Stoke Therapeutics, Inc.

The company is a late-stage clinical company focused on treating severe diseases by increasing protein expression using RNA-based medicines. They use their proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to develop antisense oligonucleotides (ASOs) that selectively restore protein levels. Their primary investigational medicine, zorevunersen (STK-001), is a potential disease-modifying medicine in late-stage clinical testing for Dravet syndrome. Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged, and refractory seizures starting within the first year of life, and is associated with developmental delays and cognitive impairment. There are currently no approved disease-modifying medicines for Dravet syndrome, which is one of many diseases caused by haploinsufficiency, where a loss of approximately 50% of normal protein levels leads to the disease. The company initially focuses on haploinsufficiencies and diseases of the central nervous system and the eye, with proof of concept also demonstrated in other organs, tissues, and systems. Zorevunersen is being evaluated in their global Phase 3 clinical study, EMPEROR, which began in May 2025, with the first patient dosed in August 2025. This study follows two completed open-label Phase 1/2a studies, MONARCH in the United States and ADMIRAL in the United Kingdom, and assesses zorevunersen's efficacy and safety in children and adolescents aged 2 to 18 with Dravet syndrome.