Ractigen Therapeutics Announces RAG-01 as the First saRNA Drug to Receive FDA Fast Track Designation for BCG-Unresponsive NMIBC

On May 21, 2024, Ractigen Therapeutics announced that its self-developed small activating RNA (saRNA) drug RAG-01 received Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA) for the treatment of BCG-unresponsive non-muscle invasive bladder cancer. Previously, the FDA had approved the clinical trial application for RAG-01 for treating non-muscle invasive bladder cancer. Meanwhile, a Phase I clinical trial of RAG-01 has been initiated in Australia, with three subjects already enrolled and dosed. The Fast Track Designation granted by the FDA will significantly accelerate the development process of this drug during the clinical trial period. RAG-01 is also the world's first saRNA project to receive FDA Fast Track Designation.

Fast Track Designation (FTD) was established by the U.S. FDA to expedite the development of new drugs for the treatment of serious diseases that address unmet clinical needs. After obtaining FTD, during the drug development phase, the applicant receives more precise and timely guidance through close communication with the FDA; meanwhile, during the marketing application process, the applicant can submit research materials to the FDA on a rolling basis. These designation benefits will facilitate the rapid development of RAG-01.Provide strong support for development, thereby accelerating the RAG-01 R&D and market launch process.

Dr. Longcheng Li, founder and CEO of Ractigen Therapeutics, stated: "We are delighted to achieve significant progress continuously in the saRNA development field. The FTD designation will accelerate the clinical development of RAG-01, also indicating that the potential of RAG-01 in treating serious diseases and addressing unmet clinical needs has been recognized by the U.S. FDA. In subsequent development, we will maintain active communication with the FDA to expedite the market launch of this product, and we hope to provide NMIBC patients worldwide with a safer and more effective treatment option as soon as possible."

AboutRAG-01

RAG-01RAG-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 to inhibit tumor cell proliferation and induce apoptosis and senescence. Preclinical efficacy studies have shown that, delivered via Ractigen Therapeutics' proprietary small nucleic acid delivery system LiCO™, RAG-01 treatment significantly inhibits the growth of bladder cancer tumors in animal models while demonstrating robust safety.

AboutBladder Cancer (BladderCancer）

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 up to 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.

AboutRNA Activation (RNAa)

RNA Activation (RNAa) is a platform technology pioneered internationally by Dr. Li Longcheng, 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 is an extremely rare platform technology in the pharmaceutical field, with broad application prospects in developing therapeutic drugs, including for cancer, genetic diseases, chronic conditions, metabolic disorders, and cardiovascular and cerebrovascular diseases.

AboutRactigen

Ractigen Therapeutics is a platform-based new drug development company based in China and targeting the global market, committed to developing breakthrough small nucleic acid drugs and disease treatment methods. Ractigen Therapeutics is one of the few global small nucleic acid drug companies that simultaneously possess both intrahepatic and extrahepatic delivery technologies, having developed multiple internationally leading proprietary small nucleic acid drug delivery platform technologies with independent intellectual property rights, 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 disorders, providing innovative therapeutic solutions for undruggable targets and currently incurable diseases across various disease areas.