DLX2.0-SCN1A

DLX2.0-SCN1A is an investigational dual-AAV gene replacement therapy being developed by **Epi-Cure**, in collaboration with the **Allen Institute** and **Seattle Children's Research Institute**, for the treatment of **Dravet syndrome**. Dravet syndrome is primarily caused by haploinsufficiency of the *SCN1A* gene, which encodes the **Nav1.1** voltage-gated sodium channel alpha subunit. Because the *SCN1A* gene exceeds the packaging capacity of a single AAV vector, DLX2.0-SCN1A utilizes a **split-intein** approach to deliver the full-length gene via two separate AAV vectors that reassemble within target cells. The therapy specifically targets **GABAergic inhibitory interneurons**—the cells most affected in Dravet syndrome—by employing the **DLX2.0 enhancer**. Preclinical data in mouse models have demonstrated that this approach can restore Nav1.1 function, significantly reduce seizures, and prevent mortality without observed toxicity.

Other names

AAV-SCN1AAAV-SCN-1AAAV-SCN 1AEpi-Cure SCN1A gene therapydual-AAV SCN1A gene therapy