PepGen Inc.

PepGen Inc. is a clinical-stage biotechnology company focused on developing advanced oligonucleotide therapeutics for severe neuromuscular and neurologic diseases. The company's proprietary Enhanced Delivery Oligonucleotide (EDO) platform uses cell-penetrating peptides (CPPs) to improve how conjugated oligonucleotide therapeutics are absorbed and become active. These EDO peptides are designed to optimize tissue penetration, cellular uptake, and delivery to the cell nucleus. Preclinical studies have shown they can transport oligonucleotides into various target tissues, including smooth, skeletal, and cardiac muscle. Clinical trials have demonstrated strong delivery and activity of oligonucleotides in skeletal muscle. PepGen is currently addressing myotonic dystrophy type 1 (DM1), a multi-systemic neuromuscular disease with no approved therapies. The company is developing PGN-EDODM1 for DM1. PGN-EDODM1's therapeutic oligonucleotide component is engineered to bind to the pathogenic cytosine-uracil-guanine (CUG) repeat expansion in the myotonic dystrophy protein kinase (DMPK) messenger RNA (mRNA) of DM1 patients. By binding to these expanded repeats, PGN-EDODM1 reduces their ability to sequester RNA binding proteins like MBNL1, a key RNA splicing factor. This liberation of MBNL1 corrects downstream mis-splicing events that cause DM1 pathology. The company anticipates expanding the EDO technology to other severe diseases with significant unmet needs.