Avidity Biosciences(US:RNA) 美股IPO·2025-08-06 16:52Core Viewpoint - Novartis is reportedly proposing to acquire Avidity Biosciences Inc. to expand its pharmaceutical pipeline, leading to a significant increase of over 24% in Avidity's stock price [1]. Company Overview - Avidity Biosciences, Inc. (NASDAQ: RNA) is a biopharmaceutical company focused on developing a new class of RNA therapies known as Antibody Oligonucleotide Conjugates (AOC™) [3][12]. - The company has received Breakthrough Therapy designation from the FDA for its drug delpacibart zotadirsen (del-zota), aimed at treating Duchenne Muscular Dystrophy (DMD) patients with exon 44 skipping mutations [3][6]. Drug Development and Clinical Trials - Del-zota is currently being evaluated in the Phase 2 EXPLORE44-OLE trial, which is an open-label extension study for DMD patients [4][8]. - The Phase 1/2 EXPLORE44 trial demonstrated significant improvements in biomarkers, including increased dystrophin production and decreased creatine kinase levels, indicating a favorable safety profile [4][11]. - Avidity plans to submit a Biologics License Application (BLA) for del-zota by the end of 2025, with commercial preparations underway for a potential U.S. launch [4][6]. Disease Context - Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle degeneration due to the absence of dystrophin, leading to severe muscle weakness and a significantly reduced lifespan [9]. - DMD primarily affects males, with an incidence of approximately 1 in every 3,500 to 5,000 male births globally [9]. Technology and Innovation - Avidity's AOC platform combines the specificity of monoclonal antibodies with the precision of oligonucleotide therapies, enabling targeted delivery of RNA to muscle tissues [12]. - The company is advancing clinical development projects for three rare neuromuscular diseases: DM1, DMD, and FSHD, while also exploring candidates for rare genetic cardiomyopathies [12].