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.

MASH – metabolic dysfunction-associated steatohepatitis
AMD - age-related macular degeneration


Metabolic dysfunction-associated steatohepatitis (MASH) is a highly prevalent disease with limited treatment options and significant unmet need. ETX-312 has shown exceptional results in Gubra’s industry-leading diet-induced obesity model of MASH, rated by experts as the most translatable murine model of the condition. ETX-312 has been assessed as both a monotherapy and in combination with leading MASH assets, including GLP-1 receptor agonists.


ETX-407 has the potential to be a transformative, patient-friendly therapeutic option for the millions of people with dry age-related macular degeneration (dAMD), a leading cause of blindness worldwide. In dAMD, the ability to modulate disease pathology with an infrequent subcutaneous injection would be a substantial improvement upon the highly invasive intravitreal injections that are current standard-of-care. ETX-407 targets a gene with human validation and demonstrates the applicability of ETX’s hepatocyte-targeting GalOmic platform to tackle indications affecting distal organs.


ETX-148 is a low burden 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.


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.

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.