Oligonucleotide therapy to raise NaV1.1 protein in Dravet syndrome
Official title: Development of an oligonucleotide therapeutic for Dravet syndrome
Funded at Allen Institute · NIH-11330318 · Data updated 2026-11-12
A new antisense therapy aims to boost the brain’s NaV1.1 protein to help people with Dravet syndrome.
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 | 2026 |
| Award (this year) | $393,239 |
| NIH project number | 5R61NS138832-02 |
| Project ID | NIH-11330318 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Dravet syndrome linked to SCN1A deficiency or impaired NaV1.1 expression, potentially benefiting from later-stage translational trials.
✕Less likely to help
Patients without Dravet syndrome or without SCN1A-related pathology may not benefit from this specific ASO strategy.
What this research studies
This project focuses on a novel antisense oligonucleotide (ASO) designed to increase NaV1.1 protein by blocking a translation-starting element, which could help address the root cause in many Dravet patients. Researchers will study how the ASO behaves in the body and how it changes NaV1.1 levels in a mouse model of Dravet syndrome. The plan spans two phases, with the first phase (R61) aimed at identifying and characterizing lead compounds and their pharmacokinetics and pharmacodynamics. If successful, results will inform a subsequent phase to further optimize and test the therapeutic approach before potential clinical development.
Why it matters
Potential benefit: If successful, this approach could provide a disease-modifying option for Dravet syndrome by increasing the brain’s protective NaV1.1 protein.
How similar studies have gone: Similar antisense approaches have shown promise in preclinical epilepsy models, though this exact mechanism and target are novel and at an early stage.
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.