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PepGen raises $113M to challenge Sarepta in DMD, build neuromuscular and neurologic pipeline

Boston-based PepGen’s lead candidate is a therapeutic oligonucleotide that targets exon 51 to treat a subset of DMD patients, putting the biotech squarely on the turf of Sarepta. Like Exondys 51, the drug is designed to mask exon 51 and thereby enable patients to produce the functional dystrophin needed to strengthen muscle fibers.