mi405

mi405 is an experimental adeno-associated virus (AAV) gene therapy designed for the treatment of facioscapulohumeral muscular dystrophy (FSHD). It utilizes an AAV vector to deliver a microRNA (miRNA) sequence, specifically mi405, which targets and silences the *DUX4* (Double Homeobox 4) gene through RNA interference (RNAi). In FSHD, the aberrant expression of the DUX4 transcription factor in skeletal muscle leads to progressive muscle weakness and degeneration. By reducing DUX4 mRNA and protein levels, mi405 aims to halt the underlying pathogenic process and preserve muscle function. Preclinical data presented at the MDA Clinical & Scientific Conference 2026 demonstrated that mi405 treatment in TIC-DUX4 mice (an inducible model of FSHD) preserved plantarflexor isometric torque and prevented disease-associated muscle weakness. The program is being developed by Armatus Bio in collaboration with Nationwide Children's Hospital.

Other names

AAV-mi405AAV-mi-405AAV-mi 405