Stoke Therapeutics Inc

Stoke Therapeutics is a clinical-stage biotechnology company that develops RNA medicines intended to restore natural protein expression. It uses its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to design antisense oligonucleotides (ASOs) for diseases caused by haploinsufficiency, meaning a loss of roughly 50% of normal protein levels. Its initial focus is on disorders of the central nervous system and the eye.

Its lead candidate, zorevunersen, is an ASO for Dravet syndrome designed to increase NaV1.1 protein from the healthy copy of the SCN1A gene. It holds the following designations:
- FDA and EMA orphan drug designation
- FDA rare pediatric disease designation
- FDA Breakthrough Therapy Designation
- Breakthrough Therapy Designation from China's Center for Drug Evaluation

Zorevunersen is in the global Phase 3 EMPEROR study. Data are expected in Q3 2027 and a U.S. NDA submission in H2 2027, with a potential U.S. launch in early 2028. Stoke co-develops zorevunersen with Biogen: Stoke keeps exclusive rights in the U.S., Canada and Mexico, and Biogen holds rest-of-world commercialization rights.

The pipeline also includes:
- **STK-002:** an ASO for Autosomal Dominant Optic Atrophy (ADOA), with FDA orphan drug designation, in the Phase 1 OSPREY dose-escalation study in Europe.
- **SYNGAP1-related disorders:** a lead optimization program.

The company currently earns revenue from collaboration and licensing agreements with Acadia and Biogen. Updated Oct 29, 2026