ETX-148

ETX-148 is an investigational small interfering RNA (siRNA) therapeutic developed by e-therapeutics for the treatment of bleeding disorders, with a primary focus on haemophilia A and B. It is designed as a pan-haemophilia rebalancing agent to provide joint protection, reduce inflammation, and minimize haemorrhage in preclinical models. The drug leverages e-therapeutics' proprietary GalOmic™ GalNAc-siRNA technology for targeted delivery to hepatocytes and was discovered using the company's HepNet™ computational platform, which identifies novel gene targets through network analysis of coagulation pathways. ETX-148 aims for infrequent subcutaneous dosing (quarterly or longer), compatibility with emergency factor replacement therapies, and a favorable safety profile without increasing thrombosis risk. As of June 2025, it is in IND-enabling studies with an IND submission anticipated in 2026[1][3][4][6].