AOC-1044

Satellos Bioscience's investigational treatment for Duchenne muscular dystrophy (DMD) has demonstrated encouraging efficacy signals in adult patients, offering potential new options in a challenging therapeutic area.

Phase I/II Deliver trial demonstrates sustained functional improvements in DMD patients treated with DYNE-251, showing mean absolute dystrophin expression of 8.72% above baseline at six months with the 20mg/kg dose.

Avidity Biosciences' phase 1/2 trial of delpacibart zotadirsen showed significant 25% increase in dystrophin production and 37% improvement in exon 44 skipping in DMD patients.

Avidity Biosciences plans to submit a Biologics License Application (BLA) for delpacibart zotadirsen (del-zota) to treat Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44) by the end of 2025.

Wave Life Sciences reported positive interim results for WVE-N531, an exon-skipping oligonucleotide, showing promising dystrophin expression in patients with Duchenne muscular dystrophy.