Stoke Therapeutics Inc Dossier
Qualitative Analysis
Business overview
Stoke Therapeutics, Inc. (NASDAQ: STOK) is a clinical-stage biotechnology company dedicated to addressing the underlying genetic causes of severe diseases by restoring protein expression. Utilizing its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform, the company designs antisense oligonucleotides (ASOs) to selectively upregulate protein levels. Stoke's lead investigational medicine, zorevunersen (STK-001), is being developed as a first-in-class potential disease-modifying treatment for Dravet syndrome, a rare and severe genetic epilepsy. The company is also developing STK-002 for the treatment of autosomal dominant optic atrophy (ADOA). Stoke is headquartered in Bedford, Massachusetts.
Research as of 20 Jun 2026
Strategic Initiatives
Growth programs, investments, and their expected impact
Advancing zorevunersen, a first-in-class potential disease-modifying antisense oligonucleotide (ASO) designed to treat the underlying cause of Dravet syndrome by upregulating NaV1.1 protein production.
Expected impact: Aims to reduce seizure frequency and improve neurodevelopment, cognition, and behavior in patients with Dravet syndrome.
Developing STK-002, an investigational antisense oligonucleotide designed to upregulate OPA1 protein expression for the treatment of Autosomal Dominant Optic Atrophy (ADOA).
Expected impact: Aims to restore OPA1 protein levels to maintain or improve vision in people with ADOA, a rare genetic disease with no currently approved treatments.
Mergers, Acquisitions & Partnerships
Recent deals and strategic collaborations
Strategic Partnerships
Broadens the global reach of zorevunersen by leveraging Biogen's deep expertise in neurology and rare genetic diseases, while providing Stoke with substantial non-dilutive funding.
Terms: Stoke received a $165 million upfront payment. Stoke is eligible to receive up to $385 million in development and commercial milestone payments, plus tiered royalties ranging from low double digits to high teens on net sales outside North America. Clinical development costs are shared (70% Stoke, 30% Biogen).