RNA Profile
Avidity Biosciences, Inc., a biopharmaceutical firm based in San Diego, California, specializes in developing innovative oligonucleotide-based therapies. The company focuses on creating antibody-oligonucleotide conjugates (AOCs), which are designed to target and treat a variety of serious diseases through a novel therapeutic approach. These conjugates combine the specificity of antibodies with the precision of oligonucleotides to address genetic and rare diseases with high unmet medical needs.
The company's lead product candidate, AOC 1001, is in development for the treatment of myotonic dystrophy type 1, a rare and debilitating genetic muscle disorder. Additionally, Avidity is advancing AOC 1044, aimed at treating Duchenne Muscular Dystrophy (DMD), a severe genetic condition characterized by progressive muscle degeneration. Another key candidate, AOC 1020, is being developed to address facioscapulohumeral muscular dystrophy (FSHD), another rare and progressive muscle disease. All three of these product candidates are currently in the preclinical stage of development.
Beyond its pipeline of AOCs, Avidity Biosciences also offers Lumizyme, a therapy used for the treatment of Pompe disease, a rare genetic disorder characterized by the buildup of glycogen in the muscles. This product is a testament to the company’s commitment to providing treatments for rare and challenging conditions, leveraging its expertise in biopharmaceutical development to bring effective therapies to market.
Founded in 2012, Avidity Biosciences has established itself as a leader in the field of oligonucleotide-based therapeutics. The company’s innovative approach and robust pipeline of product candidates reflect its dedication to addressing complex genetic diseases with novel and targeted treatments. With a strong foundation in research and development, Avidity Biosciences continues to advance its mission of transforming the treatment landscape for patients with serious and rare diseases.
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