Avidity Biosciences is a biopharmaceutical company focused on RNA therapeutics using its proprietary AOC platform. The company was founded in 2012 and is headquartered in San Diego, California.
Avidity Biosciences announced promising Phase 1/2 trial results for its myotonic dystrophy type 1 drug delpacibart etedesiran, showing 40% reduction in DMPK mRNA and functional improvements. The therapy has received multiple regulatory designations and is now in Phase 3 testing with data expected in late 2026. The company's stock has surged 128% over the past year despite reporting a net loss of approximately $550 million.
Researchers have identified KHDC1L as a circulating biomarker for DUX4 activity in facioscapulohumeral muscular dystrophy (FSHD). The discovery could enable non-invasive blood-based monitoring of disease progression instead of muscle biopsies. The finding emerged from collaboration between academic researchers and Avidity Biosciences scientists.
CAMP4 Therapeutics appoints Michael MacLean to its Board of Directors as the company advances CMP-002 toward a Phase 1/2 trial for SYNGAP1-related disorder. The company also announced an inducement stock option grant of 8,000 shares at $3.85 per share to a new employee. CAMP4 is developing regulatory RNA-targeting therapeutics to upregulate gene expression for genetic diseases.
Novartis has agreed to acquire Avidity Biosciences in the first half of 2026, obtaining its RNA therapeutics platform and neuromuscular disease pipeline. Avidity's cardiovascular RNA programs will be separated into a new independent company called Atrium.
