Washington Research Foundation (WRF) has awarded $1 million to accelerate development of a new gene therapy for Dravet syndrome, a rare and severe form of epilepsy. The funding will support researchers at the Allen Institute for Brain Science in their efforts to bring this potentially life-saving therapy to the clinic.

Dravet syndrome, which can strike before an infant’s first birthday, is characterized by frequent seizures, developmental delays, and a high risk of premature death.

“We are excited to have WRF’s support for this critical program,” said Boaz Levi, Ph.D., an associate investigator leading the research in the Allen Institute. “This will support key experiments to hone the design and approach for a clinical therapeutic. The impact of this support is to accelerate the development and clinical application of this potentially life-changing therapy.”

The therapy targets the SCN1A gene, which is typically mutated in patients with Dravet syndrome. Using a harmless virus as a delivery vehicle, researchers introduced a healthy version of this gene directly to the affected brain cells. This method successfully produced functional proteins and significantly reduced seizures and mortality in mouse models of the disease. The proof-of-concept and technology development work was done in collaboration with Franck Kalume, Ph.D., and colleagues at Seattle Children’s Research Institute.

The Phase 3 Technology Commercialization grant will support critical experiments to advance the gene therapy from proof-of-concept to a clinical product. These experiments will help the researchers choose the best therapeutic candidate, show the therapy can spread effectively in a large primate brain to provide benefits, and test other properties of the therapeutic.

The grant will hopefully accelerate development of this innovative gene therapy, bringing hope to patients and families affected by Dravet syndrome, said Meher Antia, Ph.D., director of grant programs at WRF.

“The gene therapy approach devised by the Allen Institute team and collaborators has the potential to be truly transformative,” Antia said. “This project is exactly the kind of high-quality, innovative research that our Technology Commercialization grant program is intended help de-risk on its journey to achieving genuine patient benefit.”