Pipeline

RBD4059, a GalNAc-siRNA drug based on our proprietary liver-targeting delivery technology RiboGalSTAR^TM, is being developed for treatment of thrombotic diseases. As the world's first and most advanced siRNA drug in clinical development targeting FXI (First-In-Modality), RBD4059 offers distinct advantages over both small molecules and antibodies for FXI inhibition with the extended dosing intervals up to several months as well as the reduced risk of immune reactions and anti-drug antibodies (ADA). Furthermore, RBD4059 combines the advantages of FXI-targeting with siRNA drug technology, offering reduced bleeding risk while maintaining strong efficacy compared to standard-of-care. RBD4059 has completed a phase Ⅰ clinical trial in Australia, and the results showed good safety profile, long acting and strong therapeutic effects. A Phase IIa clinical trial application was approved by the EMA, and enrollment of all participants has been completed.

RBD5044, a GalNAc-siRNA drug based on our proprietary liver-targeting delivery technology RiboGalSTAR^TM, is being developed for the treatment of triglycerides (TGs)-type hypertriglyceridemia. RBD5044 is the second siRNA globally to enter clinical development targeting ApoC3. The Phase I clinical trial conducted in Australia demonstrated a favorable safety profile, along with robust and long-acting therapeutic effects. These results support sustained TG control with quarterly or bi-annual dosing schedules, significantly improving patient adherence. A Phase II clinical trial application has been approved by the EMA, and the study is ongoing.

RBD7022, a GalNAc-siRNA drug based on our proprietary liver-targeting delivery technology RiboGalSTAR^TM, is being developed for the treatment of hyperlipidemia. By inhibiting the expression of PCSK9, RBD7022 reduces blood LDL-C levels. A Phase I clinical trial for RBD7022 has been completed in China, and a phase II clinical trial is ongoing. Development, manufacture and commercialization rights for RBD7022 in Mainland China, HK, and Macau have been licensed out to Qilu Pharmaceutical.

RBD7007, a GalNAc-siRNA drug based on our proprietary liver-targeting delivery technology RiboGalSTAR^TM, is being developed for treatment of complement-mediated diseases. RBD7007 inhibits the expression of complement components in hepatocytes to reduce the overactivation of the complement system, achieving the effect of treating complement-related diseases, such as renal and autoimmune diseases. RBD7007 has demonstrated robust and long-duration pharmacological effects in cynomolgus monkeys. A clinical trial application for a first-in-human study has been approved by EMA.

RBD2080, a GalNAc-siRNA drug based on our proprietary liver-targeting delivery technology RiboGalSTAR^TM, is being developed for treatment of complement-mediated diseases. RBD2080 inhibits the expression of complement components in hepatocytes to reduce the overactivation of the complement system, achieving the effect of treating complement-related diseases, such as renal and autoimmune diseases. A clinical trial application for a first-in-human study of RBD2080 has been approved in Australia.