A Phase I/II trial reported in the New England Journal of Medicine shows delpacibart etedesiran (del‑desiran, AOC‑1001), an antibody–siRNA conjugate, can deliver siRNA to skeletal muscle and reduce DMPK mRNA in patients with myotonic dystrophy type 1. The MARINA trial led by Nicholas Johnson at VCU Health documented target engagement despite two severe adverse events during early testing. The lead sentence: investigators demonstrated systemic muscle delivery of an siRNA payload via antibody conjugation, achieving molecular knockdown in target tissue. The report details tissue uptake, safety signals, and the modality’s potential to treat a genetic neuromuscular disease; antibody‑oligo conjugates use receptor targeting to overcome poor intrinsic uptake into muscle cells.
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