Tides Express | Xuanjing Bio's siRNA Innovative Drug Receives CDE IND Acceptance

On June 21, 2024, Rigerna announced that its self-developed small nucleic acid (siRNA) Class I innovative drug "RG002C0106 Injection" for the treatment of complement-related diseases has been officially accepted by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA).

RG002C0106 Injection is an siRNA drug developed by Rigerna based on its proprietary core technology platforms, including RIHOST®, RICMO®, and LICOD®. It utilizes second-generation N'-acetylgalactosamine (GalNAc) conjugation technology for efficient delivery to hepatocytes and inhibits the expression of complement factors in the liver, thereby treating complement-mediated diseases. Preclinical studies have demonstrated that RG002C0106 exhibits excellent efficacy, safety, and long-lasting effects, validated across various preclinical animal models, including rodents and non-human primates. Notably, significant dose-dependent improvements in a series of clinical endpoint indicators were observed in cynomolgus monkey disease models, showing great potential to become the world's first-in-class drug.

About Nucleic Acid Drugs

Nucleic acid drugs are a type of therapeutic approach that regulates at the post-gene transcription and pre-protein translation stage. Unlike traditional small-molecule or monoclonal antibody drugs, nucleic acid drugs are composed of nucleotides with specific sequences, utilizing the translation or regulatory functions of nucleic acid molecules to intervene at the source of diseases. Compared with traditional drugs, they possess both "symptomatic and radical treatment" capabilities, offer abundant targeting options, and have the advantage of digital design, which can significantly accelerate their research and development process, transforming many diseases from "untreatable" to "treatable." They cover a wide range of disease spectrums and can extend short-acting drugs into long-acting or even ultra-long-acting treatments, showing immense potential.

AboutRigerna

Suzhou Rigerna Therapeutics and Beijing Rigerna Pharmaceuticals (Rigerna) were established in 2022. The company focuses on the research, development, and industrialization of innovative small nucleic acid drugs, with a foundation in China and a vision for global impact (In China, For Global). It is committed to becoming a leader and innovator in the field of nucleic acid pharmaceuticals.