Select Publications and Presentations
Please contact medinfo@encoded.com for a copy of any of the medical or scientific conference presentations listed below.
Dravet
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May 2024Seventeenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT 2024)ETX101: A potential one-time, disease-modifying gene regulation therapy targeting the underlying cause of SCN1A+ Dravet syndrome
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July 2023International League Against Epilepsy (ILAE) 15th European Epilepsy CongressThe ENVISION Study, an International, Prospective Natural History Study in Young Children with SCN1A+ Dravet Syndrome, 18 Month Follow-up
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May 2023American Society of Gene and Cell Therapy (ASGCT) 26th Annual MeetingFine and Gross Motor Functioning in Young Children with SCN1A+ Dravet Syndrome Participating in the ENVISION Study, an International, Prospective Natural History
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May 2023American Society of Gene and Cell Therapy (ASGCT) 26th Annual MeetingCognitive and Executive Functioning Difficulties in Young Children with SCN1A+ Dravet Syndrome Participating in the ENVISION Study, an International, Prospective Natural History
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May 2023American Society of Gene and Cell Therapy (ASGCT) 26th Annual MeetingLongitudinal Assessment of Quality of Life in Caregivers and Young Children with SCN1A+ Dravet Syndrome Participating in the ENVISION Study, an International, Prospective Natural History
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May 2023American Society of Gene and Cell Therapy (ASGCT) 26th Annual MeetingSeizure Burden and Communication Issues in Young Children with SCN1A+ Dravet Syndrome Participating in the ENVISION Study, an International, Prospective Natural History
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May 2023American Society of Gene and Cell Therapy (ASGCT) 26th Annual MeetingDecoding Dravet Syndrome: The Role of Advanced EEG Analytics in Phenotypin
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December 2022American Epilepsy Society (AES) 2022 Annual MeetingBehavior, social-emotional, and sleep difficulties in young children with SCN1A+ Dravet Syndrome Participating in the ENVISION study, an international, prospective natural history study
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December 2022American Epilepsy Society (AES) 2022 Annual MeetingThe ENVISION Study, an international, prospective natural history study in young children with SCN1A+ Dravet syndrome, 18-month follow-up
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July 2022International League Against Epilepsy (ILAE) – 14th European Epilepsy CongressThe ENVISION Study, an international, prospective natural history study in young children with SCN1A+ Dravet syndrome
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April 202214th European Paediatric Neurology Society CongressThe ENVISION Study, an International, Prospective Natural History Study in Young Children with SCN1A+ Dravet Syndrome
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April 2022Epilepsy and Behavior
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December 2021American Epilepsy Society (AES) 2021 Annual MeetingENVISION: An International, Prospective Natural History Study in Young Children with SCN1A+ Dravet Syndrome
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December 2021American Epilepsy Society (AES) 2021 Annual MeetingMolecular Diagnostic and Phenotypic Findings in Individuals With SCN1A-Related Disease Identified Through a Sponsored, No-Charge Epilepsy Genetic Testing Program
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December 2021American Epilepsy Society (AES) 2021 Annual MeetingA Patient-Oriented Approach to Integrating Families’ Experiences in Clinical Trials Design and Clinical Care for Dravet Syndrome
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August 2021International League Against Epilepsy (ILAE) – 34th International Epilepsy CongressDRAVET ENGAGE. Parent Caregivers of Children with Dravet Syndrome: Perspectives, Needs, and Opportunities for Clinical Research
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August 2021International League Against Epilepsy (ILAE) – 34th International Epilepsy CongressThe Clinical, Economic, and Humanistic Burden of Dravet Syndrome — A Systematic Literature Review
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July 2021Epilepsy and Behavior
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December 2020American Epilepsy Society (AES) 2020 Annual MeetingETX101, a GABAergic Interneuron Selective AAV-mediated Gene Therapy for Treatment of SCN1A+ Dravet Syndrome: Biodistribution and Safety in Non-human Primates
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December 2020American Epilepsy Society (AES) 2020 Annual MeetingReducing the time to diagnosis and increasing the detection of individuals with SCN1A-related disease through a sponsored epilepsy genetic testing program
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December 2020Fundación Síndrome de Dravet Webinar
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July 2020Dravet Syndrome Foundation Webinar
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December 2019American Epilepsy Society (AES) 2019 Annual MeetingFrom Gene Replacement to Gene Regulation: Developing a Disease-Modifying AAV Gene Therapy Vector for SCN1A-Positive (SCN1A+) Dravet Syndrome
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December 2019American Epilepsy Society (AES) 2019 Annual MeetingProposed Design for a Natural History Study of SCN1A-Positive (SCN1A+) Dravet Syndrome
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May 2019American Society of Gene and Cell Therapy (ASGCT) 22nd Annual MeetingA GABA-Selective AAV Vector-Based Approach to Up-Regulate Endogenous Scn1a Expression Reverses Key Phenotypes in a Mouse Model of Dravet Syndrome
Pipeline
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May 2024American Society of Gene & Cell Therapy (ASGCT) 27th Annual MeetingIdentification of Potent and Selective AAV-miRNA Candidates to Knockdown Non-Monogenic Neurological Targets SCN9A (Pain) and MAPT (Tauopathies)
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May 2024American Society of Gene & Cell Therapy (ASGCT) 27th Annual MeetingAdvancing Gene Therapy for STXBP1-related Disorders Through Targeted Vector Engineering: Efficacy and Safety Results in Mice and Non-Human Primates
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May 2024American Society of Gene & Cell Therapy (ASGCT) 27th Annual MeetingGABA Selective AAV-mediated Gene Therapy Provides Durable Seizure Protection in Multiple Refractory Epilepsy Models
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May 2024American Society of Gene & Cell Therapy (ASGCT) 27th Annual MeetingA Vectorized miRNA-based Approach to Unsilence UBE3A in Angelman Syndrome
Research Platform
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October 2023European Society of Gene & Cell Therapy (ESGCT) 30th Annual CongressOptimized Human Genomic Regulatory Elements Achieve DRG-selective De-targeting in Non-human Primates and Reduce Off-Target Toxicity in AAV Gene Therapies
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May 2023American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting
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October 2022European Society of Gene and Cell Therapy (ESGCT) 29th CongressOptimized human regulatory sequences achieve targeted expression in brain and decreased off-target liver and dorsal root ganglion expression
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May 2022American Society of Gene and Cell Therapy (ASGCT) 25th Annual MeetingOptimized Human Regulatory Sequences Achieve Targeted Expression in CNS and Decreased Liver Expression in Mice
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May 2019American Society of Gene and Cell Therapy (ASGCT) 22nd Annual MeetingEfficient Delivery of Clinically-Validated Adeno-Associated Viral (AAV) Vectors to the Central Nervous System: A Systematic Evaluation of Multiple Routes of Administration and Viral Serotypes in Cynomolgus Macaques