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Recently, Rigerna announced that its self-developed small interfering RNA (siRNA) Class I innovative drug "RG002C0106 Injection," for the treatment of complement-related diseases, has officially received approval from the Australian Human Research Ethics Committee (HREC) on August 22 to commence Phase I clinical trial research. This Phase I clinical study aims to evaluate the safety, tolerability, and pharmacokinetic characteristics of RG002C0106 Injection in healthy volunteers.RG002C0106 Injection is an siRNA drug developed by Rigerna based on its independently established core technology platforms such as RIHOST®, RICMO®, and LICOD®, utilizing its self-developed second-generation N'-acetylgalactosamine (GalNAc) conjugation technology, targeting the complement factor C3. It is also the first pipeline of Rigerna to enter the clinical trial stage.Preclinical studies have demonstrated that RG002C0106 exhibits excellent efficacy, safety, and long-lasting effects, and has been validated in various preclinical animal models, including rodents and non-human primates, particularly in...Cynomolgus MonkeyA dose-dependent improvement was observed in a series of clinically significant endpoint indicators in the disease model.RG002C0106 is expected to become the best-in-class and one of the world's first marketed siRNA drugs targeting C3.Dr. Huang Yuanyu, founder of Rigerna, stated"Since its establishment in 2022, Rigerna has efficiently and rapidly built a small nucleic acid pharmaceutical technology platform with core independent intellectual property rights. The approval of RG002C0106 injection by the Australian HREC marks a significant milestone for the company, transitioning from early drug development to the clinical stage. This not only demonstrates the team's high execution ability and professionalism but also reaffirms the capabilities of Rigerna’s small nucleic acid pharmaceutical technology platform. Unlike currently marketed drugs for complement-related diseases, RG002C0106 represents an entirely new class of medication, with the potential to offer patients suffering from complement-related diseases more effective, safer, and more economical treatment options, addressing a significant unmet clinical need. Rigerna will continue to accelerate clinical trials, striving to provide patients with new therapeutic options as soon as possible."Rigerna was founded in 2022. The company focuses on the research and development and industrialization of innovative small nucleic acid drugs, with a base in China and a vision for the global market. It is committed to becoming a leading force and innovator in the field of nucleic acid pharmaceuticals.
In terms of drug design, the R&D team of Suzhou Rigerna Therapeutics, based on prior experience in designing and developing drug molecules for dozens of targets, has developed multiple siRNA design and analysis software tools. By integrating the functions of these software tools, they launched the RAIHOST platform, incorporating past design and R&D experience into the development of new targets. This has increased the efficiency of nucleic acid drug design and screening by dozens of times compared to traditional methods, while significantly enhancing the rationality and precision of drug design. The company has now registered several software copyrights and continues to iterate and improve the system.
For extrahepatic delivery, the team, based on its deep understanding, has adopted a variety of exclusive innovative strategies for parallel development. Currently, exciting animal-level data has been obtained in multiple extrahepatic tissues, which will greatly support subsequent product development for extrahepatic targets and indications. Rigerna's forward-looking strategy and systematic layout since its establishment have ensured that the company reaches important milestones in the short term, while also supporting the company’s development for the next 10, 20 years, or even longer.
Source: Rigerna
