Avidity Biosciences Announces New Precision Cardiology Development Candidates to Treat Rare Genetic Cardiomyopathies and Provides First Look at Next-Generation Technology Innovations

Core Insights - Avidity Biosciences has expanded its RNA delivery technology into precision cardiology with two new wholly-owned development candidates targeting rare genetic cardiomyopathies: AOC 1086 for PLN Cardiomyopathy and AOC 1072 for PRKAG2 Syndrome [1][2] - Preclinical data for AOC 1072 and AOC 1086 showed approximately 80% reduction in cardiac PLN mRNA and PRKAG2 mRNA, demonstrating robust siRNA delivery to the heart [1][2] - The company introduced next-generation technology innovations that enhance siRNA delivery in skeletal muscle and increase durability, allowing for less frequent dosing [3] Company Developments - Avidity is the first company to successfully deliver siRNA directly to skeletal muscle and has now demonstrated similar capabilities for the heart in preclinical studies [2] - AOC 1072 and AOC 1086 were well tolerated in preclinical studies, showing no adverse effects on electrocardiogram parameters [2] - Avidity will present preclinical data for AOC 1072 at the American Heart Association (AHA) Scientific Sessions 2024 on November 16 [2] Technology Innovations - Next-generation technology innovations include siRNA modifications and evolved antibody engineering, resulting in up to a 30-fold increase in siRNA delivery in skeletal muscle and sustained target inhibition for three months [3] - These advancements in siRNA delivery and durability are expected to improve patient convenience through less frequent dosing [3] Background on Target Diseases - PLN Cardiomyopathy is caused by a mutation in the PLN gene, leading to severe cardiac issues with no FDA-approved disease-modifying therapies available [5][6] - PRKAG2 Syndrome results from a mutation in the PRKAG2 gene, causing excessive cardiac glycogen storage and increasing the risk of sudden cardiac arrest or heart failure, with no FDA-approved therapies currently available [7] Company Mission - Avidity's mission is to improve lives by delivering a new class of RNA therapeutics, Antibody Oligonucleotide Conjugates (AOCs), which combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies [8] - The company is broadening its pipeline to include programs in cardiology and immunology through internal discovery efforts and partnerships [8]