Pharma Focus Asia

Ractigen Therapeutics Advances to Clinical Stage with Revolutionary saRNA Medication, RAG-01

Thursday, December 07, 2023

Ractigen Therapeutics, a prominent player in the realm of small activating RNA (saRNA) therapeutics, is pleased to announce the submission of a clinical trial application in Australia. This significant move sets the stage for a phase I, open-label, multi-center study designed to assess the safety, tolerability, pharmacokinetics, and preliminary efficacy of RAG-01, an innovative saRNA drug candidate. The study focuses on patients with non-muscle-invasive bladder cancer (NMIBC) who have not responded to Bacillus Calmette-Guérin (BCG) therapy. This milestone signifies Ractigen's entry into clinical development, with RAG-01 targeting NMIBC.

RAG-01 is acknowledged as the second saRNA drug globally to enter clinical trials and the first of its kind in China to achieve this significant milestone. This progress represents a remarkable leap forward in the application of RNA-based therapeutic approaches, particularly in the challenging landscape of cancer treatments.

Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, expressed his optimistic perspective on this milestone: "This achievement goes beyond a procedural advancement; it marks a critical progression in the application of RNAa technology in clinical contexts. Our proprietary RNAa platform, exemplified by the development of RAG-01, has demonstrated promising results in preclinical tumor suppression and a robust safety profile. We are confident in the potential of RAG-01 to advance bladder cancer treatment and remain committed to pushing boundaries with more innovative saRNA candidates in clinical trials, targeting historically difficult-to-treat diseases."

RAG-01 stands as a pioneering saRNA candidate from Ractigen Therapeutics, designed to target and activate the tumor suppressor gene p21. Administered intravesically using Ractigen’s advanced LiCO™ technology, the drug has exhibited significant tumor suppression in mouse orthotopic bladder cancer models. Its development marks a significant stride in RNA-based therapies, addressing the unmet needs of NMIBC patients.

LiCO™, Ractigen's proprietary delivery system, facilitates the delivery of duplex RNA into various tissues and organs. It supports multiple administration routes, including subcutaneous, intravenous, intravesical, and intravitreal, providing profound and durable activity. Its versatility and effectiveness have been validated in numerous preclinical studies, establishing it as a cornerstone in Ractigen's therapeutic arsenal.

NMIBC accounts for 50-80% of all bladder cancer cases. Despite standard treatments like transurethral resection of bladder tumor (TURBT) followed by intravesical BCG or chemotherapy, recurrence rates remain high, estimated at 50-70% within the first five years. The development of RAG-01 represents a significant step towards addressing this substantial unmet need in bladder cancer therapy.

Pioneered by Dr. Long-Cheng Li and his team, RNAa is a clinically validated platform technology. It employs saRNA to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This technology holds immense potential for developing therapeutic drugs across various diseases, particularly in areas where traditional methods fall short, including cancer, genetic disorders, chronic diseases, and metabolic and cerebrovascular disorders.



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