Rare disease drugmaker · based in Bedford, United States · founded 2014 · ticker STOK · stoketherapeutics.com
Stoke Therapeutics works on Dravet Syndrome, one of the rare diseases we track. It has 2 active clinical trials, though none are looking for new volunteers right now.
Stoke Therapeutics, based in Bedford, Massachusetts, develops antisense oligonucleotide (ASO) medicines that raise the protein made by a person's working copy of a gene, using its TANGO platform (Targeted Augmentation of Nuclear Gene Output). Its lead drug, zorevunersen (formerly STK-001) for Dravet syndrome, is in the Phase 3 EMPEROR trial, whose main group of 162 children finished enrolling in June 2026.
Stoke Therapeutics pipeline
Each row lists the diseases Stoke Therapeutics is studying at that stage, from the ones closest to approval down to the earliest work.
Phase 3
Large studies, usually the last step before a company asks the FDA for approval.
These are Stoke Therapeutics trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
2 trials underway, not taking new volunteers
Underway, not taking new volunteersPhase 3Recently updatedNCT06872125
A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
Stoke Therapeutics is evaluating the long-term safety \& tolerability of repeated doses of zorevunersen (STK-001) in patients with Dravet syndrome who previously participated in studies of zorevunersen. Change in seizure frequency and overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study.
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Trial Alerts, when a new trial opens or a trial changes
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Stoke expects results in the third quarter of 2027 and plans to complete a U.S. application in the second half of 2027. Biogen holds rights outside the United States, Canada and Mexico.
STK-002, for autosomal dominant optic atrophy (ADOA), an inherited cause of vision loss, is in the Phase 1 OSPREY study at sites in Europe, with first data expected in the first half of 2027. Stoke is also searching for a drug candidate for SYNGAP1-related disorders.
Where Stoke Therapeutics trials take place
The trials above run at 78 study sites in 8 countries. Tap a country to see its states, cities and hospitals.
United States
45▼
Japan
11▼
Germany
6▼
Italy
4▼
China
3▼
France
3▼
Spain
3▼
United Kingdom
3▼
Rare diseases Stoke Therapeutics works on
Each disease page lists every open trial, not only Stoke Therapeutics's, plus approved treatments.
Dravet Syndrome is a severe form of epilepsy that typically begins in infancy with prolonged seizures triggered by fever.
How common: Approximately 1 in 16,000 to 40,000 people, accounting for 5-10% of early infantile epileptic encephalopathies
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Stoke Therapeutics's own site is stoketherapeutics.com, and our help paying for treatment page lists drugmaker programs and charity funds.
Stoke Therapeutics, based in Bedford, Massachusetts, develops antisense oligonucleotide (ASO) medicines that raise the protein made by a person's working copy of a gene, using its TANGO platform (Targeted Augmentation of Nuclear Gene Output). Its lead drug, zorevunersen (formerly STK-001) for Dravet syndrome, is in the Phase 3 EMPEROR trial, whose main group of 162 children finished enrolling in June 2026. Stoke expects results in the third quarter of 2027 and plans to complete a U.S. application in the second half of 2027. Biogen holds rights outside the United States, Canada and Mexico.
STK-002, for autosomal dominant optic atrophy (ADOA), an inherited cause of vision loss, is in the Phase 1 OSPREY study at sites in Europe, with first data expected in the first half of 2027. Stoke is also searching for a drug candidate for SYNGAP1-related disorders.
Stoke Therapeutics has active programs across 1 rare disease: Dravet Syndrome. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Yes. Stoke Therapeutics is a rare disease specialist headquartered in Bedford, United States, founded in 2014. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Dravet Syndrome.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Stoke Therapeutics, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
None that we track as of November 2026. No Stoke Therapeutics label in the FDA's openFDA database matches a rare disease on Trial Friend, which usually means the company has no approved product yet or its approved drugs are for common conditions.
In development: the trials list on this page shows what Stoke Therapeutics is recruiting for now, and the FDA calendar lists any application once a decision date is public.
Stoke Therapeutics was founded in 2014 and is headquartered in Bedford, United States. The company has been active for 12 years. It trades publicly under the ticker symbol STOK. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.