ARM-201

ARM-201 is an investigational, adeno-associated virus (AAV)-delivered gene therapy developed by Armatus Bio for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a progressive neuromuscular disorder. The therapy uses a proprietary microRNA (miRNA) payload designed to silence or inhibit expression of DUX4, a toxic protein that drives muscle degeneration in FSHD. By reducing DUX4 levels, ARM-201 aims to arrest muscle weakening and atrophy, prevent further degeneration, and reduce inflammation and oxidative stress associated with the disease. The vector employs AAV-SLB101—a next-generation myotropic capsid licensed from Solid Biosciences—engineered for enhanced skeletal and cardiac muscle transduction while minimizing liver exposure. Preclinical studies have shown improvements in FSHD-linked biomarkers and neuromotor function. ARM-201 has received orphan drug designation in the United States[1][2][3][5][6].

Other names

AAV-based gene therapy expressing artificial microRNA targeting the human DUX4 open reading frame