Avidity Biosciences, Inc. announced positive AOC 1001 data from the preliminary assessment of the Phase 1/2 MARINA(TM) trial demonstrating the first-ever successful targeted delivery of RNA into muscle, a revolutionary advancement for the field of RNA therapeutics. The effective targeted delivery of siRNA into muscle further reinforces the broad and disruptive potential of Avidity's proprietary AOC platform and expands the ability to address targets and diseases previously unreachable with existing RNA therapies. AOC 1001, Avidity's lead clinical program utilizing its AOC platform, is designed to address the root cause of myotonic dystrophy type 1 (DM1), an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.
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- Avidity Biosciences, Inc. Announces Positive AOC 1001 Phase 1/2 Marina(Tm) Data Demonstrating First-Ever Successful Targeted Delivery of RNA to Muscle - Revolutionary Advancement for the Field of RNA Therapeutics