PepGen is a clinical-stage biotech focused entirely on developing PGN-EDODM1, a drug candidate for myotonic dystrophy type 1 (DM1) — a severe, progressive genetic neuromuscular disease with no approved treatments. DM1 is caused by a DNA mutation that produces defective RNA, which traps a protein called MBNL1 in the cell nucleus and disrupts normal RNA processing, causing muscle weakness, cardiac and respiratory problems, and cognitive impairment. PGN-EDODM1 is designed to bind to the defective RNA and free MBNL1 to restore normal function. PepGen argues this mechanism is more precise than competing approaches that simply knock down or degrade the mutated gene transcript entirely. The drug is built on PepGen's Enhanced Delivery Oligonucleotide (EDO) platform, which uses proprietary cell-penetrating peptides chemically linked to oligonucleotide drug payloads. This technology was developed over a decade at Oxford and the MRC Laboratory of Molecular Biology, and PepGen holds an exclusive license from those institutions. PGN-EDODM1 is in active clinical development: Phase 1 showed dose-dependent biological activity, and the ongoing Phase 2 FREEDOM2 trial — currently dosing outside the U.S. due to an FDA partial clinical hold — is expected to produce data in 2026. PepGen has no revenue and funds operations through capital raises. If approved, PepGen would commercialize PGN-EDODM1 as a high-priced specialty drug targeting a small, concentrated patient population of roughly 40,000 in the U.S. and 75,000 in Europe.