Avidity Biosciences is a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs). Their proprietary AOC platform combines the specificity of monoclonal antibodies with the precision of oligonucleotide therapies, enabling targeted delivery of RNA therapies to address diseases previously unreachable with existing approaches.
Avidity's lead product candidate, delpacibart etedesiran (AOC 1001), is an investigational treatment designed to address the root cause of myotonic dystrophy type one (DM1) by reducing levels of disease-related DMPK mRNA. In May 2024, AOC 1001 received Breakthrough Therapy designation from the FDA for the treatment of DM1. Avidity is initiating the global Phase 3 HARBOR study of AOC 1001 this quarter, with primary endpoints including video hand opening time and key secondary endpoints evaluating muscle strength and activities of daily living. Previous data demonstrated reversal of disease progression across multiple functional measures when compared to natural history data.
Beyond DM1, Avidity's pipeline includes clinical programs for Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD), with preclinical programs in muscle atrophy and Pompe disease. The company is also expanding into cardiology and immunology indications through internal efforts and partnerships, such as collaborations with Bristol Myers Squibb and Eli Lilly.
In June 2024, Avidity completed an upsized public offering of common stock, raising approximately USD 461 million in gross proceeds to fund development of its clinical programs and advance research on the AOC platform.
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