SAN DIEGO , Oct. 3, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA ), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCsTM), today announced that the U.
S. Food and Drug Administration (FDA) has removed the partial clinical hold on delpacibart etedesiran (del-desiran/AOC 1001), an investigational treatment designed to address the root cause of myotonic dystrophy type 1 (DM1). Del-desiran is being evaluated in the ongoing Phase 3 HARBORTM trial in patients with DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.
Del-desiran has received Breakthrough Therapy, Orphan Drug and Fast Track designations by the FDA and Orphan designation by the European Medicines Agency (EMA). About Del-desiran (AOC 1001) Del-desiran (AOC 1001), Avidity's lead product candidate utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of a disease-related mRNA called DMPK. Del-desiran consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA.
Del-desiran is currently being assessed in the global Phase 3 HARBORTM trial and in the ongoing MARINA-OLETM trial in people with DM1. Long-term data from the MARINA-OLE trial showed reversal of disease progression in people living with DM1 across multiple endpoints including video hand opening time (vHOT) a.
