
News & Events
Encoded Therapeutics to Present Nonclinical Data Showing Targeted Brain Expression While De-Targeting the Liver With Genomic Medicine Platform at the American Society of Gene and Cell Therapy 25th Annual Meeting
Encoded human genomic regulatory element prediction model employed to discover sequences among thousands of variants that modulate gene expression selectively in targeted cell types
South San Francisco, Calif. – May 2, 2022 – Encoded Therapeutics today announced the upcoming oral presentation of nonclinical data demonstrating the ability of regulatory elements to produce exquisite control of gene expression, at the American Society of Gene and Cell Therapy 25th Annual Meeting, taking place in Washington, D.C., from May 16-19, 2022. Discovered through Encoded’s expression-based functional screening and computational modeling, these sequence elements produced expression in target brain tissue and selectively and substantially decreased liver expression. Achieving highly targeted transgene expression profiles has the potential to improve the safety and efficacy of gene therapies for a broad range of monogenic and non-monogenic diseases.
Title: Optimized Human Regulatory Sequences Achieve Targeted Expression in CNS
and Decreased Liver Expression in Mice
Abstract: #864
Session: Vector Manufacturing and Engineering 3: Improving Vector Design and System Performance
Date: Wednesday, May 18th
Time: 4:30-4:45 p.m. ET
Room: #201
Presenting author: Annie Tanenhaus, Ph.D., Director of Technology, Encoded Therapeutics
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 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. For more information, please visit
Contact
Lynnea Olivarez
lolivarez@encoded.com
956-330-1917
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