Pip6a-PMO-CAG7

Pip6a-PMO-CAG7 is a peptide-conjugated antisense oligonucleotide (P-ASO) designed for the treatment of myotonic dystrophy type 1 (DM1). It consists of an arginine-rich Pip6a cell-penetrating peptide conjugated to a morpholino phosphorodiamidate oligomer (PMO) with a CAG7 antisense sequence. The drug specifically targets mutant CUG-expanded DMPK transcripts in the nucleus to abrogate the sequestration of the MBNL1 splicing factor by toxic RNA foci. By displacing MBNL1 from these foci, Pip6a-PMO-CAG7 restores the functional regulatory activity of MBNL1, thereby correcting alternative splicing defects and muscle dysfunction associated with DM1. Developed by researchers at the University of Oxford and the Institute of Myology, this modality has demonstrated high efficacy and long-lasting corrective effects in DM1 mouse models and patient-derived muscle cells.

Other names

Pip6a-conjugated morpholino phosphorodiamidate oligomerPip-6a-conjugated morpholino phosphorodiamidate oligomerPip 6a-conjugated morpholino phosphorodiamidate oligomerPip6a-PMOPip-6a-PMOPip 6a-PMO