Stoke Therapeutics Inc

Stoke Therapeutics (Nasdaq: STOK) is a clinical-stage biotechnology company focused on restoring protein expression using RNA-based medicines for severe genetic diseases caused by haploinsufficiency — conditions where a loss of approximately 50% of normal protein levels leads to disease. The company's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform develops antisense oligonucleotides (ASOs) designed to selectively upregulate protein production from the unaffected copy of a gene. Stoke's lead investigational medicine, zorevunersen, is being developed as a potential first-in-class disease-modifying treatment for Dravet syndrome, a severe developmental and epileptic encephalopathy affecting an estimated 38,000 people across the U.S., UK, EU-4, and Japan. Zorevunersen is currently in a pivotal Phase 3 study (EMPEROR) and has received Breakthrough Therapy Designation from both the FDA and China's Center for Drug Evaluation. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen, retaining exclusive U.S., Canada, and Mexico rights while Biogen holds rest-of-world commercialization rights. The company also has a collaboration with Acadia Pharmaceuticals. Beyond Dravet syndrome, Stoke is advancing STK-002 in a Phase 1 study for Autosomal Dominant Optic Atrophy (ADOA), the most common inherited optic nerve disorder, and is pursuing lead optimization for SYNGAP1-related neurodevelopmental disorders. The company's initial therapeutic focus areas are central nervous system and ocular diseases. Updated Aug 3, 2026