PepGen Inc.
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PepGen Inc. is a clinical-stage biotechnology company focused on developing oligonucleotide therapeutics for severe neuromuscular and neurological diseases. Headquartered in Boston, Massachusetts, the company utilizes its proprietary Enhanced Delivery Oligonucleotide (EDO) platform technology to improve the delivery and uptake of therapeutic molecules into cells and tissues. PepGen's lead product candidate, PGN-EDO51, targets Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping, representing approximately 13% of the DMD population. The company is also developing additional EDO-based candidates for myotonic dystrophy type 1 (DM1) and other neuromuscular conditions. PepGen completed its initial public offering in May 2022, raising approximately $100 million to advance its clinical programs. The company's EDO platform employs cell-penetrating peptides conjugated to phosphorodiamidate morpholino oligomers (PMOs), designed to enhance tissue penetration and cellular uptake compared to traditional oligonucleotides. In recent developments, PepGen has advanced multiple clinical trials evaluating its lead candidates, with ongoing studies assessing safety, tolerability, and preliminary efficacy in DMD patients. The company maintains research and development operations focused on expanding its pipeline and optimizing its EDO technology for additional genetic diseases with high unmet medical needs. PepGen competes in the oligonucleotide therapeutics space alongside other companies developing exon-skipping and genetic medicines for rare neuromuscular disorders.