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On May 21, Ractigen Therapeutics announced that its self-developed small activating RNA (saRNA) drug RAG-01 had been granted Fast Track designation by the U.S. FDA.For the treatment of BCG-unresponsive non-muscle-invasive bladder cancer。Bladder cancer is a common cancer of the urinary system, among which NMIBC accounts for 50% to 80%. The standard treatment for NMIBC is primarily transurethral resection of bladder tumor (TURBT), but the postoperative recurrence rate is high. Therefore, there is an urgent need to develop drugs that can effectively prevent the recurrence of bladder cancer and treat advanced bladder cancer.RNA Activation (RNAa) Technology Utilizes Double-Stranded RNA Targeting Gene Promoter Regions to Activate Gene Expression, Restoring Therapeutic Protein Levels. RNAa Technology Holds Broad Application Prospects in Developing Therapeutic Drugs for Tumors, Genetic Disorders, Chronic Diseases, Metabolic Disorders, and Cardiovascular and Cerebrovascular Diseases.RAG-01 is a specific targeted activator of tumor suppressor genesp21The double-stranded saRNA drug activates through the RNAa mechanismp21Gene expression to suppress tumor cell proliferation, induce apoptosis and senescencePreclinical efficacy studies have shown that, through Ractigen's self-developed small nucleic acid delivery system, RAG-01 treatment significantly inhibits the growth of bladder cancer tumors in animal models and demonstrates robust safety.Previously, the clinical trial application for RAG-01 for the treatment of non-muscle invasive bladder cancer has been approved by the FDA. Meanwhile, the phase 1 clinical trial of RAG-01 has been initiated in Australia, with three subjects currently enrolled and receiving dosing.[1] Ractigen's RAG-01 Becomes the First saRNA Drug to Receive FDA Fast Track Designation. Retrieved May 22, 2024, from https://mp.weixin.qq.com/s/6OBvsEcEi-RrIeR8Ytvbug
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