DYNE-302

DYNE-302 is an investigational therapeutic candidate developed by Dyne Therapeutics for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a rare and progressive muscle disorder characterized by skeletal muscle weakness. The drug consists of a fragment antibody (Fab) that targets transferrin receptor 1 (TfR1), which is highly expressed on muscle cells, conjugated to a small interfering RNA (siRNA) designed to suppress DUX4 mRNA expression. Aberrant expression of the DUX4 gene is the underlying cause of FSHD, leading to progressive muscle wasting. Preclinical studies have demonstrated that DYNE-302 achieves robust and durable suppression of DUX4, resulting in improved muscle structure and function in both in vitro models using patient-derived myotubes and innovative in vivo mouse models expressing human TfR1. As of June 2024, DYNE-302 is advancing through IND/CTA-enabling studies with plans for phase 1 clinical trials[1][2][5][6].