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About PEPGEN INC.
PepGen Limited is a clinical-stage biotechnology company advancing the oligonucleotide therapeutics for the treatment of severe neuromuscular and neurologic diseases. Its proprietary enhanced delivery oligonucleotides (EDOs) are designed to target the underlying causes of rare genetic diseases, such as duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The Company through its enhanced delivery oligonucleotide (EDO) platform developing a pipeline of disease-modifying peptide-conjugated oligonucleotide candidates to treat a variety of degenerative neuromuscular diseases. Its product candidates include PGN-EDO51 and PGN-EDODM1. The Company’s lead product candidate PGN-EDO51, is an EDO peptide conjugated to a phosphorodiamidate morpholino oligomer (PMO) therapeutic cargo, which is developing for the treatment of DMD patients with mutations amenable to an exon 51-skipping approach. It is developing PGN-EDODM1, an EDO peptide-conjugated PMO, for the treatment of DM1.
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