New Podcast: Oligonucleoptide Therapeutics Changing Outcomes for Rare Genetic Neuromuscular Diseases
James McArthur Ph.D., the CEO of PepGen discusses with Karen Jagoda his company"s enhanced delivery cell-penetrating peptide technology to treat rare genetic neuromuscular diseases.
The lead diseases PepGen is focused on are Duchenne muscular dystrophy, treatable with an exon 51 skipping oligonucleotide, as well as myotonic dystrophy type 1 (DM1), both rare genetic diseases with high unmet medical needs.
James says. "For a long period of time, we've known that we can fundamentally change the outcome of many diseases with oligonucleotide therapeutics. But the great challenge has been getting enough of these oligos, as they're often called, into the cells where they need to do essentially the job of changing disease outcomes."