PepGen Inc.

PepGen Inc. (Nasdaq: PEPG) is a clinical-stage biotechnology company focused on developing next-generation oligonucleotide therapeutics for severe neuromuscular and neurologic diseases. The company's proprietary Enhanced Delivery Oligonucleotide (EDO) platform leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics, enabling robust delivery to target tissues including skeletal, smooth, and cardiac muscle. The EDO platform represents over a decade of research and development and is engineered to optimize tissue penetration, cellular uptake, and nuclear delivery of therapeutic agents.

PepGen's lead product candidate is PGN-EDODM1, an EDO peptide conjugated to a phosphorodiamidate morpholino oligonucleotide (PMO) designed to treat myotonic dystrophy type 1 (DM1), a multi-systemic neuromuscular disease with no approved therapies and approximately 40,000 patients in the United States. PGN-EDODM1 binds to pathogenic cytosine-uracil-guanine repeat expansions in the DMPK messenger RNA, reducing sequestration of critical RNA binding proteins and correcting downstream mis-splicing events that drive disease pathology. The company has completed a Phase 1 clinical trial (FREEDOM) demonstrating robust, dose-dependent splicing correction and a favorable safety profile. A Phase 2 multiple ascending dose study (FREEDOM2) is currently enrolling patients across multiple geographies including Canada, the United Kingdom, South Korea, Australia, and New Zealand, with data readouts expected in 2026. An open-label extension study is also underway.

The company's manufacturing process is fully synthetic, scalable, and does not require cell-based processes, substantially reducing contamination risks. PGN-EDODM1 has received orphan drug designation and Fast Track designation from the FDA, as well as orphan medicinal product designation from the European Medicines Agency. PepGen maintains worldwide development and commercial rights to its technology and may selectively explore strategic collaborations to maximize platform value. The company website is www.pepgen.com.