PGN-EDO51

About PepGen

PepGen is a clinical-stage biotech focused on developing transformative oligonucleotide therapies for severe genetic neuromuscular and neurological diseases. Its core strategy leverages a proprietary Enhanced Delivery Oligonucleotide (EDO) platform designed to solve the critical challenge of delivering therapeutic cargo to deep tissues like muscle and nerve cells. The company's lead candidate, PGN-EDODM1 for myotonic dystrophy type 1 (DM1), has generated compelling early clinical data, including unprecedented splicing correction, and is advancing in Phase 2 studies. With a modular platform and a pipeline extending into Duchenne muscular dystrophy (DMD) and Charcot-Marie-Tooth disease (CMT1A), PepGen aims to establish a new class of disease-modifying genetic medicines.

Therapeutic Areas

Other Duchenne Muscular Dystrophy (DMD) amenable to Exon 51 Skipping Drugs