Avidity Biosciences Gets Fast Track for Facioscapulohumeral Muscular Dystrophy
The FDA has granted Fast Track status to Avidity Biosciences’ AOC 1020 candidate for the treatment of facioscapulohumeral muscular dystrophy, which affects the facial muscles and shoulders.
The drug candidate combines a monoclonal antibody with an oligonucleotide to target the DUX4 gene, an underlying cause of the disease.
AOC 1020 is currently being evaluated in a phase 1/2 clinical trial, to determine its safety and tolerability.
There are currently no FDA-approved treatments available for patients with facioscapulohumeral muscular dystrophy.
Avidity Biosciences, Inc.
Avidity Biosciences, Inc. is pioneering a new class of oligonucleotide-based therapies called AOCs designed to overcome the current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity utilizes its proprietary AOC platform to design, engineer and develop therapeutics that combine the tissueselectivity of monoclonal antibodies and the precision of oligonucleotide therapies in order to access previously undruggable tissue and cell types and more effectively target underlying genetic drivers of diseases. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, and its four other muscle programs are focused on the treatment of muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy and Pompe disease. In addition to its muscle franchise, Avidity has development efforts focused on immune and other cell types.
January 19, 2023
