Encoded Therapeutics Addresses Common Source of Gene Therapy Toxicity While Achieving Precise Brain Expression in Non-Human Primates, Presented at the American Society of Gene and Cell Therapy 26th Annual Meeting
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Friday, May 19, 2023
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, Central nervous system, Gene, NGS, Toxicity, Optimization, CNS, Confluence (abstract rewriting), Cell, Annual general meeting, DRG, American Society of Gene and Cell Therapy, NHP, Therapy, Society, Vaccine, Mouse
Encoded Therapeutics announced today the presentation of non-human primate (NHP) validation of the company’s technology to drive desired gene and protein expression patterns at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting.
Key Points:
- Encoded Therapeutics announced today the presentation of non-human primate (NHP) validation of the company’s technology to drive desired gene and protein expression patterns at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting.
- An important challenge of developing gene therapy in many central nervous system (CNS) disorders is the toxicity concern associated with high transgene expression in the dorsal root ganglion (DRG).
- This resulted in a greater than 10-fold selective reduction in DRG transcriptional activity without impacting brain expression.
- The research team analyzed top candidates in NHPs, showing successful translation to higher species while still achieving precise expression in the CNS.