Oligonucleotides have long promised to transform healthcare with their ability to specifically target RNA sequences that cause disease, but the efficacious delivery of oligonucleotide therapeutics has lagged and remains a major challenge. PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform leverages cell-penetrating peptide conjugates to improve the activity and tolerability of oligonucleotide cargo therapeutics. PepGen’s lead candidates in Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) leverage novel EDO peptides to reach key muscle targets, including cardiac tissue. The development and application of bioanalytical strategies for the determination of PPMOs from biological matrices will be discussed.
Learning Objectives:
Know strategies to develop bioanalytical assays for peptide-conjugated oligos. different available platforms for PPMO characterization
Overcome challenges, potential of PPMOs for efficient delivery