Webinar: Gene therapies for Dravet Syndrome & UK clinical trials
Led by Professor Helen Cross and Professor Andreas Brunklaus, this webinar provides an overview of current and development therapies for Dravet Syndrome.
Anti-seizure treatments
Traditionally, treatment for Dravet Syndrome focuses on managing seizures.
DEE‑p SEA Study (NCT06660394)
The DEE‑p SEA study is evaluating bexicaserin as an oral treatment for motor seizures in individuals with developmental and epileptic encephalopathies (DEEs).
Investigational compound: Bexicaserin
Phase: 3
Age: 2–65 years
Locations: Glasgow (currently recruiting); London, Newcastle, Southampton (opening soon)
Sponsor: Lundbeck
Study website: https://deepdsstudy.com/
ARGUS Study (NCT04462770)
The ARGUS study aims to evaluate clemizole as an oral add-on therapy for seizures in people with SCN1A-confirmed Dravet syndrome.
Investigational compound: Clemizole HCl
Phase: 3
Age: 2 years and older
Locations: Cardiff, Sheffield, London (Liverpool opening soon)
Sponsor: Harmony Biosciences
Study website: https://global.argustrial.com/
Disease-modifying Treatments
As the understanding of Dravet syndrome and SCN1A mutations has progressed, new therapies are being developed to target the underlying mechanisms. There are currently two compounds in clinical trials investigating disease-modifying approaches in Dravet syndrome.
EXPEDITION Study (NCT06283212)
The EXPEDITION study is evaluating ETX‑101, a one‑time AAV9 based gene therapy, designed for children with SCN1A‑confirmed Dravet syndrome.
Investigational compound: ETX‑101
Phase: 1/2
Age: 6-48 months
Locations: Glasgow, London, Sheffield
Sponsor: Encoded Therapeutics
Study website: https://encoded.com/programs/clinical-studies/
Upcoming EMPEROR Study (NCT06872125)
The EMPEROR study will assess zorevunersen, a disease‑modifying antisense therapy, in people with SCN1A-confirmed Dravet syndrome. This double‑blind, sham‑controlled trial will assess efficacy for reducing major motor seizures and improving cognition/behaviour over 52 weeks, with an open-label extension for eligible participants.
Investigational compound: Zorevunersen
Phase: 3
Sponsor: Stoke Therapeutics & Biogen
Study website: https://www.emperorstudy.com/en-GB
Advancing precision medicine: genome engineering for on-demand gene therapy in Dravet Syndrome
Lead researcher: Dr Jenna Carpenter