American Epilepsy Society Meeting 2025: Dravet syndrome, gene-targeted therapies and digital innovation

New data shaping diagnosis, treatment strategies and long-term management in epilepsy

The American Epilepsy Society Annual Meeting 2025 brought together leading experts to showcase the latest developments in epilepsy research and clinical care. The meeting highlighted significant progress in Dravet Syndrome, with growing momentum in gene-targeted therapies for epilepsy, real-world treatment data and digital innovation.

Together, these advances reflect a broader shift toward earlier diagnosis, improved disease monitoring and more personalized approaches to epilepsy management.

Rising focus on Dravet syndrome

The American Epilepsy Society Annual Meeting 2025 highlighted a rapidly evolving epilepsy landscape, with notable momentum in Dravet syndrome research, gene-targeted therapies and digital innovation in care.

One of the most striking trends was the growing focus on Dravet Syndrome. According to the Dravet Syndrome Foundation, the number of Dravet-related abstracts has increased substantially over time, with nearly 100 abstracts presented at AES 2025 compared with just 10 in 2009. This reflects both increased research activity and expanding industry engagement, with more than 10 companies actively developing therapies in this space.¹

Progress in gene-targeted therapies

Gene-targeted approaches were a central theme at AES 2025. Updates on zorevunersen demonstrated continued clinical benefit, with up to 4 years of data showing sustained seizure reduction alongside improvements in behaviour, cognition and quality of life.¹

Similarly, early data in the POLARIS trial evaluating ETX101, an AAV9-based gene regulation therapy, were presented from 19 participants, with favourable safety findings and reported neurodevelopmental improvements across initial dose cohorts.^1,2 These findings were also highlighted as the first clinical data for this investigational therapy in Dravet syndrome.

Continued role of established therapies

Despite the focus on innovation, AES 2025 reinforced the importance of established therapies in Dravet syndrome management.¹

Evidence presented included:

• Real-world data on cannabidiol suggested reductions in polypharmacy and healthcare utilisation for Dravet syndrome.³
• Long-term data from the STIRUS study indicate that real-world treatment with stiripentol confirmed sustained effectiveness as an add-on therapy, with many patients maintaining reduced seizure frequency over several years.⁴
• Long-term safety data further support the use of fenfluramine in both pediatric and adult populations with Dravet syndrome and Lennox–Gastaut syndrome, demonstrating sustained seizure reduction with no new safety signals.⁵

Digital innovation and AI in epilepsy care

Developments in digital innovation and AI in epilepsy care, point toward a more integrated and data-driven approach to epilepsy management.

Key themes included:⁶

• The growing role of artificial intelligence in clinical workflows
• Use of mobile applications for daily seizure and symptom tracking
• Advances in neuromodulation approaches, including thalamic stimulation
• Increasing adoption of genetic testing in adult epilepsy

Toward personalized epilepsy care

Overall, AES 2025 underscored a shift in epilepsy care from reactive seizure control to more proactive and personalized approaches.

Emerging gene-targeted therapies, improved diagnostic tools and digital health innovations are converging to support earlier diagnosis, better monitoring and more individualized treatment strategies. Together, these advances signal a transition toward precision medicine in epilepsy.

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References

1. Dravet Syndrome Foundation. AES 2025: Big moments and emerging insights for Dravet syndrome. Available at: https://dravetfoundation.org/aes-2025-big-moments-and-emerging-insights-for-dravet-syndrome/ (Accessed 2026).
2. American Epilepsy Society. POLARIS Phase 1/2 program: interim safety and preliminary efficacy results of ETX101, a one-time gene regulation therapy in young children with Dravet syndrome. Available at: https://aesnet.org/abstractslisting/polaris-phase-1-2-program-interim-safety-and-preliminary-efficacy-results-of-etx101-a-one-time-gene-regulation-therapy-in-young-children-with-dravet-syndrome (Accessed 2026).
3. American Epilepsy Society. Real-world effectiveness of cannabidiol on antiseizure medication cycling, polypharmacy and healthcare resource utilization: a US claims analysis. Available at: https://aesnet.org/abstractslisting/real-world-effectiveness-of-cannabidiol-on-antiseizure-medication-cycling-polypharmacy-and-healthcare-resource-utilization-a-us-claims-analysis (Accessed 2026).
4. American Epilepsy Society. Efficacy and tolerability of stiripentol across non-Dravet developmental and epileptic encephalopathies: a literature review including genetic and syndromic epilepsies. Available at: https://aesnet.org/abstractslisting/efficacy-and-tolerability-of-stiripentol-across-non-dravet-developmental-and-epileptic-encephalopathies-a-literature-review-including-genetic-and-syndromic-epilepsies (Accessed 2026).
5. American Epilepsy Society. Final results from a long-term open-label extension study (up to 4 years): tolerability of fenfluramine and global functioning of pediatric and adult patients with Dravet or Lennox–Gastaut syndromes. Available at: https://aesnet.org/abstractslisting/final-results-from-a-long-term-open-label-extension-study-up-to-4-years-tolerability-of-fenfluramine-and-global-functioning-of-pediatric-and-adult-patients-with-dravet-or-lennox-gastaut-syndromes (Accessed 2026).
6. NeurologyLive. AES conference coverage. Available at: https://www.neurologylive.com/conferences/aes (Accessed 2026).