Creating a new treatment for Dravet syndrome
Official title: Development of an oligonucleotide therapeutic for Dravet syndrome
Funded at Allen Institute · NIH-11122651 · Data updated 2026-11-12
This study is exploring a new treatment for Dravet syndrome, a serious type of epilepsy that starts in babies, by developing a special therapy that helps boost a key protein in the brain to reduce seizures and improve survival, with plans to test it in animals first before moving to people.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Allen Institute NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | May 15, 2025 |
| Project end | April 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $391,903 |
| NIH project number | 1R61NS138832-01A1 |
| Project ID | NIH-11122651 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Dravet syndrome, particularly those who have a genetic mutation affecting the SCN1A gene.
✕Less likely to help
Patients with other forms of epilepsy or those without a confirmed diagnosis of Dravet syndrome may not benefit from this research.
What this research studies
This research focuses on developing a novel therapeutic approach for Dravet syndrome, a severe form of epilepsy that begins in infancy. The project aims to create an antisense oligonucleotide (ASO) that increases the production of a crucial protein, NaV1.1, which is often deficient in patients with this condition. By blocking a specific mechanism that inhibits protein production, the ASO has shown promise in preclinical models by improving survival and reducing seizure activity. The research will involve detailed studies on how this new treatment works and its effectiveness in animal models before considering human trials.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that significantly improves the quality of life and reduces the risks associated with Dravet syndrome.
How similar studies have gone: While there have been various approaches to treating Dravet syndrome, this specific strategy using antisense oligonucleotides is novel and has not been extensively tested in clinical settings.
Where this research is happening
-
Allen Institute
Seattle, United States Active
Researchers
- Principal investigator: Levi, Boaz Pirie — Allen Institute
- Study coordinator: Levi, Boaz Pirie
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.