Dyne Therapeutics Presents New Preclinical Data for its Facioscapulohumeral Muscular Dystrophy Program During the FSHD Society International Research Congress

Core Insights - Dyne Therapeutics presented new preclinical data for DYNE-302, demonstrating robust and durable DUX4 suppression and functional benefits in models of facioscapulohumeral muscular dystrophy (FSHD) [1][7] - The data were showcased at the 31st Annual FSHD Society International Research Congress, indicating the potential of DYNE-302 as a therapeutic candidate for FSHD, a disease with no currently approved therapies [1][7] Company Overview - Dyne Therapeutics is a clinical-stage company focused on developing innovative therapeutics for genetically driven muscle diseases, utilizing its proprietary FORCE™ platform [12] - The FORCE platform is designed to enhance the delivery of oligonucleotide therapeutics to muscle tissue, addressing limitations in current treatment approaches [11][12] Product Candidate Details - DYNE-302 targets the genetic cause of FSHD by utilizing a fragment antibody that binds to transferrin receptor 1 (TfR1), conjugated with siRNA to reduce DUX4 expression [8][10] - In preclinical studies, a single intravenous dose of DYNE-302 resulted in a dose-dependent reduction of the DUX4 transcriptome lasting up to three months, with observed benefits on muscle structure and function [1][7] Disease Context - FSHD is a severe muscle disorder caused by aberrant expression of the DUX4 gene, leading to progressive muscle loss and weakness, affecting an estimated 16,000-38,000 individuals in the U.S. and approximately 35,000 in Europe [10] - There are currently no approved therapies for FSHD, highlighting the significant unmet medical need in this area [10]