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Find Clinical Drug Pipeline Developments & Deals by Encoded Therapeutics
ETX101 is an AAV9-mediated candidate gene regulation therapy designed to selectively upregulate expression of the SCN1A gene in GABAergic inhibitory interneurons. It is being evaluated for the treatment of SCN1A-positive Dravet syndrome in infants & young children.
ETX101 is an AAV9-mediated candidate gene regulation therapy designed to selectively upregulate expression of the SCN1A gene in GABAergic inhibitory interneurons. It is being evaluated in phase 1/2 clinical trials for SCN1A-positive Dravet syndrome in infants & young children.
Multiple adaptable DNA sequence-encoded human genetic elements are modality-agnostic and can be combined to customize expression profiles to optimize therapeutic approaches in and beyond the brain.
ETX101, Encoded's lead asset, was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by FDA for the treatment of SCN1A+ Dravet Syndrome.
ETX101, a gene therapy developed by Encoded Therapeutics to restore the levels of NaV1.1-alpha specifically in GABAergic neurons, is currently progressing toward clinical development for SCN1A-positive Dravet patients.