PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and ...

The treatment led to a substantial reduction in DMPK RNA along with splicing defect corrections, as measured by the composite alternative ... DYNE-251, designed with the same platform as DYNE-101, is ...

WVE-N531 is an exon skipping oligonucleotide designed to induce production of endogenous, functional dystrophin protein for the treatment of boys with DMD amenable to exon 53 skipping. Key ...

WVE-N531 is an exon skipping oligonucleotide designed to induce production of endogenous, functional dystrophin protein for the treatment of boys with DMD amenable to exon 53 skipping. Key ...

The trial data demonstrated a reduction in DMPK RNA levels, the molecular target of DYNE-101, and robust splicing correction associated with improvements in multiple ... for Duchenne muscular ...

Dyne measured splicing in all study participants using ... Dyne previously reported that DYNE-251 demonstrated unprecedented dystrophin expression and functional improvement on multiple measures ...