
RNAi Drug Developer


Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare acquired blood disorder. The core pathogenesis stems from abnormal complement activation-induced hemolysis of red blood cells, accompanied by clinical manifestations such as high incidence of thrombosis and bone marrow failure. Epidemiological data show that the global prevalence of PNH is approximately 10 to 20 per million people. Patients often suffer from severe anemia, extreme fatigue, abnormal hemoglobinuria, kidney damage, and pulmonary hypertension, among other symptoms. Thromboembolism is the most life-threatening cause of mortality in this disease, accounting for 40% to 67% of PNH-related deaths, significantly reducing patients' survival time and greatly impairing their quality of life. siRNA-targeted therapy, with its precise mechanism, prolonged efficacy, reduced dosing frequency, and enhanced safety and compliance, holds promise to offer a new optimized treatment option for PNH patients.
Immunoglobulin A Nephropathy (IgA Nephropathy) is a chronic glomerular disease caused by the deposition of abnormal IgA complexes in the renal glomeruli, continuously triggering local inflammatory damage, which leads to progressive renal function decline. As the most prevalent primary glomerular disease globally, it carries a severe prognosis, with more than half of patients developing end-stage renal failure within 10 to 20 years of diagnosis. The current global patient population is substantial, and existing clinical intervention options are very limited. The industry urgently needs innovative therapeutic drugs capable of halting disease progression and improving long-term patient outcomes.
Argo Biopharma is a clinical-stage biotechnology company dedicated to developing a new generation of RNAi therapies, providing better treatment options for patients worldwide. The company leverages its liver-targeting and extrahepatic delivery technology platforms to build a diversified RNAi drug R&D pipeline covering cardiometabolic diseases, specialty disorders, viral infections, central nervous system diseases, and rare diseases. Currently, Argo Biopharma has seven RNAi candidate drugs in clinical studies.
Source:
1.Argo