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AAV9-GADv1-Naβ1
- Development stage
- Preclinical
- Lead developer
- University of Michigan
- Modality
- Gene Therapies
- Administration
- Intrathecal
01
Overview
AAV9-GADv1-Naβ1 (also known as AAV-Navβ1) is an investigational adeno-associated virus serotype 9 (AAV9) gene therapy designed to deliver a functional copy of the SCN1B gene, which encodes the voltage-gated sodium channel β1 subunit (Navβ1). Developed by researchers at the University of Michigan and the University of Toronto, the therapy utilizes a modified mouse Gad1 promoter (GADv1) to drive expression of the β1 subunit specifically in GABAergic inhibitory interneurons and excitatory neurons. In preclinical models of SCN1B-linked developmental and epileptic encephalopathy (DEE52) and SCN1A-linked Dravet syndrome, neonatal administration of AAV9-GADv1-Naβ1 restored neuronal excitability of parvalbumin-positive (PV+) fast-spiking interneurons, normalized Scn1a mRNA expression, reduced seizure severity, and prolonged survival.
- Other names
- AAV9-GADv1-NaVβ1rAAV9-GADv1-Naβ1
02
Targets
SCN1B (Sodium channel regulatory subunit beta-1)
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