Gene Therapy Trials for Epilepsy Yield Promising Results

Groundbreaking Phase 1/2a clinical trials show first gene regulation treatment for Dravet syndrome is safe and effective.

Published on Mar. 5, 2026

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Researchers at Ann & Robert H. Lurie Children's Hospital of Chicago have reported promising results from Phase 1/2a clinical trials of a gene regulation treatment for Dravet syndrome, a rare and severe form of epilepsy. The treatment, called zorevunersen, was found to be safe and well-tolerated by patients, and led to significant reductions in seizures as well as improvements in other symptoms like language, motor skills, and behavior.

Why it matters

Dravet syndrome is a debilitating condition that typically emerges in infancy, causing seizures, cognitive deficits, communication and behavioral issues, and other developmental problems. Current treatments are often ineffective, so these gene therapy results represent a major breakthrough that could dramatically improve quality of life for Dravet patients and their families.

The details

The gene regulation treatment works by targeting the underlying genetic cause of Dravet syndrome - a mutation in the SCN1A gene that leads to a sodium channel receptor deficiency. Zorevunersen acts on the normal SCN1A gene to increase production of the receptor subunit, overcoming the deficit caused by the mutated gene. In the clinical trials, patients who received two to three doses of 70 mg zorevunersen saw motor seizures reduced by nearly 85% at three months and 73% at six months. Patients who continued in the open-label extension studies maintained significant seizure reduction of 58-90% over 20 months, and also showed improvements in communication and adaptive behavior.

  • The Phase 1/2a clinical trials were conducted in 2025.
  • Patients in the open-label extension studies have been receiving 45 mg of zorevunersen every four months since the initial trials.
  • A Phase 3 double-blind, placebo-controlled trial of zorevunersen is currently underway.

The players

Linda Laux, MD

Head of the Epilepsy Center and Associate Division Head of Neurology at Lurie Children's, as well as Associate Professor of Pediatrics at Northwestern University Feinberg School of Medicine.

Owen

A 12-year-old Lurie Children's patient with Dravet syndrome who participated in the clinical trial and continues in the open-label extension study. His seizures have been significantly reduced and he has seen marked improvement in language and behavior.

Austin

Owen's mother, who has reported that his quality of life has increased substantially and he is now able to enjoy more activities with neurotypical peers.

Ann & Robert H. Lurie Children's Hospital of Chicago

A nonprofit organization committed to providing exceptional care for children, and the site of the clinical trials. It is the only independent, research-driven children's hospital in Illinois and one of less than 35 nationally.

Stoke Therapeutics

The company that provided funding for the research.

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What they’re saying

“Our results are highly promising, especially since currently there are no approved treatments that address the underlying cause of Dravet syndrome. Since this gene regulation product targets the actual root cause of Dravet syndrome, we observed improvements in other developmental and cognitive symptoms, in addition to seizure control. This is unprecedented.”

— Linda Laux, MD, Head of the Epilepsy Center and Associate Division Head of Neurology at Lurie Children's (Mirage News)

“He is able to make friends, which is kind of a new development. His quality of life has increased substantially so that he's able to enjoy more activities with neurotypical peers.”

— Austin, Owen's mother (Mirage News)

What’s next

A Phase 3, double-blind, placebo-controlled trial of zorevunersen for Dravet syndrome is currently underway.

The takeaway

These promising gene therapy results offer new hope for Dravet syndrome patients and their families, potentially leading to a treatment that can address the underlying genetic cause of the disease and provide significant improvements in seizures, development, and quality of life.