Position:R&D > Research and Development

Research and Development

RNA interferenceRNAiis a new technology,emerged at beginning of this centurythat can inhibit efficiently of target gene expression. In theory, any disease caused by over-expression of specific gene(s), could be treated by such a small interference RNA (siRNA). siRNA can serve not only as a perfect tool for gene function, its potential in enabling RNAi pharmaceutical approaches is also tremendous. As a highly effective and specific gene silencing technology, siRNA has unique advantage in therapy application against infectious diseases and malignant tumors, with which traditional drugs can’t match. In the past few years, RNAi research and development is characterized by strong involvement of world pharmaceutical giants, and a number of siRNA drugs at different stages of clinical study, which symbolizes that siRNA as a new drug development platform is entering a phase of maturation. At present, the RNAi pharmaceutical industry is still in early stage, and there are still some technical barriers.


Suzhou Ribo is a leading siRNA drug development company in China, devoted to the development of innovative nucleic acid therapeutic drug and related products. Ribo has a professional R&D team focusing onRNAi technology, drug discovery, and other related aspects, and has made significant progress and contributions in the RNAi research and development. Over the years Ribo has built strength on various siRNA drug development platforms: siRNA design, stabilization,bio-evaluation, delivery technology, and synthesis, etc.Ribo hasdeveloped the liquid phase synthesisforsiRNA in order to meet future marketing needs. Ribo has submitted several patent applications in liquid phase synthesis and one of applications has been granted in China, USA, Europe and Japan. Currently, Ribo has developed full-length siRNA liquid phase synthesis technology.Pilot-scale process development/optimizationis on-going.Through collaboration with a number of domestic institutes and internationalBiotech and Pharmaceutical companies, Ribo has explored and evaluated varieties ofsiRNA delivery systems, such as, liposome, lipid complex, polymer, dendrimer, protein and peptide complex, etc. The RBP131, alipid nanoparticle based and liver-targeted delivery system,has been developed for systemic delivery siRNAdrug,The bio-evaluation demonstrated that RBP131 hashigh delivery efficacy with excellent duration time. No cytokines induction, no significant influence on hepatic and renal function wasobserved in animal models. With the platform and technologies establishment, Ribo has built up anenriched product pipeline, including siRNA therapeutics against Hepatitis B, Breast Carcinoma,Liver Carcinoma, Hyperlipidemia, Liver Fibrosis, and HIV, etc. In addition, Ribo has founded a joint venture RiboQuarkwith Quark Pharmaceuticals in 2012 and the first application for clinical trial of siRNA drug (QPI-1007) in China was submitted in January 2014, which has been approved by CFDAin December 2015. QPI-1007 is an anti-NAION (Non-Arteritic Anterior Ischemic Optic Neuropathy) siRNA drug. The other indications such as APACG (Acute Primary Angle-Closure Glaucoma) are also in clinical stages as demonstrated below. 


 

Platforms for siRNA Drug Development

                

 

Liquid Phase Synthesis

Liquid Phase Synthesis Scheme

 

Liver-targeted Delivery System: RBP131



 

Product Pipeline in Ribo (Apr.2017)