AAV-RNJ1

AAV-RNJ1 is a preclinical gene therapy candidate designed to target intracellular tau pathology for the treatment of Alzheimer's disease and other tauopathies. Developed by researchers at The Florey Institute of Neuroscience and Mental Health, the therapy consists of a single-chain variable fragment (scFv) derived from the N-terminal tau-specific parental antibody, RNJ1, packaged into the brain-penetrant adeno-associated virus vector PHP.eB (AAV-PHP.eB). Unlike conventional antibodies that are restricted to extracellular targets, AAV-RNJ1 is designed to express the RNJ1 intrabody directly within the cell cytoplasm, allowing it to bind to intracellular tau, reduce total and phosphorylated tau inclusions, and restore cellular homeostasis pathways such as RNA splicing, mitochondrial function, and protein degradation.

Other names

RNJ1 intrabodyRNJ-1 intrabodyRNJ 1 intrabodyiRNJ1iRNJ-1iRNJ 1