Therapeutic pipeline

We are progressing a broad therapeutic pipeline of GalOmic™ RNAi therapies that selectively target novel genes, guided by insights from our HepNet™ Computational Platform.

Lead Assets


Cardiometabolic diseases, encompassing multiple conditions such as obesity, cardiovascular disease, and Type 2 Diabetes, are a leading cause of death and morbidity. ETX-291 is a therapeutic for the treatment of cardiometabolic diseases that targets a gene with human genetic evidence of disease-modifying benefit. In preclinical studies in a representative disease model, ETX-291 impacts multiple cardiometabolic disease drivers resulting in a pleiotropic benefit and highlighting its potential to treat a broad range of cardiometabolic indications. 



ETX-148 is a pan-haemophilia treatment that addresses a key remaining unmet need in haemophilia: protection from joint bleeds. Histological data from a preclinical joint bleed model suggests ETX-148 can protect against bleed-induced joint damage. This protection is achieved without an increased risk of thrombosis, which has been characterised in a variety of safety experiments. 

We are continually adding to our pool of novel targets, ensuring our early pipeline is populated and generating additional partnering opportunities. Please get in touch for more information about our preclinical assets or platforms.