Sarepta’s Next-Gen DMD Drug SRP-5051 Shows Advantages, But Safety Concerns Too
The next-generation DMD drug showed dramatic improvement on exon dystrophin and exon skipping, but small numbers and adverse events give pause.
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Sarepta’s Exondys 51 Successor On Clinical Hold, But Quick Resolution Expected
The FDA placed a hold on the Phase II study of SRP-5051, which uses the company’s next-generation peptide conjugation technology, but Sarepta thinks it can address safety questions in short order.
Sarepta Charges Ahead With DMD Gene Therapy Despite Setback
The company plans to start its Phase III trial of SRP-9001 in the middle of this year, with a potential readout by the end of 2022.
Oxford’s PepGen Launches Into DMD With Next-Gen Oligonucleotide Platform
Arising from an Oxford and Cambridge university collaboration, the spin-out has selected Duchenne muscular dystrophy as the first target for its peptide platform.