Suzhou Ribo Life Science Co., Ltd is committed to the improvement of key technologies around siRNA therapeutic development. The liver targeting delivery technology and second-generation modification technology at the company have evolved to a level of maturation that supports rapid development of drug candidates to clinical stages. Based on the system, four compounds have been advanced into Phase II or Phase I clinical stage to date, thus have generated encouraging data about safety, efficacy, and translational properties of the system. Ribo has been investing great efforts and resources in the development of siRNA delivery technologies for extrahepatic tissues and organs, such as, tumor，central nervous system (CNS), lung, kidney and muscle, etc. In directions such as third-generation modification technology, multi-gene silencing technology, and innovative synthesis technology, Ribo has also carried out a lot of research and achieved interesting results.
siRNA modification is a very important key technology in improving the efficiency, durability, and safety of siRNA drugs. Naked or unmodified oligonucleotides not only are highly susceptible to degradation, but also can trigger different degrees of their off-target effects and/or immunogenicity. Based on a large number of experimental results and deep understanding experience, it was recognized that innovative combinations of different siRNA backbone modifications, ribose modifications, base modifications, or other modifications can greatly improve the druggability of siRNA candidates. RSC2.0（Ribo Stabilization Chemistry）is made based on such systematic studies on the mechanisms of siRNA degradation and stabilization and their correlation with potency and/or off-target effects. RSC2.0 technology has been granted its patents from major patent offices in the United States, Canada, Australia, and other important jurisdictions, making it one of the few companies globally to obtain patent authorization for the siRNA chemical modification platform. Suzhou Ribo Life Science Co., Ltd is committed to continuously exploring an optimized modification platform aiming for furthermore improved the efficiency, durability, and safety of their siRNA products.
The delivery systems based on the uptake of siRNA via interaction of conjugated N-acetylgalactosamine (GalNAc) with its receptor has tremendously accelerated the siRNA drug development for the treatment of diseases associated with any gene expression in hepatocytes. GalNAc-siRNA conjugates specifically bind to the asialoglycoprotein receptor (ASGPR) on the surface of hepatocytes and trigger fast endocytosis of the siRNA. This can result in highly specific and highly durable enrichment of the siRNA in hepatocytes. GalNAc-based System for liver TARgeting, named RIBO-GalSTARTM, is an siRNA liver targeting delivery technology that Suzhou Ribo Life Science Co., Ltd developed independently through efforts in the past decade. The patents of RIBO-GalSTARTM have been granted by the patent authorities in important territories such as China, the United States, etc, and the technology has been employed in the research and development of a full panel of siRNA drug candidates for various targets and indications related to gene expression in the liver. Four of the drug products have been advanced into the different clinical stages, among which the fastest products have entered the phase II. More differentiated innovative drugs are in preclinical and IND application stages.
Suzhou Ribo Life Science Co., Ltd has been investing great efforts and resources in the development of siRNA delivery technologies for extrahepatic tissues and organs, such as, tumor，central nervous system (CNS), lung, kidney and muscle, etc. Pipelines have been initiated in three therapeutic areas: Oncology，Renal Diseases, and Neruo-diseases.
Among these, RIBO-OncoSTAR TM (Oncology Specific TARgeting technology) is a proprietary siRNA tumor delivery technology developed independently by Ribo, which enables specific drug delivery to a variety of tumors. Ribo has made significant progress in the development of siRNA therapeutics and diagnostic projects targeting glioma based on RIBO-OncoSTARTM. Efforts are being accelerated to quickly advance towards clinical studies. RIBO-OncoSTARTM will be further explored for treatment of other cancer diseases, too.