ENTR-601-50

ENTR-601-50 is an investigational nucleic acid-based therapeutic developed by Entrada Therapeutics for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 50 skipping. It acts as an exon 50 inhibitor/modulator, targeting the DMD gene to promote exon skipping and restore dystrophin protein production. The drug is part of Entrada’s pipeline of Endosomal Escape Vehicle (EEV)-conjugated oligonucleotide therapies, which are designed to enhance intracellular delivery and efficacy in muscle tissue. As of late 2024, ENTR-601-50 remains in preclinical development with no reported clinical trial data or regulatory approvals[1][4].