GABA Biomarkers in Dravet Syndrome

计划入组 36 人 · 首次发布于 2022 年 12 月 14 日

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome

计划入组 5 人 · 首次发布于 2024 年 2 月 28 日

A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

计划入组 47 人 · 首次发布于 2022 年 6 月 15 日

SCN1A gene therapy (AAV9, Dravet syndrome)

SCN1A antisense oligonucleotide therapy (Dravet syndrome)

Dravet Syndrome (Camp4 Therapeutics)

ALPORT SYNDROME FOUNDATION

United States

International Rett Syndrome Foundation

United States

RETT SYNDROME RESEARCH TRUST

United States

Encoded Therapeutics Receives FDA RMAT Designation for ETX101 Gene Therapy in Dravet Syndrome

Encoded Therapeutics' ETX101 Gene Therapy Shows Durable Seizure Reduction and Neurodevelopmental Gains in Dravet Syndrome

Encoded Therapeutics Raises $275M Series F to Push ETX101 Gene Therapy Through Pivotal Dravet Syndrome Development