Entrada Therapeutics announces clinical candidate ENTR-601-45

Entrada Therapeutics announced the selection of a second clinical candidate within its Duchenne franchise, ENTR-601-45, for the potential treatment of people living with Duchenne who are exon 45 skipping amenable. The Company plans to submit an Investigational New Drug application in the second half of 2024. The selection of ENTR-601-45 is based on robust in vitro exon skipping and dystrophin restoration observed in patient derived skeletal and cardiac muscle cells as well as in vivo preclinical data demonstrating exon skipping levels of over 90% in skeletal muscle in a hDMD mouse model. Entrada plans to present additional data in support of ENTR-601-45 at the Muscular Dystrophy Association Clinical & Scientific Conference in March 2023. ENTR-601-45, a proprietary Endosomal Escape Vehicle conjugated phosphorodiamidate morpholino oligomer, is the third novel clinical candidate from Entrada’s growing pipeline of EEV-therapeutics. Within Entrada’s growing neuromuscular franchise, each EEV-PMO therapeutic candidate has an oligonucleotide sequence designed and optimized for the specific subpopulation of interest. ENTR-601-45 is designed to address the underlying cause of Duchenne muscular dystrophy due to mutated or missing exons in the DMD gene. ENTR-601-45 is an exon skipping investigational therapy that has the potential to restore the mRNA reading frame and allow for the translation of dystrophin protein that is slightly shortened but still functional.