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PepGen, a Cambridge, MA-based biotech, is advancing oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases has focused on delivery. SVP Head of Clinical Development Dr. Michelle Mellion explains PepGen's trial of PGN-EDO51, the company’s lead product candidate for the treatment of DMD patients whose mutations are amenable to an exon 51 skipping approach. She also explains how the delivery of oligonucleotide therapies differ from other techniques in genetic medicine, and much more.
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Cell & Gene: The Podcast