Programs
Encoded is building an internally developed pipeline of best- and potentially first-in-class therapies focused on neurological disorders.
Our Quest
We aim to deliver transformative therapies to as many people with neurological disorders as possible. Beyond targeting this disease area, our genomics-powered approach to modulating gene expression in specific cell types offers broad therapeutic potential to treat liver, metabolic and cardiovascular conditions.
Our Focus
Our initial program is for SCN1A+ 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 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
Our clinical program, POLARIS, comprises a suite of clinical studies for ETX101 in infants, children and adolescents. These studies, which include ENDEAVOR, EXPEDITION, and WAYFINDER, are being conducted at sites across the US, the United Kingdom, and Australia to assess the safety, tolerability, and efficacy of ETX101. The program was designed following ENVISION, our longitudinal natural history study, which provided the robust data foundation necessary to delineate disease outcomes and inform our current interventional trials.
Research Areas
Our expertise and independently built infrastructure will yield future efficiencies, enabling the sustainable development of innovative one-time therapies for difficult-to-treat neurological disorders beyond Dravet syndrome.
Publications & Presentations
Neurological disorders represent a significant proportion of the global burden of disease and contribute to premature mortality and lifelong disability in children and adults. 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.