Multiplex staining in the mouse brain. Encoded's cell-selective gene therapy approach enables us to tap into the diversity of different cell types in the brain.


Encoded is building an internally developed pipeline of best- and potentially first-in-class therapies focused on pediatric central nervous system (CNS) disorders.

Our Quest

We aim to deliver transformative therapies to as many people with pediatric CNS disorders as possible. Beyond targeting the CNS, our genomics-powered approach to modulating gene expression with specific cell types also offers broad therapeutic potential in the future to treat liver, metabolic and cardiovascular diseases.

Our Focus

Our initial program is for Dravet syndrome, which is a severe, developmental and epileptic encephalopathy. This disease is characterized by frequent, treatment-resistant and prolonged seizures and status epilepticus events, significant cognitive delays, sleep abnormalities, motor impairment and profound behavioral difficulties. But we’re not stopping there. We are positioned to develop and manufacture best- and potentially first-in-class, one-time gene therapies for a broad range of indications, providing long-lasting and life-changing benefits to affected people.

ETX101 for Dravet Syndrome

ETX101, our lead program, is specifically designed to address the underlying cause of Dravet syndrome, the most common developmental and epileptic encephalopathy. Modulating the SCN1A gene in a cell-selective manner has the potential to address the full range of seizure, cognitive, behavioral, developmental and motor manifestations of Dravet syndrome.

Clinical Studies

ETX101’s planned development path is expected to initially provide signals of modulating seizure burden, while tracking long-term changes to neurodevelopmental outcomes. We have initiated ENVISION, a natural history study, to delineate longitudinal outcomes in parallel to progressing toward our first-in-human clinical trial, ENDEAVOR.

Research Areas

Our expertise and independently built infrastructure will yield future efficiencies, enabling the sustainable development of innovative one-time therapies for other difficult-to-treat pediatric CNS disorders beyond Dravet syndrome. Our novel approach also holds promise to treat liver, metabolic and cardiovascular diseases.

Publications & Presentations

Pediatric CNS disorders represent a significant proportion of the global burden of disease because they contribute both to premature mortality and years lived with a disability. We’re advancing a portfolio of differentiated therapies with significant opportunities in areas of high unmet need. Check out our publications and presentations to learn more about our work.