Encoded Therapeutics Presents Nonclinical Data Showing Genomic Medicine Platform Yields Selective Expression to Optimize Gene Therapy Performance at the American Society for Cell and Gene Therapy 25th Annual Meeting

Multiple adaptable DNA sequence-encoded human genetic elements are modality-agnostic and can be combined to customize expression profiles to optimize therapeutic approaches in and beyond the brain

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Encoded Therapeutics announced nonclinical data being presented today at the American Society for Cell and Gene Therapy 25^th Annual Meeting showing how its proprietary human genomic regulatory element (RE) engineering platform has been used to develop cell-selective expression vectors for targeted gene therapy. The company efficiently discovered a spectrum of sequence-encoded genetic elements, including enhancers, promoters and UTR elements, that drive selective expression profiles in mice. These REs function within the size constraints and episomal architecture of adeno-associated viruses (AAVs) and are compatible with multiple capsids and gene delivery systems.

“By combining human genomic regulatory elements to customize expression profiles and minimize off-target effects, we aim to improve the safety and efficacy of gene therapies for a broad range of monogenic and non-monogenic diseases in the future,” said Encoded CEO Kartik Ramamoorthi, Ph.D. “Our regulatory elements engineering approach increases cell-selective expression, reducing toxicity concerns in tissues like the liver. The data we’re sharing today at ASGCT are exemplary of the Encoded platform’s ability to achieve appropriately targeted transgene expression across many other central nervous system (CNS) cell types, like dorsal root ganglia (DRG) neurons, as well as non-CNS cell types​.”

Using Encoded's genomic medicine platform, researchers applied both expression-based functional screening and computational modeling to simultaneously test thousands of genomic elements in vivo. They uncovered sequence elements that selectively decrease liver expression while maintaining CNS expression in mice. Additionally, the research team constructed predictive models to rapidly and iteratively continue to improve the discovery rate and distribution of activity profiles, resulting in further reduction of liver expression in mice, with unchanged expression in the brain.

Click here for the abstract of Encoded’s ASGCT presentation, “Optimized Human Regulatory Sequences Achieve Targeted Expression in CNS and Decreased Liver Expression in Mice.”

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.