Stoke Therapeutics
Stoke Therapeutics works on 1 rare disease tracked on Trial Friend, including Dravet Syndrome, with 2 recruiting clinical trials.
Stoke Therapeutics develops antisense oligonucleotide medicines targeting severe genetic diseases through its proprietary TANGO platform that increases protein output from healthy genes. The company's lead program STK-001 for Dravet syndrome and STK-002 for autosomal dominant optic atrophy represent treatments for rare neurodevelopmental and ophthalmic disorders.
Focus areas at Stoke Therapeutics
As a rare disease specialist, Stoke Therapeutics has active clinical trial programs and drug development efforts across 1 rare diseases, including Dravet Syndrome. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Stoke Therapeutics, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Stoke Therapeutics is headquartered in Bedford, United States, founded in 2014, publicly traded under the ticker symbol STOK. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Stoke Therapeutics Drug Pipeline
Stoke Therapeutics has 2 active clinical trials across 2 development stages, with 2 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline includes all of Stoke Therapeutics's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Stoke Therapeutics Clinical Trials (2)
Active and recruiting clinical trials sponsored by Stoke Therapeutics, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
FDA-Approved Drugs
Stoke Therapeutics Trial Locations
Stoke Therapeutics clinical trials are running at 78 sites in 8 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (1)
Diseases targeted by Stoke Therapeutics's clinical trial and drug development programs
Patient Resources
Organizations and resources related to Stoke Therapeutics's rare disease focus areas
Frequently Asked Questions About Stoke Therapeutics
Common questions about Stoke Therapeutics's rare disease programs, clinical trials, and treatments.