Science
The technology to
change lives
PepGen’s technology, powered by our Enhanced Delivery Oligonucleotide (EDO) platform, targets the root cause of severe neuromuscular and neurological disorders by optimizing the efficient delivery of therapeutic oligonucleotide cargoes into affected tissues and cells.
While conjugated oligonucleotide therapeutics have long promised in research studies to transform patient outcomes with their ability to specifically target RNA sequences that cause disease, the safe and efficacious delivery of these therapeutics has not met expectations. Our EDO platform is designed to solve the key challenges of previous generations of therapies: enabling delivery and uptake by the heart and skeletal muscle, the key affected tissues in neuromuscular disorders like Duchenne muscular dystrophy (DMD), and myotonic dystrophy type 1 (DM1).
Enhanced Delivery
Oligonucleotide
Novel cell-penetrating peptide
Oligonucleotide
PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform
Our EDO platform, founded on over a decade of research and development, is designed to overcome the key challenges facing previous attempts to target neuromuscular disorders such as DMD and DM1.
PepGen’s EDO peptides have been engineered for efficient cell delivery, robust stability and minimal immunogenicity. Our data demonstrate we can achieve high biodistribution and uptake by key muscle targets, including skeletal and cardiac tissue, while also improving tolerability. These peptides subsequently facilitate the delivery of conjugated oligonucleotide therapeutics with improved tolerability, uptake, and activity.
Safe transport means truly transformative medicines
We have demonstrated the ability to safely transport oligonucleotides into a broad range of target tissues, including smooth, skeletal and cardiac muscle, and nerve cells – indicating the potential for our approach to result in the development of truly transformative medicines for individuals in need.
Our lead product candidates in DMD and DM1 utilize our optimized, proprietary, novel EDO peptides, and we continue to build and develop this platform technology and expand into new therapeutic areas.
Enhanced Delivery
Oligonucleotide
Novel cell-penetrating peptide
Oligonucleotide
Publications
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Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment
Gait et. al., Nucleic Acid Therapeutics (2018), 29(1), 1 -
How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse
Godfrey et. al., Human Molecular Genetics (2015), 24(15), 4225 -
Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment
Betts et. al., Molecular Therapy–Nucleic Acids (2012, 1(8), e38 -
Targeting RNA to treat neuromuscular disease
Muntoni and Woods, Nature Reviews Drug Discovery (2011), 10, 612