Stoke Therapeutics, Inc. focuses on addressing the underlying causes of severe genetic diseases by upregulating protein expression. The company's proprietary TANGO approach targets pre-mRNA splicing to increase gene expression, primarily focusing on haploinsufficiency diseases of the central nervous system and the eye. Its lead product candidate, zorevunersen (STK-001), is a potential first-in-class disease-modifying treatment for Dravet syndrome, a severe developmental and epileptic encephalopathy. Stoke is also advancing early-stage programs, including STK-002 for Autosomal Dominant Optic Atrophy (ADOA). In 2025, the company entered into a strategic collaboration with Biogen to develop and commercialize zorevunersen globally, while retaining exclusive rights in the United States, Canada, and Mexico.
