BIO-Europe Spring Presenting Companies 2023
PepGen Inc. is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen’s Enhanced Delivery Oligonucleotide, or EDO, platform is founded on over a decade of research & development and leverages cell-penetrating peptides to improve uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, PepGen is generating a pipeline of treatments that target the root cause of serious diseases. PepGen’s EDOs are engineered for optimal tissue and cell penetration and have demonstrated ability to transport oligonucleotides into a broad range of target tissues, including smooth, skeletal and cardiac muscle, and the CNS. PepGen is poised to become a leader in developing disease-modifying therapies for neuromuscular diseases, with plans to initiate trials in Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) patients in 2023. DMD is a muscle-wasting disease that arises due to mutations in the gene encoding dystrophin, a protein necessary for normal muscle function. PGN-EDO51 is PepGen’s lead candidate for the treatment of DMD patients who are amenable to an exon 51 skipping approach and is designed to treat the underlying cause of DMD by “skipping” exon 51 of the dystrophin transcript, resulting in production of a truncated yet functional dystrophin protein. DM1 arises due to an abnormal repeat expansion in the DMPK gene. PepGen’s lead candidate in DM1, PGN-EDODM1, is designed to bind to repeats in the DMPK transcript and destabilize the hairpin loop structures that form, liberating critical transcription factor proteins and correcting downstream mis-splicing events. PepGen’s EDO candidates could extend the boundaries of what's possible in drug development & delivery for neuromuscular diseases, resulting in transformative impacts for patients. Initial results are included below.