Stoke Therapeutics, Inc.

Stoke Therapeutics Inc. (NASDAQ: STOK) is a late-stage clinical biopharmaceutical company dedicated to developing RNA-based medicines that address the underlying causes of severe genetic diseases by upregulating protein expression. The company operates through its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform, which uses antisense oligonucleotides to selectively restore protein levels in patients with haploinsufficiency disorders—conditions where loss of approximately fifty percent of normal protein levels leads to disease manifestation.

The company's lead investigational medicine, zorevunersen (STK-001), is in late-stage clinical development for Dravet syndrome, a severe developmental and epileptic encephalopathy characterized by frequent, prolonged seizures beginning in infancy, along with significant cognitive and behavioral impairments. Dravet syndrome affects approximately one in 15,600 babies globally, with an estimated 15,700 patients in the United States alone. Zorevunersen is designed to upregulate Nav1.1 protein expression by leveraging the non-mutant copy of the SCN1A gene. The company initiated its global Phase 3 EMPEROR study in May 2025, with enrollment expected to complete in the second quarter of 2026 and pivotal data anticipated in mid-2027 to support a New Drug Application submission to the FDA. In December 2024, zorevunersen received Breakthrough Therapy Designation from the FDA.

Stoke's second product candidate, STK-002, targets autosomal dominant optic atrophy (ADOA), a rare inherited disorder causing progressive vision loss. STK-002 is currently in Phase 1 clinical testing through the OSPREY study, which began dosing patients in February 2026.

The company's TANGO platform identifies and targets non-productive alternative splicing events in genes associated with severe diseases, focusing initially on haploinsufficiency disorders affecting the central nervous system and eye. Stoke has identified approximately 3,200 disease-associated genes containing non-productive splicing events potentially amenable to TANGO treatment. The company collaborates with Biogen on the development and commercialization of zorevunersen outside the United States, Canada, and Mexico, and maintains an ongoing collaboration with Acadia Pharmaceuticals for SYNGAP1 development. Stoke maintains its website at www.stoketherapeutics.com.