DG9-PMO

DG9-PMO is an experimental peptide-conjugated antisense oligonucleotide (ASO) being developed for the treatment of Spinal Muscular Atrophy (SMA). It consists of a Phosphorodiamidate Morpholino Oligomer (PMO) conjugated to DG9, a novel cell-penetrating peptide derived from the human Hph-1 transcription factor. This platform is designed to overcome the poor cellular uptake and limited tissue distribution of conventional PMOs. By utilizing the DG9 peptide, the drug can efficiently cross the blood-brain barrier and penetrate peripheral tissues following systemic administration, potentially eliminating the need for invasive intrathecal injections. In preclinical SMA mouse models, DG9-PMO has demonstrated the ability to restore Survival Motor Neuron (SMN) protein levels, improve motor function, and provide multi-organ phenotypic rescue, including cardiac, hepatic, and respiratory improvements.

Other names

DG9-conjugated PMODG-9-conjugated PMODG 9-conjugated PMO