Exegenesis Bio is developing next-generation engineered AAV Capsids for more efficient delivery of CNS, Ocular, Muscle and Cardiac payloads directly to targeted cells will minimal off target effects or liver interaction. (1) BBB-penetrant capsid increases transduction of neuronal cells >100-fold vs AAV9 in Mouse and NHP models. TfR1 mediated transport increases gDNA distribution up to 30-fold and mRNA expression up to 60-fold vs AAV9 in Mouse model, and up to 50-fold and 250-fold, respectively in Rhesus and Cyano NHP models. Up to 4-fold reduction in liver effects vs AAV9. High manufacturability and packaging efficiency. (2) Muscle-Tropic Capsid significantly higher transduction of skeletal muscle cells than MyoAAV2A and MyoAAV4E. Up to 60-fold increase in mRNA expression across muscle types. Low liver and off target effects; high manufacturability. (3) Ocular Capsid that enables Intravitreal delivery of genetic payloads (currently in a clinical trial for wet AMD).
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