Changing the course
PepGen is advancing the next generation of oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases.
Our Enhanced Delivery Oligonucleotide (EDO) platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. These have long promised to transform healthcare with their ability to specifically target RNA sequences that cause disease, and yet the efficacious delivery of oligonucleotide therapeutics has lagged and remains a major challenge. Our proprietary EDO platform has shown the potential to treat such genetic disorders by optimizing the safe and efficient delivery of therapeutic oligonucleotide cargos into affected tissues and cells, thereby addressing the key challenge that has impeded the clinical translation and validation of this class of molecules. We have leveraged these characteristics to develop a robust pipeline of potentially world-leading therapeutics for the treatment of serious genetic diseases.
PepGen is headquartered in Boston, Massachusetts.
Our EDO platform is the result of a decade of work by pioneering researchers and biotech leaders. The EDO peptides are engineered to optimize tissue penetration, cellular uptake and nuclear delivery: in preclinical studies we have observed their ability to transport oligonucleotides into a broad range of target tissues, including smooth, skeletal, and cardiac muscle and the central nervous system, or CNS, and in the clinic we have observed high levels of oligonucleotide delivery to muscle tissue. Furthermore, the high levels of pharmacological activity observed in preclinical and clinical studies support our belief that our EDO platform technology has the potential to deliver therapeutic agents to the cell nucleus.
We are advancing two lead programs: PGN-EDO51, a clinical-stage therapeutic candidate for people living with DMD who are amenable to an exon 51 skipping approach, and PGN-EDODM1, a preclinical-stage therapeutic candidate for people living with myotonic dystrophy type 1 (DM1). Both DMD and DM1 are rare genetic diseases with a high unmet medical need. We are also developing three further therapeutic candidates for other DMD patient populations. While our current pipeline focuses on neuromuscular diseases, we believe the broad applicability of our delivery platform should enable us to create breakthrough therapeutics targeting a wide variety of serious diseases.