Minicircle DNA (MC) is an optimized non-viral platform technology for gene therapy [Chen ZY et al, Mol Ther 2003; 8: 495; Kay MA et al, Nat Biotechnol 2010; 28: 1287]. MC has superior profiles in safety and transgene expression because it comprises almost solely the transgene expression cassette without plasmid backbone DNA and the associated detrimental effects, such as silencing of transgene expression and immunological elimination of cells hosting the DNA vector.
SynoMC has developed a proprietary and efficient skeletal muscle transfection technology – MusFX.MC, making the MC platform much more applicable in a clinical setting. Thus far, MusFX.MC has demonstrated excellent transgene expression properties, including stable expression of luciferase for hundreds of days, and therapeutic levels of bispecific antibodies in in vivo studies. Specifically, MusFX.MC has been used to correct deficiency of a specific gene in gene-knockout mice and cure multiple cancers in xenograft mouse models.