Avidity unveils positive topline data from DM1 therapy trial

Avidity Biosciences has reported positive topline results from the Phase I/II MARINA trial of AOC 1001 to treat myotonic dystrophy type 1 (DM1) patients.

The data were examined from the 3:1 randomised trial with 38 subjects, who received one dose of 1mg/kg AOC 1001, three doses of either 2mg/kg AOC 1001 or 4mg/kg of AOC 1001 or placebo.