Exploring One-Time Gene Regulation Therapy EXT101 in Dravet Syndrome: The Phase 1/2 POLARIS Program

Welcome to NeurologyLive^®'s Clinical Trial in Focus. Every month, an ongoing clinical trial in the landscape of neurology is featured, highlighting the design of the study, the targeted patient population, the enrollment criteria, the primary and secondary end points, and its potential implications for clinical care. This month’s spotlight is the phase 1/2 POLARIS program of EXT101 (Encoded Therapeutics), an adeno-associated virus (AAV)-mediated candidate for gene regulation therapy, for the treatment of SCN1A+ Dravet syndrome (DS).