Founded in 2007, we are a biopharmaceutical company engaged in oligonucleotide research and development, with a focus on siRNA therapeutics. We have one Core Product, RBD4059 (FXI-targeting siRNA), targeting thrombotic diseases, among a pipeline of seven in-house discovered drug assets in clinical trials for seven indications across cardiovascular, metabolic, renal and liver diseases, including four in phase 2 clinical trials.
WE MAY NOT BE ABLE TO SUCCESSFULLY DEVELOP, MARKET AND/OR GENERATE MEANINGFUL ECONOMIC VALUE FROM OUR PIPELINE PRODUCTS, INCLUDING OUR CORE PRODUCT RBD4059.
OUR PIPELINE
The pipeline chart below summarizes the development status of our clinical-stage drug candidates and selected preclinical assets. All drug candidates listed in this pipeline chart were discovered internally by our research and development team. Leveraging our RiboGalSTARTM platform equipped with proprietary and clinically validated GalNAc delivery technology, we have consistently advanced siRNA programs in-house from discovery through clinical development across cardiovascular, metabolic, renal and liver diseases.
Besides our Core Product RBD4059, we are advancing other clinical-stage products, including RBD5044 and RBD1016. RBD5044 is the second siRNA globally to enter clinical development that targets APOC3, a protein that plays a role in lipid metabolism. RBD1016, with its effect on HBsAg, targets to achieve functional cure of CHB, while being a differentiated siRNA candidate for CHD. Beyond our clinical pipeline, we maintain over 20 preclinical programs that we aim to advance into clinical development.
We are actively advancing our drug pipeline in each key therapeutic area:
‧ Cardiovascular, metabolic and renal diseases. Cardiovascular, metabolic and renal diseases are chronic conditions affecting vast patient populations worldwide. These interconnected diseases involve multiple organs and systems, with the liver serving as a key metabolic hub in their development and progression. Given the liver’s central role in regulating many disease pathways, our liver-targeting pipeline, powered by our proprietary RiboGalSTARTM delivery technology, offers a treatment approach to these widespread conditions.
We are developing a comprehensive siRNA franchise for the treatment of thrombosis and dyslipidemia, represented by our Core Product RBD4059 and two other pipeline assets, RBD5044 and RBD7022, all currently in phase 2 clinical trials. Leveraging three targets, namely FXI, APOC3 and PCSK9, our drug franchise provides a multi-pronged approach to treating these complex diseases with synergistic potential.
RBD4059 (FXI-targeting siRNA), our Core Product, is a clinical-stage siRNA drug that targets thrombotic diseases. Thrombotic diseases have emerged as one of the leading causes of death worldwide, claiming over 10 million lives each year. By selectively inhibiting FXI, RBD4059 can potentially reduce the risk of thrombus formation without significantly increasing bleeding risks, a common limitation of traditional anticoagulants, while providing long-lasting effects with infrequent dosing to improve patient compliance. Taken together, RBD4059 represents a therapeutic option to treat and prevent thrombosis associated with atherosclerotic cardiovascular disease (“ASCVD”) and other conditions associated with abnormal clot formation, such as atrial fibrillation, cancer-associated thrombosis, and venous thromboembolism.
We completed RBD4059’s phase 1 trial in Australia in healthy subjects in October 2024 and obtained the EMA’s CTA approval in May 2024, pursuant to which we initiated RBD4059’s phase 2a clinical trial in Sweden in August 2024. All patients in this phase 2a trial have completed treatment and are currently in the safety follow-up period. See “Business — Our Pipeline — Cardiovascular, Metabolic and Renal Diseases — RBD4059” for details.
Meanwhile, RBD5044 (APOC3-targeting siRNA) and RBD7022 (PCSK9-targeting siRNA) are two assets designed for the treatment of hypertriglyceridemia (“HTG”) and hypercholesterolemia, respectively — two common forms of dyslipidemia that significantly contribute to cardiovascular and metabolic diseases. Strategically, RBD5044 and RBD7022 serve as complementary monotherapies within our broader dyslipidemia portfolio. While each addresses distinct aspects of dyslipidemia independently, their combined use offers the potential for enhanced lipid management by addressing both elevated triglycerides and cholesterol levels.
We have completed RBD5044’s phase 1 trial in Australia and submitted a CTA to EMA for RBD5044’s phase 2 trial, which was approved in October 2024. This phase 2 trial is currently ongoing in Sweden in patients with mixed dyslipidemia. We obtained an IND approval from the NMPA for RBD7022 in September 2022 and completed the phase 1 trial of RBD7022 in March 2025 in China. See “Business — Our Pipeline — Cardiovascular, Metabolic and Renal Diseases — RBD5044” and “Business — Our Pipeline — Cardiovascular, Metabolic and Renal Diseases — RBD7022” for details.
