and effective solution for various administration routes.
NMIBC constitutes a significant portion of bladder cancer cases, with high recurrence rates despite current treatments. The development of RAG-01 represents a step forward in addressing the unmet medical needs of these patients. RNAa, the technology behind Ractigen's saRNA drugs, has been clinically validated and holds great potential for therapeutic drug development across a range of diseases, particularly in areas where conventional treatments are insufficient. Ractigen is at the cutting edge of this technology, focusing on enhancing gene expression to restore normal protein functions and treat diseases that are otherwise difficult to address.

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