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Ractigen Therapeutics Advances into Clinical Stage with Groundbreaking saRNA Drug, RAG-01

By December 6, 2023May 22nd, 2024No Comments

Ractigen Therapeutics Advances into Clinical Stage with Groundbreaking saRNA Drug, RAG-01

JIANGSU, China, Dec. 06, 2023 — Ractigen Therapeutics, a leader in the field of small activating RNA (saRNA) therapeutics, is excited to announce the submission of a clinical trial application in Australia. This application paves the way for a phase Ⅰ, open-label, multi-center study, aiming to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of RAG-01, a groundbreaking saRNA drug candidate, in patients with non-muscle-invasive bladder cancer (NMIBC) who have not responded to Bacillus Calmette-Guérin (BCG) therapy. This milestone marks Ractigen’s transition into clinical development, with RAG-01 targeting NMIBC.

RAG-01 is recognized as the second saRNA drug worldwide to enter clinical trials and the first of its kind in China to achieve this critical milestone. This development signifies a remarkable advancement in RNA-based therapeutic applications, especially in the context of challenging cancer treatments.

Reflecting on this significant development, Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, shared his confident outlook: “This milestone represents more than a procedural step for us; it marks a crucial advancement in the application of RNAa technology in clinical settings. Our proprietary RNAa platform, as evidenced by the development of RAG-01, has shown promising results in preclinical tumor suppression and a solid safety profile. We hold a strong belief in the potential of RAG-01 in advancing bladder cancer treatment and remain dedicated to pushing the boundaries with more innovative saRNA candidates in clinical trials, targeting diseases that have historically been difficult to treat.”

About RAG-01: RAG-01 stands as a pioneering saRNA candidate from Ractigen Therapeutics, engineered to target and activate the tumor suppressor gene p21. The drug, delivered intravesically using Ractigen’s advanced LiCOTM technology, has shown 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.

About LiCOTM: LiCOTM, Ractigen’s proprietary delivery system, enables 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 many preclinical studies, marking it as a cornerstone in Ractigen’s therapeutic arsenal.

About NMIBC: NMIBC represents 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. RAG-01’s development is a significant step towards addressing this substantial unmet need in bladder cancer therapy.

About RNAa: 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 has vast potential for developing therapeutic drugs across various diseases, especially where traditional methods fall short, including cancer, genetic disorders, chronic diseases, and metabolic and cerebrovascular disorders.

About Ractigen Therapeutics: Ractigen Therapeutics is at the forefront of developing saRNA drugs, utilizing RNA activation (RNAa) to up-regulate gene expression endogenously. This innovative approach involves saRNA targeting specific genes to enhance transcription, thereby restoring normal protein functions. Ractigen’s cutting-edge technology is pivotal in treating diseases unaddressable by conventional methods, such as those resulting from epigenetic silencing or gene downregulation.