Avidity Biosciences Reports Positive Data Demonstrating AOC 1044 Delivers Unprecedented Concentrations of PMO in Muscle Following a Single Dose in Healthy Volunteers from Phase 1/2 EXPLORE44™ Trial for Duchenne Muscular Dystrophy

As an early funder of Avidity Biosciences, we are pleased to share that AOC 1044, designed to skip exon 44 in the dystrophin gene, produced significant exon skipping in healthy volunteers.  AOC 1044 consists of two parts : a PMO designed to skip exon 44, coupled to an antibody to the Transferrin receptor to aid its delivery into muscle cells. Avidity is reporting that a single dose of AOC 1044 delivered up to 50-times more PMO to skeletal muscle in healthy volunteers compared to peptide-conjugated PMO, and demonstrated up to 1.5% exon 44 skipping.


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