AAV-miRNA J2J

AAV-miRNA J2J is an experimental gene therapy platform designed to treat trinucleotide repeat expansion disorders (TREDs) by targeting the junction-to-junction (J2J) regions flanking pathogenic repeats. Developed through a collaboration involving Uni-Pioneers BioMed (UP-Bio), AskBio, and the University of North Carolina at Chapel Hill, the platform uses adeno-associated virus (AAV) vectors to deliver artificial microRNAs (miRNAs). These miRNAs are engineered to bind to the conserved sequences immediately adjacent to expanded repeats—such as the CAG repeats in Huntington's disease or CTG repeats in Myotonic Dystrophy Type 1—facilitating the degradation of toxic mutant transcripts. By targeting these structurally accessible flanking domains, the platform aims to provide a universal and highly efficient gene-silencing framework that reduces the need for transcript-wide miRNA screening. Preclinical data presented at ASGCT 2026 demonstrated up to 90.8% knockdown of mutant huntingtin (HTT) and significant reduction of mutant DMPK expression in patient-derived cell models.

Other names

J2J miRNA platformJ-2J miRNA platformJ 2J miRNA platformAAV-miRNA J2J platformJunction-to-Junction miRNA platform