Description
Avidity Biosciences is a biopharmaceutical company, which is engaged in delivering a ribonucleic acid (RNA) therapeutics called antibody oligonucleotide conjugates (AOCs).
The Company utilizes its AOC platform to design, engineer and develop therapeutics, which combine specificity of monoclonal antibodies (mAbs), with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with such therapeutics. Its lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1 (DM1) a rare monogenic muscle disease.
The Company’s advancing and expanding pipeline also includes AOC 1044, the lead of three programs for the treatment of Duchenne Muscular Dystrophy (DMD), and AOC 1020, designed to treat facioscapulohumeral muscular dystrophy (FSHD). AOC 1020 consists of its mAb, which is designed to bind to TfR1 conjugated with a siRNA targeted to DUX4 mRNA to be administered as an intravenous infusion.