Therapeutic pipeline

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-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.

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.

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.