We are committed to continuous innovation in key siRNA pharmaceutical technologies. The RiboGalSTARTM liver-targeting delivery technology is now fully mature. RiboOncoSTARTM, our first extrahepatic delivery technology, provides targeting capabilities for various tumor tissues. RiboPepSTARTM represents a systematic layout for targeting delivery to tissues and organs beyond the liver, with candidate delivery molecules identified for kidney targeting, central nervous system (CNS) targeting, adipose targeting, and muscle targeting. We have also made significant progress in siRNA chemical modification platform and multi-target drug design platform.
Our liver-targeting RiboGalSTARTM platform enables the delivery of siRNA to various liver-derived targets and indications. siRNA conjugated with GalNAc specifically binds to the asialoglycoprotein receptor (ASGPR) expressed on hepatocytes. siRNA enters the liver cells via endocytosis, and initiates RNA interference (RNAi) by specifically cleaving the target gene mRNA.
Our RiboGalSTARTM platform offers competitive targeting, specificity and efficiency. To date, RiboGalSTARTM has advanced six programs into clinical development across cardiovascular, metabolic, renal and liver diseases. The high potency, long duration, and favorable safety profile have been clinically validated. This marks it as one of the most productive GalNAc platforms globally.
We are developing RiboOncoSTARTM, a leading tumor-targeted platform utilizing oligonucleotide-conjugate delivery technology, to support our development of multiple potentially first-in-class cancer treatments. This platform enables specific targeted delivery to solid tumors.In preclinical studies, RiboOncoSTARTM has shown superior anti-tumor effects and safety profiles in selected cancer types (including glioma, pancreatic cancer, etc) compared to standard-of-care treatments. These attributes the position of RiboOncoSTARTM as a globally leading technology in tumor-targeted oligonucleotide delivery. And potentially expand to the development of targeted chemotherapy, targeted radiopharmaceuticals, and other next-generation targeted therapies and diagnostic approaches.
We are delivering our siRNA drug candidates to multiple critical organs and tissues with our RiboPepSTARTM platform. RiboPepSTARTM is the world’s first delivery technology that achieves targeted delivery to multiple extrahepatic organs by conjugated molecules. The advantages of RiboPepSTARTM are its ability to obtain specific delivery technology for any extrahepatic organ, tissue, or even target cell type, with short screening cycles and good peptide stability.
The platform has demonstrated significant efficacy superior to existing therapies in multiple disease models for kidney and CNS delivery. With a favorable safety profile, the platform supports our position at the forefront of global innovation in siRNA research.
Our leadership in chemical modification complements our delivery technologies as a core competitive advantage. Chemical modifications are essential for developing effective oligonucleotide therapeutics, protecting nucleic acids from degradation while minimizing off-target effects and immunogenicity. our proprietary RSC (Stabilization Chemistry) platform is a siRNA chemical modification technology based on systematic research on the degradation mechanism, sequence activity, and off target rules of siRNA. Platform achieves potent, sustained gene suppression with enhanced target specificity, significantly improving drug safety and developability.
The synergy between RSC and our RiboGalSTARTM delivery technology is demonstrated by the favorable safety profile and sustained efficacy of RBD4059 and other clinical-stage assets. We have continued to iterate this technology, featuring broader sequence compatibility and a unique strategy to reduce off-target effects, and AI-empowered strategies.
While most siRNA drugs are designed with only one target, our multi-target siRNA drugplatform enables a single drug molecule to interfere with two or more targets simultaneously, achieving a synergistic therapeutic effect by allowing combinations of two or more targets in varying ratios, offering a technological advantage.
We have developed software dedicated to designing oligonucleotide drug sequences, capable of analyzing predefined parameters such as off-target gene identification, cross-species comparison and homology assessment to quickly select high-quality siRNA sequences with optimal specificity and activity. High-throughput screening platform for oligonucleotide compounds rapidly generates lead candidates.
By leveraging modeling and simulation techniques, we quantitatively analyze drug characteristics and disease-related data, gaining a deeper understanding of siRNA mechanisms and improving predictability at each stage of drug development.
