Ractigen Therapeutics Submits IND Application in Australia for RAG-01, a First-in-Class saRNA Therapy Targeting BCG-Unresponsive NMIBC

Dec 08, 2023 10:14 CST Updated 10:14

Ractigen

Innovative Drug R&D Developer

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Ractigen Therapeutics, an innovative biopharmaceutical company at the forefront of small activating RNA (saRNA) therapeutics, recently announced the submission of its first saRNA drug, RAG-01, for a Phase I clinical trial application in Australia. This clinical study is an open-label, multi-center Phase I trial designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of RAG-01 in patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). This milestone marks Ractigen's transition into a clinical-stage biopharmaceutical company and represents a significant breakthrough in the field of RNA activation.

RAG-01 Successfully Submits for Clinical Trials, Becoming the Second saRNA Drug Globally to Enter the Clinical Stage and the First and Only One in China to Achieve This Milestone.

Dr. Longcheng Li, founder and CEO of Ractigen, stated"Today marks not only an important moment for Ractigen Therapeutics to enter the clinical stage, but also a significant achievement in the field of global innovative drugs. Based on the original platform technology of RNA activation (RNAa), the preclinical data of RAG-01 has demonstrated excellent tumor suppression effects and safety. We look forward to the clinical performance of RAG-01 and hope that it will become a globally innovative targeted cancer treatment drug. We will actively promote more promising saRNA products into the clinical stage, providing innovative therapies for many currently difficult-to-treat diseases."

About RAG-01

RAG-01 is a first-in-class double-stranded saRNA drug that specifically targets and activates the tumor suppressor gene p21. It enhances the expression of the p21 gene through the RNAa mechanism, thereby inhibiting tumor cell proliferation and inducing apoptosis and senescence. Preclinical efficacy studies have shown that, delivered via Ractigen Therapeutics' proprietary extracellular small nucleic acid delivery system LiCO™, RAG-01 treatment significantly inhibits the growth of bladder cancer tumors in animal models while demonstrating robust safety.

About LiCO™

RAG-01 utilizes Ractigen Therapeutics' self-developed LiCO™ delivery system, which employs a unique lipid chemistry approach and supports multiple administration routes such as subcutaneous, intravenous, intravesical, and intravitreal injections. The versatility and efficacy of LiCO™ have been validated in numerous preclinical projects, enabling efficient and sustained delivery to tissues including the heart, muscle, fat, and eyes, completely revolutionizing the delivery method for oligonucleotides.

About NMIBC

Bladder Cancer is the most common cancer of the urinary system, with NMIBC accounting for 50-80%. The standard treatment for NMIBC is primarily transurethral resection of bladder tumor (TURBT), but the postoperative recurrence rate is high, reaching 50%-70% within five years. Therefore, there is an urgent need to develop drugs that can effectively prevent bladder cancer recurrence and treat advanced bladder cancer.

About RNA Activation

RNA Activation (RNAa) is a platform technology pioneered internationally by Dr. Long-Cheng Li, the founder of Ractigen Therapeutics, and his team, which has already been clinically validated. This technology uses double-stranded RNA targeting the promoter region of genes to activate gene expression, restoring the levels of therapeutic proteins. RNA Activation technology is a highly scarce platform technology in the pharmaceutical field, with broad application prospects in developing therapeutic drugs, including for cancer, genetic diseases, chronic diseases, metabolic diseases, and cardiovascular and cerebrovascular diseases.

About Ractigen

Ractigen Therapeutics is a platform-based new drug research and development company based in China and targeting the global market. It is committed to developing breakthrough small nucleic acid drugs and disease treatment methods. Ractigen Therapeutics is one of the very few small nucleic acid pharmaceutical companies globally that simultaneously masters both intrahepatic and extrahepatic delivery technologies, having developed multiple proprietary small nucleic acid drug delivery platform technologies with internationally leading standards, such as SCAD™ and LiCO™. Based on RNA activation technology and its self-developed Smart-TTC saRNA drug development platform, the company has established a highly differentiated small nucleic acid drug pipeline, with indications covering neurodegenerative and neuromuscular diseases, cancer, metabolic and hematological diseases, among others. This provides innovative therapeutic solutions for undruggable targets and currently incurable diseases across various disease areas.