PMOs

Overview

Phosphorodiamidate morpholino oligomers — uncharged synthetic oligonucleotides used for exon skipping by targeting pre-mRNA in the nucleus; the payload class used in Sarepta's eteplirsen and in Dyne's FORCE platform for DMD. PMOs are synthetic uncharged oligonucleotides — a subset of ASO-like molecules — used to target pre-mRNA in the nucleus for exon skipping therapy; they are one of the interchangeable payloads used in Dyne Therapeutics' FORCE platform for DMD. Naked PMO infusion as used by Sarepta's eteplirsen results in poor tissue distribution; conjugating PMO to the FORCE Fab platform achieved 30-50x higher delivery to muscle tissues in primate studies, enabling greater dystrophin expression. (Segment 2 (05:00-10:00))