Avidity Biosciences announced positive AOC 1001 data from the preliminary assessment of the Phase 1/2 MARINA 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, or DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. The preliminary assessment from the randomized, double-blind, placebo-controlled Phase 1/2 MARINA trial of AOC 1001 provides first in-human data and a mid-study look at the safety and tolerability of all 38 participants and key biomarkers in 19 participants. The preliminary assessment includes biomarker data six weeks after dosing. Participants received a single dose of 1 mg/kg AOC 1001, two doses of 2 mg/kg AOC 1001, or placebo.
Published first on TheFly
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