Encoded Therapeutics is focused on developing innovative genetic medicines aimed at addressing severe central nervous system (CNS) disorders. Their proprietary vector engineering platform utilizes adeno-associated virus (AAV) vectors combined with novel regulatory elements to enhance gene specificity, cell selectivity, and potency. This technology allows for targeted therapies that can effectively reach specific cell types, thereby addressing the underlying causes of various CNS conditions. Encoded's lead program, ETX101, is designed to treat Dravet syndrome by selectively upregulating the SCN1A gene, showcasing the potential for long-lasting therapeutic benefits.

In addition to ETX101, Encoded is advancing a diverse pipeline of gene therapy programs, including ETX201, which employs a vectorized microRNA approach to unsilence the paternal UBE3A gene for the treatment of Angelman syndrome. This innovative strategy aims to knock down the UBE3A antisense transcript, thereby restoring UBE3A expression in critical brain regions. The company has demonstrated promising preclinical results, including safety and efficacy in non-human primates, indicating the potential for these therapies to provide significant improvements in the lives of individuals affected by debilitating CNS disorders.

United States

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 potentially 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.

Encoded Therapeutics

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