Oxford’s PepGen Launches Into DMD With Next-Gen Oligonucleotide Platform
Aims To Challenge Sarepta
Arising from an Oxford and Cambridge university collaboration, the spin-out has selected Duchenne muscular dystrophy as the first target for its peptide platform.
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The next-generation DMD drug showed dramatic improvement on exon dystrophin and exon skipping, but small numbers and adverse events give pause.
Durable responses have continued to be seen two years after administration of Sarepta’s candidate gene therapy to four boys with Duchenne muscular dystrophy.
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