PGN-EDODM1

PGN-EDODM1 is an investigational peptide-conjugated antisense oligonucleotide (ASO) therapy developed using PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform. It is designed to treat myotonic dystrophy type 1 (DM1), a rare genetic disorder caused by toxic CUG repeat expansions in the DMPK gene. The drug works by selectively binding and blocking the pathogenic DMPK mRNA transcripts, thereby correcting RNA mis-splicing events central to DM1 pathology while preserving healthy DMPK function. Preclinical studies demonstrated robust reversal of myotonia and correction of mis-splicing, and early clinical data show dose-dependent increases in muscle tissue concentrations and splicing correction after intravenous administration. PGN-EDODM1 has received FDA Fast Track designation for DM1[2][3][5][8].