ENTR-601-45

ENTR-601-45 is an investigational therapy developed by Entrada Therapeutics for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 45 skipping. It is a proprietary Endosomal Escape Vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer (PMO). The drug is designed to restore the mRNA reading frame in the DMD gene, enabling translation of a slightly shortened but functional dystrophin protein. This approach targets the underlying genetic cause of DMD due to mutated or missing exons and aims to improve muscle function by promoting production of functional dystrophin. Preclinical studies have shown robust dose-dependent exon skipping and restoration of dystrophin production with improved skeletal muscle function in relevant models[1][3][5][6][9].