Encoded Therapeutics
Encoded Therapeutics focuses on developing precision gene therapies for pediatric central nervous system disorders, with its leading program, ETX101, targeting Dravet syndrome.
Services
Encoded Therapeutics develops transformative precision gene therapies focusing on pediatric central nervous system disorders. They utilize a novel targeting and regulation platform to create gene therapies that offer increased specificity and potency. Their research and development initiatives aim to address the underlying causes of these severe conditions and deliver substantial benefits to patients.
Lead Program ETX101
Encoded Therapeutics' lead program is ETX101, which targets Dravet syndrome, a severe developmental and epileptic encephalopathy. This gene therapy candidate is designed to modulate the SCN1A gene in a cell-selective manner, aiming to treat the underlying cause of the disorder. ETX101 has received both UK CTA approval and US IND clearance, indicating its potential as a significant therapeutic option for Dravet syndrome.
Pipeline
Encoded Therapeutics' pipeline features an array of potential therapies for various pediatric CNS disorders, along with treatments for liver, metabolic, and cardiovascular diseases. The company's approach focuses on developing gene therapies that offer greater specificity and potency, aiming to address unmet medical needs and improve patient outcomes across multiple therapeutic areas.
Research Initiative ENVISION
The ENVISION study is a natural history study conducted by Encoded Therapeutics to understand the progression of Dravet syndrome in children. This research is crucial in providing insights into the disease's development and assisting in the development of effective treatments like ETX101. ENVISION plays a fundamental role in gathering data necessary for the continued advancement of gene therapy approaches for this severe condition.
Headquarters and Research Facilities
Encoded Therapeutics is headquartered in South San Francisco, CA. Additionally, the company operates a research facility in Research Triangle Park, NC. These locations support the company's focus on innovation and research in developing gene therapies for various severe medical conditions.