Programs
Encoded is building an internally developed pipeline of best- and potentially first-in-class therapies focused on central nervous system (CNS) disorders.
Our Quest
We aim to deliver transformative therapies to as many people with CNS disorders as possible. Beyond targeting the CNS, our genomics-powered approach to modulating gene expression in specific cell types offers broad therapeutic potential to treat liver, metabolic and cardiovascular diseases.
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
ETX101’s planned development path is expected to initially provide signals of modulating seizure burden, while tracking long-term changes to neurodevelopmental outcomes. We conducted ENVISION, a natural history study, to delineate longitudinal outcomes and our first-in-human clinical trials will begin in the first half of 2024.
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 CNS disorders beyond Dravet syndrome. Our novel approach also holds promise to treat liver, metabolic and cardiovascular diseases.
Publications & Presentations
CNS 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.