Cardiometabolic diseases have a well-established association with renal disorders, wherein inflammation and autoimmunity play pivotal roles. To address these interrelated pathologies, we have established a complement factor pipeline to target various renal and autoimmune diseases. Dysfunctions in the complement system can lead to tissue damage and inflammation, contributing to complement-mediated renal and autoimmune conditions such as IgA nephropathy (“IgAN”). Our GalNAc- conjugated siRNA candidates RBD7007 and RBD2080 are engineered to specifically target complement proteins in liver cells — the primary site of their production. This approach effectively reduces the levels of these complement proteins at their source and in circulation.
We obtained the CTA approval from the EMA in September 2024 to initiate RBD7007’s phase 1 clinical trial. For RBD2080, we received the TGA’s acknowledgment of our clinical trial notification in February 2025. See “Business — Our Pipeline — Cardiovascular, Metabolic and Renal Diseases — RBD7007 and RBD2080” for details.
‧ Liver diseases. Despite medical advances, the treatment of liver diseases remains challenging. The inability of traditional treatments to target intracellular pathways within liver cells, coupled with severe side effects from systemic exposure, has left unmet need in the treatment of liver diseases and their complications, which account for approximately two million deaths annually. Our liver-targeted RiboGalSTARTM delivery technology is designed to enable siRNA therapies to leverage intracellular pathways which were previously considered undruggable.
Our liver disease strategy concentrates on two therapeutic areas with medical needs: chronic viral hepatitis, including chronic hepatitis B (“CHB”) and chronic hepatitis D (“CHD”), and metabolic dysfunction-associated steatohepatitis (“MASH”), particularly advanced diseases.
Our liver disease pipeline is led by RBD1016, an siRNA candidate in global clinical development for patients with chronic hepatitis B Virus (“HBV”) infection, including those with hepatitis D virus (“HDV”) co-infection. Current antiviral therapies, primarily interferons and nucleoside analogs, are limited with no effective functional cure. With our liver-targeted RiboGalSTARTM delivery technology, RBD1016 represents a therapeutic opportunity for CHB due to its differentiated intracellular mechanism of action that potentially exerts multiple antiviral effects, particularly the suppression of HBsAg, which is known to cause adverse CHB- associated liver complications. RBD1016, with its potent and durable effect on HBsAg, is positioned as a backbone therapy in future combination approaches to achieve functional cure of CHB, and a differentiated siRNA candidate for CHD.
For MASH, we focus on advanced disease stages, where our RiboGalSTARTM delivery technology can potentially address the lack of effective therapeutics for fibrosis and inflammation — conditions where systemic treatments not only lack efficacy but can lead to serious side effects. This approach is exemplified by our strategic collaboration with Boehringer Ingelheim to develop siRNA drugs targeting multiple novel disease pathways for the treatment of MASH.
We have completed RBD1016’s phase 2 global MRCT in CHB patients, with the last patient’s final visit achieved in October 2025, and are currently finalizing data analysis for this trial. We received IND approval from the NMPA in October 2024, which enables us to potentially expand RBD1016’s clinical trials for CHB into China. We also commenced a phase 2a trial in Sweden in August 2024 to further explore the therapeutic potential of RBD1016 for treating CHD, with trial completion expected by the end of 2026. See “Business — Our Pipeline — Liver Diseases — RBD1016” for details.
‧ Other therapeutic areas. We are also developing drug candidates for hereditary angioedema (“HAE”) and inflammatory diseases based on our RiboGalSTARTM delivery technology. We currently have over 20 other preclinical assets in our pipeline, including multiple siRNA candidates derived from RiboPepSTARTM, our proprietary platform being developed to target extra-hepatic organs and tissues like the kidney, CNS, and metabolic tissues such as adipocytes and muscles. Meanwhile, we have one drug candidate in IND-enabling studies for the treatment of glioma, leveraging RiboOncoSTARTM, our proprietary oncology-focused technology platform. We believe the agility of our technology platforms presents broad clinical potential, with the capability to advance two to four assets into clinical stage each year.
WE MAY NOT BE ABLE TO SUCCESSFULLY DEVELOP AND/OR MARKET OUR CORE PRODUCT, OR ANY OF OUR DRUG CANDIDATES.
Source: Ribo Life-B (06938) Prospectus (IPO Date : 2025/12/31) |