SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Encoded Therapeutics today announced the upcoming presentation of initial data from the ENVISION prospective natural history study of children with SCN1A+ Dravet syndrome, a rare developmental and epileptic encephalopathy, at the American Epilepsy Society 2021 Annual Meeting, taking place in Chicago from December 3-7, 2021. Additionally, Encoded will present on its ongoing DRAVET ENGAGE initiative, which aims to capture the Dravet syndrome patient community’s experiences and perspectives, and a summary of molecular diagnostic and phenotypic findings in people with SCN1A+ Dravet syndrome identified through genetic testing.
Title: ENVISION: An International, Prospective Natural History Study in Young Children With SCN1A+ Dravet Syndrome
Abstract number: 2.222
Date: Sunday, December 5th
Platform presentation details: #B.07 | 10-10:15 a.m. CT | W475, Level 4
Poster session details: 12-2 p.m. CT | Hall F1, Level 3
Presenting author: Andreas Brunklaus, M.D., Consultant Pediatric Neurologist at the Royal Hospital for Children, Glasgow, United Kingdom
Title: A Patient-Oriented Approach to Integrating Families’ Experiences in Clinical Trials Design and Clinical Care for Dravet Syndrome
Abstract number: 3.073
Date/time: Monday, December 6th | 12-1:45 p.m. CT
Location: Hall F1, Level 3
Presenting author: Emma James, Ph.D., M.F.P.M. (Hon), VP, Medical & Patient Affairs at Encoded Therapeutics
Title: Molecular Diagnostic and Phenotypic Findings in Individuals With SCN1A-Related Disease Identified Through a Sponsored, No-Charge Epilepsy Genetic Testing Program
Abstract number: 3.351
Date/time: Monday, December 6th | 12-1:45 p.m. CT
Location: Hall F1, Level 3
Co-presenting author: Jennifer Gorzelany, Head of Strategy at Encoded Therapeutics
About ETX101
Encoded is developing ETX101 as a potential one-time, disease-modifying gene regulation therapy targeting the underlying cause of SCN1A+ Dravet syndrome in hopes of addressing the full range of disease manifestations. In ETX101, a cell-selective regulatory element and a transgene coding for an engineered transcription factor are delivered in a clinically validated adeno-associated virus (AAV) capsid to upregulate, or increase, the expression of the endogenous SCN1A gene in the brain. This approach is expected to increase production of NaV1.1 protein and density of sodium channels in target neurons, leading to restored function. ETX101 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. Encoded anticipates treating the first participant in the ENDEAVOR interventional trial in 2022.
About the ENVISION Natural History Study
ENVISION is an ongoing observational study of infants and children with SCN1A+ Dravet syndrome. This prospective natural history study is designed to further define the seizure, neurodevelopmental, motor and behavioral manifestations of SCN1A+ Dravet syndrome in children aged 6 to 60 months with SCN1A mutations. The study will examine these characteristics over two years using standardized assessments and will also explore the impact of the disease on parents/caregivers and healthcare resource utilization. These findings will help identify appropriate scales and endpoints to assess ETX101 safety and efficacy in clinical trials. For more information about ENVISION (NCT04537832), visit clinicaltrials.gov and search for “Encoded Therapeutics”.
About Dravet Syndrome
Dravet syndrome is a severe, lifelong disorder of the central nervous system that occurs in approximately 1 in 16,000 births worldwide, with the majority of cases resulting from loss-of-function mutations in the SCN1A gene. This rare developmental and epileptic encephalopathy equally affects people of both sexes and all races, manifesting in a wide array of symptoms. Frequent, prolonged and difficult-to-treat seizures typically begin in the first year of life of a previously healthy baby. Additional symptoms, such as cognitive delays, sleep abnormalities, motor impairment and behavioral difficulties, compound the effects of the disease when they appear, often in the second or third year of a child’s life. More information about Dravet syndrome can be found at www.dravetfoundation.org.
About Encoded Therapeutics
Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation platform. The Encoded approach offers unprecedented gene specificity and cell selectivity to unlock novel opportunities by targeting a range of disease mechanisms. Encoded’s technology is compatible with any delivery system to control where and when therapeutic transgenes are expressed, thereby shaping the functionality of target cells and holding broader therapeutic potential beyond CNS disorders. For more information, please visit www.encoded.com, and follow us on LinkedIn, Twitter @EncodedTx and YouTube.
